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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. Read our disclaimer for details. Identifier: NCT04806451
Recruitment Status : Active, not recruiting
First Posted : March 19, 2021
Last Update Posted : April 13, 2023
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 participants 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 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 14 months for the core study and will be a variable amount of time per participant for the OLE (estimated to be approximately 3 years).

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

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 103 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
Actual Primary Completion Date : March 10, 2023
Estimated Study Completion Date : August 2027

Arm Intervention/treatment
Experimental: Crinecerfont
Crinecerfont solution or capsule, administered orally, twice daily for 28 weeks during the placebo-controlled treatment period, followed by active treatment with crinecerfont for at least 24 weeks.
Drug: Crinecerfont
CRF1-receptor antagonist
Other Name: NBI-74788

Placebo Comparator: Placebo
Placebo solution or capsule, administered orally, twice daily for 28 weeks, followed by active treatment with crinecerfont for at least 24 weeks.
Drug: Crinecerfont
CRF1-receptor antagonist
Other Name: NBI-74788

Drug: Placebo
Non-active dosage form

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

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.

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

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.
  • Participants 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 identifier (NCT number): NCT04806451

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Sponsors and Collaborators
Neurocrine Biosciences
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Study Director: Clinical Development Lead Neurocrine Biosciences
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Responsible Party: Neurocrine Biosciences 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 13, 2023
Last Verified: April 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
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
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