Achondroplasia clinical trials at University of California Health

7 in progress, 2 open to eligible people

Showing trials for

  • 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,…

    at UCSF

  • 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…

    at UCSF

  • 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 …

    at UCLA UCSF

  • Infigratinib in Children and Adolescents with Achondroplasia

    Sorry, accepting new patients by invitation only

    This is a Phase 3, multicenter, double-blind, randomized, placebo-controlled study to evaluate the efficacy and safety of infigratinib in children and adolescents with achondroplasia (ACH) who have completed at least 26 weeks of participation in the …

    at UCSF

  • 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…

    at UCLA UCSF

  • 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

    at UCLA UCSF

  • 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…

    at UCSF

Our lead scientists for Achondroplasia research studies include .