Clinical trial NCT04490915: Global Safety and Efficacy Registration Study of Crinecerfont for Congenital Adrenal Hyperplasia
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
Overall status: ACTIVE_NOT_RECRUITING
Phase: PHASE3
Conditions: Congenital Adrenal Hyperplasia
Lead sponsor: Neurocrine Biosciences
Sponsor class: INDUSTRY
First posted: 2020-07-29
Enrollment: 182
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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 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).


Eligibility criteria: 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.

Primary outcomes:
measure: Percent Change From Baseline in Glucocorticoid Daily Dose at Week 24
timeFrame: Baseline, Week 24
Secondary outcomes:
measure: Change From Baseline in Serum Androstenedione at Week 4
timeFrame: Baseline, Week 4
measure: Number of Participants Who Achieved a Reduction to Physiologic Glucocorticoid Dose While Maintaining Androstenedione Control at Week 24
timeFrame: Week 24
measure: Change From Baseline in Homeostatic Model Assessment of Insulin Resistance (HOMA-IR) at Week 24
timeFrame: Baseline, Week 24
measure: Percent Change From Baseline in Body Weight at Week 24
timeFrame: Baseline, Week 24
measure: Change From Baseline in Percent Total Fat Mass at Week 24
timeFrame: Baseline, Week 24
measure: Change From Baseline in Serum 17-hydroxyprogesterone (17-OHP) at Week 4
timeFrame: Baseline, Week 4
measure: Change From Baseline in Blood Pressure at Week 24
timeFrame: Baseline, Week 24
measure: Change From Baseline in Glucose Tolerance at Week 24
timeFrame: Baseline, Week 24
measure: Change From Baseline in Waist Circumference at Week 24
timeFrame: Baseline, Week 24
measure: Change From Baseline in Menstrual Regularity at Week 24
timeFrame: Baseline, Week 24
measure: Change From Baseline in Testicular Adrenal Rest Tumor (TART) Volume at Week 24
timeFrame: Baseline, Week 24
Other outcomes:
Locations:
city: Los Angeles
country: United States
facility: Neurocrine Clinical Site
state: California
city: San Diego
country: United States
facility: Neurocrine Clinical Site
state: California
city: San Francisco
country: United States
facility: Neurocrine Clinical Site
state: California
city: Aurora
country: United States
facility: Neurocrine Clinical Site
state: Colorado
city: Atlanta
country: United States
facility: Neurocrine Clinical Site
state: Georgia
city: Indianapolis
country: United States
facility: Neurocrine Clinical Site
state: Indiana
city: Bethesda
country: United States
facility: Neurocrine Clinical Site
state: Maryland
city: Boston
country: United States
facility: Neurocrine Clinical Site
state: Massachusetts
city: Ann Arbor
country: United States
facility: Neurocrine Clinical Site
state: Michigan
city: Minneapolis
country: United States
facility: Neurocrine Clinical Site
state: Minnesota
city: Rochester
country: United States
facility: Neurocrine Clinical Site
state: Minnesota
city: Saint Louis
country: United States
facility: Neurocrine Clinical Site
state: Missouri
city: Great Neck
country: United States
facility: Neurocrine Clinical Site
state: New York
city: New York
country: United States
facility: Neurocrine Clinical Site
state: New York
city: Winston-Salem
country: United States
facility: Neurocrine Clinical Site
state: North Carolina
city: Tulsa
country: United States
facility: Neurocrine Clinical Site
state: Oklahoma
city: Philadelphia
country: United States
facility: Neurocrine Clinical Site
state: Pennsylvania
city: Pittsburgh
country: United States
facility: Neurocrine Clinical Site
state: Pennsylvania
city: Dallas
country: United States
facility: Neurocrine Clinical Site
state: Texas
city: Seattle
country: United States
facility: Neurocrine Clinical Site
state: Washington
city: Graz
country: Austria
facility: Neurocrine Clinical Site
city: Vienna
country: Austria
facility: Neurocrine Clinical Site
city: Brussels
country: Belgium
facility: Neurocrine Clinical Site
city: Leuven
country: Belgium
facility: Neurocrine Clinical Site
city: Sofia
country: Bulgaria
facility: Neurocrine Clinical Site
city: Sofia
country: Bulgaria
facility: Neurocrine Clinical Site
city: Halifax
country: Canada
facility: Neurocrine Clinical Site
state: Nova Scotia
city: Hradec Králové
country: Czechia
facility: Neurocrine Clinical Site
city: Angers
country: France
facility: Neurocrine Clinical Site
city: Grenoble
country: France
facility: Neurocrine Clinical Site
city: Le Kremlin-Bicêtre
country: France
facility: Neurocrine Clinical Site
city: Nantes
country: France
facility: Neurocrine Clinical Site
city: Paris
country: France
facility: Neurocrine Clinical Site
city: Paris
country: France
facility: Neurocrine Clinical Site
city: Dresden
country: Germany
facility: Neurocrine Clinical Site
city: Essen
country: Germany
facility: Neurocrine Clinical Site
city: Frankfurt
country: Germany
facility: Neurocrine Clinical Site
city: Leipzig
country: Germany
facility: Neurocrine Clinical Site
city: Munich
country: Germany
facility: Neurocrine Clinical Site
city: Athens
country: Greece
facility: Neurocrine Clinical Site
city: Athens
country: Greece
facility: Neurocrine Clinical Site
city: Athens
country: Greece
facility: Neurocrine Clinical Site
city: Thessaloníki
country: Greece
facility: Neurocrine Clinical Site
city: Afula
country: Israel
facility: Neurocrine Clinical Site
city: Beer Sheva
country: Israel
facility: Neurocrine Clinical Site
city: Petah Tikva
country: Israel
facility: Neurocrine Clinical Site
city: Tel Aviv
country: Israel
facility: Neurocrine Clinical Site
city: Ancona
country: Italy
facility: Neurocrine Clinical Site
city: Bologna
country: Italy
facility: Neurocrine Clinical Site
city: Florence
country: Italy
facility: Neurocrine Clinical Site
city: Messina
country: Italy
facility: Neurocrine Clinical Site
city: Milan
country: Italy
facility: Neurocrine Clinical Site
city: Milan
country: Italy
facility: Neurocrine Clinical Site
city: Naples
country: Italy
facility: Neurocrine Clinical Site
city: Padova
country: Italy
facility: Neurocrine Clinical Site
city: Roma
country: Italy
facility: Neurocrine Clinical Site
city: Leiden
country: Netherlands
facility: Neurocrine Clinical Site
city: Kraków
country: Poland
facility: Neurocrine Clinical Site
city: Poznań
country: Poland
facility: Neurocrine Clinical Site
city: Warszawa
country: Poland
facility: Neurocrine Clinical Site
city: Porto
country: Portugal
facility: Neurocrine Clinical Site
city: Belgrade
country: Serbia
facility: Neurocrine Clinical Site
city: Madrid
country: Spain
facility: Neurocrine Clinical Site
city: Sevilla
country: Spain
facility: Neurocrine Clinical Site
city: Gothenburg
country: Sweden
facility: Neurocrine Clinical Site
city: Stockholm
country: Sweden
facility: Neurocrine Clinical Site
city: Cardiff
country: United Kingdom
facility: Neurocrine Clinical Site
city: London
country: United Kingdom
facility: Neurocrine Clinical Site
city: Manchester
country: United Kingdom
facility: Neurocrine Clinical Site
city: Salford
country: United Kingdom
facility: Neurocrine Clinical Site
Study officials:
affiliation: Neurocrine Biosciences
name: Clinical Development Lead
role: STUDY_DIRECTOR
References: