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

Primary Purpose

Congenital Adrenal Hyperplasia

Status
Active
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
Crinecerfont
Placebo
Sponsored by
Neurocrine Biosciences
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Congenital Adrenal Hyperplasia

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

  • Neurocrine Clinical Site
  • Neurocrine Clinical Site
  • Neurocrine Clinical Site
  • Neurocrine Clinical Site
  • Neurocrine Clinical Site
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Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Placebo Comparator

Arm Label

Crinecerfont

Placebo

Arm Description

Crinecerfont capsule, administered orally, twice daily for 24 weeks during the placebo-controlled treatment period, followed by active treatment with crinecerfont for at least 1 year.

Placebo capsule, administered orally, twice daily for 24 weeks, followed by active treatment with crinecerfont for at least 1 year.

Outcomes

Primary Outcome Measures

Percent change from baseline in glucocorticoid daily dose at Week 24

Secondary Outcome Measures

Change from baseline in serum androstenedione at Week 4
Achievement of a reduction in glucocorticoid daily dose to physiologic levels at Week 24
Change from baseline in homeostatic model assessment of insulin resistance (HOMA-IR) index at Week 24
Change from baseline in body weight at Week 24
Change from baseline in fat mass at Week 24
Change from baseline in blood pressure at Week 24
Change from baseline in glucose tolerance at Week 24
Change from baseline in waist circumference at Week 24
Change from baseline in menstrual regularity at Week 24
Change from baseline in testicular adrenal rest tumor size at Week 24

Full Information

First Posted
July 24, 2020
Last Updated
August 17, 2023
Sponsor
Neurocrine Biosciences
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1. Study Identification

Unique Protocol Identification Number
NCT04490915
Brief Title
Global Safety and Efficacy Registration Study of Crinecerfont for Congenital Adrenal Hyperplasia
Acronym
CAHtalyst
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
Study Type
Interventional

2. Study Status

Record Verification Date
August 2023
Overall Recruitment Status
Active, not recruiting
Study Start Date
July 23, 2020 (Actual)
Primary Completion Date
July 19, 2023 (Actual)
Study Completion Date
August 2027 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Neurocrine Biosciences

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. 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 participants 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 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 20 months for the core study and will be a variable amount of time per subject for the OLE (estimated to be approximately 3 years).

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Congenital Adrenal Hyperplasia

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigator
Allocation
Randomized
Enrollment
182 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Crinecerfont
Arm Type
Experimental
Arm Description
Crinecerfont capsule, administered orally, twice daily for 24 weeks during the placebo-controlled treatment period, followed by active treatment with crinecerfont for at least 1 year.
Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Description
Placebo capsule, administered orally, twice daily for 24 weeks, followed by active treatment with crinecerfont for at least 1 year.
Intervention Type
Drug
Intervention Name(s)
Crinecerfont
Other Intervention Name(s)
NBI-74788
Intervention Description
CRF1-receptor antagonist
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
Non-active dosage form
Primary Outcome Measure Information:
Title
Percent change from baseline in glucocorticoid daily dose at Week 24
Time Frame
Baseline and Week 24
Secondary Outcome Measure Information:
Title
Change from baseline in serum androstenedione at Week 4
Time Frame
Baseline and Week 4
Title
Achievement of a reduction in glucocorticoid daily dose to physiologic levels at Week 24
Time Frame
Baseline and Week 24
Title
Change from baseline in homeostatic model assessment of insulin resistance (HOMA-IR) index at Week 24
Time Frame
Baseline and Week 24
Title
Change from baseline in body weight at Week 24
Time Frame
Baseline and Week 24
Title
Change from baseline in fat mass at Week 24
Time Frame
Baseline and Week 24
Title
Change from baseline in blood pressure at Week 24
Time Frame
Baseline and Week 24
Title
Change from baseline in glucose tolerance at Week 24
Time Frame
Baseline and Week 24
Title
Change from baseline in waist circumference at Week 24
Time Frame
Baseline and Week 24
Title
Change from baseline in menstrual regularity at Week 24
Time Frame
Baseline and Week 24
Title
Change from baseline in testicular adrenal rest tumor size at Week 24
Time Frame
Baseline and Week 24

