A Study to Learn About Recifercept in Patients With Achondroplasia
Study Details
Study Description
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.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
|
Study Design
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| Recifercept cohort Achondroplasia patients enrolled in the recifercept phase 2 clinical trial |
Other: Recifercept
Patients received recifercept intervention in the phase 2 clinical trial
|
| Natural history cohort Achondroplasia patients enrolled in the achondroplasia natural history study |
Outcome Measures
Primary Outcome Measures
- Height growth [6 months]
Increase in height growth above expected growth in reference population, defined as the height growth in the achondroplasia reference population +50%
Secondary Outcome Measures
- Standing height [6 months]
standing height in centimeters
- Sitting height [6 months]
sitting height in centimeters
- Knee height [6 months]
knee height in centimeters
- Arm span [6 months]
arm span in centimeters
- Length of the legs [6 months]
length of the legs in centimeters
- Sitting height to standing height ratio [6 months]
sitting height to standing height ratio (unitless)
- arm span to height or length difference [6 months]
arm span to height or length difference in centimeters
- knee height to low segment ratio [6 months]
knee height to low segment ratio (unitless)
- Achondroplasia-related orthopaedic complications [6 months]
lordosis, kyphoscoliosis, small foramen magnum, spinal stenosis
- Other chondroplasia-related complications [6 months]
sleep disordered breathing, acute otitis media, hydrocephalus
Eligibility Criteria
Criteria
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)
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Aged ≥ 3 months to <11 years (up to the day before 11th birthday inclusive) at time of enrollment into the observational natural history study.
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Havecompleted at least 2 valid height/length measurements (at least 3 months apart)
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Assessed for Tanner stage 1 during physical examination before or at enrollment (must include assessment of breast development for females, testicular stage for males)
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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.
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Have at least 6 months of available follow-up data after enrollment into the natural history study
Exclusion Criteria:
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Patients meeting any of the following criteria will not be included in the study:
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Presence of severe obesity (BMI>95% percentile on Hoover-Fong BMI charts);
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Body weight <7kg or >30kg
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History of chronic kidney disease (CKD) or renal impairment
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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.
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Less than 6 months since fracture or surgical procedure of any bone determined from the baseline visit date.
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Presence of any internal guided growth plates/devices
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History of removal of internal guided growth plates/devices within 6 months prior to enrollment
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | Pfizer | New York | New York | United States | 10017 |
Sponsors and Collaborators
- Pfizer
Investigators
- Study Director: Pfizer CT.gov Call Center, Pfizer
Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- C4181010