Interventional Study of Infigratinib in Children < 3 Years Old With Achondroplasia (ACH)

Recruiting now · Phase 2 · Has a placebo group

Conditions studied: Achondroplasia

In brief

This is a Phase 2, multicenter, randomized, placebo-controlled study to evaluate the safety and efficacy of infigratinib in participants \< 3 years old with ACH. The purposes of the SAD and Phase 2 portions are to identify and confirm the dose of infigratinib to be used in the Phase 2b portion, based on safety and PK. The purpose of the Phase 2b, placebo-controlled portion is to evaluate the safety and efficacy of infigratinib in children \< 3 years old with ACH at the selected dose.

Key facts

Study ID: NCT07169279
Run by: QED Therapeutics, a BridgeBio company
People needed: 77
Starts: 2025-11-19
Expected to finish: 2032-03-01
Last updated by the study team: 2026-07-13

Who can join

Age: any, up to 3. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

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).

You may not qualify if…

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.

Where it is running

UCSF Benioff Children's Hospital — Oakland, California, United States (enrolling)
Johns Hopkins University — Baltimore, Maryland, United States (enrolling)
University of Wisconsin Madison - Waisman Center Bone Dysplasia Clinic — Madison, Wisconsin, United States (enrolling)
Murdoch Children's Research Institute — Parkville, Victoria, Australia (enrolling)
Children's Hospital of Eastern Ontario Research Institute — Ottawa, Ontario, Canada (enrolling)
Université de Montréal - Centre Hospitalier Universitaire Sainte-Justine — Montreal, Quebec, Canada (enrolling)
Paediatric Clinical Research Unit at Oslo University Hospital — Oslo, Norway (enrolling)
KK Women's and Children's Hospital — Singapore, Singapore (enrolling)
Unidad de Cirugía Artroscópica (UCA) — Vitoria-Gasteiz, Spain (enrolling)
NHS Greater Glasgow and Clyde — Glasgow, United Kingdom (enrolling)
Manchester University NHS Foundation Trust — Manchester, United Kingdom (enrolling)
Sheffield Children's NHS Foundation Trust — Sheffield, United Kingdom (enrolling)

Full record on ClinicalTrials.gov

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