New pill could help kids with dwarfism grow better

NCT ID NCT07169279

First seen Jun 26, 2026 · Last updated Jun 27, 2026 · Updated 1 time

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.

Disclaimer Read more

This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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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.

Contacts and locations

Study contacts

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••

Locations

  • Children's Hospital of Eastern Ontario Research Institute

    RECRUITING

    Ottawa, Ontario, K1H 8L1, Canada

  • Guy's and Saint Thomas' NHS Foundation Trust

    NOT_YET_RECRUITING

    London, SE1 7EH, United Kingdom

  • 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

    NOT_YET_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 Cirugia Artroscopica (UCA)

    NOT_YET_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