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

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What is this study about?

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.

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.

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.

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.

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.

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 Study?

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 Study?

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

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name	City	Country	Status
Fondazione Policlinico Universitario Agostino Gemelli IRCCS	Rome	Italy

Other Sites

Site Name	City	Country
Vseobecna Fakultni Nemocnice V Praze	Prague	Czechia
Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico	Milan	Italy
Fakultni Nemocnice Brno	Brno	Czechia
Hospital Sant Joan De Deu Barcelona	Esplugues De Llobregat	Spain
Hospital San Jose	Vitoria	Spain

Trial status

Country	Status	Recruitment Start
Czechia	Not recruiting	01.07.2023
Italy	Not recruiting	01.07.2023
Spain	Not recruiting	01.07.2023

Trial locations

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.

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.

Trial ID:

2023-503677-37-00

Protocol code:

DRI16646

Trial Phase:

Therapeutic exploratory (Phase II)

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

What is this study about?

Who Can Join the Study?

Who Cannot Join the Study?