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

NCT07850739 | Not Yet Recruiting Phase 2 FDA Drug

• 1 other identifier: NBIP-01435-CAH2039
• Study Type: interventional
• Target: 36
• Locations: 0 countries
• Sites: N/A

Brief 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.

Conditions

Congenital Adrenal Hyperplasia

Keywords

Classic congenital adrenal hyperplasia; CAH; Autosomal recessive disorder; NBIP-01435

Outcome Measures

Primary Outcomes (1)

• Change From Baseline in A4 Level at Week 8

  Baseline, Week 8

Study Arms (2)

Part 1: NBIP-01435

EXPERIMENTAL

Participants will receive NBIP-01435 administered once a week (QW) subcutaneously (SC) for a minimum of 8 weeks.

Drug: NBIP-01435

Part 2: NBIP-01435

EXPERIMENTAL

Participants will receive NBIP-01435 administered SC for a minimum of 8 weeks.

Drug: NBIP-01435

Interventions

NBIP-01435 DRUG

NBIP-01435 will be administered per schedule specified in the arm description.

Part 1: NBIP-01435; Part 2: NBIP-01435

Eligibility Criteria

• Age: 18 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Neurocrine Biosciences: lead

MeSH Terms

Conditions

Adrenal Hyperplasia, Congenital

Condition Hierarchy (Ancestors)

Adrenogenital Syndrome; Disorders of Sex Development; Urogenital Abnormalities; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Male Urogenital Diseases; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, Inborn; Steroid Metabolism, Inborn Errors; Metabolism, Inborn Errors; Metabolic Diseases; Nutritional and Metabolic Diseases; Adrenal Gland Diseases; Endocrine System Diseases; Gonadal Disorders

Study Officials

• Clinical Development Lead

  Neurocrine Biosciences

  STUDY DIRECTOR

Central Study Contacts

Neurocrine Medical Information Call Center

CONTACT

1-877-641-3461; medinfo@neurocrine.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 23, 2026
• First Posted: September 30, 2026
• Study Start (Estimated): December 1, 2026
• Primary Completion (Estimated): March 16, 2028
• Study Completion (Estimated): May 11, 2028
• Last Updated: September 30, 2026

Record last verified: 2026-09

Data Sharing

• IPD Sharing: Will not share