CONGENITAL ADRENAL HYPERPLASIA
Clinical trials for CONGENITAL ADRENAL HYPERPLASIA explained in plain language.
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One-Shot gene therapy trial aims to fix rare hormone disorder
Disease control OngoingThis is the first-ever study in people to test a gene therapy for congenital adrenal hyperplasia (CAH), a genetic disorder where the body can't make enough cortisol. Eight adults with classic CAH will receive a single intravenous dose of the experimental therapy, called BBP-631. …
Matched conditions: CONGENITAL ADRENAL HYPERPLASIA
Phase: PHASE1, PHASE2 • Sponsor: Adrenas Therapeutics Inc • Aim: Disease control
Last updated Mar 10, 2026 12:52 UTC
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Can a short video help teens take charge of their rare disease care?
Knowledge-focused TerminatedThis study aimed to test if a virtual education program could help teenagers and young adults with congenital adrenal hyperplasia (CAH) prepare to manage their own healthcare as they transition from pediatric to adult doctors. Participants, aged 16-22, would have been randomly as…
Matched conditions: CONGENITAL ADRENAL HYPERPLASIA
Phase: NA • Sponsor: National Institutes of Health Clinical Center (CC) • Aim: Knowledge-focused
Last updated Apr 01, 2026 14:42 UTC