A Study Of Safety, Tolerability And Effectiveness Of Recifercept In Children With Achondroplasia
Public ClinicalTrials.gov record NCT04638153. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
A PHASE 2 MULTIPLE DOSE, RANDOMIZED STUDY TO ASSESS THE SAFETY, TOLERABILITY, PHARMACOKINETICS AND EFFICACY OF RECIFERCEPT IN CHILDREN WITH ACHONDROPLASIA
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
Approximately 63 participants will be randomized to one of three doses to receive Recifercept either * Low Dose * Medium Dose * High Dose Participants will will attend the clinic at baseline and at Day 1, 4, 8, 15, 29 \& then Month 2, 3 6, 9 \& 12. Assessments include safety, blood sampling, physical examination, vital signs, anthropometric body measurements \& patient/caregiver quality of life questionnaires Participants will received treatment with Recifercept for 12 months. All participants who complete the study and in the opinion of the investigator, continue to have a positive risk:benefit profile, will be offered to enroll into an open-label extension (OLE) study. A PK cohort will include 12 participants who will randomly receive a single dose of 3 mg/kg of Phase 2 study (process 1c) formulation and a single dose of 3 mg/kg of the proposed Phase 3 (process 2) study formulation in a cross over study. Dose of the cohort could be changed due to emerging safety and efficacy data in the study.
Study identification
- NCT ID
- NCT04638153
- Recruitment status
- Terminated
- Study type
- Interventional
- Phase
- Phase 2
- Enrollment
- 60 participants
Conditions and interventions
Conditions
Eligibility (public fields only)
- Age range
- 3 Months to 10 Years
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Dec 1, 2020
- Primary completion
- Jan 15, 2023
- Completion
- Mar 26, 2023
- Last update posted
- Feb 14, 2024
2020 – 2023
United States locations
- U.S. sites
- 6
- U.S. states
- 3
- U.S. cities
- 6
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Ocean Sleep Medicine | Aliso Viejo | California | 92656 | — |
| Ocean Sleep Medicine | Irvine | California | 92604 | — |
| MemorialCare Sleep Disorders Center at Long Beach Memorial Medical Center | Long Beach | California | 90806 | — |
| Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center | Torrance | California | 90502 | — |
| Nemours Alfred I duPont Hospital for Children | Wilmington | Delaware | 19803 | — |
| Texas Children's Hospital | Houston | Texas | 77030 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
Non-U.S. locations
This page focuses on the U.S. directory. The official record also lists 10 non-U.S. sites.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT04638153, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Feb 14, 2024 · Synced Sep 6, 2026
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Open the official record
The complete protocol, eligibility criteria, and contact information for NCT04638153 live on ClinicalTrials.gov.