A Study to Learn About Recifercept in Patients With Achondroplasia

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

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Basic Information

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Recruitment Status

COMPLETED

Total Enrollment

248 participants

Study Classification

OBSERVATIONAL

Study Start Date

2022-10-19

Study Completion Date

2023-05-16

Brief Summary

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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.

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Detailed Description

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Conditions

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Achondroplasia

Study Design

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Observational Model Type

COHORT

Study Time Perspective

RETROSPECTIVE

Study Groups

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Recifercept cohort

Achondroplasia patients enrolled in the recifercept phase 2 clinical trial

Recifercept

Intervention Type OTHER

Patients received recifercept intervention in the phase 2 clinical trial

Natural history cohort

Achondroplasia patients enrolled in the achondroplasia natural history study

No interventions assigned to this group

Interventions

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Recifercept

Patients received recifercept intervention in the phase 2 clinical trial

Intervention Type OTHER

Eligibility Criteria

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Inclusion Criteria

* 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

Exclusion Criteria

* 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

Minimum Eligible Age

3 Months

Maximum Eligible Age

10 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No