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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 21-hydroxylase deficiency CAH. Be on a stable regimen of steroidal treatment for CAH. Participants of childbearing potential must agree to use an acceptable method of contraception during the study. Exclusion Criteria: Have a diagnosis of any of the other known 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 cured. Are pregnant. Have a known history of clinically significant arrhythmia or abnormalities on ECG. Have a known hypersensitivity to any corticotropin releasing hormone antagonists. 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. Have current substance dependence, or current substance (drug) or alcohol abuse. Have had a blood loss ≥550 mL or donated blood or blood products within 8 weeks prior to the study.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Clinical Development Lead
Organizational Affiliation
Neurocrine Biosciences
Official's Role
Study Director
Facility Information:
Facility Name
Neurocrine Clinical Site
City
Los Angeles
State/Province
California
ZIP/Postal Code
90027
Country
United States
Facility Name
Neurocrine Clinical Site
City
San Diego
State/Province
California
ZIP/Postal Code
92123
Country
United States
Facility Name
Neurocrine Clinical Site
City
San Francisco
State/Province
California
ZIP/Postal Code
94143
Country
United States
Facility Name
Neurocrine Clinical Site
City
Aurora
State/Province
Colorado
ZIP/Postal Code
80045
Country
United States
Facility Name
Neurocrine Clinical Site
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30329
Country
United States
Facility Name
Neurocrine Clinical Site
City
Indianapolis
State/Province
Indiana
ZIP/Postal Code
46202
Country
United States
Facility Name
Neurocrine Clinical Site
City
Bethesda
State/Province
Maryland
ZIP/Postal Code
20982
Country
United States
Facility Name
Neurocrine Clinical Site
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02115
Country
United States
Facility Name
Neurocrine Clinical Site
City
Ann Arbor
State/Province
Michigan
ZIP/Postal Code
48109
Country
United States
Facility Name
Neurocrine Clinical Site
City
Minneapolis
State/Province
Minnesota
ZIP/Postal Code
55454
Country
United States
Facility Name
Neurocrine Clinical Site
City
Rochester
State/Province
Minnesota
ZIP/Postal Code
55905
Country
United States
Facility Name
Neurocrine Clinical Site
City
Saint Louis
State/Province
Missouri
ZIP/Postal Code
63110
Country
United States
Facility Name
Neurocrine Clinical Site
City
Great Neck
State/Province
New York
ZIP/Postal Code
11021
Country
United States
Facility Name
Neurocrine Clinical Site
City
New York
State/Province
New York
ZIP/Postal Code
10029
Country
United States
Facility Name
Neurocrine Clinical Site
City
Winston-Salem
State/Province
North Carolina
ZIP/Postal Code
27157
Country
United States
Facility Name
Neurocrine Clinical Site
City
Tulsa
State/Province
Oklahoma
ZIP/Postal Code
74135
Country
United States
Facility Name
Neurocrine Clinical Site
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19104
Country
United States
Facility Name
Neurocrine Clinical Site
City
Pittsburgh
State/Province
Pennsylvania
ZIP/Postal Code
15224
Country
United States
Facility Name
Neurocrine Clinical Site
City
Dallas
State/Province
Texas
ZIP/Postal Code
75390
Country
United States
Facility Name
Neurocrine Clinical Site
City
Seattle
State/Province
Washington
ZIP/Postal Code
98105
Country
United States
Facility Name
Neurocrine Clinical Site
City
Graz
ZIP/Postal Code
8036
Country
Austria
Facility Name
Neurocrine Clinical Site
City
Vienna
ZIP/Postal Code
1090
Country
Austria
Facility Name
Neurocrine Clinical Site
City
Brussels
ZIP/Postal Code
1070
Country
Belgium
Facility Name
Neurocrine Clinical Site
City
Leuven
ZIP/Postal Code
3000
Country
Belgium
Facility Name
Neurocrine Clinical Site
City
Sofia
ZIP/Postal Code
1431
Country
Bulgaria
Facility Name
Neurocrine Clinical Site
City
Sofia
ZIP/Postal Code
1606
Country
Bulgaria
Facility Name
Neurocrine Clinical Site
City
Halifax
State/Province
Nova Scotia
ZIP/Postal Code
B3H 2Y9
Country
Canada
Facility Name
