A Study to Learn About Recifercept in Patients With Achondroplasia

NCT05659719 | Completed Results

• 1 other identifier: C4181010
• Study Type: observational
• Target: 248
• Locations: 1 country
• Sites: 1

Brief Summary

The purpose of this study is to learn about the study medicine (called recifercept) in people with achondroplasia. Achondroplasia is a very rare disease and patients of achondroplasia have short arms and legs. The study will include data already collected from a recifercept clinical trial and data collected from a separate study of achondroplasia. This study will compare patient experiences and will help the investigators determine if the study medicine, recifercept, is effective.

Conditions

Achondroplasia

Outcome Measures

Primary Outcomes (4)

• Mean Height Growth at Month 3

  Height growth was defined as ratio of observed change from baseline in standing height (centimeter) to expected change from baseline (centimeter) in reference population. For C4181001 cohort, baseline value was defined as the value recorded at the date of informed consent, whereas for C4181005 cohort the baseline value was the value taken at Day 1 before dosing.

  Baseline, Month 3 [Eligible data extracted and observed in this retrospective cohort study from 19-October-2022 to 16-May-2023 (approximately 7 months)]

• Mean Height Growth at Month 6

  Height growth was defined as ratio of observed change from baseline in standing height (centimeter) to expected change from baseline (centimeter) in reference population. For C4181001 cohort, baseline value was defined as the value recorded at the date of informed consent, whereas for C4181005 cohort the baseline value was the value taken at Day 1 before dosing.

  Baseline, Month 6 [Eligible data extracted and observed in this retrospective cohort study from 19-October-2022 to 16-May-2023 (approximately 7 months)]

• Mean Height Growth at Month 9

  Height growth was defined as ratio of observed change from baseline in standing height (centimeter) to expected change from baseline (centimeter) in reference population. For C4181001 cohort, baseline value was defined as the value recorded at the date of informed consent, whereas for C4181005 cohort the baseline value was the value taken at Day 1 before dosing.

  Baseline, Month 9 [Eligible data extracted and observed in this retrospective cohort study from 19-October-2022 to 16-May-2023 (approximately 7 months)]

• Mean Height Growth at Month 12

  Height growth was defined as ratio of observed change from baseline in standing height (centimeter) to expected change from baseline (centimeter) in reference population. For C4181001 cohort, baseline value was defined as the value recorded at the date of informed consent, whereas for C4181005 cohort the baseline value was the value taken at Day 1 before dosing.

  Baseline, Month 12 [Eligible data extracted and observed in this retrospective cohort study from 19-October-2022 to 16-May-2023 (approximately 7 months)]

Secondary Outcomes (5)

• Mean Change From Baseline in Arm Span to Height/Length Difference at Months 3, 6, 9 and 12

  Baseline, Months 3, 6, 9, 12 [Eligible data extracted and observed in this retrospective cohort study from 19-October-2022 to 16-May-2023 (approximately 7 months)]

• Mean Change From Baseline in Knee Height: Lower Segment Ratio at Months 3, 6, 9 and 12

  Baseline, Months 3, 6, 9, 12 [Eligible data extracted and observed in this retrospective cohort study from 19-October-2022 to 16-May-2023 (approximately 7 months)]

• Mean Change From Baseline in Height Standard Deviation Score (Z-Score) at Months 3, 6, 9, 12

  Baseline, Months 3, 6, 9, 12 [Eligible data extracted and observed in this retrospective cohort study from 19-October-2022 to 16-May-2023 (approximately 7 months)]

• Number of Participants With Achondroplasia Related Orthopedic Complications

  C4181005 cohorts: 12 months; C4181001 cohort: 5 years [Eligible data extracted and observed in this retrospective cohort study from 19-October-2022 to 16-May-2023 (approximately 7 months)]

• Number of Participants With Other Achondroplasia Related Orthopedic Complications

  C4181005 cohorts: 12 months; C4181001 cohort: 5 years [Eligible data extracted and observed in this retrospective cohort study from 19-October-2022 to 16-May-2023 (approximately 7 months)]

Study Arms (2)

Recifercept cohort

Achondroplasia patients enrolled in the recifercept phase 2 clinical trial

Other: Recifercept

Natural history cohort

Achondroplasia patients enrolled in the achondroplasia natural history study

Interventions

Recifercept OTHER

Patients received recifercept intervention in the phase 2 clinical trial

Recifercept cohort

Eligibility Criteria

• Age: 3 Months - 10 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)
• Sampling Method: Probability Sample

Study Population

Achondroplasia patients, aged 3 months to 11 years old with documented and confirmed genetic diagnosis of achondroplasia, who have completed at least 2 valid height and length measurement in the natural history study and have at least 6 months of follow-up data in either the natural history study or the phase 2 recifercept trial.

You may qualify if:

• All patients from Study C4181005 who have completed Visits 1 through 11 (at D183) will be included in this project.
• Documented, confirmed genetic diagnosis of achondroplasia from historical medical records (test must have been performed at a laboratory fully accredited for genetic testing under local regulations)
• Aged ≥ 3 months to \<11 years (up to the day before 11th birthday inclusive) at time of enrollment into the observational natural history study.
• Havecompleted at least 2 valid height/length measurements (at least 3 months apart)
• Assessed for Tanner stage 1 during physical examination before or at enrollment (must include assessment of breast development for females, testicular stage for males)
• Able to stand independently for height measurements (if ≥ 2 years of age at enrollment); If aged \<2 years at enrollment, has a documented historical MRI brain/cervical spine performed in the previous 12 months.
• Have at least 6 months of available follow-up data after enrollment into the natural history study

You may not qualify if:

• Patients meeting any of the following criteria will not be included in the study:
• Presence of severe obesity (BMI\>95% percentile on Hoover-Fong BMI charts);
• Body weight \<7kg or \>30kg
• History of chronic kidney disease (CKD) or renal impairment
• History of receipt of any treatment that are known to potentially affect growth (including oral steroids \> 5 days in the last 6 months before enrollment, high dose inhaled corticosteroids (\>800 mcg/day beclametasone equivalent) and medication for attention deficient hyperactivity disorder.
• Less than 6 months since fracture or surgical procedure of any bone determined from the baseline visit date.
• Presence of any internal guided growth plates/devices
• History of removal of internal guided growth plates/devices within 6 months prior to enrollment

Sponsors & Collaborators

• Pfizer: lead

Study Sites (1)

Pfizer

New York, New York, 10017, United States

Location

Related Links

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

MeSH Terms

Conditions

Achondroplasia

Condition Hierarchy (Ancestors)

Dwarfism; Bone Diseases, Developmental; Bone Diseases; Musculoskeletal Diseases; Osteochondrodysplasias; 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: observational
• Observational Model: COHORT
• Time Perspective: RETROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 12, 2022
• First Posted: December 21, 2022
• Study Start: October 19, 2022
• Primary Completion: May 16, 2023
• Study Completion: May 16, 2023
• Last Updated: September 20, 2024
• Results First Posted: September 20, 2024

Record last verified: 2024-05

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)