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

Sponsor
Neurocrine Biosciences (Industry)
Overall Status
Recruiting
CT.gov ID
NCT04806451
Collaborator
(none)
81
Enrollment
36
Locations
2
Arms
33.2
Anticipated Duration (Months)
2.3
Patients Per Site
0.1
Patients Per Site Per Month

Study Details

Study Description

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 patients 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 treatment with crinecerfont. Duration of participation is approximately 14 months.

Condition or Disease Intervention/Treatment Phase
Phase 3

Study Design

Study Type:
Interventional
Anticipated Enrollment :
81 participants
Allocation:
Randomized
Intervention Model:
Parallel Assignment
Masking:
Triple (Participant, Care Provider, Investigator)
Primary Purpose:
Treatment
Official 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
Actual Study Start Date :
Jun 24, 2021
Anticipated Primary Completion Date :
Apr 1, 2023
Anticipated Study Completion Date :
Apr 1, 2024

Arms and Interventions

Arm Intervention/Treatment
Experimental: Crinecerfont

Solution or capsule, administered orally, twice daily for 28 weeks, followed by active treatment for 24 weeks.

Drug: Crinecerfont
CRF1-receptor antagonist
Other Names:
  • NBI-74788

    		•
    Placebo Comparator: Placebo

    Solution or capsule, administered orally, twice daily for 28 weeks, followed by active treatment for 24 weeks.

    Drug: Placebo
    Non-active dosage form

    Drug: Crinecerfont
    CRF1-receptor antagonist
    Other Names:
  • NBI-74788

    		•

    Outcome Measures

    Primary Outcome Measures

    1. Change from Baseline in Serum Androstenedione (A4) at Week 4 [Baseline to Week 4]

    Secondary Outcome Measures

    1. Change from Baseline in Serum 17-hydroxyprogesterone (17-OHP) at Week 4 [Baseline to Week 4]

    2. Percent Change from Baseline in Glucocorticoid Daily Dose at Week 28 [Baseline to Week 28]

    3. Achievement of a reduction in glucocorticoid daily dose to physiologic levels at Week 28 [Baseline to Week 28]

    4. Change from baseline in body mass index at Week 28 [Baseline to Week 28]

    5. Change from baseline in salivary 17-OHP at Week 28 [Baseline to Week 28]

    6. Change in bone age advancement at Week 28 [Baseline to Week 28]

    7. Change from baseline in predicted adult height at Week 52 [Baseline to Week 52]

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    2 Years to 17 Years
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    No
    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.

    • Patients 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.

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 Neurocrine Clinical Site Los Angeles California United States 90027
    2 Neurocrine Clinical Site San Diego California United States 92123
    3 Neurocrine Clinical Site San Francisco California United States 94158
    4 Neurocrine Clinical Site Aurora Colorado United States 80045
    5 Neurocrine Clinical Site Hartford Connecticut United States 06106
    6 Neurocrine Clinical Site Washington District of Columbia United States 20010
    7 Neurocrine Clinical Site Atlanta Georgia United States 30329
    8 Neurocrine Clinical Site Indianapolis Indiana United States 46202
    9 Neurocrine Clinical Site Boston Massachusetts United States 02115
    10 Neurocrine Clinical Site Ann Arbor Michigan United States 48109
    11 Neurocrine Clinical Site Minneapolis Minnesota United States 55454
    12 Neurocrine Clinical Site New Hyde Park New York United States 11040
    13 Neurocrine Clinical Site New York New York United States 10065
    14 Neurocrine Clinical Site Oklahoma City Oklahoma United States 73104
    15 Neurocrine Clinical Site Tulsa Oklahoma United States 74135
    16 Neurocrine Clinical Site Philadelphia Pennsylvania United States 19104
    17 Neurocrine Clinical Site Pittsburgh Pennsylvania United States 15224
    18 Neurocrine Clinical Site Dallas Texas United States 75235
    19 Neurocrine Clinical Site Seattle Washington United States 98105
    20 Neurocrine Clinical Site Brussels Belgium 1200
    21 Neurocrine Clinical Site Ghent Belgium 9000
    22 Neurocrine Clinical Site Edmonton Alberta Canada T6G 1C9
    23 Neurocrine Clinical Site Vancouver British Columbia Canada V6H 3V4
    24 Neurocrine Clinical Site Montréal Quebec Canada H3T 1C5
    25 Neurocrine Clinical Site Angers France 49933
    26 Neurocrine Clinical Site Bordeau France 33076
    27 Neurocrine Clinical Site Le Kremlin-Bicêtre France 94270
    28 Neurocrine Clinical Site Paris France 75019
    29 Neurocrine Clinical Site Athens Greece 115 27
    30 Neurocrine Clinical Site Athens Greece 11527
    31 Neurocrine Clinical Site Bologna Italy 40138
    32 Neurocrine Clinical Site Milan Italy 20132
    33 Neurocrine Clinical Site Gdańsk Poland 80-214
    34 Neurocrine Clinical Site Rzeszów Poland 35-301
    35 Neurocrine Clinical Site Barcelona Spain 08035
    36 Neurocrine Clinical Site Sevilla Spain 41013

    Sponsors and Collaborators

    • Neurocrine Biosciences

    Investigators

    • Study Director: Clinical Development Lead, Neurocrine Biosciences

    Study Documents (Full-Text)

    None provided.

    More Information

    Additional Information:

    Publications

    None provided.
    Responsible Party:
    Neurocrine Biosciences
    ClinicalTrials.gov Identifier:
    NCT04806451
    Other Study ID Numbers:
    • NBI-74788-CAH2006
    • 2020-004381-19
    First Posted:
    Mar 19, 2021
    Last Update Posted:
    Jun 30, 2022
    Last Verified:
    Jun 1, 2022
    Individual Participant Data (IPD) Sharing Statement:
    No
    Plan to Share IPD:
    No
    Studies a U.S. FDA-regulated Drug Product:
    Yes
    Studies a U.S. FDA-regulated Device Product:
    No
    Keywords provided by Neurocrine Biosciences
    classic congenital adrenal hyperplasia (CAH)
    21-hydroxylase deficiency
    Additional relevant MeSH terms:
    Adrenal Hyperplasia, Congenital
    Adrenogenital Syndrome
    Adrenocortical Hyperfunction
    Hyperplasia

    Study Results

    No Results Posted as of Jun 30, 2022