ClinicalTrials.gov record NCT07850739

Efficacy, Safety, Tolerability, and Pharmacokinetics of NBIP-01435 in Adult Participants With Classic Congenital Adrenal Hyperplasia (CAH)

Record copied from ClinicalTrials.gov: Oct 10, 2026AI summaries generated: Oct 10, 2026

A Phase 2, Open-Label Study to Evaluate the Efficacy, Safety, Tolerability, and Pharmacokinetics of NBIP-01435 in Adults With Classic Congenital Adrenal Hyperplasia

About this page. This page shows the study's official ClinicalTrials.gov record ↗, with added help to make it easier to understand. We explain medical terms, highlight key details in plain language, and suggest questions you may want to ask the study team.

The record is periodically copied from ClinicalTrials.gov and may lag behind changes. AI summaries are checked by software, not a clinician.Read moreShow less

The official record is written by the study sponsor and published on ClinicalTrials.gov. It may not always reflect the latest changes. Before making any decisions, please confirm important information with the study team and your own healthcare team.

Where you see "AI summary", AI was used to create a plain-language explanation and software checked it against the source. It has not been reviewed by a clinician, so it may contain mistakes or leave out important details.

StatusNot yet recruitingThe study has been registered but has not started enrolling. Sites and contacts may not be listed yet.

Study typeInterventionalA study in which researchers assign participants to one or more interventions, such as a drug, procedure, device, behavioural programme or other approach, to measure their effects.

PhasePhase 2A study that tests whether a treatment has the intended effect and continues to check safety, usually in a larger group than phase 1.

Age18 years old to 65 years old

SexAll

AI summaryWhat this study is about, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

This Phase 2, randomized, open-label study evaluates NBIP-01435 in adults with classic congenital adrenal hyperplasia (21‑hydroxylase deficiency). Participants receive NBIP-01435 by subcutaneous injection for a minimum of 8 weeks (Part 1: once weekly; Part 2: subcutaneous administration as described). The main measure is change in blood androstenedione (A4) at Week 8.

What it is studying. The study is testing the effects of different dose levels of the investigational drug NBIP-01435 on blood androstenedione (A4) levels, and is also assessing safety, tolerability, and pharmacokinetics in adults with classic CAH.

What the researchers hope to learn. Scientifically, the study aims to measure whether NBIP-01435 lowers A4 levels in adults with classic CAH and to describe the drug's safety, tolerability, and pharmacokinetic profile.

What this study is about

The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.

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.

Interventions being studied

Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.

Drug: NBIP-01435

Eligibility

The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.

Inclusion criteria

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.
Key

Exclusion criteria

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.

Study description and what participation involves

The registry record has no detailed description beyond the summary above.

AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

Participants will receive the study drug NBIP-01435 by subcutaneous injection for a minimum of 8 weeks (Part 1: given once weekly; Part 2: subcutaneous dosing for at least 8 weeks). Screening will include a blood sample to confirm elevated androstenedione (A4), and A4 level is measured at baseline and again at Week 8. Screening also requires a negative pregnancy test at screening and on Day 1, and participants taking fludrocortisone must have upright plasma renin activity checked as stated in the eligibility criteria. The registry record does not provide a full visit schedule or additional testing details beyond these items.

How the study is structured. Phase 2, interventional treatment study with randomized, parallel assignment and no masking (open-label). There are two experimental groups (Part 1 and Part 2); both receive NBIP-01435 by subcutaneous injection for at least 8 weeks. The primary outcome is change from baseline in A4 level at Week 8.

Questions you could ask

These questions are suggestions for a conversation with the study team or your own clinician, not advice.

The registry says recruitment has not started. When is it expected to open, and can I register interest now?
No sites are listed on the registry yet. Where will the study take place?
Which of the eligibility criteria would need to be checked with tests before I could join?

More on congenital adrenal hyperplasia

Clinical trials43 trials listed for congenital adrenal hyperplasiaEvery trial the registry lists for congenital adrenal hyperplasia, with recruitment status, phase and sites, updated from ClinicalTrials.gov.See all trials →Research updatesWhat changed in congenital adrenal hyperplasia researchLatest monthly update: September 2026. Includes: Bedtime hydrocortisone. New research summarized each month in plain language, with links to every source paper.Read the latest update →

Important dates

Study start: Dec 1, 2026 (estimated)
Primary completion: Mar 16, 2028 (estimated)
Study completion: May 11, 2028 (estimated)
Target enrollment: 36 participants (estimated)
Last updated on registry: Sep 30, 2026
Data verified: Oct 10, 2026

Sponsor and research team

The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.

Lead sponsor: Neurocrine Biosciences

Central study contact

If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.

Neurocrine Medical Information Call Center 📞 1-877-641-3461 ✉ medinfo@neurocrine.com

Participating sites

Study locations have not been posted on ClinicalTrials.gov yet. This study is not yet recruiting, so sites may be added when recruitment begins. The central study contact above may be able to tell you about planned sites.

Terms used on this page · explanations by MedicalUpdates.ai

glucocorticoid: Steroid medicines, such as prednisone, that reduce inflammation. Studies often require a stable dose at entry or measure whether the study treatment allows the dose to be reduced.
before screening: The checks done before enrolment to see whether a person qualifies, such as tests, scans or a review of medical history.
upper limit of normal: The highest value of a blood test that a laboratory considers normal. Criteria such as '≤ 3 × ULN' mean the result must be no more than three times that value.
childbearing potential: Studies often ask participants who could become pregnant, or could father a child, to use contraception during the study and for a period afterwards, because the effect of the treatment on a pregnancy is unknown.
exclusion criteria: The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.
Key Inclusion Criteria: The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.

The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.

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