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NCT Number: NCT04490915

Global Safety and Efficacy Registration Study of Crinecerfont for Congenital Adrenal Hyperplasia

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 24-week randomized, double-blind, placebo-controlled period, followed by 1 year of active 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 participant for the OLE (estimated to be approximately 3 years).

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Key information

Conditions

Congenital Adrenal Hyperplasia Adrenal Gland Diseases Adrenal Hyperplasia, Congenital Adrenogenital Syndrome Congenital Abnormalities Congenital, Hereditary, and Neonatal Diseases and Abnormalities Disorders of Sex Development Endocrine System Diseases Female Urogenital Diseases Female Urogenital Diseases and Pregnancy Complications Genetic Diseases, Inborn Gonadal Disorders Male Urogenital Diseases Metabolic Diseases Metabolism, Inborn Errors Nutritional and Metabolic Diseases Steroid Metabolism, Inborn Errors Urogenital Abnormalities Urogenital Diseases

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

Neurocrine Clinical Site, Graz, Austria

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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 CAH due to 21-hydroxylase deficiency.
  • Be on a stable steroid regimen.
  • 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 receptor 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.

Treatment and study plan

Crinecerfont

Drug

CRF type 1 receptor antagonist

Other names: NBI-74788, Crenessity

Placebo

Drug

Non-active dosage form

Primary outcomes

  1. Percent Change From Baseline in Glucocorticoid Daily Dose at Week 24

    Time frame: Baseline, Week 24

    Least square (LS) mean and standard error (SE) were calculated using analysis of covariance (ANCOVA) model.

Secondary outcomes

  1. Change From Baseline in Serum Androstenedione at Week 4

    Time frame: Baseline, Week 4

  2. Number of Participants Who Achieved a Reduction to Physiologic Glucocorticoid Dose While Maintaining Androstenedione Control at Week 24

    Time frame: Week 24

  3. Change From Baseline in Homeostatic Model Assessment of Insulin Resistance (HOMA-IR) at Week 24

    Time frame: Baseline, Week 24

  4. Percent Change From Baseline in Body Weight at Week 24

    Time frame: Baseline, Week 24

  5. Change From Baseline in Percent Total Fat Mass at Week 24

    Time frame: Baseline, Week 24

  6. Change From Baseline in Serum 17-hydroxyprogesterone (17-OHP) at Week 4

    Time frame: Baseline, Week 4

  7. Change From Baseline in Blood Pressure at Week 24

    Time frame: Baseline, Week 24

  8. Change From Baseline in Glucose Tolerance at Week 24

    Time frame: Baseline, Week 24

  9. Change From Baseline in Waist Circumference at Week 24

    Time frame: Baseline, Week 24

  10. Change From Baseline in Menstrual Regularity at Week 24

    Time frame: Baseline, Week 24

  11. Change From Baseline in Testicular Adrenal Rest Tumor (TART) Volume at Week 24

    Time frame: Baseline, Week 24

Sponsors and collaborators

Lead sponsor

Neurocrine Biosciences

Industry

Registry information

Official study 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

Acronym: CAHtalyst

Important dates

Study start
2020
Primary completion
2023
Study completion
2027
First posted
Jul 29, 2020
Registry last updated
May 11, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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