Achondroplasia clinical trials at UCSF

6 in progress, 1 open to eligible people

Achondroplasia is a disorder that affects bone growth. UCSF is conducting a virtual study of children with achondroplasia across the United States. The study aims to collect information about these children who are receiving treatment.

Showing trials for

  • VIrtual STudy in Achondroplasia for the US (VISTA)

    open to eligible people ages 0 months to 18 years

    This is an observational study of individuals with achondroplasia in the United States. The primary study population consists of pediatric individuals treated and untreated with VOXZOGO™. Study enrollment started in February 2023. The projected…

    Oakland, California and other locations

  • 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 …

    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…

    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

  • Infigratinib in Children With Achondroplasia (ACH)

    Sorry, accepting new patients by invitation only

    This is a Phase 2, multicenter, open-label, extension (OLE) study to evaluate the long-term safety, tolerability, and efficacy of infigratinib, an FGFR 1-3-selective tyrosine kinase inhibitor, in subjects with ACH who previously completed a…

    Oakland, California and other locations

  • Clinical Assessment Study in Children With Achondroplasia (ACH)

    Sorry, in progress, not accepting new patients

    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,…

    Oakland, California and other locations

Our lead scientists for Achondroplasia research studies include .

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