Global Safety and Efficacy Registration Study of Crinecerfont for Congenital Adrenal Hyperplasia (CAHtalyst)
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ClinicalTrials.gov Identifier: NCT04490915 |
Recruitment Status :
Recruiting
First Posted : July 29, 2020
Last Update Posted : February 1, 2021
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Condition or disease | Intervention/treatment | Phase |
---|---|---|
Congenital Adrenal Hyperplasia | Drug: Crinecerfont Drug: Placebo | Phase 3 |
Study Type : | Interventional (Clinical Trial) |
Estimated Enrollment : | 165 participants |
Allocation: | Randomized |
Intervention Model: | Parallel Assignment |
Masking: | Triple (Participant, Care Provider, Investigator) |
Primary Purpose: | Treatment |
Official Title: | A Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Crinecerfont (NBI-74788) in Adult Subjects With Classic Congenital Adrenal Hyperplasia, Followed by Open-Label Treatment |
Actual Study Start Date : | July 23, 2020 |
Estimated Primary Completion Date : | February 2023 |
Estimated Study Completion Date : | February 2024 |

Arm | Intervention/treatment |
---|---|
Experimental: Crinecerfont
Capsule, administered orally, twice daily for 24 weeks, followed by active treatment for 1 year.
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Drug: Crinecerfont
CRF1 Inhibitor
Other Name: NBI-74788 |
Placebo Comparator: Placebo
Capsule, administered orally, twice daily for 24 weeks, followed by active treatment for 1 year.
|
Drug: Crinecerfont
CRF1 Inhibitor
Other Name: NBI-74788 Drug: Placebo Non-active dosage form |
- Percent change from baseline in glucocorticoid daily dose at Week 24 [ Time Frame: Baseline and Week 24 ]
- Change from baseline in serum androstenedione at Week 4 [ Time Frame: Baseline and Week 4 ]
- Achievement of a reduction in glucocorticoid daily dose to physiologic levels at Week 24 [ Time Frame: Baseline and Week 24 ]
- Change from baseline in homeostatic model assessment of insulin resistance (HOMA-IR) index at Week 24 [ Time Frame: Baseline and Week 24 ]
- Change from baseline in body weight at Week 24 [ Time Frame: Baseline and Week 24 ]
- Change from baseline in fat mass at Week 24 [ Time Frame: Baseline and Week 24 ]

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Ages Eligible for Study: | 18 Years and older (Adult, Older Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- Be willing and able to adhere to the study procedures, including all requirements at the study center and return for the follow-up visit.
- Have a medically confirmed diagnosis of classic 21-hydroxylase deficiency CAH.
- Be on a stable regimen of steroidal treatment for CAH.
- 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:
- Have a diagnosis of any of the other known forms of classic CAH.
- Have a history of bilateral adrenalectomy, hypopituitarism, or other condition requiring chronic glucocorticoid therapy.
- Have a clinically significant unstable medical condition or chronic disease other than CAH.
- Have a history of cancer unless considered cured.
- Are pregnant.
- Have a known history of clinically significant arrhythmia or abnormalities on ECG.
- Have a known hypersensitivity to any corticotropin releasing hormone antagonists.
- 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.
- Have current substance dependence, or current substance (drug) or alcohol abuse.
- Have had a blood loss ≥550 mL or donated blood or blood products within 8 weeks prior to the study.

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04490915
Contact: Cheryl Chen | 858-617-7744 | cechen@neurocrine.com | |
Contact: Szecho Lin | 858-617-7192 | slin@neurocrine.com |
United States, California | |
Neurocrine Clinical Site | Recruiting |
Pasadena, California, United States, 91105 | |
Neurocrine Clinical Site | Recruiting |
San Diego, California, United States, 92123 | |
United States, Georgia | |
Neurocrine Clinical Site | Recruiting |
Atlanta, Georgia, United States, 30329 | |
United States, Michigan | |
Neurocrine Clinical Site | Recruiting |
Ann Arbor, Michigan, United States, 48109 | |
United States, Minnesota | |
Neurocrine Clinical Site | Recruiting |
Minneapolis, Minnesota, United States, 55454 | |
United States, Missouri | |
Neurocrine Clinical Site | Recruiting |
Saint Louis, Missouri, United States, 63110 | |
United States, New York | |
Neurocrine Clinical Site | Recruiting |
Great Neck, New York, United States, 11021 | |
United States, North Carolina | |
Neurocrine Clinical Site | Recruiting |
Hickory, North Carolina, United States, 28601 | |
United States, Oklahoma | |
Neurocrine Clinical Site | Recruiting |
Oklahoma City, Oklahoma, United States, 73104 | |
Canada, Nova Scotia | |
Neurocrine Clinical Site | Recruiting |
Halifax, Nova Scotia, Canada, B3H 2Y9 | |
Greece | |
Neurocrine Clinical Site | Recruiting |
Athens, Greece, 10676 | |
Neurocrine Clinical Site | Recruiting |
Thessaloníki, Greece, 54642 | |
Spain | |
Neurocrine Clinical Site | Recruiting |
Madrid, Spain, 28034 | |
Neurocrine Clinical Site | Recruiting |
Sevilla, Spain, 41013 | |
Sweden | |
Neurocrine Clinical Site | Recruiting |
Gothenburg, Sweden, 41766 |
Responsible Party: | Neurocrine Biosciences |
ClinicalTrials.gov Identifier: | NCT04490915 |
Other Study ID Numbers: |
NBI-74788-CAH3003 2019-004873-17 ( EudraCT Number ) |
First Posted: | July 29, 2020 Key Record Dates |
Last Update Posted: | February 1, 2021 |
Last Verified: | January 2021 |
Individual Participant Data (IPD) Sharing Statement: | |
Plan to Share IPD: | No |
Studies a U.S. FDA-regulated Drug Product: | Yes |
Studies a U.S. FDA-regulated Device Product: | No |
Adrenal Hyperplasia, Congenital Adrenogenital Syndrome Hyperplasia Congenital Abnormalities Adrenal Gland Diseases Adrenocortical Hyperfunction Pathologic Processes Disorders of Sex Development |
Urogenital Abnormalities Genetic Diseases, Inborn Steroid Metabolism, Inborn Errors Metabolism, Inborn Errors Metabolic Diseases Endocrine System Diseases Gonadal Disorders |