Achondroplasia clinical trials at UCSF

5 in progress, 2 open to eligible people

Achondroplasia is a condition that affects bones and causes short stature. UCSF is conducting clinical trials for children with Achondroplasia. These trials study growth, health-related quality of life, and the safety and effectiveness of a drug called infigratinib in children aged 3 to 11 years. If interested, families can enroll their children to participate in these studies.

Showing trials for

  • Infigratinib in Children With Achondroplasia

    open to eligible people ages 3-11

    This is a Phase 2, multicenter, open-label, dose-escalation and dose-expansion study to evaluate the safety, tolerability, and efficacy of infigratinib, a fibroblast growth factor receptor (FGFR) 1-3-selective tyrosine kinase inhibitor, in children 3 to 11 years of age with Achondroplasia (ACH) who previously participated in the PROPEL study (Protocol QBGJ398-001) for at least 6 months. The study includes dose escalation with extended treatment, and dose expansion. The study also includes a PK Substudy to fully characterize the pharmacokinetics of infigratinib in children with ACH.

    Oakland, California and other locations

  • Clinical Assessment Study in Children With Achondroplasia (ACH)

    open to eligible people ages 30 months to 17 years

    This is a long-term, multi-center, observational study in children 2.5 to <17 years with achondroplasia (ACH). The objective is to evaluate growth, ACH-related medical complications, assessments of health-related quality of life, body pain, functional abilities, cognitive functions, and treatments of study participants. No study medication will be administered.

    Oakland, California and other locations

  • Long-Term Safety, Tolerability, & Efficacy of BMN 111 in Children With Achondroplasia (ACH)

    Sorry, in progress, not accepting new patients

    This is a Phase 2, Open-Label, Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of BMN 111 in Children with Achondroplasia. The primary objective is to evaluate the long-term safety and tolerability of daily SC injections of BMN 111 in children with ACH who have completed two years of treatment in the 111-202 study.

    Oakland, California and other locations

  • BMN 111 in Children With Achondroplasia

    Sorry, in progress, not accepting new patients

    This is a Phase 2, open-label multi-center long-term extension study, with approximately 70 subjects, to evaluate the safety and efficacy of BMN111 in children with Achondroplasia until subjects reach near-adult final height. Eligible subjects will have completed 1 year of BMN111 or placebo treatment in the 111-206 study and once enrolled in the 111-208 extension study will receive a daily dose of BMN111 by subcutaneous injection according to their age as determined by 111-206.

    Oakland, California and other locations

  • BMN 111 in Children With Achondroplasia

    Sorry, in progress, not accepting new patients

    The intent and design of this Phase 3 study is to assess BMN 111 as a therapeutic option for the treatment of children with Achondroplasia

    Oakland, California and other locations

Our lead scientists for Achondroplasia research studies include .

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