A Natural History Study In Chinese Male Patients With Duchenne Muscular Dystrophy

NCT03760029 | Completed Results

• 1 other identifier: C3391004
• Study Type: interventional
• Target: 312
• Locations: 1 country
• Sites: 8

Brief Summary

This is a multicenter, prospective, single cohort study designed to describe the natural history of DMD in Chinese male patients. A total of approximately 330 subjects will be enrolled with the target number of subjects in each group as below:

• Group 1, Ambulatory subjects aged \<6 years, approximately 100 subjects;
• Group 2, Ambulatory subjects aged \>=6 years, approximately 180 subjects;
• Group 3, Non-ambulatory subjects, approximately 50 subjects. Subjects will visit sites every 6 months. Each subject will be observed for at least 24 months. All subjects will remain enrolled until the study completion date, such that some will have data collected after Month 24. Subjects, who complete Visit 5/Month 24 at least 6 months prior to study completion, will be asked to complete an additional visit at Month 30.

Conditions

Duchenne Muscular Dystrophy

Outcome Measures

Primary Outcomes (84)

• Age of Participants When They Failed to Walk

  Participant's age at life-altering clinical milestones- failure to walk was calculated based on the birthdate and the date of failure to walk as reported by caregiver during 30 months of this study. Participants who were not reported being failure to walk by their caregivers were censored on the day of their last visit. Kaplan-Meier method was used for analysis.

  Up to Month 30

• Age of Participants When They Failed to Stand

  Participant's age at life-altering clinical milestones- failure to stand was calculated based on the birthdate and the date of failure to stand as reported by caregiver during 30 months of this study. Participants who were not reported being failure to stand by their caregivers were censored on the day of their last visit. Kaplan-Meier method was used for analysis.

  Up to Month 30

• Age of Participants When They Failed to Self-feed

  Participant's age at life-altering clinical milestones- failure to self-feed during 30 months of this study was analyzed using the Kaplan-Meier method. Age was summarized in years.

  Up to Month 30

• Change From Baseline in Northstar Ambulatory Assessment (NSAA) Total Score at Month 6: Ambulatory Participants Aged >=3 Years

  NSAA is a 17-item test that grades performance of various functional skills using the following scale: 0 (unable to achieve goal independently), 1 (modified method but achieves goal with no physical assistance), or 2 ("normal"- no obvious modification of activity). The scale assesses activities required to remain functionally ambulant (e.g. rise from the floor), activities that can be difficult even early in the disease (example \[e.g.\] standing on heels) and activities that are known to progressively deteriorate over time (stand from a chair, walk). NSAA total score was calculated by adding the responses of all 17 items and ranged from 0 to 34, with higher scores indicating better function. NSAA was only performed in ambulatory participants aged \>=3 years old as pre-specified in protocol.

  Baseline (Day 1) and Month 6

• Change From Baseline in NSAA Total Score at Month 12: Ambulatory Participants Aged >=3 Years

  NSAA is a 17-item test that grades performance of various functional skills using the following scale: 0 (unable to achieve goal independently), 1 (modified method but achieves goal with no physical assistance), or 2 ("normal"- no obvious modification of activity). The scale assesses activities required to remain functionally ambulant (e.g. rise from the floor), activities that can be difficult even early in the disease (e.g. standing on heels) and activities that are known to progressively deteriorate over time (stand from a chair, walk). NSAA total score was calculated by adding the responses of all 17 items and ranged from 0 to 34, with higher scores indicating better function. NSAA was only performed in ambulatory participants aged \>=3 years old as pre-specified in protocol.

  Baseline (Day 1) and Month 12

• Change From Baseline in NSAA Total Score at Month 18: Ambulatory Participants Aged >=3 Years

  NSAA is a 17-item test that grades performance of various functional skills using the following scale: 0 (unable to achieve goal independently), 1 (modified method but achieves goal with no physical assistance), or 2 ("normal"- no obvious modification of activity). The scale assesses activities required to remain functionally ambulant (e.g. rise from the floor), activities that can be difficult even early in the disease (e.g. standing on heels) and activities that are known to progressively deteriorate over time (stand from a chair, walk). NSAA total score was calculated by adding the responses of all 17 items and ranged from 0 to 34, with higher scores indicating better function. NSAA was only performed in ambulatory participants aged \>=3 years old as pre-specified in protocol.

  Baseline (Day 1) and Month 18

• Change From Baseline in NSAA Total Score at Month 24: Ambulatory Participants Aged >=3 Years

  NSAA is a 17-item test that grades performance of various functional skills using the following scale: 0 (unable to achieve goal independently), 1 (modified method but achieves goal with no physical assistance), or 2 ("normal"- no obvious modification of activity). The scale assesses activities required to remain functionally ambulant (e.g. rise from the floor), activities that can be difficult even early in the disease (e.g. standing on heels) and activities that are known to progressively deteriorate over time (stand from a chair, walk). NSAA total score was calculated by adding the responses of all 17 items and ranged from 0 to 34, with higher scores indicating better function. NSAA was only performed in ambulatory participants aged \>=3 years old as pre-specified in protocol.

