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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/ct2/show/NCT04783181




Registration number
NCT04783181
Ethics application status
Date submitted
1/03/2021
Date registered
5/03/2021
Date last updated
17/05/2023

Titles & IDs
Public title
A Study of Gene Therapy for Classic Congenital Adrenal Hyperplasia (CAH)
Scientific 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
Secondary ID [1] 0 0
CAH-301
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Congenital Adrenal Hyperplasia 0 0
Condition category
Condition code
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Renal and Urogenital 0 0 0 0
Other renal and urogenital disorders
Metabolic and Endocrine 0 0 0 0
Other endocrine disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Other interventions - AAV BBP-631

Experimental: Dose Level 1 - BBP-631 lowest dose, administered once, intravenously (IV)

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

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


Other interventions: AAV BBP-631
intravenous
Intervention code [1] 0 0
Other interventions
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Number of participants with Treatment-emergent Adverse Events that Led to Study Discontinuation
Timepoint [1] 0 0
up to 3 years
Primary outcome [2] 0 0
To select the optimum dose or dose range of BBP 631 for future studies
Timepoint [2] 0 0
up to 3 years
Secondary outcome [1] 0 0
Change from Baseline in 17-OHP (hydroxyprogesterone) levels
Timepoint [1] 0 0
Baseline, Week 52
Secondary outcome [2] 0 0
Change from Baseline in endogenous cortisol levels
Timepoint [2] 0 0
Baseline, Week 52
Secondary outcome [3] 0 0
Change from Baseline in androstenedione (A4) levels
Timepoint [3] 0 0
Baseline, Week 52

Eligibility
Key inclusion criteria
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
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
Exclusion Criteria

1. Positive for anti-AAV5 (Adeno-Associated Virus Type 5) antibodies

2. History of adrenalectomy and/or significant liver disease

Study design
Purpose of the study
Treatment
Allocation to intervention
Non-randomised trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Other
Other design features
Phase
Phase 1/Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
1/07/2021
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/11/2028
Actual
Sample size
Target
25
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
Illinois
Country [3] 0 0
United States of America
State/province [3] 0 0
Maryland
Country [4] 0 0
United States of America
State/province [4] 0 0
Minnesota
Country [5] 0 0
United States of America
State/province [5] 0 0
North Carolina
Country [6] 0 0
United States of America
State/province [6] 0 0
Pennsylvania

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Adrenas Therapeutics Inc
Address
Country

Ethics approval
Ethics application status

Summary
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.
Trial website
https://clinicaltrials.gov/ct2/show/NCT04783181
Trial related presentations / publications
Public notes
This record is viewable in the ANZCTR as it had previously listed Australia and/or New Zealand as a recruitment site, however these sites have since been removed

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Patient Advocacy
Address 0 0
Country 0 0
Phone 0 0
650-391-9740
Fax 0 0
Email 0 0
[email protected]
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/ct2/show/NCT04783181