Not recruitingRare disease

Study on the Effectiveness and Safety of Infigratinib for Children Aged 3 to 17 with Achondroplasia

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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 a condition called Achondroplasia, which is a genetic disorder affecting bone growth and resulting in shorter stature. The study is investigating a treatment called Infigratinib, which is a type of medication known as a tyrosine kinase inhibitor. This medication works by blocking certain proteins that can affect bone growth. In this study, Infigratinib is given in the form of capsules that are taken orally.

The purpose of the study is to evaluate how effective and safe Infigratinib is for children aged 3 to under 18 years who have Achondroplasia and still have the potential to grow. The study will compare the effects of Infigratinib to a placebo, which is a substance with no active medication. Participants will be randomly assigned to receive either Infigratinib or the placebo, and neither the participants nor the researchers will know who is receiving which treatment. This is known as a double-blind study. The treatment period will last up to 52 weeks, during which various measurements related to growth and development will be taken to assess the medication's impact.

Throughout the study, researchers will monitor changes in growth rates, body proportions, and other health indicators to determine the effectiveness of Infigratinib. The study will also track any side effects or adverse events to ensure the safety of the participants. By the end of the study, the goal is to gather enough information to understand how Infigratinib can help children with Achondroplasia grow and develop more effectively.

The research process

The trial runs in 4 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 is required to meet specific criteria, including being between 3 to less than 18 years of age, having growth potential, and being able to swallow oral medication.
The participant must have completed at least 26 weeks in a previous study and must not have any contraindications to the licensed treatment option.
Step 2
Medication administration
The participant will receive infigratinib in the form of oral capsules. The dosage and frequency will be determined by the study protocol.
The medication is administered to evaluate its effectiveness and safety in children with achondroplasia.
Step 3
Monitoring and assessments
Throughout the study, the participant's growth and development will be monitored. This includes measuring changes in height, body proportions, and other physical parameters.
Regular assessments will be conducted to evaluate the participant's response to the medication and to monitor for any side effects.
Step 4
Completion of the study
The study is designed to last until April 2026, with the primary endpoint being the change in annual height velocity after 52 weeks.
Upon completion, the data collected will be analyzed to determine the efficacy and safety of infigratinib compared to a placebo.

Who can join the trial?

10 criteria

The child must be between 3 and less than 18 years old and still have the potential to grow. This means they should grow more than 1.5 cm in a year, be in a certain stage of puberty (Tanner stage 4 or less), and have a bone age of 13 years or less for girls and 15 years or less for boys.
The usual treatment option, called vosoritide, should not be suitable for the child. This could be because the doctor thinks it's not safe, it's not available, or the family cannot or does not want to use it.
The child must have a confirmed diagnosis of **Achondroplasia** through clinical examination and genetic testing.
The child must have completed at least 26 weeks in a previous study called PROPEL (QBGJ398-001) before joining this study.
The child must be able to swallow pills.
The child and their parents, legal guardians, or caregivers must be willing and able to attend study visits and follow study procedures.
The child must be able to walk and stand without help.
Girls aged 10 and older, or any girl who has started menstruating, must have a negative pregnancy test.
If the child is sexually active, they must agree to use a highly effective method of birth control while taking the study drug and for 3 months after the last dose.
Parents or legal guardians must sign a consent form agreeing to the study's requirements and restrictions. The child must also sign if they are able to understand and agree.

Who cannot join the trial?

3 criteria

Patients who are not diagnosed with Achondroplasia, a condition affecting bone growth, cannot participate.
Patients who are not between the ages of 3 and under 18 years old cannot participate.
Patients who do not have the potential to grow cannot participate.

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Verified sites

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Centre Hospitalier Lyon Sud
Verified
Pierre Benite, France
Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Verified
Rome, Italy
Fundacion Para La Investigacion Biomedica Del Hospital Universitario La Paz
Responsive
Madrid, Spain

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Investigated drugs

Infigratinib is a medication being studied for its potential to help children with achondroplasia, a condition that affects bone growth. The trial aims to see if infigratinib can improve growth in children aged 3 to under 18 years who have this condition.

Infigratinib

What is already known about the treatment

Infigratinib – Infigratinib is administered orally and is currently being studied in clinical trials for its potential use in treating achondroplasia, a condition characterized by abnormal bone growth. The medication is in the investigational stage, with ongoing research to assess its safety and effectiveness in children. It works by inhibiting specific cellular pathways that affect bone growth, aiming to promote normal development. Infigratinib is classified as a tyrosine kinase inhibitor, targeting molecular mechanisms involved in growth regulation.

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, bowed legs, and a sway of the lower back. Despite these challenges, individuals with achondroplasia typically have normal intelligence and life expectancy. The progression of the condition is generally stable after childhood, with most growth occurring in the first few years of life.