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

Study of Investigational 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 the safety, tolerability, and effectiveness of an investigational medication in children with achondroplasia. This study is open-label, meaning both the researchers and participants know what treatment is being given, and it involves children from birth to 12 years old.

Participants in the study will undergo various procedures to assess how the investigational medication affects their condition. This includes monitoring how the medication is processed in the body and its impact on growth and development. Participants and their families will be required to follow the study procedures as best as they can.

  • Who can participate: Children from birth to 12 years old with achondroplasia and a confirmed mutation in the FGFR3 gene can participate. Participants must be able to perform study procedures, and consent must be given by parents or legal representatives.
  • Study details: Participants will take part in assessments to evaluate the investigational medication's effects on their condition. They must adhere to study procedures.
  • Study timelines: The study will last 275 weeks.
Updated on 04 Apr 2025. Study ID: NCT06067425