Global Safety and Efficacy Registration Study of Crinecerfont for Congenital Adrenal Hyperplasia (CAHtalyst)
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|ClinicalTrials.gov Identifier: NCT04490915|
Recruitment Status : Active, not recruiting
First Posted : July 29, 2020
Last Update Posted : February 14, 2023
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|Condition or disease||Intervention/treatment||Phase|
|Congenital Adrenal Hyperplasia||Drug: Crinecerfont Drug: Placebo||Phase 3|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||182 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||Triple (Participant, Care Provider, Investigator)|
|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 :||August 2023|
|Estimated Study Completion Date :||August 2027|
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.
Other Name: NBI-74788
Placebo Comparator: Placebo
Placebo capsule, administered orally, twice daily for 24 weeks, followed by active treatment with crinecerfont for at least 1 year.
Other Name: NBI-74788
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 ]
- Change from baseline in blood pressure at Week 24 [ Time Frame: Baseline and Week 24 ]
- Change from baseline in glucose tolerance at Week 24 [ Time Frame: Baseline and Week 24 ]
- Change from baseline in waist circumference at Week 24 [ Time Frame: Baseline and Week 24 ]
- Change from baseline in menstrual regularity at Week 24 [ Time Frame: Baseline and Week 24 ]
- Change from baseline in testicular adrenal rest tumor size at Week 24 [ Time Frame: Baseline and Week 24 ]
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
|Ages Eligible for Study:||18 Years and older (Adult, Older Adult)|
|Sexes Eligible for Study:||All|
|Accepts Healthy Volunteers:||No|
- 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.
- Participants of childbearing potential must agree to use an acceptable method of contraception during the study.
- 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
|Study Director:||Clinical Development Lead||Neurocrine Biosciences|
|Responsible Party:||Neurocrine Biosciences|
|Other Study ID Numbers:||
2019-004873-17 ( EudraCT Number )
|First Posted:||July 29, 2020 Key Record Dates|
|Last Update Posted:||February 14, 2023|
|Last Verified:||February 2023|
|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
Disorders of Sex Development
Genetic Diseases, Inborn
Steroid Metabolism, Inborn Errors
Metabolism, Inborn Errors
Adrenal Gland Diseases
Endocrine System Diseases