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Global Safety and Efficacy Registration Study of Crinecerfont in Pediatric Patients With Classic Congenital Adrenal Hyperplasia (CAHtalyst Pediatric Study)

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.
 
ClinicalTrials.gov Identifier: NCT04806451
Recruitment Status : Recruiting
First Posted : March 19, 2021
Last Update Posted : April 11, 2022
Sponsor:
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 28 weeks in approximately 81 pediatric patients with classic congenital adrenal hyperplasia (CAH) due to 21-hydroxylase deficiency. The study consists of a 28-week double blind, placebo-controlled period, followed by 24 weeks of treatment with crinecerfont. Duration of participation is approximately 14 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 : 81 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 Pediatric Subjects With Classic Congenital Adrenal Hyperplasia, Followed by Open-Label Treatment
Actual Study Start Date : June 24, 2021
Estimated Primary Completion Date : April 2023
Estimated Study Completion Date : April 2024


Arm Intervention/treatment
Experimental: Crinecerfont
Solution or capsule, administered orally, twice daily for 28 weeks, followed by active treatment for 24 weeks.
Drug: Crinecerfont
CRF1-receptor antagonist
Other Name: NBI-74788

Placebo Comparator: Placebo
Solution or capsule, administered orally, twice daily for 28 weeks, followed by active treatment for 24 weeks.
Drug: Placebo
Non-active dosage form

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




Primary Outcome Measures :
  1. Change from Baseline in Serum Androstenedione (A4) at Week 4 [ Time Frame: Baseline to Week 4 ]

Secondary Outcome Measures :
  1. Change from Baseline in Serum 17-hydroxyprogesterone (17-OHP) at Week 4 [ Time Frame: Baseline to Week 4 ]
  2. Percent Change from Baseline in Glucocorticoid Daily Dose at Week 28 [ Time Frame: Baseline to Week 28 ]
  3. Achievement of a reduction in glucocorticoid daily dose to physiologic levels at Week 28 [ Time Frame: Baseline to Week 28 ]
  4. Change from baseline in body mass index at Week 28 [ Time Frame: Baseline to Week 28 ]
  5. Change from baseline in salivary 17-OHP at Week 28 [ Time Frame: Baseline to Week 28 ]
  6. Change in bone age advancement at Week 28 [ Time Frame: Baseline to Week 28 ]
  7. Change from baseline in predicted adult height at Week 52 [ Time Frame: Baseline to Week 52 ]


Information from the National Library of Medicine

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Ages Eligible for Study:   2 Years to 17 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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 21-hydroxylase deficiency CAH.
  • Be on a stable regimen of steroidal treatment for CAH.
  • Have elevated androgen levels.
  • Patients of childbearing potential must be abstinent or agree to use appropriate birth control during the study.

Exclusion Criteria:

  • Have a diagnosis of any of the other 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 to be cured.
  • Have a known history of clinically significant arrhythmia or abnormalities on ECG.
  • Have a known hypersensitivity to any corticotropin-releasing hormone antagonist.
  • Have received an 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 substance (drug) or alcohol abuse.
  • Have had a significant blood loss 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 ClinicalTrials.gov identifier (NCT number): NCT04806451


Contacts
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Contact: Neurocrine Medical Information Call Center 877-641-3461 medinfo@neurocrine.com

Locations
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United States, California
Neurocrine Clinical Site Recruiting
Los Angeles, California, United States, 90027
Neurocrine Clinical Site Recruiting
San Diego, California, United States, 92123
Neurocrine Clinical Site Recruiting
San Francisco, California, United States, 94158
United States, Colorado
Neurocrine Clinical Site Recruiting
Aurora, Colorado, United States, 80045
United States, District of Columbia
Neurocrine Clinical Site Recruiting
Washington, District of Columbia, United States, 20010
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, Massachusetts
Neurocrine Clinical Site Recruiting
Boston, Massachusetts, United States, 02115
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, New York
Neurocrine Clinical Site Recruiting
New Hyde Park, New York, United States, 11040
Neurocrine Clinical Site Recruiting
New York, New York, United States, 10065
United States, Oklahoma
Neurocrine Clinical Site Recruiting
Tulsa, Oklahoma, United States, 74135
United States, Pennsylvania
Neurocrine Clinical Site Recruiting
Philadelphia, Pennsylvania, United States, 19104
Neurocrine Clinical Site Recruiting
Pittsburgh, Pennsylvania, United States, 15224
United States, Texas
Neurocrine Clinical Site Recruiting
Dallas, Texas, United States, 75235
United States, Washington
Neurocrine Clinical Site Recruiting
Seattle, Washington, United States, 98105
Canada, Alberta
Neurocrine Clinical Site Recruiting
Edmonton, Alberta, Canada, T6G 1C9
Canada, British Columbia
Neurocrine Clinical Site Recruiting
Vancouver, British Columbia, Canada, V6H 3V4
Canada, Quebec
Neurocrine Clinical Site Recruiting
Montréal, Quebec, Canada, H3T 1C5
France
Neurocrine Clinical Site Recruiting
Le Kremlin-Bicêtre, France, 94270
Neurocrine Clinical Site Recruiting
Paris, France, 75019
Greece
Neurocrine Clinical Site Recruiting
Athens, Greece, 115 27
Neurocrine Clinical Site Recruiting
Athens, Greece, 11527
Poland
Neurocrine Clinical Site Recruiting
Gdańsk, Poland, 80-214
Spain
Neurocrine Clinical Site Recruiting
Barcelona, Spain, 08035
Neurocrine Clinical Site Recruiting
Sevilla, Spain, 41013
Sponsors and Collaborators
Neurocrine Biosciences
Additional Information:
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Responsible Party: Neurocrine Biosciences
ClinicalTrials.gov Identifier: NCT04806451    
Other Study ID Numbers: NBI-74788-CAH2006
2020-004381-19 ( EudraCT Number )
First Posted: March 19, 2021    Key Record Dates
Last Update Posted: April 11, 2022
Last Verified: April 2022
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
Keywords provided by Neurocrine Biosciences:
classic congenital adrenal hyperplasia (CAH)
21-hydroxylase deficiency
Additional relevant MeSH terms:
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Adrenal Hyperplasia, Congenital
Adrenogenital Syndrome
Adrenocortical Hyperfunction
Hyperplasia
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