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 |
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Recruitment Status :
Active, not recruiting
First Posted : March 19, 2021
Last Update Posted : April 13, 2023
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Sponsor:
Neurocrine Biosciences
Information provided by (Responsible Party):
Neurocrine Biosciences
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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 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 |
| 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 |
Resource links provided by the National Library of Medicine
MedlinePlus Genetics related topics:
Congenital adrenal hyperplasia due to 11-beta-hydroxylase deficiency
21-hydroxylase deficiency
17 alpha-hydroxylase/17,20-lyase deficiency
3-beta-hydroxysteroid dehydrogenase deficiency
| Arm | Intervention/treatment |
|---|---|
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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.
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Drug: Crinecerfont
CRF1-receptor antagonist
Other Name: NBI-74788 |
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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.
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Drug: Crinecerfont
CRF1-receptor antagonist
Other Name: NBI-74788 Drug: Placebo Non-active dosage form |
Primary Outcome Measures :
- Change from Baseline in Serum Androstenedione (A4) at Week 4 [ Time Frame: Baseline to Week 4 ]
Secondary Outcome Measures :
- 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 ]
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.
- 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 ClinicalTrials.gov identifier (NCT number): NCT04806451
Locations
Show 46 study locations
Sponsors and Collaborators
Neurocrine Biosciences
Investigators
| Study Director: | Clinical Development Lead | Neurocrine Biosciences |
Additional Information:
| 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 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 |
Keywords provided by Neurocrine Biosciences:
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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 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 |