RecruitingRare disease

Study on Long-Term Use of Infigratinib 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 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.

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, 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.
Step 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.
Step 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.
Step 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.
Step 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 trial?

9 criteria

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 trial?

9 criteria

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.

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Verified sites

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

Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico
Verified
Milan, Italy
Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Verified
Rome, Italy
Fundacion Para La Investigacion Biomedica Del Hospital Universitario La Paz
Responsive
Madrid, Spain

Trial locations

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Investigated drugs

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.

Infigratinib

What is already known about the treatment

Infigratinib – Infigratinib is administered orally and is currently being studied in clinical trials for its use in treating achondroplasia, a condition characterized by abnormal bone growth. It is a selective inhibitor of FGFR 1-3 tyrosine kinases, which are proteins involved in cell growth and division. By blocking these proteins, infigratinib aims to normalize bone growth in individuals with achondroplasia. It is classified as a tyrosine kinase inhibitor and is being evaluated for its safety, tolerability, and effectiveness in improving height outcomes in children with this condition.

Investigated diseases

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.