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Long‑Term Safety and Efficacy of Infigratinib in Children with Hypochondroplasia

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What is this trial about?

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

Hypochondroplasia is a rare condition that leads to short stature and proportionate body parts because of changes in genes that affect bone growth. The study examines a medicine called Infigratinib, which is a FGFR 1-3-Selective Tyrosine Kinase Inhibitor that works by blocking activity of proteins involved in bone development. The drug is given as an oral capsule once a day.

The aim of the study is to evaluate the long‑term safety of the medicine and to see if it can help increase height. Participants will take the capsule each day and attend regular visits where doctors check vital signs, perform blood tests, eye and dental exams, and take simple scans such as X‑rays and a bone‑density scan called dual x‑ray absorptiometry to monitor growth. Height will be measured over time and compared with standard growth charts.

The research process

The trial runs in 7 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Initial visit and baseline assessments

    After enrollment you attend an initial visit where baseline measurements are taken.

    Measurements include standing height, weight, body proportions, and head circumference.

    Laboratory tests, eye examination, dental examination, x‑ray imaging and a dual x‑ray absorptiometry (dx a) scan are performed to record starting values.

  2. Step 2

    Start of study medication

    You begin taking infigratinib capsules.

    The dose is 0.25 mg/kg (milligrams per kilogram of body weight) taken by mouth once each day.

    The medication is taken for the entire duration of the study as instructed by the study team.

  3. Step 3

    Daily dosing

    Each day you swallow the prescribed amount of infigratinib capsule with water.

    The dose remains the same unless a dose adjustment is required because of safety findings.

  4. Step 4

    Regular follow‑up visits

    You attend scheduled clinic visits at intervals defined by the study protocol.

    At each visit vital signs, laboratory tests, eye and dental examinations, and imaging (x‑ray and dx a scan) are repeated to monitor safety.

    Standing height and related growth measurements are recorded to track changes in height z‑score over time.

  5. Step 5

    Questionnaire assessments

    During follow‑up visits you complete age‑appropriate quality‑of‑life questionnaires as instructed.

    These questionnaires help evaluate how the treatment affects daily life and well‑being.

  6. Step 6

    Dose adjustment or interruption if needed

    If any adverse event (undesired medical occurrence) is identified, the study protocol may require a reduction in the dose of infigratinib or a temporary stop of the medication.

    Such decisions are made based on the type, severity, and relationship of the event to the study drug.

  7. Step 7

    Final assessment and study completion

    At the end of the study period you undergo a final set of measurements, laboratory tests, imaging studies, and questionnaires.

    The final data are used to evaluate long‑term safety, tolerability, and the effect of infigratinib on growth outcomes.

Who can join the trial?

5 criteria

  • You are between 3 years old and younger than 18 years at the time of screening and still have the ability to grow taller.
  • You have been diagnosed with hypochondroplasia (HCH), which means you have short stature that is not in proportion to the rest of your body, and this diagnosis has been confirmed with a genetic or other laboratory molecular test.
  • You have completed the earlier ACCEL 2/3 study and have at least six months of growth measurements recorded from that study.
  • You can walk and stand on your own without needing help (you are ambulatory).
  • If you are a girl who is 10 years old or older, or if you have started menstruating (menarche), you must have a negative pregnancy test before joining the study.

Who cannot join the trial?

13 criteria

  • If you have another medical condition that the doctor thinks could make the study unsafe or interfere with the tests, you cannot join.
  • If doctors find a serious health problem (referred to as a “clinically significant X”) that could affect the study, you cannot join.
  • If you develop a new medical condition that requires you to start a medication that is not allowed in this study, you cannot join.
  • If you stopped the earlier ACCEL 2/3 trial early, you cannot join.
  • If you have already reached your final adult height or are very close to it (your growth plates have closed), you cannot join.
  • If you are currently taking strong medicines that change the pH (acid level) of your stomach or intestines for more than one week, you cannot join.
  • If you are taking any medication that could increase the level of a substance referred to as “X” and affect the study, you cannot join.
  • If you have been on high‑dose glucocorticoid (steroid) therapy for three weeks or more, or on regular anti‑inflammatory steroid doses for more than three weeks within the past six months, you cannot join.
  • If you have had a broken long bone (such as the thigh or shin bone) or a spine fracture within the last 12 months, you cannot join.
  • If you are currently participating in another clinical study that is sponsored by a company other than QED, you cannot join.
  • If you have another serious disease or condition that the doctor believes would increase your risk or interfere with the study, you cannot join.
  • If you have a history of or current signs of extensive ectopic tissue calcification (abnormal calcium deposits in tissues where they don’t belong), you cannot join.
  • If you have ever had a malignancy (cancer), you cannot join.
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Investigated drugs

Infigratinib is a medicine taken as an oral capsule once a day. It works by blocking certain signals (called FGFR 1‑3) that help control bone growth. In this study, the drug is being given to children with hypochondroplasia to see if it can safely help them grow taller over time. Researchers will watch closely for any side effects and will measure changes in the children’s height compared with normal growth patterns.

What is already known about the treatment

Infigratinib - Infigratinib is taken as an oral capsule once daily. It is an FDA‑approved orphan drug for certain cancers and is now being studied in medical literature for long‑term use in children with hypochondroplasia. The medication works by selectively blocking FGFR1‑3 enzymes, which are signals that control bone growth. This makes it a selective FGFR tyrosine‑kinase inhibitor, a type of targeted drug repurposed for a rare growth disorder.

Investigated diseases

Hypochondroplasia - Hypochondroplasia is a rare inherited disorder that affects bone growth, leading to short stature and a relatively normal head size. It is caused by mutations in the FGFR3 gene, which slows the development of long bones. Children with the condition typically show slower growth during early childhood, resulting in proportionally short arms and legs. The spine may develop a slight curvature, and the fingers can appear slightly short. As the individual ages, growth may continue at a reduced rate, maintaining a shorter height compared to peers. The condition does not affect intelligence and many individuals lead normal lives.
Trial detailsLast updated 2 Oct 2026
Age0-17PhasePhase IITrial ID2025-523509-13-00Protocol codeQBGJ398-205Estimated enrolment98 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.

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