  Baseline (Day 1) and Month 24

• Change From Baseline in NSAA Total Score at Month 30: Ambulatory Participants Aged >=3 Years

  NSAA is a 17-item test that grades performance of various functional skills using the following scale: 0 (unable to achieve goal independently), 1 (modified method but achieves goal with no physical assistance), or 2 ("normal"- no obvious modification of activity). The scale assesses activities required to remain functionally ambulant (e.g. rise from the floor), activities that can be difficult even early in the disease (e.g. standing on heels) and activities that are known to progressively deteriorate over time (stand from a chair, walk). NSAA total score was calculated by adding the responses of all 17 items and ranged from 0 to 34, with higher scores indicating better function. NSAA was only performed in ambulatory participants aged \>=3 years old as pre-specified in protocol.

  Baseline (Day 1) and Month 30

• Change From Baseline in Performance of Upper Limb (PUL) 2.0 Total Score at Month 6: Participants Aged >=10 Years

  PUL 2.0 scale is a 22-item scale used to assess the change that occurs in motor performance of the upper limb overtime from when a participant is still ambulant to the time participant loses all arm function when non-ambulant. PUL 2.0 includes an entry item to define broad starting functional level and 22 items subdivided into shoulder level (six items), mid-level (nine items), and distal level (seven items). Each dimension (shoulder, mid, distal) can be scored separately. There is maximum score of 12 for shoulder level, 17 for mid-level, and 13 for distal level. The total score was calculated by adding three level scores and ranged from 0-42. Higher score indicates better upper limb function. PUL 2.0 total score was assessed in participants aged \>=10 years only as pre-specified in protocol.

  Baseline (Day 1) and Month 6

• Change From Baseline in PUL 2.0 Total Score at Month 12: Participants Aged >=10 Years

  PUL 2.0 scale is a 22-item scale used to assess the change that occurs in motor performance of the upper limb overtime from when a participant is still ambulant to the time participant loses all arm function when non-ambulant. PUL 2.0 includes an entry item to define broad starting functional level and 22 items subdivided into shoulder level (six items), mid-level (nine items), and distal level (seven items). Each dimension (shoulder, mid, distal) can be scored separately. There is maximum score of 12 for shoulder level, 17 for mid-level, and 13 for distal level. The total score was calculated by adding three level scores and ranged from 0-42. Higher score indicates better upper limb function. PUL 2.0 total score was assessed in participants aged \>=10 years only as pre-specified in protocol.

  Baseline (Day 1) and Month 12

• Change From Baseline in PUL 2.0 Total Score at Month 18: Participants Aged >=10 Years

  PUL 2.0 scale is a 22-item scale used to assess the change that occurs in motor performance of the upper limb overtime from when a participant is still ambulant to the time participant loses all arm function when non-ambulant. PUL 2.0 includes an entry item to define broad starting functional level and 22 items subdivided into shoulder level (six items), mid-level (nine items), and distal level (seven items). Each dimension (shoulder, mid, distal) can be scored separately. There is maximum score of 12 for shoulder level, 17 for mid-level, and 13 for distal level. The total score was calculated by adding three level scores and ranged from 0-42. Higher score indicates better upper limb function. PUL 2.0 total score was assessed in participants aged \>=10 years only as pre-specified in protocol.

  Baseline (Day 1) and Month 18

• Change From Baseline in PUL 2.0 Total Score at Month 24: Participants Aged >=10 Years

  PUL 2.0 scale is a 22-item scale used to assess the change that occurs in motor performance of the upper limb overtime from when a participant is still ambulant to the time participant loses all arm function when non-ambulant. PUL 2.0 includes an entry item to define broad starting functional level and 22 items subdivided into shoulder level (six items), mid-level (nine items), and distal level (seven items). Each dimension (shoulder, mid, distal) can be scored separately. There is maximum score of 12 for shoulder level, 17 for mid-level, and 13 for distal level. The total score was calculated by adding three level scores and ranged from 0-42. Higher score indicates better upper limb function. PUL 2.0 total score was assessed in participants aged \>=10 years only as pre-specified in protocol.

  Baseline (Day 1) and Month 24

• Change From Baseline in PUL 2.0 Total Score at Month 30: Participants Aged >=10 Years

  PUL 2.0 scale is a 22-item scale used to assess the change that occurs in motor performance of the upper limb overtime from when a participant is still ambulant to the time participant loses all arm function when non-ambulant. PUL 2.0 includes an entry item to define broad starting functional level and 22 items subdivided into shoulder level (six items), mid-level (nine items), and distal level (seven items). Each dimension (shoulder, mid, distal) can be scored separately. There is maximum score of 12 for shoulder level, 17 for mid-level, and 13 for distal level. The total score was calculated by adding three level scores and ranged from 0-42. Higher score indicates better upper limb function. PUL 2.0 total score was assessed in participants aged \>=10 years only as pre-specified in protocol.

