Global Safety and Efficacy Registration Study of Crinecerfont for Congenital Adrenal Hyperplasia

Phase 3

Active, not recruiting

• Conditions: Congenital Adrenal Hyperplasia
• Interventions: Drug: Crinecerfont; Drug: Placebo

• Registration Number: NCT04490915
• Lead Sponsor: Neurocrine Biosciences

Brief Summary

This is a Phase 3 study to evaluate the efficacy, safety, and tolerability of crinecerfont versus placebo administered for 24 weeks in approximately 165 adult participants with classic CAH due to 21-hydroxylase deficiency. The study consists of a 24-week randomized, double-blind, placebo-controlled period, followed by 1 year of active treatment with crinecerfont. Subsequently, participants may elect to participate in the open-label extension (OLE) period. The duration of participation in the study is approximately 20 months for the core study and will be a variable amount of time per participant for the OLE (estimated to be approximately 3 years).

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2020-07-24
• Study First Submitted QC: 2020-07-24
• Study First Posted: 2020-07-29
• Study Start: 2020-12-16
• Primary Completion: 2023-07-19
• Disposition First Posted: 2024-05-07
• Disposition First Submitted: 2024-07-11
• Status Verified: 2025-01
• Results First Submitted: 2025-01-10
• Results First Submitted QC: 2025-01-10
• Last Update Submitted: 2025-01-10
• Results First Posted: 2025-02-05
• Last Update Posted: 2025-02-05
• Study Completion: 2027-08

Recruitment & Eligibility

• Status: ACTIVE_NOT_RECRUITING
• Sex: All
• Target Recruitment: 182

Inclusion Criteria

1. Be willing and able to adhere to the study procedures, including all requirements at the study center and return for the follow-up visit.
2. Have a medically confirmed diagnosis of classic CAH due to 21-hydroxylase deficiency.
3. Be on a stable steroid regimen.
4. Participants of childbearing potential must agree to use an acceptable method of contraception during the study.

Exclusion Criteria

1. Have a diagnosis of any of the other known forms of classic CAH.
2. Have a history of bilateral adrenalectomy, hypopituitarism, or other condition requiring chronic glucocorticoid therapy.
3. Have a clinically significant unstable medical condition or chronic disease other than CAH.
4. Have a history of cancer unless considered cured.
5. Are pregnant.
6. Have a known history of clinically significant arrhythmia or abnormalities on ECG.
7. Have a known hypersensitivity to any corticotropin releasing hormone receptor antagonists.
8. Have received any other investigational drug within 30 days before initial screening or plan to use an investigational drug (other than the study drug) during the study.
9. Have current substance dependence, or current substance (drug) or alcohol abuse.
10. Have had a blood loss ≥550 mL or donated blood or blood products within 8 weeks prior to the study.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Placebo	Crinecerfont	Placebo capsule, administered orally, twice daily for 24 weeks, followed by active treatment with crinecerfont for at least 1 year.
Placebo	Placebo	Placebo capsule, administered orally, twice daily for 24 weeks, followed by active treatment with crinecerfont for at least 1 year.
Crinecerfont	Crinecerfont	Crinecerfont capsule, administered orally, twice daily for 24 weeks during the placebo-controlled treatment period, followed by active treatment with crinecerfont for at least 1 year.

Primary Outcome Measures

Name	Time	Method
Percent Change From Baseline in Glucocorticoid Daily Dose at Week 24	Baseline, Week 24	Least square (LS) mean and standard error (SE) were calculated using analysis of covariance (ANCOVA) model.

Secondary Outcome Measures

Name	Time	Method
Change From Baseline in Serum Androstenedione at Week 4	Baseline, Week 4
Number of Participants Who Achieved a Reduction to Physiologic Glucocorticoid Dose While Maintaining Androstenedione Control at Week 24	Week 24
Change From Baseline in Homeostatic Model Assessment of Insulin Resistance (HOMA-IR) at Week 24	Baseline, Week 24
Percent Change From Baseline in Body Weight at Week 24	Baseline, Week 24
Change From Baseline in Percent Total Fat Mass at Week 24	Baseline, Week 24
Change From Baseline in Serum 17-hydroxyprogesterone (17-OHP) at Week 4	Baseline, Week 4
Change From Baseline in Blood Pressure at Week 24	Baseline, Week 24
Change From Baseline in Glucose Tolerance at Week 24	Baseline, Week 24
Change From Baseline in Waist Circumference at Week 24	Baseline, Week 24
Change From Baseline in Menstrual Regularity at Week 24	Baseline, Week 24
Change From Baseline in Testicular Adrenal Rest Tumor (TART) Volume at Week 24	Baseline, Week 24

Trial Locations

Locations (1)

Neurocrine Clinical Site; 🇬🇧 Salford, United Kingdom