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

Investigating the Safety and Efficacy of an Investigational Medication for Achondroplasia in Children

Recruiting
12 years or below
All
Phase 2
36 participants needed
2 Locations

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.

Updated on 30 May 2024. Study ID: NCT06067425

This study investigates the safety and effectiveness of an investigational medication in children with achondroplasia, a genetic condition that affects bone growth. Achondroplasia is caused by a mutation in the FGFR3 gene, leading to shorter stature and other skeletal abnormalities. The purpose of this study is to evaluate how well the investigational medication works and how safe it is for children from birth up to 12 years old.

Participants in the study will receive the investigational medication and will be monitored for any side effects. They will undergo a series of medical tests and assessments to evaluate the medication's effects on their growth and development. This will help researchers understand the medication's impact on achondroplasia.

  • Who can participate: Children from birth up to 12 years old with achondroplasia and a confirmed FGFR3 gene mutation can participate. Participants must be able to perform study procedures, and their parents or guardians must provide consent.
  • Study details: Participants will receive the investigational medication and undergo medical tests to monitor their growth. A placebo is not used in this study, meaning all participants receive the active treatment.
  • Study timelines: The study will last 275 weeks.

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