  Baseline (Day 1) and Month 30

• Change From Baseline in Rise From Floor Velocity at Month 6: Ambulatory Participants Aged >=3 Years Only

  The rise from floor velocity was defined as the reciprocal of the time (in seconds) to rise from floor. The rise from floor test was performed only in ambulatory participants aged \>=3 years old as pre-specified in the protocol.

  Baseline (Day 1) and Month 6

• Change From Baseline in Rise From Floor Velocity at Month 12: Ambulatory Participants Aged >=3 Years Only

  The rise from floor velocity was defined as the reciprocal of the time (in seconds) to rise from floor. The rise from floor test was performed only in ambulatory participants aged \>=3 years old as pre-specified in the protocol.

  Baseline (Day 1) and Month 12

• Change From Baseline in Rise From Floor Velocity at Month 18: Ambulatory Participants Aged >=3 Years Only

  The rise from floor velocity was defined as the reciprocal of the time (in seconds) to rise from floor. The rise from floor test was performed only in ambulatory participants aged \>=3 years old as pre-specified in the protocol.

  Baseline (Day 1) and Month 18

• Change From Baseline in Rise From Floor Velocity at Month 24: Ambulatory Participants Aged >=3 Years Only

  The rise from floor velocity was defined as the reciprocal of the time (in seconds) to rise from floor. The rise from floor test was performed only in ambulatory participants aged \>=3 years old as pre-specified in the protocol.

  Baseline (Day 1) and Month 24

• Change From Baseline in Rise From Floor Velocity at Month 30: Ambulatory Participants Aged >=3 Years Only

  The rise from floor velocity was defined as the reciprocal of the time (in seconds) to rise from floor. The rise from floor test was performed only in ambulatory participants aged \>=3 years old as pre-specified in the protocol.

  Baseline (Day 1) and Month 30

• Change From Baseline in 10 Meter Walk or Run Velocity at Month 6: Ambulatory Participants Aged >=3 Years

  The 10 meter walk or run test was performed as part of NSAA. The 10 meter walk or run velocity was defined as the reciprocal of the time (in seconds) to complete the 10 meter run or walk test. The 10 meter walk or run test was performed in ambulatory children \>=3 years old only as pre-specified in the protocol.

  Baseline (Day 1) and Month 6

• Change From Baseline in 10 Meter Walk or Run Velocity at Month 12: Ambulatory Participants Aged >=3 Years

  The 10 meter walk or run test was performed as part of NSAA. The 10 meter walk or run velocity was defined as the reciprocal of the time (in seconds) to complete the 10 meter run or walk test. The 10 meter walk or run test was performed in ambulatory children \>=3 years old only as pre-specified in the protocol.

  Baseline (Day 1) and Month 12

• Change From Baseline in 10 Meter Walk or Run Velocity at Month 18: Ambulatory Participants Aged >=3 Years

  The 10 meter walk or run test was performed as part of NSAA. The 10 meter walk or run velocity was defined as the reciprocal of the time (in seconds) to complete the 10 meter run or walk test. The 10 meter walk or run test was performed in ambulatory children \>=3 years old only as pre-specified in the protocol.

  Baseline (Day 1) and Month 18

• Change From Baseline in 10 Meter Walk or Run Velocity at Month 24: Ambulatory Participants Aged >=3 Years

  The 10 meter walk or run test was performed as part of NSAA. The 10 meter walk or run velocity was defined as the reciprocal of the time (in seconds) to complete the 10 meter run or walk test. The 10 meter walk or run test was performed in ambulatory children \>=3 years old only as pre-specified in the protocol.

  Baseline (Day 1) and Month 24

• Change From Baseline in 10 Meter Walk or Run Velocity at Month 30: Ambulatory Participants Aged >=3 Years

  The 10 meter walk or run test was performed as part of NSAA. The 10 meter walk or run velocity was defined as the reciprocal of the time (in seconds) to complete the 10 meter run or walk test. The 10 meter walk or run test was performed in ambulatory children \>=3 years old only as pre-specified in the protocol.

  Baseline (Day 1) and Month 30

• Change From Baseline in Knee Extension of Muscle Strength at Month 6: Participants Aged >=5 Years

  Muscle strength was recorded by handheld myometry. Left and right knee extension was analyzed. The muscle strength test was only performed in participants \>=5 years old as pre-specified in protocol.

  Baseline (Day 1) and Month 6

• Change From Baseline in Knee Extension of Muscle Strength at Month 12: Participants Aged >=5 Years

  Muscle strength was recorded by handheld myometry. Left and right knee extension was analyzed. The muscle strength test was only performed in participants \>=5 years old as pre-specified in protocol.

  Baseline (Day 1) and Month 12

• Change From Baseline in Knee Extension of Muscle Strength at Month 18: Participants Aged >=5 Years

  Muscle strength was recorded by handheld myometry. Left and right knee extension was analyzed. The muscle strength test was only performed in participants \>=5 years old as pre-specified in protocol.

  Baseline (Day 1) and Month 18

• Change From Baseline in Knee Extension of Muscle Strength at Month 24: Participants Aged >=5 Years

  Muscle strength was recorded by handheld myometry. Left and right knee extension was analyzed. The muscle strength test was only performed in participants \>=5 years old as pre-specified in protocol.

