Congenital Adrenal HyperplasiaPhase 2NCT07850739
Efficacy, Safety, Tolerability, and Pharmacokinetics of NBIP-01435 in Adult Participants With Classic Congenital Adrenal Hyperplasia (CAH)
A Phase 2 study testing NBIP-01435 in people with Congenital Adrenal Hyperplasia, run by Neurocrine Biosciences.
Trial summary
The main objective of this study is to evaluate the efficacy of different dose levels of NBIP-01435 on reducing elevated androstenedione (A4) levels in adults with classic CAH. Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Congenital Adrenal Hyperplasia
- What is being tested?
- NBIP-01435
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 36 participants
- Where
- No sites listed yet
- Study dates
- 1 Dec 2026to 11 May 2028
- Phase
- Phase 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 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Medically confirmed diagnosis of classic 21-hydroxylase deficiency (21-OHD) CAH, based on standard, medically accepted criteria.
- Stable glucocorticoid (GC) dose regimen for classic CAH for a minimum of 30 days before screening.
- If treated with fludrocortisone, the dose should be stable for at least 30 days before screening with an upright plasma renin activity (PRA) <upper limit of normal (ULN) on participant's usual sodium intake.
- Have elevated A4, based on a blood sample collected at screening.
- Body weight ≥50 kilograms (kg) for males and ≥45 kg for females.
- Negative pregnancy test at screening visit and Day 1.
- Participants with ovaries of childbearing potential must agree to use contraception consistently.
- Unless considered permanently sterile or vasectomized at least 3 months prior to screening with medically confirmed successful procedure, participants with testes must agree to use effective barrier contraception (that is, condom with spermicide [cream, spray, foam, gel, suppository, or polymer film]) consistently.
Who cannot join
- Pregnant (that is, positive pregnancy test), or lactating, or plan to become pregnant during the study.
- Had a clinically significant illness within 30 days of screening.
- Have a known or suspected diagnosis of any of the other known forms of classic CAH.
- Note: Other protocol-defined inclusion and exclusion criteria may apply.
Trial locations and contacts
No sites are listed yet. Contact the study team or a site near you to ask about taking part.
About this study
Official title: A Phase 2, Open-Label Study to Evaluate the Efficacy, Safety, Tolerability, and Pharmacokinetics of NBIP-01435 in Adults With Classic Congenital Adrenal Hyperplasia
The main objective of this study is to evaluate the efficacy of different dose levels of NBIP-01435 on reducing elevated androstenedione (A4) levels in adults with classic CAH.
- Conditions
- Congenital Adrenal Hyperplasia
- Being tested
- NBIP-01435
- Keywords
- Classic congenital adrenal hyperplasia, CAH, Autosomal recessive disorder, NBIP-01435
- 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.
NBIP-01435Drug
NBIP-01435 will be administered per schedule specified in the arm description.
| Study group | Role | What they receive |
|---|---|---|
| Part 1: NBIP-01435 | Experimental | Participants will receive NBIP-01435 administered once a week (QW) subcutaneously (SC) for a minimum of 8 weeks. Receives: Drug: NBIP-01435 |
| Part 2: NBIP-01435 | Experimental | Participants will receive NBIP-01435 administered SC for a minimum of 8 weeks. Receives: Drug: NBIP-01435 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change From Baseline in A4 Level at Week 8
Measured: Baseline, Week 8
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 Sep 2026
- Study start1 Dec 2026 (estimated)
- Primary completion16 Mar 2028 (estimated)
- Study completion11 May 2028 (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.
- More trials run by Neurocrine BiosciencesThe sponsor's other registered studies.
- 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 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 36 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Neurocrine Biosciences
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Clinical Development Lead, Study director, Neurocrine Biosciences
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07850739
- Sponsor's protocol ID
- NBIP-01435-CAH2039
- Status last verified
- Sep 2026
- First submitted
- 23 Sep 2026
- Last updated
- 30 Sep 2026
- Results posted
- No
- 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 30 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2, Open-Label Study to Evaluate the Efficacy, Safety, Tolerability, and Pharmacokinetics of NBIP-01435 in Adults With Classic Congenital Adrenal Hyperplasia
- ClinicalTrials.gov ID
- NCT07850739
- Acronym / sponsor ID
- NBIP-01435-CAH2039
- Conditions
- Congenital Adrenal Hyperplasia
- Keywords
- Classic congenital adrenal hyperplasia, CAH, Autosomal recessive disorder, NBIP-01435
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 36 participants (estimated)
- Lead sponsor
- Neurocrine BiosciencesIndustry (pharma, biotech, devices)
- Study start
- 1 Dec 2026 (estimated)
- Primary completion
- 16 Mar 2028 (estimated)
- Study completion
- 11 May 2028 (estimated)
- First posted
- 30 Sep 2026 (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.
The main objective of this study is to evaluate the efficacy of different dose levels of NBIP-01435 on reducing elevated androstenedione (A4) levels in adults with classic CAH.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Part 1: NBIP-01435 | Experimental | Drug: NBIP-01435Participants will receive NBIP-01435 administered once a week (QW) subcutaneously (SC) for a minimum of 8 weeks. |
| Part 2: NBIP-01435 | Experimental | Drug: NBIP-01435Participants will receive NBIP-01435 administered SC for a minimum of 8 weeks. |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- Medically confirmed diagnosis of classic 21-hydroxylase deficiency (21-OHD) CAH, based on standard, medically accepted criteria.
- Stable glucocorticoid (GC) dose regimen for classic CAH for a minimum of 30 days before screening.
- If treated with fludrocortisone, the dose should be stable for at least 30 days before screening with an upright plasma renin activity (PRA) <upper limit of normal (ULN) on participant's usual sodium intake.
- Have elevated A4, based on a blood sample collected at screening.
- Body weight ≥50 kilograms (kg) for males and ≥45 kg for females.
Key exclusion criteria (4 listed)
- Pregnant (that is, positive pregnancy test), or lactating, or plan to become pregnant during the study.
- Had a clinically significant illness within 30 days of screening.
- Have a known or suspected diagnosis of any of the other known forms of classic CAH.
- Note: Other protocol-defined inclusion and exclusion criteria may apply.
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 |
|---|---|---|
| NBIP-01435NBIP-01435 will be administered per schedule specified in the arm description. | Drug | Part 1: NBIP-01435, Part 2: NBIP-01435 |
Primary outcome
Change From Baseline in A4 Level at Week 8
Time frame: Baseline, Week 8
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 30 Sep 2026.
- 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.
Clinical Development Lead study director
Neurocrine Biosciences
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing NBIP-01435 in people with Congenital Adrenal Hyperplasia, run by Neurocrine Biosciences.
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 8 inclusion and 4 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: opening soon, as of the sponsor's last update on 30 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
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 phase mean?
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?
Contact Neurocrine Medical Information Call Center (1-877-641-3461, medinfo@neurocrine.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change From Baseline in A4 Level at Week 8 (time frame: Baseline, Week 8). 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 30 Sep 2026.