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

NCT04806451 | Active Not Recruiting Phase 3 Results DMC FDA Drug

• 3 other identifiers: NBI-74788-CAH20062020-004381-192023-509170-33-00
• Study Type: interventional
• Target: 103
• Locations: 10 countries
• Sites: 46

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 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).

Conditions

Congenital Adrenal Hyperplasia

Keywords

classic congenital adrenal hyperplasia (CAH); 21-hydroxylase deficiency

Outcome Measures

Primary Outcomes (1)

• Change From Baseline in Serum Androstenedione at 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.

  Baseline, Week 4

Secondary Outcomes (6)

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

  Baseline, Week 4

• Percent Change From Baseline in Glucocorticoid Daily Dose at Week 28

  Baseline, Week 28

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

  Week 28

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

  Baseline, Week 28

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

  Baseline, Week 28

Study Arms (2)

Crinecerfont

EXPERIMENTAL

Crinecerfont solution or capsule, administered orally, twice daily for 28 weeks during the placebo-controlled treatment period, followed by active treatment with crinecerfont for at least 24 weeks.

Drug: Crinecerfont

Placebo

PLACEBO COMPARATOR

Placebo solution or capsule, administered orally, twice daily for 28 weeks, followed by active treatment with crinecerfont for at least 24 weeks.

Drug: Crinecerfont; Drug: Placebo

Interventions

Crinecerfont DRUG

CRF type 1 receptor antagonist

Also known as: NBI-74788, Crenessity

Crinecerfont; Placebo

Placebo DRUG

Non-active dosage form

Placebo

Eligibility Criteria

• Age: 2 Years - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Neurocrine Biosciences: lead

Study Sites (46)

Neurocrine Clinical Site

Birmingham, Alabama, 35233, United States

Location

Neurocrine Clinical Site

Los Angeles, California, 90027, United States

Location

Neurocrine Clinical Site

Orange, California, 92868, United States

Location

Neurocrine Clinical Site

San Diego, California, 92123, United States

Location

Neurocrine Clinical Site

San Francisco, California, 94158, United States

Location

Neurocrine Clinical Site

Aurora, Colorado, 80045, United States

Location

Neurocrine Clinical Site

Hartford, Connecticut, 06106, United States

Location

Neurocrine Clinical Site

Washington D.C., District of Columbia, 20010, United States

Location

Neurocrine Clinical Site

Atlanta, Georgia, 30329, United States

Location

Neurocrine Clinical Site

Indianapolis, Indiana, 46202, United States

Location

Neurocrine Clinical Site

Boston, Massachusetts, 02115, United States

Location

Neurocrine Clinical Site

Ann Arbor, Michigan, 48109, United States

Location

Neurocrine Clinical Site

Minneapolis, Minnesota, 55454, United States

Location

Neurocrine Clinical site

St Louis, Missouri, 63104, United States

Location

Neurocrine Clinical Site

New Hyde Park, New York, 11040, United States

Location

Neurocrine Clinical Site

New York, New York, 10065, United States

Location

Neurocrine Clinical Site

Oklahoma City, Oklahoma, 73104, United States

Location

Neurocrine Clinical Site

Tulsa, Oklahoma, 74135, United States

Location

Neurocrine Clinical Site

Philadelphia, Pennsylvania, 19104, United States

Location

Neurocrine Clinical Site

Pittsburgh, Pennsylvania, 15224, United States

Location

Neurocrine Clinical Site

Dallas, Texas, 75235, United States

Location

Neurocrine Clinical Site

Seattle, Washington, 98105, United States

Location

Neurocrine Clinical Site

Brussels, 1200, Belgium

Location

Neurocrine Clinical Site

Ghent, 9000, Belgium

Location

Neurocrine Clinical Site

Edmonton, Alberta, T6G 1C9, Canada

Location

Neurocrine Clinical Site

Vancouver, British Columbia, V6H 3V4, Canada

Location

Neurocrine Clinical Site

Montreal, Quebec, H3T 1C5, Canada

Location

Neurocrine Clinical Site

Angers, 49933, France

Location

Neurocrine Clinical Site

Bordeau, 33076, France

Location

Neurocrine Clinical Site

Le Kremlin-Bicêtre, 94270, France

Location

Neurocrine Clinical Site

Paris, 75015, France

Location

Neurocrine Clinical Site

Paris, 75019, France

Location

Neurocrine Clinical Site

Berlin, 13353, Germany

Location

Neurocrine Clinical Site

Heidelberg, 69120, Germany

Location

Neurocrine Clinical Site

Magdeburg, 39120, Germany

Location

Neurocrine Clinical Site

Athens, 115 27, Greece

Location

Neurocrine Clinical Site

Athens, 11527, Greece

Location

Neurocrine Clinical Site

Bologna, 40138, Italy

Location

Neurocrine Clinical Site

Milan, 20132, Italy

Location

Neurocrine Clinical Site

Napoli, 80131, Italy

Location

Neurocrine Clinical Site

Roma, 00165, Italy

Location

Neurocrine Clinical Site

Gdansk, 80-214, Poland

Location

Neurocrine Clinical Site

Rzeszów, 35-301, Poland

Location

Neurocrine Clinical Site

Barcelona, 08035, Spain

Location

Neurocrine Clinical Site

Seville, 41013, Spain

Location

Neurocrine Clinical Site

London, WC1N 3JH, United Kingdom

Location

Related Publications (1)

• Sarafoglou K, Kim MS, Lodish M, Felner EI, Martinerie L, Nokoff NJ, Clemente M, Fechner PY, Vogiatzi MG, Speiser PW, Auchus RJ, Rosales GBG, Roberts E, Jeha GS, Farber RH, Chan JL; CAHtalyst Pediatric Trial Investigators. Phase 3 Trial of Crinecerfont in Pediatric Congenital Adrenal Hyperplasia. N Engl J Med. 2024 Aug 8;391(6):493-503. doi: 10.1056/NEJMoa2404655. Epub 2024 Jun 2.

  PMID: 38828945 DERIVED

MeSH Terms

Conditions

Adrenal Hyperplasia, Congenital; Congenital adrenal hyperplasia due to 21 hydroxylase deficiency

Interventions

crinecerfont

Condition Hierarchy (Ancestors)

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

Results Point of Contact

• Title: Neurocrine Medical Information Call Center
• Organization: Neurocrine Biosciences

medinfo@neurocrine.com

877-641-3461

Study Officials

• Clinical Development Lead

  Neurocrine Biosciences

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 16, 2021
• First Posted: March 19, 2021
• Study Start: June 25, 2021
• Primary Completion: March 10, 2023
• Study Completion (Estimated): August 1, 2027
• Last Updated: February 5, 2025
• Results First Posted: February 5, 2025

Record last verified: 2025-01

Data Sharing

• IPD Sharing: Will not share

Locations

PL (2); US (22); BE (2); CA (3); IT (4); GB (1); ES (2); FR (5); GR (2); DE (3)