Achondroplasia clinical trials at University of California Health

6 in progress, 1 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, functional abilities, cognitive functions, and treatments of study participants. No study medication will be administered.

    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 of BMN 111 in children with ACH who have completed two years of treatment in the 111-202 study.

    at UCLA 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 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.

    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 QED-sponsored interventional study, and potentially in additional subjects who are naïve to infigratinib treatment. Quality of Life assessments for this subject population will also be evaluated. Treatment-naïve subjects must have at least a 6-month period of growth assessment in the PROPEL study (Protocol QBGJ398 001) and will be enrolled in this OLE study only after a dose to be explored further is identified in Phase 2 Study QBGJ398-201.

    at UCSF

  • Infigratinib in Children With Achondroplasia

    Sorry, in progress, not accepting new patients

    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.

    at UCSF

Our lead scientists for Achondroplasia research studies include .

Last updated: