Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of SAR442501 in Pediatric Participants With Achondroplasia (upreACH-2)

Sanofi

Status and phase: Terminated; Phase 2

Conditions: Osteochondrodysplasia

Treatments: Drug: SAR442501

Study type: Interventional

Funder types: Industry

Identifiers

NCT06067425

U1111-1280-5374 (Registry Identifier)

2023-503677-37 (Registry Identifier)

DRI16646

Details and patient eligibility

About

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.

Full description

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.

Enrollment: 16 patients

Sex: All

Ages: Under 12 years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Sequential Assignment

Masking: None (Open label)

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16 participants in 3 patient groups

Cohort 1

Experimental group

Treatment:

Drug: SAR442501

Cohort 2

Experimental group

Treatment:

Drug: SAR442501

Cohort 3

Experimental group

Treatment:

Drug: SAR442501