Active — Not RecruitingPhase 1/2

A Study of Gene Therapy for Classic Congenital Adrenal Hyperplasia (CAH)

United States8 participantsStarted 2021-07-01

Plain-language summary

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.

Who can participate

Age range18 Years

SexALL

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Inclusion criteria

✓. Adult male and non-pregnant females with classic CAH (simple virilizing or salt-wasting) due to 21-OHD
✓. Screening/baseline 17-OHP levels \> 5-10 × ULN and \< 40 × ULN (upper limit of normal)
✓. Stable oral hydrocortisone (HC) regimen as the only glucocorticoid (GC) maintenance therapy
✓. Naïve to prior gene therapy or AAV-mediated therapy

Exclusion criteria

✕. Positive for anti-AAV5 (Adeno-Associated Virus Type 5) antibodies
✕. History of adrenalectomy and/or significant liver disease

What they're measuring

Number of participants with Treatment-emergent Adverse Events that Led to Study Discontinuation

Timeframe: up to 5 years

To select the optimum dose or dose range of BBP 631 for future studies

Timeframe: up to 5 years

Trial details

NCT IDNCT04783181

SponsorAdrenas Therapeutics Inc

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2029-02

View on ClinicalTrials.gov More Congenital Adrenal Hyperplasia trials