A Study of Gene Therapy for Classic Congenital Adrenal Hyperplasia (CAH)
NCT ID: NCT04783181
Last Updated: 2025-12-23
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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ACTIVE_NOT_RECRUITING
PHASE1/PHASE2
8 participants
INTERVENTIONAL
2021-07-01
2029-02-28
Brief Summary
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Detailed Description
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Conditions
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Keywords
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Study Design
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NON_RANDOMIZED
SEQUENTIAL
TREATMENT
NONE
Study Groups
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Dose Level 1
BBP-631 lowest dose, administered once, intravenously (IV)
AAV BBP-631
intravenous
Dose Level 2
BBP-631 middle dose, administered once, IV
AAV BBP-631
intravenous
Dose Level 3
BBP-631, high dose, administered once, IV
AAV BBP-631
intravenous
Dose Level 4
BBP-631, highest dose, administered once, IV
AAV BBP-631
intravenous
Interventions
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AAV BBP-631
intravenous
Eligibility Criteria
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Inclusion Criteria
2. Screening/baseline 17-OHP levels \> 5-10 × ULN and \< 40 × ULN (upper limit of normal)
3. Stable oral hydrocortisone (HC) regimen as the only glucocorticoid (GC) maintenance therapy
4. Naïve to prior gene therapy or AAV-mediated therapy
Exclusion Criteria
2. History of adrenalectomy and/or significant liver disease
18 Years
ALL
No
Sponsors
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Adrenas Therapeutics Inc
INDUSTRY
Responsible Party
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Locations
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Ann and Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States
National Institutes of Health Clinical Center
Bethesda, Maryland, United States
University of Minnesota
Minneapolis, Minnesota, United States
Lucas Research, Inc.
Morehead City, North Carolina, United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
Countries
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Related Links
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CAH Gene Therapy Website
Adrenas Therapeutics Website
Other Identifiers
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CAH-301
Identifier Type: -
Identifier Source: org_study_id