  Baseline (Day 1) and Month 24

• Change From Baseline in Knee Extension of Muscle Strength at Month 30: Participants Aged >=5 Years

  Muscle strength was recorded by handheld myometry. Left and right knee extension was analyzed. The muscle strength test was only performed in participants \>=5 years old as pre-specified in protocol.

  Baseline (Day 1) and Month 30

• Change From Baseline in Elbow Flexion of Muscle Strength at Month 6: Participants Aged >=5 Years

  Muscle strength was recorded by handheld myometry. Left and right elbow flexion were analyzed. The muscle strength test was only performed in participants \>=5 years old as pre-specified in the protocol.

  Baseline (Day 1) and Month 6

• Change From Baseline in Elbow Flexion of Muscle Strength at Month 12: Participants Aged >=5 Years

  Muscle strength was recorded by handheld myometry. Left and right elbow flexion were analyzed. The muscle strength test was only performed in participants \>=5 years old as pre-specified in the protocol.

  Baseline (Day 1) and Month 12

• Change From Baseline in Elbow Flexion of Muscle Strength at Month 18: Participants Aged >=5 Years

  Muscle strength was recorded by handheld myometry. Left and right elbow flexion were analyzed. The muscle strength test was only performed in participants \>=5 years old as pre-specified in the protocol.

  Baseline (Day 1) and Month 18

• Change From Baseline in Elbow Flexion of Muscle Strength at Month 24: Participants Aged >=5 Years

  Muscle strength was recorded by handheld myometry. Left and right elbow flexion were analyzed. The muscle strength test was only performed in participants \>=5 years old as pre-specified in the protocol.

  Baseline (Day 1) and Month 24

• Change From Baseline in Elbow Flexion of Muscle Strength at Month 30: Participants Aged >=5 Years

  Muscle strength was recorded by handheld myometry. Left and right elbow flexion were analyzed. The muscle strength test was only performed in participants \>=5 years old as pre-specified in the protocol.

  Baseline (Day 1) and Month 30

• Change From Baseline in Elbow Extension of Muscle Strength at Month 6: Participants Aged >=5 Years

  Muscle strength was recorded by handheld myometry. Left and right elbow extension were analyzed. The muscle strength test was only performed in participants \>=5 years old as pre-specified in protocol.

  Baseline (Day 1) and Month 6

• Change From Baseline in Elbow Extension Muscle Strength at Month 12: Participants Aged >=5 Years

  Muscle strength was recorded by handheld myometry. Left and right elbow extension were analyzed. The muscle strength test was only performed in participants \>=5 years old as pre-specified in protocol.

  Baseline (Day 1) and Month 12

• Change From Baseline in Elbow Extension Muscle Strength at Month 18: Participants Aged >=5 Years

  Muscle strength was recorded by handheld myometry. Left and right elbow extension were analyzed. The muscle strength test was only performed in participants \>=5 years old as pre-specified in protocol.

  Baseline (Day 1) and Month 18

• Change From Baseline in Elbow Extension Muscle Strength at Month 24: Participants Aged >=5 Years

  Muscle strength was recorded by handheld myometry. Left and right elbow extension were analyzed. The muscle strength test was only performed in participants \>=5 years old as pre-specified in protocol.

  Baseline (Day 1) and Month 24

• Change From Baseline in Elbow Extension Muscle Strength at Month 30: Participants Aged >=5 Years

  Muscle strength was recorded by handheld myometry. Left and right elbow extension were analyzed. The muscle strength test was only performed in participants \>=5 years old as pre-specified in protocol.

  Baseline (Day 1) and Month 30

• Change From Baseline in Shoulder Abduction of Muscle Strength at Month 6: Participants Aged >=5 Years

  Muscle strength was recorded by handheld myometry. Left and right shoulder abduction were analyzed. The muscle strength test was only performed in participants \>=5 years old as pre-specified in the protocol.

  Baseline (Day 1) and Month 6

• Change From Baseline in Shoulder Abduction of Muscle Strength at Month 12: Participants Aged >=5 Years

  Muscle strength was recorded by handheld myometry. Left and right shoulder abduction were analyzed. The muscle strength test was only performed in participants \>=5 years old as pre-specified in the protocol.

  Baseline (Day 1) and Month 12

• Change From Baseline in Shoulder Abduction of Muscle Strength at Month 18: Participants Aged >=5 Years

  Muscle strength was recorded by handheld myometry. Left and right shoulder abduction were analyzed. The muscle strength test was only performed in participants \>=5 years old as pre-specified in the protocol.

  Baseline (Day 1) and Month 18

• Change From Baseline in Shoulder Abduction of Muscle Strength at Month 24: Participants Aged >=5 Years

  Muscle strength was recorded by handheld myometry. Left and right shoulder abduction were analyzed. The muscle strength test was only performed in participants \>=5 years old as pre-specified in the protocol.

