A Study to Learn About Recifercept in Patients With Achondroplasia (NCT05659719) | Clinical Trial Compass
CompletedNot Applicable
A Study to Learn About Recifercept in Patients With Achondroplasia
United States248 participantsStarted 2022-10-19
Plain-language 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.
Who can participate
Age range
3 Months – 10 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
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 …
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Mean Height Growth at Month 3
Timeframe: Baseline, Month 3 [Eligible data extracted and observed in this retrospective cohort study from 19-October-2022 to 16-May-2023 (approximately 7 months)]
2
Mean Height Growth at Month 6
Timeframe: Baseline, Month 6 [Eligible data extracted and observed in this retrospective cohort study from 19-October-2022 to 16-May-2023 (approximately 7 months)]
3
Mean Height Growth at Month 9
Timeframe: Baseline, Month 9 [Eligible data extracted and observed in this retrospective cohort study from 19-October-2022 to 16-May-2023 (approximately 7 months)]
4
Mean Height Growth at Month 12
Timeframe: Baseline, Month 12 [Eligible data extracted and observed in this retrospective cohort study from 19-October-2022 to 16-May-2023 (approximately 7 months)]