Status:
RECRUITING
Interventional Study of Infigratinib in Children < 3 Years Old With Achondroplasia (ACH)
Lead Sponsor:
QED Therapeutics, a BridgeBio company
Conditions:
Achondroplasia
Eligibility:
All Genders
Up to 32 years
Phase:
PHASE2
Brief 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 po...
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 (op...
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.
Key Trial Info
Start Date :
November 19 2025
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
March 1 2032
Estimated Enrollment :
77 Patients enrolled
Trial Details
Trial ID
NCT07169279
Start Date
November 19 2025
End Date
March 1 2032
Last Update
December 15 2025
Active Locations (13)
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1
UCSF Benioff Children's Hospital
Oakland, California, United States, 94609
2
Johns Hopkins University
Baltimore, Maryland, United States, 21218
3
University of Wisconsin Madison - Waisman Center Bone Dysplasia Clinic
Madison, Wisconsin, United States, 53705
4
Murdoch Children's Research Institute
Parkville, Victoria, Australia, 3052