  Baseline (Day 1) and Month 24

• Change From Baseline in Shoulder Abduction of Muscle Strength at Month 30: Participants Aged >=5 Years

  Muscle strength was recorded by handheld myometry. Left and right shoulder abduction were analyzed. The muscle strength test was only performed in participants \>=5 years old as pre-specified in the protocol.

  Baseline (Day 1) and Month 30

• Change From Baseline in Range of Motion (ROM) at Bilateral Ankles at Month 6

  Range of motion was evaluated by using goniometry to record any occurrences of ankle contractures. The ROM at left and right ankles were measured in degrees of passive dorsiflexion.

  Baseline (Day 1) and Month 6

• Change From Baseline in ROM at Bilateral Ankles at Month 12

  Range of motion was evaluated by using goniometry to record any occurrences of ankle contractures. The ROM at left and right ankles were measured in degrees of passive dorsiflexion.

  Baseline (Day 1) and Month 12

• Change From Baseline in ROM at Bilateral Ankles at Month 18

  Range of motion was evaluated by using goniometry to record any occurrences of ankle contractures. The ROM at left and right ankles were measured in degrees of passive dorsiflexion.

  Baseline (Day 1) and Month 18

• Change From Baseline in ROM at Bilateral Ankles at Month 24

  Range of motion was evaluated by using goniometry to record any occurrences of ankle contractures. The ROM at left and right ankles were measured in degrees of passive dorsiflexion.

  Baseline (Day 1) and Month 24

• Change From Baseline in ROM at Bilateral Ankles at Month 30

  Range of motion was evaluated by using goniometry to record any occurrences of ankle contractures. The ROM at left and right ankles were measured in degrees of passive dorsiflexion.

  Baseline (Day 1) and Month 30

• Change From Baseline in ROM at Bilateral Elbows at Month 6

  Range of motion was evaluated by using goniometry to record any occurrences of elbow contractures. The ROM at left and right elbows were measured in degrees of passive extension.

  Baseline (Day 1) and Month 6

• Change From Baseline in ROM at Bilateral Elbows at Month 12

  Range of motion was evaluated by using goniometry to record any occurrences of elbow contractures. The ROM at left and right elbows were measured in degrees of passive extension.

  Baseline (Day 1) and Month 12

• Change From Baseline in ROM at Bilateral Elbows at Month 18

  Range of motion was evaluated by using goniometry to record any occurrences of elbow contractures. The ROM at left and right elbows were measured in degrees of passive extension.

  Baseline (Day 1) and Month 18

• Change From Baseline in ROM at Bilateral Elbows at Month 24

  Range of motion was evaluated by using goniometry to record any occurrences of elbow contractures. The ROM at left and right elbows were measured in degrees of passive extension.

  Baseline (Day 1) and Month 24

• Change From Baseline in ROM at Bilateral Elbows at Month 30

  Range of motion was evaluated by using goniometry to record any occurrences of elbow contractures. The ROM at left and right elbows were measured in degrees of passive extension.

  Baseline (Day 1) and Month 30

• Change From Baseline in Percent Predicted Forced Vital Capacity (%pFVC) at Month 12: Participants Aged >=6 Years

  Forced vital capacity (FVC) is the volume of air that can be maximally forcefully exhaled after taking the deepest breath possible and was measured using spirometry. The percent predicted FVC was calculated from FVC (measured in liter) according to age, height (estimated height as derived from the ulna length for non-ambulatory participants), ethnicity, and gender using multi-ethnic reference values for spirometry. The pulmonary function assessments were performed in participants aged \>=6 years as pre-specified in the protocol.

  Baseline (Day 1) and Month 12

• Change From Baseline in %pFVC at Month 24: Participants Aged >=6 Years

  FVC is the volume of air that can be maximally forcefully exhaled after taking the deepest breath possible and was measured using spirometry. The percent predicted FVC was calculated from FVC (measured in liter) according to age, height (estimated height as derived from the ulna length for non-ambulatory participants), ethnicity, and gender using multi-ethnic reference values for spirometry. The pulmonary function assessments were performed in participants aged \>=6 years as pre-specified in the protocol.

  Baseline (Day 1) and Month 24

• Change From Baseline in %pFVC at Month 30: Participants Aged >=6 Years

  FVC is the volume of air that can be maximally forcefully exhaled after taking the deepest breath possible and was measured using spirometry. The percent predicted FVC was calculated from FVC (measured in liter) according to age, height (estimated height as derived from the ulna length for non-ambulatory participants), ethnicity, and gender using multi-ethnic reference values for spirometry. The pulmonary function assessments were performed in participants aged \>=6 years as pre-specified in the protocol.

  Baseline (Day 1) and Month 30

• Change From Baseline in %pFVC at Month 12: Participants Aged >=6 Years (Unplanned Analysis)

  FVC is the volume of air that can be maximally forcefully exhaled after taking the deepest breath possible and was measured using spirometry. The percent predicted FVC was calculated from FVC (measured in liter) according to age, height (estimated height as derived from the ulna length for non-ambulatory participants), ethnicity, and gender using multi-ethnic reference values for spirometry. The pulmonary function assessments were performed in participants aged \>=6 years as pre-specified in the protocol. Incorrect data were addressed by unplanned analysis.

