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Congenital Adrenal Hyperplasia clinical trials at UCSF

2 in progress, 1 open to eligible people

Congenital adrenal hyperplasia is a condition where the adrenal glands don't work properly due to a gene problem. UCSF is doing studies to see if a new gene therapy, BBP-631, is safe and works well for adults with this disease. These studies will help us learn more about this new treatment.

Showing trials for
  • Gene Therapy for Classic Congenital Adrenal Hyperplasia (CAH)

    open to eligible people ages 18 years and up

    This study is designed to evaluate the safety, tolerability, and efficacy of AAV5 based BBP-631 in adult participants diagnosed with classic congenital adrenal hyperplasia.

    Oakland, California and other locations

  • Global Safety and Efficacy Registration Study of Crinecerfont for Congenital Adrenal Hyperplasia

    Sorry, in progress, not accepting new patients

    This is a Phase 3 study to evaluate the efficacy, safety, and tolerability of crinecerfont versus placebo administered for 24 weeks in approximately 165 adult participants with classic CAH due to 21-hydroxylase deficiency. The study consists of a 6-month randomized, double-blind, placebo-controlled period, followed by 1 year of open-label treatment with crinecerfont. Subsequently, participants may elect to participate in the open-label extension (OLE) period. The duration of participation in the study is approximately 20 months for the core study and will be a variable amount of time per subject for the OLE (estimated to be approximately 3 years).

    San Francisco, California and other locations

Our lead scientists for Congenital Adrenal Hyperplasia research studies include .

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