Actively Recruiting
Phase 2b, Randomized, Double-Blind, Placebo-Controlled Clinical Trial of Oral Infigratinib in Infants and Young Children With Achondroplasia, Following Single Ascending Dose and Open-Label Phases
Led by QED Therapeutics, a BridgeBio company · Updated on 2026-05-12
77
Participants Needed
13
Research Sites
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are studying the safety and effects of the drug infigratinib in children younger than 3 years old who have achondroplasia (ACH), a form of dwarfism. This Phase 2, multicenter, randomized, placebo-controlled trial aims to find the right dose of infigratinib for young children and then evaluate its safety and effectiveness compared to a placebo. The study includes multiple phases to carefully assess how the drug works in this age group. The trial has four parts: a single ascending dose (SAD) portion where different doses are tested openly, a Phase 2 open-label portion to confirm dosing, a Phase 2b placebo-controlled portion to compare infigratinib to placebo, and an open-label extension to continue monitoring safety and effects until the children reach 3 years old plus 6 months. Infigratinib is given orally as minitablets or sprinkle capsules, with doses adjusted based on the child's age and weight throughout the study. Participants will be regularly assessed through various methods including blood tests, physical exams, brain and bone imaging (MRI and x-rays), sleep studies, and development milestone charts. Researchers will monitor growth, body proportions, quality of life, motor and language skills, and any surgical interventions or sleep apnea. The study includes safety monitoring by tracking adverse events over periods ranging from 2 weeks in early dose testing up to over 3 years in the extension phase. Parents or guardians will attend all visits and support adherence to study procedures.
CONDITIONS
Brief 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 from a certified laboratory
- Age between 0 to 32 months (2 years and 8 months) at screening
- Signed informed consent obtained from parent(s) or legal guardian
- Parent(s)/guardian(s) willing and able to attend all study visits and follow study requirements
- Parent(s)/guardian(s) willing to follow routine care guidelines for infants and young children with achondroplasia
- Ability to swallow age-appropriate oral medication
- For participants under 1 year old, compliance with recommended vitamin D supplementation (5-10 micrograms/day or as per local guidelines)
You will not qualify if you...
- Diagnosis of hypochondroplasia or any other genetic condition affecting growth besides achondroplasia
- Gestational age at birth under 37 weeks or birth weight less than 2500 grams
- Gastroesophageal reflux disease requiring prolonged treatment with prohibited medications
- Evidence of cervicomedullary compression diagnosed by MRI (score 4), with or without symptoms
- History of fracture in long bones or spine within 6 months before screening
- Any other significant disease or condition that could affect treatment assessment or compliance
- Previous or planned treatment with investigational or approved products for achondroplasia or short stature
- Regular long-term treatment with high-dose glucocorticoids or recent anti-inflammatory glucocorticoid use
- Significant abnormal lab results at screening
- Allergy or hypersensitivity to any components of the study drug
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - 2 weeks
Participants receive a single dose of infigratinib to evaluate safety and pharmacokinetics in different age cohorts.
1 to 2 visits depending on cohort assignment
Duration - 52 weeks
Participants receive daily oral doses of infigratinib for up to 52 weeks to assess safety, pharmacokinetics, and effects on growth and development.
Regular visits approximately every 3 months for dose adjustments and assessments
Duration - 52 weeks
Participants are randomized to receive daily oral infigratinib or placebo for 52 weeks to evaluate safety and efficacy.
Visits approximately every 3 months for dose adjustments and assessments
Duration - Up to 3 years and 6 months
Participants who completed prior study phases continue to receive daily oral infigratinib with ongoing safety and efficacy monitoring until they reach 3 years old (+6 months).
Visits every 3 to 6 months for dose adjustments and long-term assessments
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.
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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