Global Safety and Efficacy Registration Study of Crinecerfont in Pediatric Patients With Classic Congenital Adrenal Hyperplasia (CAHtalyst Pediatric Study)
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|ClinicalTrials.gov Identifier: NCT04806451|
Recruitment Status : Active, not recruiting
First Posted : March 19, 2021
Last Update Posted : April 13, 2023
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|Condition or disease||Intervention/treatment||Phase|
|Congenital Adrenal Hyperplasia||Drug: Crinecerfont Drug: Placebo||Phase 3|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||103 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||Triple (Participant, Care Provider, Investigator)|
|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|
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.
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.
Other Name: NBI-74788
Non-active dosage form
- Change from Baseline in Serum Androstenedione (A4) at Week 4 [ Time Frame: Baseline to Week 4 ]
- Change from Baseline in Serum 17-hydroxyprogesterone (17-OHP) at Week 4 [ Time Frame: Baseline to Week 4 ]
- Percent Change from Baseline in Glucocorticoid Daily Dose at Week 28 [ Time Frame: Baseline to Week 28 ]
- Achievement of a reduction in glucocorticoid daily dose to physiologic levels at Week 28 [ Time Frame: Baseline to Week 28 ]
- Change from baseline in body mass index at Week 28 [ Time Frame: Baseline to Week 28 ]
- Change from baseline in salivary 17-OHP at Week 28 [ Time Frame: Baseline to Week 28 ]
- Change in bone age advancement at Week 28 [ Time Frame: Baseline to Week 28 ]
- Change from baseline in predicted adult height at Week 52 [ Time Frame: Baseline to Week 52 ]
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|Ages Eligible for Study:||2 Years to 17 Years (Child)|
|Sexes Eligible for Study:||All|
|Accepts Healthy Volunteers:||No|
- 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.
- 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.
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
|Study Director:||Clinical Development Lead||Neurocrine Biosciences|
|Responsible Party:||Neurocrine Biosciences|
|Other Study ID Numbers:||
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|
|Studies a U.S. FDA-regulated Drug Product:||Yes|
|Studies a U.S. FDA-regulated Device Product:||No|
classic congenital adrenal hyperplasia (CAH)
Adrenal Hyperplasia, Congenital
Disorders of Sex Development
Female Urogenital Diseases
Female Urogenital Diseases and Pregnancy Complications
Male Urogenital Diseases
Genetic Diseases, Inborn
Steroid Metabolism, Inborn Errors
Metabolism, Inborn Errors
Adrenal Gland Diseases
Endocrine System Diseases