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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 : April 5, 2021
Sponsor:
Information provided by (Responsible Party):
Neurocrine Biosciences

Study Description
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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

Study Design
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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

Resource links provided by the National Library of Medicine


Arms and Interventions
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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


Outcome Measures
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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 ]

Eligibility Criteria
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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.
Contacts and Locations
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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 ClinicalTrials.gov identifier (NCT number): NCT04490915


Contacts
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Contact: Cheryl Chen 858-617-7744 cechen@neurocrine.com
Contact: Szecho Lin 858-617-7192 slin@neurocrine.com

Locations
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United States, California
Neurocrine Clinical Site Recruiting
Los Angeles, California, United States, 90027
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, Indiana
Neurocrine Clinical Site Recruiting
Indianapolis, Indiana, United States, 46202
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
Neurocrine Clinical Site Recruiting
New York, New York, United States, 10029
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
United States, Pennsylvania
Neurocrine Clinical Site Recruiting
Pittsburgh, Pennsylvania, United States, 15224
United States, Texas
Neurocrine Clinical Site Recruiting
Dallas, Texas, United States, 75390
Belgium
Neurocrine Clinical Site Recruiting
Brussels, Belgium, 1070
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
Athens, Greece, 115 27
Neurocrine Clinical Site Recruiting
Thessaloníki, Greece, 54642
Poland
Neurocrine Clinical Site Recruiting
Kraków, Poland, 31-0111
Neurocrine Clinical Site Recruiting
Poznań, Poland, 60-355
Neurocrine Clinical Site Recruiting
Warszawa, Poland, 01-809
Spain
Neurocrine Clinical Site Recruiting
Madrid, Spain, 28034
Neurocrine Clinical Site Recruiting
Sevilla, Spain, 41013
Sweden
Neurocrine Clinical Site Recruiting
Gothenburg, Sweden, 41766
Sponsors and Collaborators
Neurocrine Biosciences
More Information
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Additional Information:

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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: April 5, 2021
Last Verified: April 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
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