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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT04490915
Recruitment Status : Recruiting
First Posted : July 29, 2020
Last Update Posted : November 1, 2021
Information provided by (Responsible Party):
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 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.

Condition or disease Intervention/treatment Phase
Congenital Adrenal Hyperplasia Drug: Crinecerfont Drug: Placebo Phase 3

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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.
Drug: Crinecerfont
CRF1-receptor antagonist
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-receptor antagonist
Other Name: NBI-74788

Drug: Placebo
Non-active dosage form

Primary Outcome Measures :
  1. Percent change from baseline in glucocorticoid daily dose at Week 24 [ Time Frame: Baseline and Week 24 ]

Secondary Outcome Measures :
  1. Change from baseline in serum androstenedione at Week 4 [ Time Frame: Baseline and Week 4 ]
  2. Achievement of a reduction in glucocorticoid daily dose to physiologic levels at Week 24 [ Time Frame: Baseline and Week 24 ]
  3. Change from baseline in homeostatic model assessment of insulin resistance (HOMA-IR) index at Week 24 [ Time Frame: Baseline and Week 24 ]
  4. Change from baseline in body weight at Week 24 [ Time Frame: Baseline and Week 24 ]
  5. Change from baseline in fat mass at Week 24 [ Time Frame: Baseline and Week 24 ]
  6. Change from baseline in blood pressure at Week 24 [ Time Frame: Baseline and Week 24 ]
  7. Change from baseline in glucose tolerance at Week 24 [ Time Frame: Baseline and Week 24 ]
  8. Change from baseline in waist circumference at Week 24 [ Time Frame: Baseline and Week 24 ]
  9. Change from baseline in menstrual regularity at Week 24 [ Time Frame: Baseline and Week 24 ]
  10. Change from baseline in testicular adrenal rest tumor at Week 24 [ Time Frame: Baseline and Week 24 ]

Information from the National Library of Medicine

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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:

  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.

Information from the National Library of Medicine

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 identifier (NCT number): NCT04490915

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Contact: Neurocrine Medical Information Call Center 877-641-3461

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Sponsors and Collaborators
Neurocrine Biosciences
Additional Information:
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Responsible Party: Neurocrine Biosciences 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: November 1, 2021
Last Verified: October 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Adrenal Hyperplasia, Congenital
Adrenogenital Syndrome
Adrenocortical Hyperfunction
Pathologic Processes
Disorders of Sex Development
Urogenital Abnormalities
Congenital Abnormalities
Genetic Diseases, Inborn
Steroid Metabolism, Inborn Errors
Metabolism, Inborn Errors
Metabolic Diseases
Adrenal Gland Diseases
Endocrine System Diseases
Gonadal Disorders