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Safety, tolerability, pharmacokinetics, pharmacodynamics, and efficacy of SAR442501 in pediatric participants with Achondroplasia
EU CTIS ID: 2023-503677-37-00
What this study is testing
To assess the safety and tolerability of SAR442501 administered subcutaneously (SC) in participants with achondroplasia (ACH)
- Therapeutic exploratory (Phase II)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- Participants must have ACH with a confirmed mutation in the FGFR3 gene
- Participants and/or parent(s) or legal representative must be willing and able to perform all the study procedures to the best of their physical ability.
- Parent(s) or legal representative capable of giving signed informed consent and participants capable of giving assent when applicable.
You likely can't join if
- 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).
See the full eligibility criteria
- Participants must have ACH with a confirmed mutation in the FGFR3 gene
- Participants and/or parent(s) or legal representative must be willing and able to perform all the study procedures to the best of their physical ability.
- Parent(s) or legal representative capable of giving signed informed consent and participants capable of giving assent when applicable.
- 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 study team makes the final eligibility decision.
Where it's taking place
- Australia
- Korea, Republic of
- United States
- United Kingdom
- China
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 0-17 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Australia; Korea, Republic of; United States; United Kingdom; China. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.