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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. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04490915
Recruitment Status : Active, not recruiting
First Posted : July 29, 2020
Last Update Posted : August 21, 2023
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 participants 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 open-label treatment with crinecerfont. Subsequently, participants may elect to participate in the open-label extension (OLE) period. The duration of participation in the study is approximately 20 months for the core study and will be a variable amount of time per subject for the OLE (estimated to be approximately 3 years).

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)
Actual Enrollment : 182 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
Actual Primary Completion Date : July 19, 2023
Estimated Study Completion Date : August 2027

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: Crinecerfont
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.
 Drug: Crinecerfont
CRF1-receptor antagonist
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.
 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 size 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. Participants of childbearing potential must agree to use an acceptable method of 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


Locations
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Sponsors and Collaborators
Neurocrine Biosciences
Investigators
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Study Director: Clinical Development Lead 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: August 21, 2023
Last Verified: August 2023
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
Hyperplasia
Pathologic Processes
Disorders of Sex Development
Urogenital Abnormalities
Female Urogenital Diseases
Female Urogenital Diseases and Pregnancy Complications
Urogenital Diseases
 Male Urogenital Diseases
Congenital Abnormalities
Genetic Diseases, Inborn
Steroid Metabolism, Inborn Errors
Metabolism, Inborn Errors
Metabolic Diseases
Adrenal Gland Diseases
Endocrine System Diseases
Gonadal Disorders