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

March 5, 2024 updated by: Neurocrine Biosciences

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

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).

Study Overview

Status

Active, not recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

103

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Belgium
      • Brussels, Belgium, 1200
        • Neurocrine Clinical Site
      • Ghent, Belgium, 9000
        • Neurocrine Clinical Site
  • Canada
    • Alberta
      • Edmonton, Alberta, Canada, T6G 1C9
        • Neurocrine Clinical Site
    • British Columbia
      • Vancouver, British Columbia, Canada, V6H 3V4
        • Neurocrine Clinical Site
    • Quebec
      • Montréal, Quebec, Canada, H3T 1C5
        • Neurocrine Clinical Site
  • France
      • Angers, France, 49933
        • Neurocrine Clinical Site
      • Bordeau, France, 33076
        • Neurocrine Clinical Site
      • Le Kremlin-Bicêtre, France, 94270
        • Neurocrine Clinical Site
      • Paris, France, 75015
        • Neurocrine Clinical Site
      • Paris, France, 75019
        • Neurocrine Clinical Site
  • Germany
      • Berlin, Germany, 13353
        • Neurocrine Clinical Site
      • Heidelberg, Germany, 69120
        • Neurocrine Clinical Site
      • Magdeburg, Germany, 39120
        • Neurocrine Clinical Site
  • Greece
      • Athens, Greece, 115 27
        • Neurocrine Clinical Site
      • Athens, Greece, 11527
        • Neurocrine Clinical Site
  • Italy
      • Bologna, Italy, 40138
        • Neurocrine Clinical Site
      • Milan, Italy, 20132
        • Neurocrine Clinical Site
      • Napoli, Italy, 80131
        • Neurocrine Clinical Site
      • Roma, Italy, 00165
        • Neurocrine Clinical Site
  • Poland
      • Gdańsk, Poland, 80-214
        • Neurocrine Clinical Site
      • Rzeszów, Poland, 35-301
        • Neurocrine Clinical Site
  • Spain
      • Barcelona, Spain, 08035
        • Neurocrine Clinical Site
      • Sevilla, Spain, 41013
        • Neurocrine Clinical Site
  • United Kingdom
      • London, United Kingdom, WC1N 3JH
        • Neurocrine Clinical Site
  • United States
    • Alabama
      • Birmingham, Alabama, United States, 35233
        • Neurocrine Clinical Site
    • California
      • Los Angeles, California, United States, 90027
        • Neurocrine Clinical Site
      • Orange, California, United States, 92868
        • Neurocrine Clinical Site
      • San Diego, California, United States, 92123
        • Neurocrine Clinical Site
      • San Francisco, California, United States, 94158
        • Neurocrine Clinical Site
    • Colorado
      • Aurora, Colorado, United States, 80045
        • Neurocrine Clinical Site
    • Connecticut
      • Hartford, Connecticut, United States, 06106
        • Neurocrine Clinical Site
    • District of Columbia
      • Washington, District of Columbia, United States, 20010
        • Neurocrine Clinical Site
    • Georgia
      • Atlanta, Georgia, United States, 30329
        • Neurocrine Clinical Site
    • Indiana
      • Indianapolis, Indiana, United States, 46202
        • Neurocrine Clinical Site
    • Massachusetts
      • Boston, Massachusetts, United States, 02115
        • Neurocrine Clinical Site
    • Michigan
      • Ann Arbor, Michigan, United States, 48109
        • Neurocrine Clinical Site
    • Minnesota
      • Minneapolis, Minnesota, United States, 55454
        • Neurocrine Clinical Site
    • Missouri
      • Saint Louis, Missouri, United States, 63104
        • Neurocrine Clinical Site
    • New York
      • New Hyde Park, New York, United States, 11040
        • Neurocrine Clinical Site
      • New York, New York, United States, 10065
        • Neurocrine Clinical Site
    • Oklahoma
      • Oklahoma City, Oklahoma, United States, 73104
        • Neurocrine Clinical Site
      • Tulsa, Oklahoma, United States, 74135
        • Neurocrine Clinical Site
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19104
        • Neurocrine Clinical Site
      • Pittsburgh, Pennsylvania, United States, 15224
        • Neurocrine Clinical Site
    • Texas
      • Dallas, Texas, United States, 75235
        • Neurocrine Clinical Site
    • Washington
      • Seattle, Washington, United States, 98105
        • Neurocrine Clinical Site

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

2 years to 17 years (Child)

Accepts Healthy Volunteers

No

Description

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.
  • 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 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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Triple

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Crinecerfont
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
CRF1-receptor antagonist
Other Names:
  • NBI-74788
Placebo Comparator: Placebo
Placebo solution or capsule, administered orally, twice daily for 28 weeks, followed by active treatment with crinecerfont for at least 24 weeks.
Drug: Crinecerfont
CRF1-receptor antagonist
Other Names:
  • NBI-74788
Drug: Placebo
Non-active dosage form

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Change from Baseline in Serum Androstenedione (A4) at Week 4
Time Frame: Baseline to Week 4
Baseline to Week 4

Secondary Outcome Measures

Outcome Measure
Time Frame
Change from Baseline in Serum 17-hydroxyprogesterone (17-OHP) at Week 4
Time Frame: Baseline to Week 4
Baseline to Week 4
Percent Change from Baseline in Glucocorticoid Daily Dose at Week 28
Time Frame: Baseline to Week 28
Baseline to Week 28
Achievement of a reduction in glucocorticoid daily dose to physiologic levels at Week 28
Time Frame: Baseline to Week 28
Baseline to Week 28
Change from baseline in body mass index at Week 28
Time Frame: Baseline to Week 28
Baseline to Week 28
Change from baseline in salivary 17-OHP at Week 28
Time Frame: Baseline to Week 28
Baseline to Week 28
Change in bone age advancement at Week 28
Time Frame: Baseline to Week 28
Baseline to Week 28
Change from baseline in predicted adult height at Week 52
Time Frame: Baseline to Week 52
Baseline to Week 52

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Neurocrine Biosciences

Investigators

  • Study Director: Clinical Development Lead, Neurocrine Biosciences

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

June 24, 2021

Primary Completion (Actual)

March 10, 2023

Study Completion (Estimated)

August 1, 2027

Study Registration Dates

First Submitted

March 16, 2021

First Submitted That Met QC Criteria

March 16, 2021

First Posted (Actual)

March 19, 2021

Study Record Updates

Last Update Posted (Actual)

March 7, 2024

Last Update Submitted That Met QC Criteria

March 5, 2024

Last Verified

March 1, 2024

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • NBI-74788-CAH2006
  • 2020-004381-19 (EudraCT Number)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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