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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT04783181
Recruitment Status : Not yet recruiting
First Posted : March 5, 2021
Last Update Posted : June 29, 2021
Information provided by (Responsible Party):
Adrenas Therapeutics Inc

Brief 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.

Condition or disease Intervention/treatment Phase
Congenital Adrenal Hyperplasia Biological: AAV BBP-631 Phase 1 Phase 2

Detailed Description:
Participants will receive a single dose of intravenous (IV) BBP-631 and are monitored for 52 weeks post-treatment. All participants who receive AAV5 based BBP-631 will be followed for an additional 4 years for safety and efficacy in a separate long-term follow-up study. In total, all participants will be followed for at least 5 years after the date of treatment with BBP-631.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 25 participants
Allocation: Non-Randomized
Intervention Model: Sequential Assignment
Intervention Model Description: Participants will be assigned sequentially to one of 3 dose levels depending on the date of determination of eligibility.
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase 1/2, First-in-Human, Open-Label, Dose-Escalation Study of the Safety and Efficacy of Gene Therapy for Congenital Adrenal Hyperplasia Through Administration of an Adeno-Associated Virus (AAV) Serotype 5-Based Recombinant Vector Encoding the Human CYP21A2 Gene
Estimated Study Start Date : July 2021
Estimated Primary Completion Date : August 2024
Estimated Study Completion Date : December 2024

Arm Intervention/treatment
Experimental: Dose Level 1
BBP-631 lowest dose, administered once, intravenously (IV)
Biological: AAV BBP-631

Experimental: Dose Level 2
BBP-631 middle dose, administered once, IV
Biological: AAV BBP-631

Experimental: Dose Level 3
BBP-631, highest dose, administered once, IV
Biological: AAV BBP-631

Primary Outcome Measures :
  1. Number of participants with Treatment-emergent Adverse Events that Led to Study Discontinuation [ Time Frame: up to 3 years ]
  2. To select the optimum dose or dose range of BBP 631 for future studies [ Time Frame: up to 3 years ]

Secondary Outcome Measures :
  1. Change from Baseline in 17-OHP (hydroxyprogesterone) levels [ Time Frame: Baseline, Week 52 ]
  2. Change from Baseline in endogenous cortisol levels [ Time Frame: Baseline, Week 52 ]
  3. Change from Baseline in androstenedione (A4) levels [ Time Frame: Baseline, Week 52 ]

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Key Inclusion Criteria

  1. Adult male and non-pregnant females with classic CAH (simple virilizing or salt-wasting) due to 21-OHD
  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

Key Exclusion Criteria

  1. Positive for anti-AAV5 (Adeno-Associated Virus Type 5) antibodies
  2. History of adrenalectomy and has no significant liver disease

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT04783181

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Contact: Kathleen M Kirby +1.978.479.7640
Contact: Mel Pozun

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United States, California
Children's Hospital Los Angeles
Los Angeles, California, United States, 90027
Contact: Norma Martinez    323-361-5627   
Principal Investigator: Mimi Kim, MD MSc         
United States, Maryland
National Institutes of Health Clinical Center
Bethesda, Maryland, United States, 20892-1932
Contact: Office of Patient Recruitment    800-411-1222   
Contact: Elizabeth Joyal (Study Coordinator), CRNP    301-496-8542   
Principal Investigator: Deborah Merke, M.D., M.S.         
United States, Minnesota
University of Minnesota
Minneapolis, Minnesota, United States, 55455
Contact: Coordinator Office    612-624-8672   
Contact: Coordinator Office    612-626-7024      
Principal Investigator: Kyriakie Sarafoglou, M.D.         
Australia, South Australia
PARC Clinical Research, CALHN Royal Adelaide Hospital
Adelaide, South Australia, Australia, 5000
Contact: Kathy Heyman    +61 8 7074 4404   
Principal Investigator: David Torpy, MD         
Sponsors and Collaborators
Adrenas Therapeutics Inc
Additional Information:
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Responsible Party: Adrenas Therapeutics Inc Identifier: NCT04783181    
Other Study ID Numbers: CAH-301
First Posted: March 5, 2021    Key Record Dates
Last Update Posted: June 29, 2021
Last Verified: May 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Adrenas Therapeutics Inc:
Gene therapy
Additional relevant MeSH terms:
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Adrenal Hyperplasia, Congenital
Adrenogenital Syndrome
Adrenocortical Hyperfunction
Pathologic Processes
Disorders of Sex Development
Urogenital Abnormalities
Congenital Abnormalities
Genetic Diseases, Inborn
Steroid Metabolism, Inborn Errors
Metabolism, Inborn Errors
Metabolic Diseases
Adrenal Gland Diseases
Endocrine System Diseases
Gonadal Disorders