Trial Review

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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/NCT05101902




Registration number
NCT05101902
Ethics application status
Date submitted
20/10/2021
Date registered
1/11/2021
Date last updated
20/07/2023

Titles & IDs
Public title
A Study to Determine Eligibility for CAH-301 (A Study of Gene Therapy for Classic Congenital Adrenal Hyperplasia (CAH) [NCT04783181])
Scientific title
Pre-screening Study to Identify Adult Participants With Classic Congenital Adrenal Hyperplasia Due to 21-Hydroxylase Deficiency Who May Be Eligible for Treatment in the CAH-301 Trial With BBP-631, an Adeno-associated Virus (AAV) Serotype 5-Based Recombinant Vector Encoding the Human CYP21A2 Gene
Secondary ID [1] 0 0
CAH-300
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
Observational
Patient registry
Target follow-up duration
Target follow-up type
Description of intervention(s) / exposure
Other interventions - Pre-Screening

Overall Study - Participants will undergo antibody and genetic screen, initial pre-screening, eligibility monitoring and eligibility confirmation for potential transition to the treatment trial center once a treatment assignment in the study CAH-301 becomes available. Participation in Study CAH-300 is permitted to continue until treatment assignment is available in the treatment trial or unless, during eligibility monitoring or eligibility


Other interventions: Pre-Screening
No intervention given
Intervention code [1] 0 0
Other interventions
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Number of Participants Potentially Eligible for CAH-301 Study
Timepoint [1] 0 0
Up to 3 years

Eligibility
Key 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

- Is on a daily regimen of glucocorticoid

- Naïve to prior gene therapy or AAV-mediated therapy
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
Key exclusion criteria
- Positive for anti-AAV5 antibodies

- History of adrenalectomy and has no significant liver disease

Study design
Purpose
Duration
Selection
Timing
Prospective
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
1/06/2024
Actual
Sample size
Target
300
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

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

Ethics approval
Ethics application status

Summary
Brief summary
This pre-screening study is designed to determine potential eligibility of adults with
classic CAH due to 21-hydroxylase deficiency (21-OHD) for participation in the CAH-301
[NCT04783181] gene therapy trial with BBP-631.
Trial website
https://clinicaltrials.gov/ct2/show/NCT05101902
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
ClinicalTrials@AdrenasTX.com
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/ct2/show/NCT05101902