Trial record 1 of 1 for:
CAH3003 | CAH - Congenital Adrenal Hyperplasia
Global Safety and Efficacy Registration Study of Crinecerfont for Congenital Adrenal Hyperplasia (CAHtalyst)
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| ClinicalTrials.gov Identifier: NCT04490915 |
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Recruitment Status :
Active, not recruiting
First Posted : July 29, 2020
Last Update Posted : May 7, 2024
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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 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 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 : | November 16, 2020 |
| Actual Primary Completion Date : | July 19, 2023 |
| Estimated Study Completion Date : | August 2027 |
Resource links provided by the National Library of Medicine
MedlinePlus Genetics related topics:
21-hydroxylase deficiency
| Arm | Intervention/treatment |
|---|---|
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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.
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Drug: Crinecerfont
CRF1-receptor antagonist
Other Name: NBI-74788 |
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Placebo Comparator: Placebo
Placebo capsule, administered orally, twice daily for 24 weeks, followed by active treatment with crinecerfont for at least 1 year.
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Drug: Crinecerfont
CRF1-receptor antagonist
Other Name: NBI-74788 Drug: Placebo Non-active dosage form |
Primary Outcome Measures :
- Percent change from baseline in glucocorticoid daily dose at Week 24 [ Time Frame: Baseline and Week 24 ]
Secondary Outcome Measures :
- Change from baseline in serum androstenedione at Week 4 [ Time Frame: Baseline and Week 4 ]
- Achievement of a reduction in glucocorticoid daily dose to physiologic levels at Week 24 [ Time Frame: Baseline and Week 24 ]
- Change from baseline in homeostatic model assessment of insulin resistance (HOMA-IR) index at Week 24 [ Time Frame: Baseline and Week 24 ]
- Change from baseline in body weight at Week 24 [ Time Frame: Baseline and Week 24 ]
- Change from baseline in fat mass at Week 24 [ Time Frame: Baseline and Week 24 ]
- Change from baseline in blood pressure at Week 24 [ Time Frame: Baseline and Week 24 ]
- Change from baseline in glucose tolerance at Week 24 [ Time Frame: Baseline and Week 24 ]
- Change from baseline in waist circumference at Week 24 [ Time Frame: Baseline and Week 24 ]
- Change from baseline in menstrual regularity at Week 24 [ Time Frame: Baseline and Week 24 ]
- Change from baseline in testicular adrenal rest tumor size at Week 24 [ Time Frame: Baseline and Week 24 ]
Information from the National Library of Medicine
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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:
- 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 classic 21-hydroxylase deficiency CAH.
- Be on a stable regimen of steroidal treatment for CAH.
- Participants of childbearing potential must agree to use an acceptable method of contraception during the study.
Exclusion Criteria:
- Have a diagnosis of any of the other known 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 cured.
- Are pregnant.
- Have a known history of clinically significant arrhythmia or abnormalities on ECG.
- Have a known hypersensitivity to any corticotropin releasing hormone antagonists.
- 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.
- Have current substance dependence, or current substance (drug) or alcohol abuse.
- Have had a blood loss ≥550 mL 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): NCT04490915
Locations
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Sponsors and Collaborators
Neurocrine Biosciences
Investigators
| Study Director: | Clinical Development Lead | Neurocrine Biosciences |
Additional Information:
| 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: | May 7, 2024 |
| Last Verified: | May 2024 |
| 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 |
Additional relevant MeSH terms:
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Adrenal Hyperplasia, Congenital Adrenogenital Syndrome Hyperplasia Congenital Abnormalities Adrenal Gland Diseases Adrenocortical Hyperfunction Pathologic Processes Disorders of Sex Development Urogenital Abnormalities Female Urogenital Diseases |
Female Urogenital Diseases and Pregnancy Complications Urogenital Diseases Male Urogenital Diseases Genetic Diseases, Inborn Steroid Metabolism, Inborn Errors Metabolism, Inborn Errors Metabolic Diseases Endocrine System Diseases Gonadal Disorders |