  Baseline (Day 1) and Month 12

• Change From Baseline in %pFVC at Month 24: Participants Aged >=6 Years (Unplanned Analysis)

  FVC is the volume of air that can be maximally forcefully exhaled after taking the deepest breath possible and was measured using spirometry. The percent predicted FVC was calculated from FVC (measured in liter) according to age, height (estimated height as derived from the ulna length for non-ambulatory participants), ethnicity, and gender using multi-ethnic reference values for spirometry. The pulmonary function assessments were performed in participants aged \>=6 years as pre-specified in the protocol. Incorrect data were addressed by unplanned analysis.

  Baseline (Day 1) and Month 24

• Change From Baseline in %pFVC at Month 30: Participants Aged >=6 Years (Unplanned Analysis)

  FVC is the volume of air that can be maximally forcefully exhaled after taking the deepest breath possible and was measured using spirometry. The percent predicted FVC was calculated from FVC (measured in liter) according to age, height (estimated height as derived from the ulna length for non-ambulatory participants), ethnicity, and gender using multi-ethnic reference values for spirometry. The pulmonary function assessments were performed in participants aged \>=6 years as pre-specified in the protocol. Incorrect data were addressed by unplanned analysis.

  Baseline (Day 1) and Month 30

• Change From Baseline in Percent Predicted Forced Expiratory Volume in One Second (%pFEV1) at Month 12: Participants Aged >=6 Years

  Forced expiratory volume in one second (FEV1) is the volume of air forcefully exhaled in 1 second and was measured using spirometry. The %pFEV1 was calculated from FEV1 (measured in liter) according to age, height (estimated height as derived from the ulna length for non-ambulatory participants), ethnicity, and gender using multi-ethnic reference values for Spirometry. The pulmonary function assessments were performed only in participants \>=6 years old as pre-specified in protocol.

  Baseline (Day 1) and Month 12

• Change From Baseline in %pFEV1 at Month 24: Participants Aged >=6 Years

  FEV1 is the volume of air forcefully exhaled in 1 second and was measured using spirometry. The %pFEV1 was calculated from FEV1 (measured in liter) according to age, height (estimated height as derived from the ulna length for non-ambulatory participants), ethnicity, and gender using multi-ethnic reference values for Spirometry. The pulmonary function assessments were performed only in participants \>=6 years old as pre-specified in protocol.

  Baseline (Day 1) and Month 24

• Change From Baseline in %pFEV1 at Month 30: Participants Aged >=6 Years

  FEV1 is the volume of air forcefully exhaled in 1 second and was measured using spirometry. The %pFEV1 was calculated from FEV1 (measured in liter) according to age, height (estimated height as derived from the ulna length for non-ambulatory participants), ethnicity, and gender using multi-ethnic reference values for Spirometry. The pulmonary function assessments were performed only in participants \>=6 years old as pre-specified in protocol.

  Baseline (Day 1) and Month 30

• Change From Baseline in %pFEV1 at Month 12: Participants Aged >=6 Years (Unplanned Analysis)

  FEV1 is the volume of air forcefully exhaled in 1 second and was measured using spirometry. The %pFEV1 was calculated from FEV1 (measured in liter) according to age, height (estimated height as derived from the ulna length for non-ambulatory participants), ethnicity, and gender using multi-ethnic reference values for Spirometry. The pulmonary function assessments were performed only in participants \>=6 years old as pre-specified in protocol. Incorrect data were addressed by unplanned analysis.

  Baseline (Day 1) and Month 12

• Change From Baseline in %pFEV1 at Month 24: Participants Aged >=6 Years (Unplanned Analysis)

  FEV1 is the volume of air forcefully exhaled in 1 second and was measured using spirometry. The %pFEV1 was calculated from FEV1 (measured in liter) according to age, height (estimated height as derived from the ulna length for non-ambulatory participants), ethnicity, and gender using multi-ethnic reference values for Spirometry. The pulmonary function assessments were performed only in participants \>=6 years old as pre-specified in protocol. Incorrect data were addressed by unplanned analysis.

  Baseline (Day 1) and Month 24

• Change From Baseline in %pFEV1 at Month 30: Participants Aged >=6 Years (Unplanned Analysis)

  FEV1 is the volume of air forcefully exhaled in 1 second and was measured using spirometry. The %pFEV1 was calculated from FEV1 (measured in liter) according to age, height (estimated height as derived from the ulna length for non-ambulatory participants), ethnicity, and gender using multi-ethnic reference values for Spirometry. The pulmonary function assessments were performed only in participants \>=6 years old as pre-specified in protocol. Incorrect data were addressed by unplanned analysis.

  Baseline (Day 1) and Month 30

• Change From Baseline in Maximum Inspiratory Pressure at Month 12: Participants Aged >=6 Years

  The pulmonary function assessments were performed only in participants \>=6 years old as pre-specified in protocol.

