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

Congenital Adrenal Hyperplasia Clinical Trial

— 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

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT04490915
• Other study ID #: NBI-74788-CAH3003
• Secondary ID: 2019-004873-17
• Status: Active, not recruiting
• Phase: Phase 3
• Start date: November 16, 2020
• Est. completion date: August 2027

Study information

• Verified date: May 2024
• Source: Neurocrine Biosciences
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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).

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 182
• Est. completion date: August 2027
• Est. primary completion date: July 19, 2023
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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. 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 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.

Related Conditions & MeSH terms

• Adrenal Hyperplasia, Congenital
• Adrenocortical Hyperfunction
• Adrenogenital Syndrome
• Congenital Adrenal Hyperplasia
• Hyperplasia

Intervention

Drug:
• Crinecerfont
CRF1-receptor antagonist
• Placebo
Non-active dosage form

Locations

Country	Name	City	State
Austria	Neurocrine Clinical Site	Graz
Austria	Neurocrine Clinical Site	Vienna
Belgium	Neurocrine Clinical Site	Brussels
Belgium	Neurocrine Clinical Site	Leuven
Bulgaria	Neurocrine Clinical Site	Sofia
Bulgaria	Neurocrine Clinical Site	Sofia
Canada	Neurocrine Clinical Site	Halifax	Nova Scotia
Czechia	Neurocrine Clinical Site	Hradec Králové
France	Neurocrine Clinical Site	Angers
France	Neurocrine Clinical Site	Grenoble
France	Neurocrine Clinical Site	Le Kremlin-Bicêtre
France	Neurocrine Clinical Site	Nantes
France	Neurocrine Clinical Site	Paris
France	Neurocrine Clinical Site	Paris
Germany	Neurocrine Clinical Site	Dresden
Germany	Neurocrine Clinical Site	Essen
Germany	Neurocrine Clinical Site	Frankfurt
Germany	Neurocrine Clinical Site	Leipzig
Germany	Neurocrine Clinical Site	Munich
Greece	Neurocrine Clinical Site	Athens
Greece	Neurocrine Clinical Site	Athens
Greece	Neurocrine Clinical Site	Athens
Greece	Neurocrine Clinical Site	Thessaloníki
Israel	Neurocrine Clinical Site	Afula
Israel	Neurocrine Clinical Site	Beer Sheva
Israel	Neurocrine Clinical Site	Petah Tikva
Israel	Neurocrine Clinical Site	Tel Aviv
Italy	Neurocrine Clinical Site	Ancona
Italy	Neurocrine Clinical Site	Bologna
Italy	Neurocrine Clinical Site	Florence
Italy	Neurocrine Clinical Site	Messina
Italy	Neurocrine Clinical Site	Milan
Italy	Neurocrine Clinical Site	Milan
Italy	Neurocrine Clinical Site	Naples
Italy	Neurocrine Clinical Site	Padova
Italy	Neurocrine Clinical Site	Roma
Netherlands	Neurocrine Clinical Site	Leiden
Poland	Neurocrine Clinical Site	Kraków
Poland	Neurocrine Clinical Site	Poznan
Poland	Neurocrine Clinical Site	Warszawa
Portugal	Neurocrine Clinical Site	Porto
Serbia	Neurocrine Clinical Site	Belgrade
Spain	Neurocrine Clinical Site	Madrid
Spain	Neurocrine Clinical Site	Sevilla
Sweden	Neurocrine Clinical Site	Gothenburg
Sweden	Neurocrine Clinical Site	Stockholm
United Kingdom	Neurocrine Clinical Site	Cardiff
United Kingdom	Neurocrine Clinical Site	London
United Kingdom	Neurocrine Clinical Site	Manchester
United Kingdom	Neurocrine Clinical Site	Salford
United States	Neurocrine Clinical Site	Ann Arbor	Michigan
United States	Neurocrine Clinical Site	Atlanta	Georgia
United States	Neurocrine Clinical Site	Aurora	Colorado
United States	Neurocrine Clinical Site	Bethesda	Maryland
United States	Neurocrine Clinical Site	Boston	Massachusetts
United States	Neurocrine Clinical Site	Dallas	Texas
United States	Neurocrine Clinical Site	Great Neck	New York
United States	Neurocrine Clinical Site	Indianapolis	Indiana
United States	Neurocrine Clinical Site	Los Angeles	California
United States	Neurocrine Clinical Site	Minneapolis	Minnesota
United States	Neurocrine Clinical Site	New York	New York
United States	Neurocrine Clinical Site	Philadelphia	Pennsylvania
United States	Neurocrine Clinical Site	Pittsburgh	Pennsylvania
United States	Neurocrine Clinical Site	Rochester	Minnesota
United States	Neurocrine Clinical Site	Saint Louis	Missouri
United States	Neurocrine Clinical Site	San Diego	California
United States	Neurocrine Clinical Site	San Francisco	California
United States	Neurocrine Clinical Site	Seattle	Washington
United States	Neurocrine Clinical Site	Tulsa	Oklahoma
United States	Neurocrine Clinical Site	Winston-Salem	North Carolina

Sponsors (1)

Lead Sponsor	Collaborator
Neurocrine Biosciences

Countries where clinical trial is conducted

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

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Percent change from baseline in glucocorticoid daily dose at Week 24		Baseline and Week 24
Secondary	Change from baseline in serum androstenedione at Week 4		Baseline and Week 4
Secondary	Achievement of a reduction in glucocorticoid daily dose to physiologic levels at Week 24		Baseline and Week 24
Secondary	Change from baseline in homeostatic model assessment of insulin resistance (HOMA-IR) index at Week 24		Baseline and Week 24
Secondary	Change from baseline in body weight at Week 24		Baseline and Week 24
Secondary	Change from baseline in fat mass at Week 24		Baseline and Week 24
Secondary	Change from baseline in blood pressure at Week 24		Baseline and Week 24
Secondary	Change from baseline in glucose tolerance at Week 24		Baseline and Week 24
Secondary	Change from baseline in waist circumference at Week 24		Baseline and Week 24
Secondary	Change from baseline in menstrual regularity at Week 24		Baseline and Week 24
Secondary	Change from baseline in testicular adrenal rest tumor size at Week 24		Baseline and Week 24

External Links

•   Study website - Cahtalyst Study