N/A
N=248
A Study to Learn About Recifercept in Patients With Achondroplasia
Achondroplasia
Bottom Line
View on ClinicalTrials.gov: NCT05659719 ↗Enrolled (actual)
248
Serious AEs
—
Results posted
Sep 2024
Primary outcome: Primary: Mean Height Growth at Month 3 — 1.11; 0.84; 0.75; 1.07 Ratio
Study Design & Population
- Study type
- Observational
- Phase
- N/A
- Interventions
- Recifercept (Other)
- Age
- Pediatric · 0+ yrs
- Sex
- All
- Sponsor
- Pfizer
- Primary completion
- May 2023
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Mean Height Growth at Month 3 |
1.11; 0.84; 0.75; 1.07 | — |
| PRIMARY Mean Height Growth at Month 6 |
0.94; 1.06; 1.01; 1.11 | — |
| PRIMARY Mean Height Growth at Month 9 |
1.01; 0.97; 1.03; 1.10 | — |
| PRIMARY Mean Height Growth at Month 12 |
0.94; 1.06; 0.84; 1.10 | — |
| SECONDARY Mean Change From Baseline in Arm Span to Height/Length Difference at Months 3, 6, 9 and 12 |
0.46; 0.02; -0.61; 0.09; 0.19; 0.72 | — |
| SECONDARY Mean Change From Baseline in Knee Height: Lower Segment Ratio at Months 3, 6, 9 and 12 |
-0.00; -0.01; 0.02; -0.09; -0.00; -0.02 | — |
| SECONDARY Mean Change From Baseline in Height Standard Deviation Score (Z-Score) at Months 3, 6, 9, 12 |
0.03; -0.04; -0.02; 0.03; -0.03; 0.03 | — |
| SECONDARY Number of Participants With Achondroplasia Related Orthopedic Complications |
0; 0; 0; 79; 0; 0 | — |
| SECONDARY Number of Participants With Other Achondroplasia Related Orthopedic Complications |
3; 1; 1; 0; 0; 1 | — |
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.
Eligibility 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)
- Aged ≥ 3 months to 95% percentile on Hoover-Fong BMI charts);
- Body weight 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
Data sourced from ClinicalTrials.gov (NCT05659719). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.