  Baseline (Day 1) and Month 12

• Change From Baseline in Maximum Inspiratory Pressure at Month 24: Participants Aged >=6 Years

  The pulmonary function assessments were performed only in participants \>=6 years old as pre-specified in protocol.

  Baseline (Day 1) and Month 24

• Change From Baseline in Maximum Inspiratory Pressure at Month 30: Participants Aged >=6 Years

  The pulmonary function assessments were performed only in participants \>=6 years old as pre-specified in protocol.

  Baseline (Day 1) and Month 30

• Change From Baseline in Maximum Expiratory Pressure at Month 12: Participants Aged >=6 Years

  The pulmonary function assessments were performed only in participants \>=6 years old as pre-specified in SAP.

  Baseline (Day 1) and Month 12

• Change From Baseline in Maximum Expiratory Pressure at Month 24: Participants Aged >=6 Years

  The pulmonary function assessments were performed only in participants \>=6 years old as pre-specified in SAP.

  Baseline (Day 1) and Month 24

• Change From Baseline in Maximum Expiratory Pressure at Month 30: Participants Aged >=6 Years

  The pulmonary function assessments were performed only in participants \>=6 years old as pre-specified in SAP.

  Baseline (Day 1) and Month 30

• Change From Baseline in Peak Cough Flow at Month 12: Participants Aged >=6 Years

  The pulmonary function assessments were performed only in participants \>=6 years old as pre-specified in protocol.

  Baseline (Day 1) and Month 12

• Change From Baseline in Peak Cough Flow at Month 24: Participants Aged >=6 Years

  The pulmonary function assessments were performed only in participants \>=6 years old as pre-specified in protocol.

  Baseline (Day 1) and Month 24

• Change From Baseline in Peak Cough Flow at Month 30: Participants Aged >=6 Years

  The pulmonary function assessments were performed only in participants \>=6 years old as pre-specified in protocol.

  Baseline (Day 1) and Month 30

• Change From Baseline in Peak Cough Flow at Month 12: Participants Aged >=6 Years (Unplanned Analysis)

  The pulmonary function assessments were performed only in participants \>=6 years old as pre-specified in protocol. Incorrect data were addressed by unplanned analysis.

  Baseline (Day 1) and Month 12

• Change From Baseline in Peak Cough Flow at Month 24: Participants Aged >=6 Years (Unplanned Analysis)

  The pulmonary function assessments were performed only in participants \>=6 years old as pre-specified in protocol. Incorrect data were addressed by unplanned analysis.

  Baseline (Day 1) and Month 24

• Change From Baseline in Peak Cough Flow at Month 30: Participants Aged >=6 Years (Unplanned Analysis)

  The pulmonary function assessments were performed only in participants \>=6 years old as pre-specified in protocol. Incorrect data were addressed by unplanned analysis.

  Baseline (Day 1) and Month 30

• Change From Baseline in Left Ventricular Ejection Fraction (LVEF) at Month 12: Participants Aged >=6 Years

  LVEF was the percentage of blood that was ejected out of left ventricle with each contraction, estimated by echocardiography. The LVEF was only performed in participants \>=6 years old as pre-specified in protocol.

  Baseline (Day 1) and Month 12

• Change From Baseline in LVEF at Month 24: Participants Aged >=6 Years

  LVEF was the percentage of blood that was ejected out of left ventricle with each contraction, estimated by echocardiography. The LVEF was only performed in participants \>=6 years old as pre-specified in protocol.

  Baseline (Day 1) and Month 24

• Change From Baseline in LVEF at Month 30: Participants Aged >=6 Years

  LVEF was the percentage of blood that was ejected out of left ventricle with each contraction, estimated by echocardiography. The LVEF was only performed in participants \>=6 years old as pre-specified in protocol.

  Baseline (Day 1) and Month 30

• Change From Baseline in LVEF at Month 12: Participants Aged >=6 Years (Unplanned Analysis)

  LVEF was the percentage of blood that was ejected out of left ventricle with each contraction, estimated by echocardiography. The LVEF was only performed in participants \>=6 years old as pre-specified in protocol. Incorrect data were addressed by unplanned analysis.

  Baseline (Day 1) and Month 12

• Change From Baseline in LVEF at Month 24: Participants Aged >=6 Years (Unplanned Analysis)

  LVEF was the percentage of blood that was ejected out of left ventricle with each contraction, estimated by echocardiography. The LVEF was only performed in participants \>=6 years old as pre-specified in protocol. Incorrect data were addressed by unplanned analysis.

  Baseline (Day 1) and Month 24

• Change From Baseline in LVEF at Month 30: Participants Aged >=6 Years (Unplanned Analysis)

  LVEF was the percentage of blood that was ejected out of left ventricle with each contraction, estimated by echocardiography. The LVEF was only performed in participants \>=6 years old as pre-specified in protocol. Incorrect data were addressed by unplanned analysis.

