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.
Who can participate
Age range0 Days – 12 Years
SexALL
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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
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.
What they're measuring
1
Number of participants with adverse events (AE), serious adverse events (SAE), and adverse events of special interest (AESI) during the treatment-emergent period