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

NCT ID: NCT04490915

Last Updated: 2025-02-05

Study Results

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Basic Information

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Recruitment Status

ACTIVE_NOT_RECRUITING

Clinical Phase

PHASE3

Total Enrollment

182 participants

Study Classification

INTERVENTIONAL

Study Start Date

2020-12-16

Study Completion Date

2027-08-31

Brief Summary

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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 24-week randomized, double-blind, placebo-controlled period, followed by 1 year of active 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 participant for the OLE (estimated to be approximately 3 years).

Detailed Description

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Conditions

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Congenital Adrenal Hyperplasia

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

TRIPLE

Participants Caregivers Investigators

Study Groups

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Placebo

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

Group Type PLACEBO_COMPARATOR

Crinecerfont

Intervention Type DRUG

CRF type 1 receptor antagonist

Placebo

Intervention Type DRUG

Non-active dosage form

Crinecerfont

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.

Group Type EXPERIMENTAL

Crinecerfont

Intervention Type DRUG

CRF type 1 receptor antagonist

Interventions

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Crinecerfont

CRF type 1 receptor antagonist

Intervention Type DRUG

Placebo

Non-active dosage form

Intervention Type DRUG

Other Intervention Names

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NBI-74788 Crenessity

Eligibility Criteria

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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 CAH due to 21-hydroxylase deficiency.
3. Be on a stable steroid regimen.
4. Participants of childbearing potential must agree to use an acceptable method of 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 receptor 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.
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Neurocrine Biosciences

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Clinical Development Lead

Role: STUDY_DIRECTOR

Neurocrine Biosciences

Locations

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Neurocrine Clinical Site

Los Angeles, California, United States

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San Diego, California, United States

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San Francisco, California, United States

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Aurora, Colorado, United States

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Atlanta, Georgia, United States

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Indianapolis, Indiana, United States

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Bethesda, Maryland, United States

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Boston, Massachusetts, United States

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Ann Arbor, Michigan, United States

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Minneapolis, Minnesota, United States

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Rochester, Minnesota, United States

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St Louis, Missouri, United States

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Great Neck, New York, United States

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New York, New York, United States

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Winston-Salem, North Carolina, United States

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Tulsa, Oklahoma, United States

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Philadelphia, Pennsylvania, United States

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Pittsburgh, Pennsylvania, United States

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Dallas, Texas, United States

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Seattle, Washington, United States

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Graz, , Austria

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Vienna, , Austria

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Brussels, , Belgium

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Leuven, , Belgium

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Sofia, , Bulgaria

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Sofia, , Bulgaria

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Halifax, Nova Scotia, Canada

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Hradec Králové, , Czechia

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Angers, , France

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Grenoble, , France

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Le Kremlin-Bicêtre, , France

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Nantes, , France

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Paris, , France

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Paris, , France

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Dresden, , Germany

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Essen, , Germany

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Frankfurt, , Germany

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Leipzig, , Germany

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Munich, , Germany

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Athens, , Greece

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Athens, , Greece

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Athens, , Greece

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Thessaloniki, , Greece

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Afula, , Israel

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Beersheba, , Israel

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Petah Tikva, , Israel

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Tel Aviv, , Israel

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Ancona, , Italy

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Bologna, , Italy

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Florence, , Italy

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Messina, , Italy

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Milan, , Italy

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Milan, , Italy

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Naples, , Italy

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Padua, , Italy

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Roma, , Italy

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Leiden, , Netherlands

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Krakow, , Poland

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Poznan, , Poland

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Warsaw, , Poland

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Porto, , Portugal

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Belgrade, , Serbia

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Madrid, , Spain

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Seville, , Spain

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Gothenburg, , Sweden

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Stockholm, , Sweden

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Cardiff, , United Kingdom

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London, , United Kingdom

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Manchester, , United Kingdom

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Salford, , United Kingdom

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Countries

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United States Austria Belgium Bulgaria Canada Czechia France Germany Greece Israel Italy Netherlands Poland Portugal Serbia Spain Sweden United Kingdom

References

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Auchus RJ, Hamidi O, Pivonello R, Bancos I, Russo G, Witchel SF, Isidori AM, Rodien P, Srirangalingam U, Kiefer FW, Falhammar H, Merke DP, Reisch N, Sarafoglou K, Cutler GB Jr, Sturgeon J, Roberts E, Lin VH, Chan JL, Farber RH; CAHtalyst Adult Trial Investigators. Phase 3 Trial of Crinecerfont in Adult Congenital Adrenal Hyperplasia. N Engl J Med. 2024 Aug 8;391(6):504-514. doi: 10.1056/NEJMoa2404656. Epub 2024 Jun 1.

Reference Type DERIVED
PMID: 38828955 (View on PubMed)

Provided Documents

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Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Related Links

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https://www.cahtalyststudy.com/

Study website - CAHtalyst Study

Other Identifiers

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2019-004873-17

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

2023-509171-16-00

Identifier Type: CTIS

Identifier Source: secondary_id

NBI-74788-CAH3003

Identifier Type: -

Identifier Source: org_study_id

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