A Low Interventional Study to Monitor Activity Using Wearable Sensors in Duchenne Muscular Dystrophy

NCT04254172 | Terminated FDA Device

• 1 other identifier: C3391005
• Study Type: observational
• Target: 2
• Locations: 1 country
• Sites: 1

Brief Summary

The purpose of this low interventional study is to collect data on everyday movement in boys with Duchenne muscular dystrophy (DMD) using wearable activity sensors. The activity sensors could provide useful information beyond what is currently collected by functional (movement, strength) assessments in clinic. This information can help with the understanding of the impact of DMD, and perhaps with how possible treatments can affect this impact.

Conditions

Duchenne Muscular Dystrophy (DMD)

Outcome Measures

Primary Outcomes (1)

• Mean change from baseline and variability of activity measures

  baseline, 3, 6, 9, and 12 months

Secondary Outcomes (2)

• Mean change from baseline in functional assessment scores obtained in the clinic

  baseline, 3, 6, 9, and 12 months

• Comparison of mean changes from baseline and correlation coefficient between activity monitoring data and functional data obtained in clinic

  baseline, 3, 6, 9, and 12 months

Study Arms (1)

Single cohort

There is no randomization or stratification in this study. All subjects will complete the same study assessments.

Device: Activity Monitor

Interventions

Activity Monitor DEVICE

Wrist and ankle sensors to be worn continuously for 2-week intervals.

Single cohort

Eligibility Criteria

• Age: 4 Years - 12 Years
• Healthy Volunteers: No
• Age Groups: Child (0-17)
• Sampling Method: Non-Probability Sample

Study Population

Ambulatory boys with Duchenne muscular dystrophy who receive care at Nationwide Children's Hospital

You may qualify if:

• Diagnosis of Duchenne muscular dystrophy confirmed by medical history and genetic testing
• Body weight between 15 and 50 kg
• Receipt of glucocorticoids for 6 months and a stable daily dose for at least 3 months prior to study entry
• Ability to rise from floor within seven (7) seconds and ability to walk

You may not qualify if:

• Current exposure to systemic immunosuppressant agents other than glucocorticoids.
• Prior exposure to any gene therapy agent, including exon-skipping and missense agents.
• Exposure to other investigational drugs within 30 days or 5 half-lives, whichever is longer.
• Any injury which may impact functional testing per investigator's judgement. 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 at screening.
• Any planned surgeries which may impact physical activity and performance.
• Presence or history of musculoskeletal or neurological disease in addition to DMD.
• Any known allergies or skin reactions to stainless steel, versaflex, and silicon that may cause possible discomfort by wearable sensors.
• Evidence or history of clinically significant hematological, renal, endocrine, pulmonary, gastrointestinal, cardiovascular, hepatic, cancer, autoimmune or allergic disease that may interfere with the study conduct as per investigator's judgment, excluding untreated, asymptomatic, seasonal allergies at time of screening.
• Investigator site staff members directly involved in the conduct of the study and their family members, site staff members otherwise supervised by the investigator, or participants who are Pfizer employees, including their family members, directly involved in the conduct of the study.

Sponsors & Collaborators

• Pfizer: lead

Study Sites (1)

Nationwide Children's Hospital

Columbus, Ohio, 43205, United States

Location

Related Links

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

MeSH Terms

Conditions

Muscular Dystrophy, Duchenne

Interventions

Fitness Trackers

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

Intervention Hierarchy (Ancestors)

Diagnostic Equipment; Equipment and Supplies; Wearable Electronic Devices; Electrical Equipment and Supplies

Study Officials

• Pfizer CT.gov Call Center

  Pfizer

  STUDY DIRECTOR

Study Design

• Study Type: observational
• Observational Model: CASE ONLY
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 31, 2020
• First Posted: February 5, 2020
• Study Start: February 19, 2020
• Primary Completion: August 19, 2020
• Study Completion: August 19, 2020
• Last Updated: October 15, 2021

Record last verified: 2021-10

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)