Trial record 1 of 4 for:
Recruiting Studies | congenital adrenal hyperplasia | Phase 3
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:
Neurocrine Biosciences
Information provided by (Responsible Party):
Neurocrine Biosciences
- Study Details
- Tabular View
- No Results Posted
- Disclaimer
- How to Read a Study Record
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 |
| 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 |
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 |
|---|---|
|
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 :
- 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
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.
| 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
| Contact: Neurocrine Medical Information Call Center | 877-641-3461 | medinfo@neurocrine.com |
Locations
| 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:
| 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 |
| 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:
|
Adrenal Hyperplasia, Congenital Adrenogenital Syndrome Hyperplasia Congenital Abnormalities Adrenal Gland Diseases Adrenocortical Hyperfunction Pathologic Processes Disorders of Sex Development |
Urogenital Abnormalities Genetic Diseases, Inborn Steroid Metabolism, Inborn Errors Metabolism, Inborn Errors Metabolic Diseases Endocrine System Diseases Gonadal Disorders |