This study is currently not recruiting participants.

Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of SAR442501 in Pediatric Participants With Achondroplasia

Research Study on Safety and Efficacy of Medication in Children with Achondroplasia

Not Recruiting
12 years or below
All
Phase 2
16 participants needed

Study Overview

This is a Phase 2, open-label, multicenter, study to evaluate safety, tolerability and efficacy of SAR442501 in children from birth up to 12 years of age with Achondroplasia.

Study Details

Up to approximately 275 weeks: 3 weeks Screening + 52 weeks primary treatment period + up to approximately 216 weeks extended treatment period+ 4 weeks follow-up.

Eligibility Criteria

You may be eligible for this study if you meet the following criteria:

  • Conditions: Osteochondrodysplasia
  • Age: 12 years or below
  • Gender: All

Inclusion Criteria:

  • Participants must have ACH with a confirmed mutation in the FGFR3 gene
  • Participants and/or parent(s) or legal representative(s) must be willing and able to perform all the study procedures to the best of their physical ability.
  • Parent(s) or legal representative(s) capable of giving signed informed consent and participants capable of giving assent when applicable.

Exclusion Criteria:

  • Have hypochondroplasia (or the N540K mutation) or short stature condition other than ACH (eg, trisomy 21, pseudochondroplasia)
  • Participants have received any dose of medications or investigational product, including human growth hormone, IGF-1, intended to affect participants' stature or body proportions between the completion of OBS16647 and enrollment (Week 0/Day 1/Visit 2).
  • Have a history of growth plate closure.
  • Long bone fracture within 3 months of enrollment (Week 0/Day 1/Visit 2)
  • Current evidence of corneal or retinal disorder/keratopathy.
  • Participants have had a previous surgical intervention involving the foramen magnum (Stage 2 only).
  • Hyperphosphatemia.

The above information is not intended to contain all considerations relevant to a potential participation in a clinical trial.

This study investigates achondroplasia in children. The purpose is to evaluate an investigational medication for its effects on this condition.

Researchers are assessing the safety and effectiveness of the investigational medication. The study also looks at how the medication behaves in the body and its overall impact on health.

  • Who can participate: Children from birth to 12 years with a confirmed FGFR3 gene mutation can participate.
  • Study details: Participants will receive the investigational medication during the study. Some may receive a placebo, which is an inactive substance that looks like the investigational medicine but does not contain any medicine.
  • Study timelines: The study will last up to approximately 275 weeks.
Updated on 02 Jul 2026. Study ID: NCT06067425