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

Sponsor
Neurocrine Biosciences (Industry)
Overall Status
Recruiting
CT.gov ID
NCT04490915
Collaborator
(none)
165
Enrollment
63
Locations
2
Arms
42.3
Anticipated Duration (Months)
2.6
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 24 weeks in approximately 165 adult subjects with classic CAH due to 21-hydroxylase deficiency. The study consists of a 6 month randomized, double blind, placebo-controlled period, followed by 1 year of treatment with crinecerfont. Duration of participation is approximately 20 months.

Condition or Disease Intervention/Treatment Phase
Phase 3

Study Design

Study Type:
Interventional
Anticipated Enrollment :
165 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 Adult Subjects With Classic Congenital Adrenal Hyperplasia, Followed by Open-Label Treatment
Actual Study Start Date :
Jul 23, 2020
Anticipated Primary Completion Date :
Feb 1, 2023
Anticipated Study Completion Date :
Feb 1, 2024

Arms and Interventions

Arm Intervention/Treatment
Experimental: Crinecerfont

Capsule, administered orally, twice daily for 24 weeks, followed by active treatment for 1 year.

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

    		•
    Placebo Comparator: Placebo

    Capsule, administered orally, twice daily for 24 weeks, followed by active treatment for 1 year.

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

    • Drug: Placebo
    Non-active dosage form

    Outcome Measures

    Primary Outcome Measures

    1. Percent change from baseline in glucocorticoid daily dose at Week 24 [Baseline and Week 24]

    Secondary Outcome Measures

    1. Change from baseline in serum androstenedione at Week 4 [Baseline and Week 4]

    2. Achievement of a reduction in glucocorticoid daily dose to physiologic levels at Week 24 [Baseline and Week 24]

    3. Change from baseline in homeostatic model assessment of insulin resistance (HOMA-IR) index at Week 24 [Baseline and Week 24]

    4. Change from baseline in body weight at Week 24 [Baseline and Week 24]

    5. Change from baseline in fat mass at Week 24 [Baseline and Week 24]

    6. Change from baseline in blood pressure at Week 24 [Baseline and Week 24]

    7. Change from baseline in glucose tolerance at Week 24 [Baseline and Week 24]

    8. Change from baseline in waist circumference at Week 24 [Baseline and Week 24]

    9. Change from baseline in menstrual regularity at Week 24 [Baseline and Week 24]

    10. Change from baseline in testicular adrenal rest tumor at Week 24 [Baseline and Week 24]

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    18 Years and Older
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    No
    Inclusion Criteria:

    1. Be willing and able to adhere to the study procedures, including all requirements at the study center and return for the follow-up visit.

    2. Have a medically confirmed diagnosis of classic 21-hydroxylase deficiency CAH.

    3. Be on a stable regimen of steroidal treatment for CAH.

    4. Patients of childbearing potential must agree to use hormonal or two forms of nonhormonal contraception (dual contraception) or other highly effective contraception during the study.

    Exclusion Criteria:

    1. Have a diagnosis of any of the other known forms of classic CAH.

    2. Have a history of bilateral adrenalectomy, hypopituitarism, or other condition requiring chronic glucocorticoid therapy.

    3. Have a clinically significant unstable medical condition or chronic disease other than CAH.

    4. Have a history of cancer unless considered cured.

    5. Are pregnant.

    6. Have a known history of clinically significant arrhythmia or abnormalities on ECG.

    7. Have a known hypersensitivity to any corticotropin releasing hormone antagonists.

    8. 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.

