Interventional Study of Infigratinib in Children < 3 Years Old With Achondroplasia
This study is testing a medication called infigratinib for children under 3 years old with achondroplasia (ACH), a genetic bone growth disorder. The main goals are to find the safest and most effective dose of infigratinib for this age group, and then to see how well it works and if it's safe. You may be eligible if your child is between birth and 32 months old and has a confirmed genetic diagnosis of ACH. The study will enroll about 77 participants. The current recruitment status is unclear.
- Study design
- This is a Phase 2, multicenter, randomized, placebo-controlled study with about 77 participants, including open-label and placebo-controlled portions.
- What's involved
- Not specified in the trial record.
- Compensation
- Not stated in the trial record.
- Follow-up
- The study will assess safety and effectiveness over 52 weeks in the main portions of the study.
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NCT07169279
Interventional Study of Infigratinib in Children < 3 Years Old With Achondroplasia (ACH)
Recruiting
PHASE2Ages 0–32InterventionalTreatmentQED Therapeutics, a BridgeBio company
~77 participants
Updated 2026-07-13 on ClinicalTrials.gov
What's tested:Infigratinib is provided as a single dose of minitablets for oral administrationInfigratinib is provided as sprinkle capsules for daily oral administrationInfigratinib or placebo comparator is provided as sprinkle capsules for daily oral administration
At a glance
Recruiting sites
12 of 12 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Single Ascending Dose Portion: Identify the dose of infigratinib to be used in each age cohort of the Phase 2 potion of the study (by assessing safety and PK of infigratinib and its active metabolites)
Measured over 2 weeks
+3 more outcomes measured
Conditions
Where it's being run
12 sites across 10 statesUnited Kingdom3
California1
Maryland1
Wisconsin1
Victoria1
Ontario1
Quebec1
Norway1
Who to contact
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Do you actually qualify for this trial?
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Eligibility criteria
Inclusion
Diagnosis of ACH confirmed by genetic testing. If prospective participants had prior genetic testing, the diagnosis must be confirmed by a report from a certified laboratory, documenting the specific mutation.
Age 0 to 32 months (2 years and 8 months) at screening.
Signed informed consent, which must be obtained from each participant's parent(s) or legal guardian.
Parent(s)/Guardian(s) willing and able to attend all study visits and comply with all study requirements.
Parent(s)/Guardian(s) willing and able to comply with the routine care of the study participants according to local guidance for the management of infants and young children with ACH.
Able to swallow age-appropriate oral medication.
In participants \<1 year old, be compliant with recommended vitamin D supplementation of 5 10 μg/day or higher (or as recommended by country specific guidelines).
Exclusion
Participants who have hypochondroplasia or diagnosis of genetic condition other than ACH, or any clinical condition that can affect growth.
Gestational age at birth \<37 weeks and/or birth weight \<2500 grams.
Gastroesophageal reflux disease requiring prolonged treatment (\>1 week) with prohibited medications.
Evidence of cervicomedullary compression, as defined by an Achondroplasia Foramen Magnum Score (AFMS) 4, symptomatic or asymptomatic, diagnosed during MRI done at screening or a previous MRI done at any time if the participant had not undergone decompression surgery.
History of fracture of a long bone or spine within 6 months prior to screening.
Any other significant concurrent disease or condition that, in the view of the investigator and/or sponsor, would confound assessment of efficacy or safety of infigratinib and/or would require treatment with a prohibited medication (per protocol), and/or would place the participant at high risk for poor treatment compliance or for failure to complete the study.
Having received or planning to receive treatment with any other investigational or approved product for the treatment of ACH or short stature, including (but not limited to) r-hGH, IGF-1, CNP analog, FGF ligand trap, or treatment targeting FGFR inhibition at any time.
Regular long-term (\>3 weeks; more than twice/year) treatment with supraphysiologic doses of glucocorticoid therapy (ie, \>15 mg/m2/day of hydrocortisone or equivalent) or treatment with glucocorticoids at anti-inflammatory doses (for over 3 weeks within 6 months of the screening visit. NOTE: Low-dose topical, inhaled, or intranasal corticosteroids are acceptable.
Significant abnormality in screening laboratory results,
Allergy or hypersensitivity to any components of the study drug.
What this trial measures
- Single Ascending Dose Portion: Identify the dose of infigratinib to be used in each age cohort of the Phase 2 potion of the study (by assessing safety and PK of infigratinib and its active metabolites)2 weeks
- Phase 2 Portion: Confirm the doses to be used in each age cohort in the Phase 2b portion of the study (by assessing safety and PK of infigratinib and its active metabolites).52 weeks
- Phase 2b Portion: Evaluate the safety and efficacy of infigratinib in infants and children < 3 years old with ACH (by assessing AE's & SAE's)52 weeks
- Extension Portion: Evaluate the safety and efficacy of infigratinib in participants who completed the Phase 2 or Phase 2b portion of the study until they have reached 3 years old (+6 months) (by assessing AE's and SAE's)3 years and 6 months