Congenital Adrenal HyperplasiaPhase 1/2NCT04783181
A Study of Gene Therapy for Classic Congenital Adrenal Hyperplasia (CAH)
A Phase 1/2 study testing AAV BBP-631 in people with Congenital Adrenal Hyperplasia, run by Adrenas Therapeutics Inc at 5 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Congenital Adrenal Hyperplasia
- What is being tested?
- AAV BBP-631
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 8 participants
- Where
- 5 sitesUnited States
- Study dates
- 1 Jul 2021to Feb 2029
- Phase
- Phase 1/2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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 and < 40 × ULN (upper limit of normal)
- Stable oral hydrocortisone (HC) regimen as the only glucocorticoid (GC) maintenance therapy
- Naïve to prior gene therapy or AAV-mediated therapy
Who cannot join
- Positive for anti-AAV5 (Adeno-Associated Virus Type 5) antibodies
- History of adrenalectomy and/or significant liver disease
Trial locations and contacts
5 sites in 1 country.
5 sites in 1 country
United States5 sites
National Institutes of Health Clinical Center
Bethesda, Maryland, 20892-1932
Ann and Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611
University of Minnesota
Minneapolis, Minnesota, 55455
Lucas Research, Inc.
Morehead City, North Carolina, 28557
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
About this study
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
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.
Read the detailed description
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.
- Conditions
- Congenital Adrenal Hyperplasia
- Being tested
- AAV BBP-631
- Keywords
- CAH, Gene therapy, AAV, AAV5
- Medical subject headings
- Adrenal Hyperplasia, Congenital
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AAV BBP-631Biological
intravenous
| Study group | Role | What they receive |
|---|---|---|
| Dose Level 1 | Experimental | BBP-631 lowest dose, administered once, intravenously (IV) Receives: Biological: AAV BBP-631 |
| Dose Level 2 | Experimental | BBP-631 middle dose, administered once, IV Receives: Biological: AAV BBP-631 |
| Dose Level 3 | Experimental | BBP-631, high dose, administered once, IV Receives: Biological: AAV BBP-631 |
| Dose Level 4 | Experimental | BBP-631, highest dose, administered once, IV Receives: Biological: AAV BBP-631 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of participants with Treatment-emergent Adverse Events that Led to Study Discontinuation
Measured: up to 5 years
To select the optimum dose or dose range of BBP 631 for future studies
Measured: up to 5 years
Secondary outcomes (3)
Change from Baseline in 17-OHP (hydroxyprogesterone) levels
Measured: Baseline, Week 52 and through study completion, an average of 5 years
Change from Baseline in androstenedione (A4) levels
Measured: Baseline, Week 52 and through study completion, an average of 5 years
Change from Baseline in endogenous cortisol levels
Measured: Baseline, Week 52 and through study completion, an average of 5 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Mar 2021
- Study start1 Jul 2021
- Primary completionFeb 2029 (estimated)
- Study completionFeb 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Congenital Adrenal HyperplasiaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Model description
- Participants will be assigned sequentially to one of 4 dose levels depending on the date of determination of eligibility.
- Masking
- None (open label)
- Enrolment
- 8 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Adrenas Therapeutics Inc
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04783181
- Sponsor's protocol ID
- CAH-301
- Status last verified
- Dec 2025
- First submitted
- 1 Mar 2021
- Last updated
- 23 Dec 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 23 Dec 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT04783181
- Acronym / sponsor ID
- CAH-301
- Conditions
- Congenital Adrenal Hyperplasia
- Keywords
- CAH, Gene therapy, AAV, AAV5
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 8 participants (estimated)
- Lead sponsor
- Adrenas Therapeutics IncIndustry (pharma, biotech, devices)
- Study start
- 1 Jul 2021 (actual)
- Primary completion
- Feb 2029 (estimated)
- Study completion
- Feb 2029 (estimated)
- First posted
- 5 Mar 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 4
- Model description
- Participants will be assigned sequentially to one of 4 dose levels depending on the date of determination of eligibility.
| Arm | Type | Receives |
|---|---|---|
| Dose Level 1 | Experimental | Biological: AAV BBP-631BBP-631 lowest dose, administered once, intravenously (IV) |
| Dose Level 2 | Experimental | Biological: AAV BBP-631BBP-631 middle dose, administered once, IV |
| Dose Level 3 | Experimental | Biological: AAV BBP-631BBP-631, high dose, administered once, IV |
| Dose Level 4 | Experimental | Biological: AAV BBP-631BBP-631, highest dose, administered once, IV |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- 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 and < 40 × ULN (upper limit of normal)
- Stable oral hydrocortisone (HC) regimen as the only glucocorticoid (GC) maintenance therapy
- Naïve to prior gene therapy or AAV-mediated therapy
Key exclusion criteria (2 listed)
- Positive for anti-AAV5 (Adeno-Associated Virus Type 5) antibodies
- History of adrenalectomy and/or significant liver disease
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| AAV BBP-631intravenous | Biological | Dose Level 1, Dose Level 2, Dose Level 3, Dose Level 4 |
Primary outcome
Number of participants with Treatment-emergent Adverse Events that Led to Study Discontinuation
Time frame: up to 5 years
To select the optimum dose or dose range of BBP 631 for future studies
Time frame: up to 5 years
3 secondary outcomes
Change from Baseline in 17-OHP (hydroxyprogesterone) levels
Time frame: Baseline, Week 52 and through study completion, an average of 5 years
Change from Baseline in androstenedione (A4) levels
Time frame: Baseline, Week 52 and through study completion, an average of 5 years
Change from Baseline in endogenous cortisol levels
Time frame: Baseline, Week 52 and through study completion, an average of 5 years
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Dec 2025.
- Adrenas Therapeutics WebsiteLink provided by the sponsor.
- CAH Gene Therapy WebsiteLink provided by the sponsor.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
5 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing AAV BBP-631 in people with Congenital Adrenal Hyperplasia, run by Adrenas Therapeutics Inc at 5 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 23 Dec 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of participants with Treatment-emergent Adverse Events that Led to Study Discontinuation (time frame: up to 5 years). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 23 Dec 2025.