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NCT Number: NCT04806451

Global Safety and Efficacy Registration Study of Crinecerfont in Pediatric Participants With Classic Congenital Adrenal Hyperplasia (CAHtalyst Pediatric Study)

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).

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Key information

Conditions

Congenital Adrenal Hyperplasia Adrenal Gland Diseases Adrenal Hyperplasia, Congenital Adrenogenital Syndrome Congenital Abnormalities Congenital adrenal hyperplasia due to 21 hydroxylase deficiency 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

2 year–17 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

Neurocrine Clinical Site, Brussels, Belgium

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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.
  • 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 electrocardiogram (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.

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. Change From Baseline in Serum Androstenedione at Week 4

    Time frame: Baseline, Week 4

    Blood serum samples were collected for the analysis of serum androstenedione concentrations. Least square (LS) mean and standard error (SE) were calculated using analysis of covariance (ANCOVA) model.

Secondary outcomes

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

    Time frame: Baseline, Week 4

  2. Percent Change From Baseline in Glucocorticoid Daily Dose at Week 28

    Time frame: Baseline, Week 28

  3. Number of Participants Who Achieved a Reduction to Physiologic Glucocorticoid Dose While Maintaining Androstenedione Control at Week 28

    Time frame: Week 28

  4. Change From Baseline in Body Mass Index (BMI) Standard Deviation Score (SDS) at Week 28

    Time frame: Baseline, Week 28

  5. Change From Baseline in Mean 24-hour Salivary 17-OHP at Week 28

    Time frame: Baseline, Week 28

  6. Change From Baseline in the Ratio of Bone Age to Chronological Age (BA:CA) at Week 28

    Time frame: Baseline, Week 28

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 Pediatric Subjects With Classic Congenital Adrenal Hyperplasia, Followed by Open-Label Treatment

Important dates

Study start
2021
Primary completion
2023
Study completion
2027
First posted
Mar 19, 2021
Registry last updated
Feb 5, 2025

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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