Study on Long-Term Use of Infigratinib for Children with Achondroplasia

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What is this study about?

This clinical trial is focused on studying the effects of a medication called Infigratinib in children with a condition known as Achondroplasia. Achondroplasia is a genetic disorder that affects bone growth, leading to shorter stature and other physical differences. The medication being tested, Infigratinib, is taken orally in the form of capsules or tablets. It is a type of drug known as a tyrosine kinase inhibitor, which works by targeting specific proteins involved in cell growth.

The purpose of this study is to evaluate the long-term safety and effectiveness of Infigratinib in children with Achondroplasia. Participants in the study will receive daily doses of the medication over an extended period. The study will monitor changes in the children’s growth, specifically looking at their height over time, as well as any potential side effects that may occur. The study aims to understand how Infigratinib can help improve growth and overall health in children with this condition.

Throughout the study, various aspects of the children’s health will be assessed, including their body proportions, weight, and any changes in their physical abilities. The study will also look at the impact of the medication on the children’s quality of life, including their ability to perform daily activities and any changes in their cognitive functions. The study is designed to provide valuable information on the potential benefits and risks of using Infigratinib for treating Achondroplasia in children.

1 joining the study

Upon joining the study, the participant must meet specific criteria, including being a child with achondroplasia who has completed a previous study with infigratinib or is treatment-naïve.

The participant must be able to swallow oral medication and, if applicable, have a negative pregnancy test. Consent from parents or guardians is required.

2 medication administration

The participant will take infigratinib orally. The medication is available in capsule or tablet form.

The dosage and frequency will be determined by the study protocol, focusing on daily administration.

3 monitoring and assessments

Throughout the study, the participant’s health and growth will be monitored. This includes regular assessments of height, weight, and body proportions.

Additional evaluations may include the number of episodes of otitis media, sleep apnea, and changes in skeletal abnormalities.

4 evaluation of quality of life

The study will assess changes in health-related quality of life using specific questionnaires designed for children with short stature.

Functional abilities and cognitive functions will also be evaluated through various tests and interviews.

5 completion of the study

The study is expected to continue until January 2029. Upon completion, the participant’s overall experience and any changes in their condition will be reviewed.

The study aims to provide insights into the long-term safety and effectiveness of infigratinib for children with achondroplasia.

Who Can Join the Study?

For those who have participated in a previous study with the medication, they must have completed that study.
Participants and their parents, legal guardians, or caregivers must be willing and able to attend study visits and follow study procedures.
Participants must be able to swallow pills.
Girls aged 10 and older, or any girl who has started menstruating, must have a negative pregnancy test.
If participants are sexually active, they must agree to use a very effective form of birth control while taking the study medication and for one month after the last dose.
Written permission must be obtained from the participant’s parents, legal guardians, or caregivers, and the participant must agree to participate if applicable, before any study activities begin.
For those who have not participated in a previous study, they must be between 3 and under 18 years old and still have the potential to grow.
Participants must have a confirmed diagnosis of Achondroplasia (ACH), which is a genetic condition affecting bone growth.
Participants must have had their growth measured for at least six months in a specific study before joining this one.

Who Cannot Join the Study?

Patients who have any other serious health conditions that could interfere with the study.
Patients who are currently taking other medications that might affect the study results.
Patients who have had a recent surgery or medical procedure that could impact their participation.
Patients who are pregnant or breastfeeding.
Patients who have a history of allergic reactions to similar medications.
Patients who are unable to follow the study procedures or instructions.
Patients who have participated in another clinical trial recently.
Patients who have a history of substance abuse or addiction.
Patients who have any condition that the study doctors believe would make it unsafe for them to participate.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name	City	Country
Fondazione Policlinico Universitario Agostino Gemelli IRCCS	Rome	Italy
Oslo Universitetssykehus HF	Oslo	Norway
Hopital Necker Enfants Malades	Paris	France

Other Sites

Site Name	City	Country
Hopital Des Enfants	Toulouse	France
Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico	Milan	Italy
Hospital Femme Mere Enfant	Bron	France
Hospital Universitario Virgen De La Victoria	Malaga	Spain
Hnfww Bjwvry Ht	Bergen	Norway
Fcqvazqra Pucs Lf Ihuezktcfophr Bjpadcxfm Dbi Hsqrngpi Ubejfptqmwxxq Le Pwh	Madrid	Spain

Trial status

Country	Status	Recruitment Start
France	Recruiting	08.02.2022
Italy	Recruiting	08.02.2022
Norway	Recruiting	08.02.2022
Spain	Recruiting	08.02.2022

Trial locations

Investigated drugs:

Infigratinib

Infigratinib is a medication being studied for its effects on children with achondroplasia, a condition that affects bone growth. It works by selectively inhibiting certain proteins known as FGFR 1-3, which are involved in the development and maintenance of bone tissue. The trial aims to assess the safety and effectiveness of taking infigratinib over a long period, focusing on how it impacts the growth in height of the participants.

Achondroplasia – Achondroplasia is a genetic disorder that affects bone growth, leading to dwarfism. It is characterized by short stature with disproportionately short arms and legs, a large head, and distinctive facial features. The condition is caused by a mutation in the FGFR3 gene, which affects the conversion of cartilage to bone, particularly in the long bones. As individuals with achondroplasia grow, they may experience a range of physical challenges, including limited range of motion at the elbows, recurrent ear infections, and potential breathing difficulties during sleep. The condition is present from birth and continues to affect growth and development throughout childhood and into adulthood. Despite these challenges, individuals with achondroplasia can lead full and active lives.

Trial ID:

2024-513857-55-00

Protocol code:

QBGJ398-203

NCT ID:

NCT05145010

Trial Phase:

Therapeutic exploratory (Phase II)

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Study on Long-Term Use of Infigratinib for Children with Achondroplasia

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