Actively Recruiting
Interventional Study of Infigratinib in Children < 3 Years Old With Achondroplasia (ACH)
Led by QED Therapeutics, a BridgeBio company · Updated on 2026-05-12
77
Participants Needed
13
Research Sites
327 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
Interventional Study of Infigratinib in Children < 3 Years Old With Achondroplasia (ACH)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Diagnosis of achondroplasia confirmed by genetic testing with a report from a certified laboratory
- Age between 0 and 32 months (2 years and 8 months) at screening
- Signed informed consent from parent(s) or legal guardian
- Parent(s)/Guardian(s) willing and able to attend all study visits and comply with study requirements
- Parent(s)/Guardian(s) willing and able to follow routine care guidelines for infants and young children with achondroplasia
- Able to swallow age-appropriate oral medication
- For participants under 1 year old, compliant with recommended vitamin D supplementation of 5-10 micrograms/day or higher as per local guidelines
You will not qualify if you...
- Diagnosis of hypochondroplasia or any genetic condition other than achondroplasia or any clinical condition affecting growth
- Born before 37 weeks gestation or with birth weight less than 2500 grams
- Gastroesophageal reflux disease requiring prolonged treatment (>1 week) with prohibited medications
- Evidence of cervicomedullary compression diagnosed by MRI with Achondroplasia Foramen Magnum Score 4, symptomatic or asymptomatic, unless decompression surgery performed
- History of fracture of a long bone or spine within 6 months before screening
- Any other significant disease or condition that may affect safety or efficacy assessment or require prohibited medications
- Previous or planned treatment with other investigational or approved products for achondroplasia or short stature
- Regular long-term treatment (>3 weeks; more than twice per year) with high-dose glucocorticoid therapy or anti-inflammatory doses within 6 months before screening (low-dose topical, inhaled, or intranasal corticosteroids allowed)
- Significant abnormalities in screening laboratory tests
- Allergy or hypersensitivity to any components of the study drug
AI-Screening
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Trial Site Locations
Total: 13 locations
1
UCSF Benioff Children's Hospital
Oakland, California, United States, 94609
Actively Recruiting
2
Johns Hopkins University
Baltimore, Maryland, United States, 21218
Actively Recruiting
3
University of Wisconsin Madison - Waisman Center Bone Dysplasia Clinic
Madison, Wisconsin, United States, 53705
Actively Recruiting
4
Murdoch Children's Research Institute
Parkville, Victoria, Australia, 3052
Actively Recruiting
5
Children's Hospital of Eastern Ontario Research Institute
Ottawa, Ontario, Canada, K1H 8L1
Actively Recruiting
6
Université de Montréal - Centre Hospitalier Universitaire Sainte-Justine
Montreal, Quebec, Canada, H3T 1C5
Actively Recruiting
7
Paediatric Clinical Research Unit at Oslo University Hospital
Oslo, Norway, 0372
Actively Recruiting
8
KK Women's and Children's Hospital
Singapore, Singapore, 2298899
Actively Recruiting
9
Unidad de Cirugia Artroscopica (UCA)
Vitoria-Gasteiz, Spain, 01010
Not Yet Recruiting
10
NHS Greater Glasgow and Clyde
Glasgow, United Kingdom, G3 8SJ
Not Yet Recruiting
11
Guy's and Saint Thomas' NHS Foundation Trust
London, United Kingdom, SE1 7EH
Not Yet Recruiting
12
Manchester University NHS Foundation Trust
Manchester, United Kingdom, M13 9WL
Not Yet Recruiting
13
Sheffield Children's NHS Foundation Trust
Sheffield, United Kingdom, S10 2TH
Actively Recruiting
Research Team
Q
QED Therapeutics Inc.
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
DOUBLE
Allocation
RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
15
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