Not recruitingRare disease

Study on the Safety and Effects of SAR442501 for Children with Achondroplasia

Verified siteInvestigationalNo placebo

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying Achondroplasia, a condition that affects bone growth, leading to shorter stature. The study will use a treatment called SAR442501, which is a special type of protein known as a humanised monoclonal antibody derivative against fibroblast growth factor receptor 3. This treatment is given as a powder that is mixed into a solution and then injected under the skin.

The purpose of the study is to understand how safe and tolerable SAR442501 is for children with Achondroplasia. Participants will receive the treatment through subcutaneous injections, which means the medicine is injected just under the skin. The study will take place over a period of time, and participants will be monitored for any side effects or changes in their condition. The study will also look at how the treatment affects growth and development in children with Achondroplasia.

Throughout the study, researchers will collect information on various aspects of the participants' health, including growth rates, body proportions, and overall quality of life. This information will help determine how the treatment works in the body and its potential benefits for children with Achondroplasia. The study aims to provide valuable insights into the treatment's effectiveness and safety, contributing to better care for those with this condition.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

Step 1
Joining the study
Upon joining the study, participants must have achondroplasia with a confirmed mutation in the FGFR3 gene.
Participants and their parent(s) or legal representative must be willing and able to perform all study procedures to the best of their physical ability.
Step 2
Treatment administration
The treatment involves the administration of a humanised monoclonal antibody derivative against fibroblast growth factor receptor 3.
The medication is provided as a powder for solution for injection and is administered via subcutaneous injection.
Step 3
Monitoring and assessment
Participants will be monitored for any adverse events, including serious adverse events and adverse events of special interest during the treatment-emergent period.
Various changes will be assessed, such as growth velocity, body segment ratios, and overall health-related quality of life.
Step 4
Pharmacokinetics and pharmacodynamics
The study will assess pharmacokinetic parameters, including plasma concentration of SAR442501, maximum plasma concentration, and time to reach maximum concentration.
Pharmacodynamic parameters will also be evaluated, such as changes in collagen X biomarker levels and other bone-related markers.
Step 5
Completion of the study
The estimated end date for the study is September 1, 2027.
Participants will complete all required assessments and procedures as outlined in the study protocol.

Who can join the trial?

5 criteria

Participants must have **achondroplasia (ACH)**, which is a condition affecting bone growth, with a confirmed change in the **FGFR3 gene**, which is a specific gene related to this condition.
Participants and/or their 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 must be capable of giving signed informed consent, which means they agree to the participant joining the study after understanding all the details. Participants must be capable of giving assent, which means they agree to participate when applicable.
Participants can be of any gender, meaning both males and females are eligible.
Participants must be at least 2 years old.

Who cannot join the trial?

1 criterion

Participants with a condition called osteochondrodysplasia, which affects bone and cartilage development, cannot take part in the study.

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Verified sites

All sites with verified contact details – recruitment status may not be available; ask directly

Fakultni Nemocnice Brno
Verified
Brno, Czechia
Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico
Verified
Milan, Italy
Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Verified
Rome, Italy

Trial locations

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Investigated drugs

SAR442501 is a medication being studied for its potential to treat achondroplasia, a condition characterized by abnormal bone growth leading to short stature. In this clinical trial, SAR442501 is administered through an injection under the skin. The study aims to evaluate how safe and tolerable this medication is for children with achondroplasia, as well as to understand how the body processes the drug and its effects on the condition.

What is already known about the treatment

SAR442501 – This medication is administered through subcutaneous injection, meaning it is injected under the skin. It is currently being studied in clinical trials, specifically in a Phase 2 study, to evaluate its safety and effectiveness in treating achondroplasia, a form of osteochondrodysplasia. The main therapeutic indication for SAR442501 is to address growth issues associated with achondroplasia. At the molecular level, it works by targeting specific pathways that influence bone growth and development. SAR442501 is classified pharmacologically as a growth modulator, aiming to improve bone growth in affected individuals.

Investigated diseases

Osteochondrodysplasia – This is a group of disorders characterized by abnormal development of bone and cartilage. These conditions often result in disproportionate short stature and can affect the shape and size of bones throughout the body. The progression of osteochondrodysplasia varies depending on the specific type, but it generally involves issues with bone growth and joint function. Individuals may experience joint pain, limited mobility, and skeletal deformities as they grow. The condition is typically present from birth and can be identified through physical characteristics and growth patterns.