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Study on the Effectiveness and Safety of Infigratinib for Children with Hypochondroplasia and Short Stature

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What is this trial about?

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

This clinical study is investigating the effects of infigratinib in children with hypochondroplasia. Hypochondroplasia is a genetic disorder that causes short stature and disproportionate body growth. The study will examine whether infigratinib can help children with this condition who have short stature but still have growth potential.

The purpose of this research is to evaluate how effective and safe infigratinib is for treating children with hypochondroplasia. The study has two parts - an initial open-label phase followed by a double-blind, randomized, placebo-controlled phase. During the study, researchers will measure changes in height velocity (how quickly children grow), height in relation to others with the same condition, and body proportions.

Children participating in the study will need to take oral medication and attend scheduled study visits. The study includes children of different age groups (from 3 to under 18 years old) and requires that participants have already been part of an observational study for at least 26 weeks prior to starting this treatment study.

The research process

The trial runs in 6 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Beginning of the Study

    After joining the study, you will start either in the open-label phase (where everyone receives the active medication) or proceed to the double-blind phase (where you might receive either the active medication or a placebo).

    This clinical trial is designed for children with hypochondroplasia (HCH), a condition that causes disproportionate short stature.

  2. Step 2

    Medication Details

    During the study, you will receive either infigratinib capsules or placebo capsules (inactive medication that looks the same as the active drug).

    You will need to be able to swallow the capsules as part of the treatment.

    The study team will provide specific instructions on when and how to take the medication.

  3. Step 3

    Regular Measurements and Assessments

    Throughout the study, your height will be measured regularly to track your height velocity (how quickly you grow in cm/year).

    Doctors will also measure your upper to lower body segment ratio to assess body proportions.

    These measurements will help determine if the medication is effective in improving growth patterns.

  4. Step 4

    Safety Monitoring

    Regular check-ups will be conducted to monitor your health and any potential side effects from the medication.

    For girls who are 10 years or older, or who have begun menstruation, pregnancy tests will be performed as part of safety monitoring.

  5. Step 5

    Duration and Follow-up

    The primary evaluation period is 52 weeks (one year), during which the change in your annualized height velocity (AHV) will be measured.

    The study includes regular visits to the study center for assessments and monitoring.

    You must remain ambulatory (able to walk without assistance) throughout the study period.

  6. Step 6

    Study Completion

    After completing the treatment period, there may be follow-up visits to monitor your long-term health and development.

    The study is expected to continue until 2025, with possible long-term follow-up until 2028.

Who can join the trial?

10 criteria

  • You must have been participating in the observational study (QBGJ398-004) for at least 26 weeks and still be enrolled when screening for this study begins.
  • Girls who are 10 years or older, or girls of any age who have started their periods (menarche), must have a negative pregnancy test.
  • If you are sexually active, regardless of gender, you must be willing to use highly effective birth control while taking the study medication and for 1 month after your last dose.
  • You and your parent/guardian must sign an informed consent form.
  • For Phase 2 of the study: You must be between 5-11 years of age (inclusive).
  • You must have a diagnosis of hypochondroplasia (a genetic condition causing short stature) confirmed both by clinical examination and a molecular test.
  • For Phase 2 portion: Your annualized height velocity (rate of growth per year) must be greater than a specific measurement over a period of at least 26 weeks before screening, based on measurements from the observational study.
  • You must be able to swallow oral medication.
  • You and your parent(s), guardian(s), or caregiver(s) must be willing and able to attend all study visits and complete all study procedures.
  • You must be able to walk and stand without assistance.

Who cannot join the trial?

14 criteria

  • Growth plates that are fused or almost fused (which means your bones are no longer growing in length).
  • Disorders affecting bone growth other than hypochondroplasia.
  • History of growth hormone therapy within 6 months before starting the study.
  • Current use of medications that might affect growth.
  • Severe kidney problems or impaired kidney function.
  • Significant liver disease or abnormal liver function.
  • History of eye problems such as retinal detachment (when the light-sensitive tissue at the back of the eye pulls away from its normal position).
  • Heart rhythm problems or other significant heart conditions.
  • Current or recent participation in another clinical trial.
  • Pregnancy, breastfeeding, or planning to become pregnant during the study period.
  • Unable to swallow pills or take the study medication as required.
  • Known allergies to the study drug or similar medications.
  • History of certain cancers or currently having cancer.
  • Certain genetic mutations that would interfere with the study results.
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Investigated drugs

Infigratinib is a medication being studied for children with hypochondroplasia (HCH), a genetic condition that causes short stature. It targets the underlying cause of the condition by blocking a specific growth pathway that is overactive in hypochondroplasia. The study aims to see if this medication can help improve growth rates in children with this condition.

What is already known about the treatment

Infigratinib – An oral medication administered as tablets or capsules, currently under investigation in Phase 2/3 clinical trials for treating hypochondroplasia in children with short stature. Infigratinib belongs to the class of fibroblast growth factor receptor (FGFR) inhibitors and works by selectively blocking FGFR3, which is overactive in hypochondroplasia, potentially allowing for improved bone growth and development. While already approved for certain cancers, its use in skeletal growth disorders represents a novel application that aims to address the underlying genetic cause of hypochondroplasia rather than just managing symptoms.

Investigated diseases

Hypochondroplasia is a genetic skeletal disorder characterized by short stature and disproportionately short arms and legs. It is caused by mutations in the fibroblast growth factor receptor 3 (FGFR3) gene, the same gene affected in achondroplasia, though hypochondroplasia typically presents with milder symptoms. Children with hypochondroplasia typically have normal trunk length but shortened limbs, with the distal segments (forearms and lower legs) more affected than the proximal segments. The condition progresses gradually during childhood growth, resulting in adult heights typically between 4'8" and 5'4" (142-162 cm). Individuals may also experience mild lordosis (curvature of the lower spine), bowed legs, and limited elbow extension. Hypochondroplasia does not typically affect intelligence or life expectancy.
Trial detailsLast updated 2 Oct 2026
Age0-17PhasePhase IVTrial ID2024-516822-67-00Protocol codeQBGJ398-304Estimated enrolment139 patientsSponsorQed Therapeutics Inc.

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On this site, “treatment” means an investigational medicine being studied in a clinical trial. Its safety and efficacy for the use being studied have not yet been confirmed, some participants may receive a placebo or a comparator medicine, and taking part does not guarantee any health benefit. The decision to take part is made by the doctor at the research site. This site is for information only and does not replace medical advice.

This service is not affiliated with the European Commission, the EMA, or the official CTIS system. Most information comes from publicly available international clinical-trial registries, supplemented by data from academic sites, national regulators and commercial sponsors. On this site, “treatment” and “therapy” mean a medicine being tested in a clinical trial. Its safety and effectiveness in the use being studied are not yet confirmed, some participants may receive a placebo or a comparator, and taking part does not guarantee a health benefit. The doctor at the research site decides who can take part. This site provides information, not medical advice. Certain content and visual elements on this website have been generated or enhanced using artificial intelligence (AI).