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

• Status: Recruiting
• Trial end date: 2024-02-01
• Target enrollment: 0
• Participant gender: All

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 subjects with classic CAH due to 21-hydroxylase deficiency. The study consists of a 6 month randomized, double blind, placebo-controlled period, followed by 1 year of treatment with crinecerfont. Duration of participation is approximately 20 months.

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Neurocrine Biosciences

Criteria

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 21-hydroxylase deficiency CAH.

3. Be on a stable regimen of steroidal treatment for CAH.

4. Patients of childbearing potential must agree to use hormonal or two forms of nonhormonal contraception (dual contraception) or other highly effective 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 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.