  Baseline (Day 1) and Month 30

• Change From Baseline in Wechsler Intelligence Scale for Children (WISC)-IV Score at Month 24: Ambulatory Participants >= 6 to <=16 Years

  WISC-IV is an individually administered intelligence test for children between the ages of 6 and 16. The WISC-IV Composites are: Verbal Comprehension, Perceptual Reasoning, Working Memory, and Processing Speed. Scores from the Composites constitute the WISC-IV Full Scale IQ score which ranges from 40 (Exceptionally Low) to 160 (Exceptionally Superior), higher scores indicated more intelligence. The WISC was only performed in ambulatory participants \>= 6 to \<=16 years old as pre-specified in the protocol.

  Baseline (Day 1) and Month 24

Secondary Outcomes (29)

• Number of Participants With Type of DMD Mutation

  Up to Month 30

• Number of Participants With Each Affected Exon by Mutation Types

  Up to Month 30

• Number of Participants With DMD Mutations Affecting Any Exon Between Exon 9 and Exon 13 or Deletion That Affects Both Exon 29 and Exon 30

  Up to Month 30

• Change From Baseline in Pediatric Outcomes Data Collection Instrument (PODCI) Global Functioning Scale and Each Core Scale Score (Pediatric Parent Report) at Months 6, 12, 18, 24 and 30

  Baseline (Day 1) and Months 6, 12, 18, 24 and 30

• Change From Baseline in PODCI Global Functioning Scale and Each Core Scale Score (Adolescent Parent Report) at Months 6, 12, 18, 24 and 30

  Baseline (Day 1) and Months 6, 12, 18, 24 and 30

Study Arms (1)

Study arm

OTHER

All subjects in this study will be observed for 24-30 months.

Other: Visit frequency

Interventions

Visit frequency OTHER

All subjects need to visit sites more frequently than in routine clinical practice.

Study arm

Eligibility Criteria

• Age: 0 Years+
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Chinese male patients with any age, diagnosed with DMD. Diagnosis must be confirmed in subject's medical history and by genetic testing obtained during routine clinical care for diagnostic purposes as reported from an appropriate regulated laboratory using a clinically validated genetic test (genetic testing is not provided by the sponsor).
• Subjects who are \>=4 years old must be receiving glucocorticosteroids for a minimum of 6 months prior to signing informed consent. There should be no significant change (\<0.2 mg/kg) in dosage or dose regimen (not related to body weight change) for at least 3 months immediately prior to signing the informed consent. Subjects who are aged \>4 years will be exempt from this requirement; those not taking GC will be eligible if the initiation of GC treatment in these subjects is considered inappropriate in the opinion of Investigators.

You may not qualify if:

• Any injury which may impact functional testing. Previous injuries must be fully healed prior to consenting. Prior lower limb fractures must be fully healed and at least 3 months from injury date.
• Presence or history of other musculoskeletal or neurologic disease or somatic disorder not related to DMD including pulmonary, cardiac, and cognitive diseases.
• Subjects \>=4 years old who have not completed the varicella vaccination.
• Participation in other studies involving investigational drug(s) for a minimum of 90 days prior to signing the informed consent and/or during study participation.

Sponsors & Collaborators

• Pfizer: lead

Study Sites (8)

Children's Hospital of Chongqing Medical University

Chongqing, Chongqing Municipality, 400014, China

Location

Children's Hospital of Chongqing Medical University (Liangjiang Branch)

Chongqing, Chongqing Municipality, 401122, China

Location

The First Affiliated Hospital Of Fujian Medical University

Fuzhou, Fujian, 350005, China

Location

Peking University First Hospital

Beijing, 100034, China

Location

Beijing Children's Hospital, Capital Medical University

Beijing, 100045, China

Location

Huashan Hospital, Fudan University

Shanghai, 200040, China

Location

Affiliated children's hospital of fudan university

Shanghai, 201102, China

Location

Children's Hospital of Fudan University

Shanghai, 201102, China

Location

Related Publications (1)

• Li X, Lv J, Zhu W, Hong S, Wang Z, Chang X, Gao YX, Zhou Y, Jia C, Fang J, Patterson TA. A 1-year analysis from a natural history study in Chinese individuals with Duchenne muscular dystrophy. Lancet Reg Health West Pac. 2023 Nov 28;42:100944. doi: 10.1016/j.lanwpc.2023.100944. eCollection 2024 Jan.

  PMID: 38089167 DERIVED

Related Links

• To obtain contact information for a study center near you, click here.

MeSH Terms

Conditions

Muscular Dystrophy, Duchenne

Condition Hierarchy (Ancestors)

Muscular Dystrophies; Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Results Point of Contact

• Title: Pfizer ClinicalTrials.gov Call Center
• Organization: Pfizer Inc.

ClinicalTrials.gov_Inquiries@pfizer.com

1-800-718-1021

Study Officials

• Pfizer CT.gov Call Center

  Pfizer

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: OTHER
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 15, 2018
• First Posted: November 30, 2018
• Study Start: July 24, 2019
• Primary Completion: March 21, 2023
• Study Completion: March 21, 2023
• Last Updated: September 19, 2024
• Results First Posted: September 19, 2024

Record last verified: 2024-09

Data Sharing

• IPD Sharing: Will not share

Locations

CN (8)