    9. Have current substance dependence, or current substance (drug) or alcohol abuse.

    10. Have had a blood loss ≥550 mL 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 Pasadena California United States 91105
    3 Neurocrine Clinical Site San Diego California United States 92123
    4 Neurocrine Clinical Site San Francisco California United States 94143
    5 Neurocrine Clinical Site Aurora Colorado United States 80045
    6 Neurocrine Clinical Site Atlanta Georgia United States 30329
    7 Neurocrine Clinical Site Indianapolis Indiana United States 46202
    8 Neurocrine Clinical Site Boston Massachusetts United States 02115
    9 Neurocrine Clinical Site Ann Arbor Michigan United States 48109
    10 Neurocrine Clinical Site Minneapolis Minnesota United States 55454
    11 Neurocrine Clinical Site Rochester Minnesota United States 55905
    12 Neurocrine Clinical Site Saint Louis Missouri United States 63110
    13 Neurocrine Clinical Site Great Neck New York United States 11021
    14 Neurocrine Clinical Site New York New York United States 10029
    15 Neurocrine Clinical Site Hickory North Carolina United States 28601
    16 Neurocrine Clinical Site Winston-Salem North Carolina United States 27157
    17 Neurocrine Clinical Site Oklahoma City Oklahoma United States 73104
    18 Neurocrine Clinical Site Tulsa Oklahoma United States 74135
    19 Neurocrine Clinical Site Philadelphia Pennsylvania United States 19104
    20 Neurocrine Clinical Site Pittsburgh Pennsylvania United States 15224
    21 Neurocrine Clinical Site Dallas Texas United States 75390
    22 Neurocrine Clinical Site Seattle Washington United States 98105
    23 Neurocrine Clinical Site Graz Austria 8036
    24 Neurocrine Clinical Site Vienna Austria 1090
    25 Neurocrine Clinical Site Brussels Belgium 1070
    26 Neurocrine Clinical Site Leuven Belgium 3000
    27 Neurocrine Clinical Site Sofia Bulgaria 1431
    28 Neurocrine Clinical Site Sofia Bulgaria 1606
    29 Neurocrine Clinical Site Halifax Nova Scotia Canada B3H 2Y9
    30 Neurocrine Clinical Site Hradec Králové Czechia 50005
    31 Neurocrine Clinical Site Angers France 49933
    32 Neurocrine Clinical Site Grenoble France 38700
    33 Neurocrine Clinical Site Le Kremlin-Bicêtre France 94270
    34 Neurocrine Clinical Site Nantes France 44093
    35 Neurocrine Clinical Site Paris France 75651
    36 Neurocrine Clinical Site Paris France 75679
    37 Neurocrine Clinical Site Dresden Germany 01307
    38 Neurocrine Clinical Site Essen Germany 45147
    39 Neurocrine Clinical Site Frankfurt Germany 60590
    40 Neurocrine Clinical Site Leipzig Germany 04103
    41 Neurocrine Clinical Site Munich Germany 80336
    42 Neurocrine Clinical Site Athens Greece 106 76
    43 Neurocrine Clinical Site Athens Greece 115 27
    44 Neurocrine Clinical Site Athens Greece 11527
    45 Neurocrine Clinical Site Thessaloníki Greece 54642
    46 Neurocrine Clinical Site Florence Italy 50139
    47 Neurocrine Clinical Site Milan Italy 20132
    48 Neurocrine Clinical Site Milan Italy 20149
    49 Neurocrine Clinical Site Naples Italy 80131
    50 Neurocrine Clinical Site Padova Italy 35128
    51 Neurocrine Clinical Site Roma Italy 00161
    52 Neurocrine Clinical Site Leiden Netherlands 2333
    53 Neurocrine Clinical Site Kraków Poland 31-011
    54 Neurocrine Clinical Site Poznań Poland 60-355
    55 Neurocrine Clinical Site Warszawa Poland 01-809
    56 Neurocrine Clinical Site Porto Portugal 4200-319
    57 Neurocrine Clinical Site Madrid Spain 28034
    58 Neurocrine Clinical Site Sevilla Spain 41013
    59 Neurocrine Clinical Site Gothenburg Sweden 41345
    60 Neurocrine Clinical Site Stockholm Sweden 17176
    61 Neurocrine Clinical Site Cardiff United Kingdom CF14 4HH
    62 Neurocrine Clinical Site London United Kingdom WC1B 5EH
    63 Neurocrine Clinical Site Salford United Kingdom M6 8HD

    Sponsors and Collaborators

    • Neurocrine Biosciences

    Investigators

    None specified.

    Study Documents (Full-Text)

    None provided.

    More Information

    Additional Information:

    Publications

    None provided.
    Responsible Party:
    Neurocrine Biosciences
    ClinicalTrials.gov Identifier:
    NCT04490915
    Other Study ID Numbers:
    • NBI-74788-CAH3003
    • 2019-004873-17
    First Posted:
    Jul 29, 2020
    Last Update Posted:
    Apr 11, 2022
    Last Verified:
    Apr 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
    Additional relevant MeSH terms:
    Adrenal Hyperplasia, Congenital
    Adrenogenital Syndrome
    Adrenocortical Hyperfunction
    Hyperplasia

    Study Results

    No Results Posted as of Apr 11, 2022