Neurocrine Clinical Site
City
Hradec Králové
ZIP/Postal Code
50005
Country
Czechia
Facility Name
Neurocrine Clinical Site
City
Angers
ZIP/Postal Code
49933
Country
France
Facility Name
Neurocrine Clinical Site
City
Grenoble
ZIP/Postal Code
38700
Country
France
Facility Name
Neurocrine Clinical Site
City
Le Kremlin-Bicêtre
ZIP/Postal Code
94270
Country
France
Facility Name
Neurocrine Clinical Site
City
Nantes
ZIP/Postal Code
44093
Country
France
Facility Name
Neurocrine Clinical Site
City
Paris
ZIP/Postal Code
75651
Country
France
Facility Name
Neurocrine Clinical Site
City
Paris
ZIP/Postal Code
75679
Country
France
Facility Name
Neurocrine Clinical Site
City
Dresden
ZIP/Postal Code
01307
Country
Germany
Facility Name
Neurocrine Clinical Site
City
Essen
ZIP/Postal Code
45147
Country
Germany
Facility Name
Neurocrine Clinical Site
City
Frankfurt
ZIP/Postal Code
60590
Country
Germany
Facility Name
Neurocrine Clinical Site
City
Leipzig
ZIP/Postal Code
04103
Country
Germany
Facility Name
Neurocrine Clinical Site
City
Munich
ZIP/Postal Code
80336
Country
Germany
Facility Name
Neurocrine Clinical Site
City
Athens
ZIP/Postal Code
106 76
Country
Greece
Facility Name
Neurocrine Clinical Site
City
Athens
ZIP/Postal Code
115 27
Country
Greece
Facility Name
Neurocrine Clinical Site
City
Athens
ZIP/Postal Code
11527
Country
Greece
Facility Name
Neurocrine Clinical Site
City
Thessaloníki
ZIP/Postal Code
54642
Country
Greece
Facility Name
Neurocrine Clinical Site
City
Afula
ZIP/Postal Code
1834111
Country
Israel
Facility Name
Neurocrine Clinical Site
City
Beer Sheva
ZIP/Postal Code
8410101
Country
Israel
Facility Name
Neurocrine Clinical Site
City
Petah Tikva
ZIP/Postal Code
4941480
Country
Israel
Facility Name
Neurocrine Clinical Site
City
Tel Aviv
ZIP/Postal Code
64239
Country
Israel
Facility Name
Neurocrine Clinical Site
City
Ancona
ZIP/Postal Code
60126
Country
Italy
Facility Name
Neurocrine Clinical Site
City
Bologna
ZIP/Postal Code
40138
Country
Italy
Facility Name
Neurocrine Clinical Site
City
Florence
ZIP/Postal Code
50139
Country
Italy
Facility Name
Neurocrine Clinical Site
City
Messina
ZIP/Postal Code
98125
Country
Italy
Facility Name
Neurocrine Clinical Site
City
Milan
ZIP/Postal Code
20132
Country
Italy
Facility Name
Neurocrine Clinical Site
City
Milan
ZIP/Postal Code
20149
Country
Italy
Facility Name
Neurocrine Clinical Site
City
Naples
ZIP/Postal Code
80131
Country
Italy
Facility Name
Neurocrine Clinical Site
City
Padova
ZIP/Postal Code
35128
Country
Italy
Facility Name
Neurocrine Clinical Site
City
Roma
ZIP/Postal Code
00161
Country
Italy
Facility Name
Neurocrine Clinical Site
City
Leiden
ZIP/Postal Code
2333
Country
Netherlands
Facility Name
Neurocrine Clinical Site
City
Kraków
ZIP/Postal Code
31-011
Country
Poland
Facility Name
Neurocrine Clinical Site
City
Poznań
ZIP/Postal Code
60-355
Country
Poland
Facility Name
Neurocrine Clinical Site
City
Warszawa
ZIP/Postal Code
01-809
Country
Poland
Facility Name
Neurocrine Clinical Site
City
Porto
ZIP/Postal Code
4200-319
Country
Portugal
Facility Name
Neurocrine Clinical Site
City
Belgrade
ZIP/Postal Code
11000
Country
Serbia
Facility Name
Neurocrine Clinical Site
City
Madrid
ZIP/Postal Code
28034
Country
Spain
Facility Name
Neurocrine Clinical Site
City
Sevilla
ZIP/Postal Code
41013
Country
Spain
Facility Name
Neurocrine Clinical Site
City
Gothenburg
ZIP/Postal Code
41345
Country
Sweden
Facility Name
Neurocrine Clinical Site
City
Stockholm
ZIP/Postal Code
17176
Country
Sweden
Facility Name
Neurocrine Clinical Site
City
Cardiff
ZIP/Postal Code
CF14 4HH
Country
United Kingdom
Facility Name
Neurocrine Clinical Site
City
London
ZIP/Postal Code
WC1B 5EH
Country
United Kingdom
Facility Name
Neurocrine Clinical Site
City
Manchester
ZIP/Postal Code
M20 4BX
Country
United Kingdom
Facility Name
Neurocrine Clinical Site
City
Salford
ZIP/Postal Code
M6 8HD
Country
United Kingdom

12. IPD Sharing Statement

Plan to Share IPD
No
Links:
URL
https://www.cahtalyststudy.com/
Description
Study website - Cahtalyst Study

Learn more about this trial

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

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