Infigratinib treatment for children under 3 with achondroplasia

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

PROPEL Infant & Toddler (I&T) is a Phase 2, multicenter, randomized, placebo-controlled program that includes a single ascending dose (SAD) portion, an open-label Phase 2 portion, a randomized placebo-controlled Phase 2b portion, and an open-label extension to study oral infigratinib in children under 3 with achondroplasia. The SAD and Phase 2 portions are used to identify and confirm a safe dose based on safety and pharmacokinetics before advancing to the placebo-controlled Phase 2b. Participants receive age-appropriate oral formulations (minitablets or sprinkle capsules) and attend regular clinic visits for safety, growth measurements, and PK sampling. Genetic confirmation of achondroplasia and caregiver ability to comply with visits and routine care are required for enrollment.

Why it matters

Who can join: the full rules

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.

Where it's running

• UCSF Benioff Children's Hospital
  Oakland, California, United States Recruiting
• Johns Hopkins University
  Baltimore, Maryland, United States Recruiting
• University of Wisconsin Madison - Waisman Center Bone Dysplasia Clinic
  Madison, Wisconsin, United States Recruiting
• Murdoch Children's Research Institute
  Parkville, Victoria, Australia Recruiting
• Children's Hospital of Eastern Ontario Research Institute
  Ottawa, Ontario, Canada Recruiting
• Université de Montréal - Centre Hospitalier Universitaire Sainte-Justine
  Montreal, Quebec, Canada Recruiting

Study contacts

• Study coordinator: QED Therapeutics Inc.
• Email: medinfo@qedtx.com
• Phone: 18772805655

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

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• Evaluating a new treatment for infants with achondroplasia

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.