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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.
Participants will receive a single dose of AAV5 based intravenous (IV) BBP-631 and will be followed for safety and efficacy for at least 5 years after the date of treatment with BBP-631.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Dose Level 1 | Experimental | BBP-631 lowest dose, administered once, intravenously (IV) |
|
| Dose Level 2 | Experimental | BBP-631 middle dose, administered once, IV |
|
| Dose Level 3 | Experimental | BBP-631, high dose, administered once, IV |
|
| Dose Level 4 | Experimental | BBP-631, highest dose, administered once, IV |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| AAV BBP-631 | Biological | intravenous |
|
| Measure | Description | Time Frame |
|---|---|---|
| Number of participants with Treatment-emergent Adverse Events that Led to Study Discontinuation | up to 5 years | |
| To select the optimum dose or dose range of BBP 631 for future studies | up to 5 years |
| Measure | Description | Time Frame |
|---|---|---|
| Change from Baseline in 17-OHP (hydroxyprogesterone) levels | Baseline, Week 52 and through study completion, an average of 5 years | |
| Change from Baseline in androstenedione (A4) levels | Baseline, Week 52 and through study completion, an average of 5 years |
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Key Inclusion Criteria
Key Exclusion Criteria
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Ann and Robert H. Lurie Children's Hospital of Chicago | Chicago | Illinois | 60611 | United States | ||
| National Institutes of Health Clinical Center |
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| Label | URL |
|---|---|
| Adrenas Therapeutics Website | View source |
| CAH Gene Therapy Website | View source |
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| ID | Term |
|---|---|
| D000312 | Adrenal Hyperplasia, Congenital |
| ID | Term |
|---|---|
| D047808 | Adrenogenital Syndrome |
| D012734 | Disorders of Sex Development |
| D014564 | Urogenital Abnormalities |
| D052776 | Female Urogenital Diseases |
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Participants will be assigned sequentially to one of 4 dose levels depending on the date of determination of eligibility.
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| Change from Baseline in endogenous cortisol levels | Baseline, Week 52 and through study completion, an average of 5 years |
| Bethesda |
| Maryland |
| 20892-1932 |
| United States |
| University of Minnesota | Minneapolis | Minnesota | 55455 | United States |
| Lucas Research, Inc. | Morehead City | North Carolina | 28557 | United States |
| Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | 19104 | United States |
| D005261 | Female Urogenital Diseases and Pregnancy Complications |
| D000091642 | Urogenital Diseases |
| D052801 | Male Urogenital Diseases |
| D000013 | Congenital Abnormalities |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D030342 | Genetic Diseases, Inborn |
| D043202 | Steroid Metabolism, Inborn Errors |
| D008661 | Metabolism, Inborn Errors |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
| D000307 | Adrenal Gland Diseases |
| D004700 | Endocrine System Diseases |
| D006058 | Gonadal Disorders |