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NCT07169279PHASE2Recruiting

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

QED Therapeutics, a BridgeBio company

Start Date

11/19/2025

Completion Date

3/1/2032

Summary

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.

Detailed Description

PROPEL Infant \& Toddler (I\&T) is a Phase 2, multicenter, randomized, placebo-controlled study that comprises 4 portions: the single ascending dose (SAD) portion (open-label), the Phase 2 portion (open-label), the Phase 2b portion (placebo-controlled), and an Extension Portion (open-label). The study will evaluate children with ACH \< 3 years old being administered oral infigratinib.

Eligibility Criteria

Age Range: No minimum to No maximum

Inclusion Criteria: * 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 Criteria: * 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.

Interventions

DRUG

Infigratinib is provided as a single dose of minitablets for oral administration

DRUG

Infigratinib is provided as sprinkle capsules for daily oral administration

DRUG

Infigratinib or placebo comparator is provided as sprinkle capsules for daily oral administration

DRUG

Infigratinib is provided as sprinkle capsules for daily oral administration

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Conditions

Achondroplasia

Locations

UCSF Benioff Children's Hospital

Oakland, California 94609

United States

Johns Hopkins University

Baltimore, Maryland 21218

United States

University of Wisconsin Madison - Waisman Center Bone Dysplasia Clinic

Madison, Wisconsin 53705

United States

Murdoch Children's Research Institute

Parkville, Victoria 3052

Australia

Children's Hospital of Eastern Ontario Research Institute

Ottawa, Ontario K1H 8L1

Canada

Université de Montréal - Centre Hospitalier Universitaire Sainte-Justine

Montreal, Quebec H3T 1C5

Canada

Paediatric Clinical Research Unit at Oslo University Hospital

Oslo, 0372

Norway

KK Women's and Children's Hospital

Singapore, 2298899

Singapore

Unidad de Cirugía Artroscópica (UCA)

Vitoria-Gasteiz, 01010

Spain

NHS Greater Glasgow and Clyde

Glasgow, G3 8SJ

United Kingdom

Manchester University NHS Foundation Trust

Manchester, M13 9WL

United Kingdom

Sheffield Children's NHS Foundation Trust

Sheffield, S10 2TH

United Kingdom