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.
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 Paul Harmatz.
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