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Trial record 1 of 1 for:    CAH3003 | CAH - Congenital Adrenal Hyperplasia
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Global Safety and Efficacy Registration Study of Crinecerfont for Congenital Adrenal Hyperplasia (CAHtalyst)

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ClinicalTrials.gov Identifier: NCT04490915
Recruitment Status : Recruiting
First Posted : July 29, 2020
Last Update Posted : July 14, 2021
Sponsor:
Information provided by (Responsible Party):
Neurocrine Biosciences

Tracking Information
First Submitted Date  ICMJE July 24, 2020
First Posted Date  ICMJE July 29, 2020
Last Update Posted Date July 14, 2021
Actual Study Start Date  ICMJE July 23, 2020
Estimated Primary Completion Date February 2023   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: July 24, 2020)
Percent change from baseline in glucocorticoid daily dose at Week 24 [ Time Frame: Baseline and Week 24 ]
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: April 1, 2021)
  • 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 at Week 24 [ Time Frame: Baseline and Week 24 ]
Original Secondary Outcome Measures  ICMJE
 (submitted: July 24, 2020)
  • 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 ]
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Global Safety and Efficacy Registration Study of Crinecerfont for Congenital Adrenal Hyperplasia
Official Title  ICMJE 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
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 subjects 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 treatment with crinecerfont. Duration of participation is approximately 20 months.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple (Participant, Care Provider, Investigator)
Primary Purpose: Treatment
Condition  ICMJE Congenital Adrenal Hyperplasia
Intervention  ICMJE
  • Drug: Crinecerfont
    CRF1-receptor antagonist
    Other Name: NBI-74788
  • Drug: Placebo
    Non-active dosage form
Study Arms  ICMJE
  • Experimental: Crinecerfont
    Capsule, administered orally, twice daily for 24 weeks, followed by active treatment for 1 year.
    Intervention: Drug: Crinecerfont
  • Placebo Comparator: Placebo
    Capsule, administered orally, twice daily for 24 weeks, followed by active treatment for 1 year.
    Interventions:
    • Drug: Crinecerfont
    • Drug: Placebo
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: July 24, 2020)
165
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE February 2024
Estimated Primary Completion Date February 2023   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  1. Be willing and able to adhere to the study procedures, including all requirements at the study center and return for the follow-up visit.
  2. Have a medically confirmed diagnosis of classic 21-hydroxylase deficiency CAH.
  3. Be on a stable regimen of steroidal treatment for CAH.
  4. Patients of childbearing potential must agree to use hormonal or two forms of nonhormonal contraception (dual contraception) or other highly effective contraception during the study.

Exclusion Criteria:

  1. Have a diagnosis of any of the other known forms of classic CAH.
  2. Have a history of bilateral adrenalectomy, hypopituitarism, or other condition requiring chronic glucocorticoid therapy.
  3. Have a clinically significant unstable medical condition or chronic disease other than CAH.
  4. Have a history of cancer unless considered cured.
  5. Are pregnant.
  6. Have a known history of clinically significant arrhythmia or abnormalities on ECG.
  7. Have a known hypersensitivity to any corticotropin releasing hormone antagonists.
  8. 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.
  9. Have current substance dependence, or current substance (drug) or alcohol abuse.
  10. Have had a blood loss ≥550 mL or donated blood or blood products within 8 weeks prior to the study.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Cheryl Chen 858-617-7744 cechen@neurocrine.com
Contact: Szecho Lin 858-617-7192 slin@neurocrine.com
Listed Location Countries  ICMJE Austria,   Belgium,   Bulgaria,   Canada,   Czechia,   France,   Germany,   Greece,   Italy,   Poland,   Portugal,   Spain,   Sweden,   United Kingdom,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT04490915
Other Study ID Numbers  ICMJE NBI-74788-CAH3003
2019-004873-17 ( EudraCT Number )
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party Neurocrine Biosciences
Study Sponsor  ICMJE Neurocrine Biosciences
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account Neurocrine Biosciences
Verification Date July 2021

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP