New pill could help kids with dwarfism grow better

NCT ID NCT07169279

Recruiting now Disease control Sponsor: QED Therapeutics, a BridgeBio company Source: ClinicalTrials.gov ↗

First seen Jun 26, 2026 · Last updated Jul 14, 2026 · Updated 3 times

Summary

This study tests an oral drug called infigratinib in infants and children under 3 years old with achondroplasia, the most common form of dwarfism. The goal is to see if it safely improves growth and body proportions. The trial has several phases to find the right dose and then compare the drug to a placebo over time.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance: infigratinib (oral drug)
What this could lead to: If successful, this could provide a daily oral treatment to improve growth and body proportions in young children with achondroplasia.
What could go wrong: This is an early Phase 2 trial with only 77 participants, so results may not confirm benefit. Side effects are still being studied, and the drug requires daily dosing.

This is an AI summary of the original study and may miss details. Read our disclaimer.

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Conditions

The condition(s) this trial relates to.

achondroplasia bone disorder Dwarfism hereditary endocrine growth disease Mucopolysaccharidosis IV musculoskeletal system disorder osteochondrodysplasia

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

ACHONDROPLASIA

Contacts and locations

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Study contacts

Contact

Email: •••••@•••••

Locations

Children's Hospital of Eastern Ontario Research Institute
RECRUITING

Ottawa, Ontario, K1H 8L1, Canada
Johns Hopkins University
RECRUITING

Baltimore, Maryland, 21218, United States
KK Women's and Children's Hospital
RECRUITING

Singapore, 2298899, Singapore
Manchester University NHS Foundation Trust
RECRUITING

Manchester, M13 9WL, United Kingdom
Murdoch Children's Research Institute
RECRUITING

Parkville, Victoria, 3052, Australia
NHS Greater Glasgow and Clyde
RECRUITING

Glasgow, G3 8SJ, United Kingdom
Paediatric Clinical Research Unit at Oslo University Hospital
RECRUITING

Oslo, 0372, Norway
Sheffield Children's NHS Foundation Trust
RECRUITING

Sheffield, S10 2TH, United Kingdom
UCSF Benioff Children's Hospital
RECRUITING

Oakland, California, 94609, United States
Unidad de Cirugía Artroscópica (UCA)
RECRUITING

Vitoria-Gasteiz, 01010, Spain
University of Wisconsin Madison - Waisman Center Bone Dysplasia Clinic
RECRUITING

Madison, Wisconsin, 53705, United States
Université de Montréal - Centre Hospitalier Universitaire Sainte-Justine
RECRUITING

Montreal, Quebec, H3T 1C5, Canada

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