Interventional Study of Infigratinib in Children < 3 Years Old With Achondroplasia (ACH) (NCT07169279)
Phase 2b, Randomized, Double-Blind, Placebo-Controlled Clinical Trial, Preceded by a Single Ascending Dose Portion and a Phase 2 Open-Label Portion, to Evaluate the Safety and Efficacy of Oral Infigratinib in Infants and Young Children With Achondroplasia
🩺 Plain-English Summary (8th-Grade Level)
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.
Who can join: Review the eligibility criteria below.
Location: UCSF Benioff Children's Hospital — Oakland, California
Age range: 0 Years to 32 Months
🧪 Interventions in This Study
- 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 (DRUG)
📋 "Do I Qualify?" — 1-Minute Self Screener
Instant ChecklistFull eligibility criteria are available on the official NIH protocol. View the criteria ↗
📍 All Participating Trial Locations (12 Sites)
- UCSF Benioff Children's Hospital — Oakland, California
- Johns Hopkins University — Baltimore, Maryland
- University of Wisconsin Madison - Waisman Center Bone Dysplasia Clinic — Madison, Wisconsin
- Murdoch Children's Research Institute — Parkville, Victoria
- Children's Hospital of Eastern Ontario Research Institute — Ottawa, Ontario
- Université de Montréal - Centre Hospitalier Universitaire Sainte-Justine — Montreal, Quebec
- Paediatric Clinical Research Unit at Oslo University Hospital — Oslo,
- KK Women's and Children's Hospital — Singapore,
- Unidad de Cirugía Artroscópica (UCA) — Vitoria-Gasteiz,
- NHS Greater Glasgow and Clyde — Glasgow,
- Manchester University NHS Foundation Trust — Manchester,
- Sheffield Children's NHS Foundation Trust — Sheffield,
📞 Contact the Study Coordinator
Phone: (877) 280-5655
Email: medinfo@qedtx.com
Coordinator details come directly from the registered NIH protocol. Confirm the study is still recruiting before traveling.
💰 Cost, Insurance & Patient Rights
- 100% Free Items: The investigational treatment and protocol-required procedures are supplied at zero cost by the sponsor.
- Routine Patient Care: Under federal law (ACA § 2709) and many state statutes, routine care costs are covered during participation.
- Voluntary Participation: You may withdraw at any time without affecting your standard medical care.
More studies in Achondroplasia
- A Study to Evaluate Long-Term Safety, Tolerability, & Efficacy of BMN 111 in Children With Achondroplasia (ACH) — Oakland, California
- An Extension Study to Evaluate Safety and Efficacy of BMN 111 in Children With Achondroplasia — Oakland, California
- An Extension Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia — Oakland, California
- A Study of TYRA-300 in Children With Achondroplasia: BEACH301 — Torrance, California
- VIrtual STudy in Achondroplasia for the US (VISTA) — Oakland, California
Explore by condition: All Achondroplasia clinical trials
💡 How to Participate
- Check the checklist above to see if you may qualify.
- Contact the study coordinator using the phone/email above.
- Ask about the visit schedule, what is covered at no cost, and any travel support.
- Confirm with your own doctor before making a decision.