A Study to Learn About Recifercept in Patients With Achondroplasia

Pfizer

Status

Completed

Conditions

Achondroplasia

Treatments

Other: Recifercept

Study type

Observational

Funder types

Industry

Identifiers

NCT05659719

C4181010

Details and patient eligibility

About

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.

Enrollment

248 patients

Sex

All

Ages

3 months to 10 years old

Volunteers

No Healthy Volunteers

Inclusion criteria

All patients from Study C4181005 who have completed Visits 1 through 11 (at D183) will be included in this project.

To construct a concurrent external control, patients from Study C4181001 will need to meet the following inclusion criteria from Study C4181005 to be eligible for inclusion:

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