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
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