Please note that the copy function is not enabled for this field.
If you wish to
modify
existing outcomes, please copy and paste the current outcome text into the Update field.
LOGIN
CREATE ACCOUNT
LOGIN
CREATE ACCOUNT
MY TRIALS
REGISTER TRIAL
FAQs
HINTS AND TIPS
DEFINITIONS
Trial Review
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 endorsed by the ANZCTR. Before participating in a study, talk to your health care provider and refer to this
information for consumers
Download to PDF
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])
Query!
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
Query!
Secondary ID [1]
0
0
CAH-300
Query!
Universal Trial Number (UTN)
Query!
Trial acronym
Query!
Linked study record
Query!
Health condition
Health condition(s) or problem(s) studied:
Congenital Adrenal Hyperplasia
0
0
Query!
Condition category
Condition code
Human Genetics and Inherited Disorders
0
0
0
0
Query!
Other human genetics and inherited disorders
Query!
Renal and Urogenital
0
0
0
0
Query!
Other renal and urogenital disorders
Query!
Metabolic and Endocrine
0
0
0
0
Query!
Other endocrine disorders
Query!
Intervention/exposure
Study type
Observational
Query!
Patient registry
Query!
Target follow-up duration
Query!
Target follow-up type
Query!
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
Query!
Intervention code [1]
0
0
Other interventions
Query!
Comparator / control treatment
Query!
Control group
Query!
Outcomes
Primary outcome [1]
0
0
Number of Participants Potentially Eligible for CAH-301 Study
Query!
Assessment method [1]
0
0
Query!
Timepoint [1]
0
0
Up to 3 years
Query!
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
Query!
Minimum age
18
Years
Query!
Query!
Maximum age
No limit
Query!
Query!
Sex
Both males and females
Query!
Can healthy volunteers participate?
Query!
Key exclusion criteria
- Positive for anti-AAV5 antibodies
- History of adrenalectomy and has no significant liver disease
Query!
Study design
Purpose
Query!
Duration
Query!
Selection
Query!
Timing
Prospective
Query!
Statistical methods / analysis
Query!
Recruitment
Recruitment status
Recruiting
Query!
Data analysis
Query!
Reason for early stopping/withdrawal
Query!
Other reasons
Query!
Date of first participant enrolment
Anticipated
Query!
Actual
1/07/2021
Query!
Date of last participant enrolment
Anticipated
Query!
Actual
Query!
Date of last data collection
Anticipated
1/06/2024
Query!
Actual
Query!
Sample size
Target
300
Query!
Accrual to date
Query!
Final
Query!
Recruitment in Australia
Recruitment state(s)
Query!
Recruitment outside Australia
Country [1]
0
0
United States of America
Query!
State/province [1]
0
0
California
Query!
Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Query!
Name
Adrenas Therapeutics Inc
Query!
Address
Query!
Country
Query!
Ethics approval
Ethics application status
Query!
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.
Query!
Trial website
https://clinicaltrials.gov/ct2/show/NCT05101902
Query!
Trial related presentations / publications
Query!
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
Query!
Contacts
Principal investigator
Name
0
0
Query!
Address
0
0
Query!
Country
0
0
Query!
Phone
0
0
Query!
Fax
0
0
Query!
Email
0
0
Query!
Contact person for public queries
Name
0
0
Patient Advocacy
Query!
Address
0
0
Query!
Country
0
0
Query!
Phone
0
0
650-391-9740
Query!
Fax
0
0
Query!
Email
0
0
[email protected]
Query!
Contact person for scientific queries
Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT05101902
Download to PDF