New oral drug shows promise for helping children with dwarfism grow

NCT ID NCT05145010

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

First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This study tests a daily oral drug called infigratinib in 300 children with achondroplasia, the most common form of dwarfism. The drug aims to improve bone growth by targeting a specific protein. Researchers will monitor safety and growth over several years, including children who have taken the drug before and new participants.

What this could mean

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

Active substance: Infigratinib (an oral drug that targets a specific protein to promote bone growth)
What this could lead to: If successful, this could provide a long-term treatment option to improve growth and quality of life for children with achondroplasia.
What could go wrong: This is an early-phase extension study, so results are preliminary. The drug may cause side effects, and not all children may benefit. Long-term safety is still being evaluated.

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 hereditary 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

Locations

Birmingham Women's and Children's NHS Foundation Trust

Birmingham, B5 6NH, United Kingdom
Centre Hospitalier Universitaire Sainte-Justine

Montreal, Quebec, H3T 1C5, Canada
Children's Hospital - London Health Sciences Centre

London, Ontario, N6A 5W9, Canada
Children's Hospital Colorado

Aurora, Colorado, 80045, United States
Children's Hospital of Eastern Ontario

Ottawa, Ontario, K1H 8L1, Canada
Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States
Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico

Milan, 20122, Italy
Haukeland University Hospital

Bergen, 5021, Norway
Hopital Femme Mere Enfant

Lyon, 69500, France
Hopital Necker-Enfants Malades

Paris, 75743, France
Hopital des Enfants

Toulouse, 31300, France
Hospital Universitario La Paz

Madrid, 28046, Spain
Hospital Universitario Virgen de la Victoria

Málaga, 29010, Spain
Hospital Vithas San José

Vitoria-Gasteiz, Álava, 01012, Spain
Hospital de Pediatría SAMIC Prof. Dr. Juan P. Garrahan

Ciudad Autonoma Buenos Aires, Buenos Aires, C1245AAM, Argentina
Johns Hopkins University

Baltimore, Maryland, 21205, United States
KK Women's and Children's Hosptial

Singapore, 229899, Singapore
Manchester University Children's Hospital

Manchester, M13 9WL, United Kingdom
Murdoch Children's Hospital

Parkville, Victoria, 3052, Australia
Nemours Alfred I. Dupont Hospital for Children

Wilmington, Delaware, 19803, United States
Oslo University Hospital

Oslo, 0372, Norway
Policlinico A. Gemelli IRCCS

Roma, 00168, Italy
Queen Elizabeth University Hospital

Glasgow, G12 0XH, United Kingdom
Sheffield Children's Hospital

Sheffield, S10 2TH, United Kingdom
St. Thomas' Hospital

London, SE1 7EH, United Kingdom
Stollery Children's Hospital

Edmonton, Alberta, T6C 2B7, Canada
USCF Benioff Children's Hospital, Oakland

Oakland, California, 94609, United States
University Hospital and UW Health Clinics

Madison, Wisconsin, 53705, United States
University Hospitals Bristol and Weston NHS Foundation Trust

Bristol, BS2 8AE, United Kingdom
University of Missouri

Columbia, Missouri, 65212, United States
Vanderbilt University Medical Center

Nashville, Tennessee, 37232, United States

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