A Study of the Efficacy and Safety of Relacorilant in Patients With Endogenous Cushing Syndrome

NCT03697109 | Completed Phase 3 Results DMC FDA Drug

• 1 other identifier: CORT125134-455
• Study Type: interventional
• Target: 152
• Locations: 11 countries
• Sites: 64

Brief Summary

This is a Phase 3, double-blind, placebo-controlled, randomized-withdrawal study to assess the efficacy, safety and pharmacokinetics (PK) of relacorilant in patients with endogenous Cushing syndrome and concurrent type 2 diabetes mellitus/impaired glucose tolerance (DM/IGT) and/or uncontrolled hypertension (HTN).

Conditions

Cushing Syndrome

Keywords

Cushing syndrome; Cushing disease; Hypercortisolemia; Cushingoid; Type 2 Diabetes; Impaired Glucose Intolerance; Hypertension; Adrenocorticotropic hormone; Primary Pigmented Nodular Adrenal Disease; Moon Facies; Dorsocervical Fat Pad; Adrenal Adenoma; Adrenal Autonomy; Cortisol; Cushing

Outcome Measures

Primary Outcomes (2)

• Number of Patients With Loss of Response With Respect to Hypertension During the RW Phase.

  Loss of response with respect to HTN was measured using 6 criteria: 1) an increase in SBP of at least 5 mm Hg, 2) an increase in DBP of at least 5 mm Hg, 3) an increase in SBP and/or DBP of at least 5 mm Hg, 4) use of HTN rescue medication, 5) treatment discontinuation, and 6) missing 24-hour ambulatory blood pressure monitoring (ABPM) measurement at the end of the RW Phase. Blood pressure was measured using ABPM. Use of rescue medication was defined as any increase, modification, or addition of antihypertensive medication due to worsening HTN. Treatment discontinuation reports the number of patients who discontinued study treatment in the RW Phase for any reason.

  Week 22 (end of OL Phase) and Week 36 (Week 12 of RW Phase)

• Number of Patients With 1 or More Treatment-emergent Adverse Events (TEAEs) as Graded by CTCAE v5.0.

  OL Phase: Up to 22 weeks; RW Phase: up to 18 weeks after completion of the OL Phase

Secondary Outcomes (6)

• Change in Area Under the Concentration-time Curve of Blood Glucose (AUCglucose) During the RW Phase

  Before and at time intervals up to 2 hours post glucose drink at Week 22 (end of OL Phase) and Week 36 (Week 12 of RW Phase)

• Change in Hemoglobin HbA1c During the RW Phase

  Week 22 (end of OL Phase) and Week 36 (Week 12 of RW Phase)

• Change in 2-hour Plasma Glucose During the RW Phase

  Before and 2 hours post glucose drink at Week 22 (end of OL Phase) and Week 36 (Week 12 of RW Phase)

• Change in SBP and DBP During the RW Phase

  Week 22 (end of OL Phase) and Week 36 (Week 12 of RW Phase)

• Change in Body Weight During the RW Phase

  Week 22 (end of OL Phase) and Week 36 (Week 12 of RW Phase)

Other Outcomes (10)

• Change in Cushing Quality of Life (QoL) Normalized Total Score During the RW Phase

  Week 22 (end of OL Phase) and Week 36 (Week 12 of RW Phase)

• Percent Change in Tissue Fat Mass During the RW Phase

  Week 22 (end of OL Phase) and Week 36 (Week 12 of RW Phase)

• Number of Patients Who Worsened, as Assessed by the Global Clinical Response, During the RW Phase

  Week 22 (end of OL Phase) and up to Week 36 (Week 12 of RW Phase)

Study Arms (3)

Relacorilant (OL Phase)

EXPERIMENTAL

Patients will receive relacorilant increased sequentially from 100 mg once daily to a maximum dose of 400 mg once daily.

Drug: Relacorilant

Relacorilant (RW Phase)

EXPERIMENTAL

Patients who meet any of the response criteria will advance to the RW Phase of the study and receive the same dose of relacorilant as the last dose administered in the OL Phase.

Drug: Relacorilant

Placebo (RW Phase)

PLACEBO COMPARATOR

Patients who meet any of the response criteria will advance to the RW Phase of the study and receive placebo matched to study drug.

Other: Placebo

Interventions

Relacorilant DRUG

Relacorilant is supplied as 100 mg capsules for oral dosing.

Also known as: CORT125134

Relacorilant (OL Phase); Relacorilant (RW Phase)

Placebo OTHER

Placebo matched to study drug

Placebo (RW Phase)

Eligibility Criteria

• Age: 18 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Has a confirmed diagnosis of endogenous Cushing syndrome
• Meets at least 1 of the following criteria:
• Has Type 2 diabetes mellitus
• Has impaired glucose tolerance
• Has hypertension.

You may not qualify if:

• Has non-endogenous source of hypercortisolism
• Has uncontrolled, clinically significant hypothyroidism or hyperthyroidism
• Has poorly controlled hypertension
• Has poorly controlled diabetes mellitus
• Has severe renal insufficiency.

Sponsors & Collaborators

• Corcept Therapeutics: lead

Study Sites (64)

Site 21

Phoenix, Arizona, 85013, United States

Location

Site 36

Los Angeles, California, 90095, United States

Location

Site 68

Torrance, California, 90502, United States

Location

Site 10

Miami, Florida, 33136, United States

Location

Site 14

Atlanta, Georgia, 30318, United States

Location

Site 41

Chicago, Illinois, 60611, United States

Location

Site 7

Indianapolis, Indiana, 46202, United States

Location

Site 2

Metairie, Louisiana, 70006, United States

Location

Site 45

Baltimore, Maryland, 21205, United States

Location

Site 46

Boston, Massachusetts, 02115, United States

Location

Site 20

Ann Arbor, Michigan, 48109, United States

Location

Site 4

Jackson, Mississippi, 39202, United States

Location

Site 13

St Louis, Missouri, 63110, United States

Location

Site 53

Omaha, Nebraska, 68198, United States

Location

Site 72

Reno, Nevada, 89511, United States

Location

Site 8

Albany, New York, 12203, United States

Location

Site 6

Jamaica, New York, 11432, United States

Location

Site 57

New York, New York, 10021, United States

Location

Site 35

New York, New York, 10029, United States

Location

Site 39

New York, New York, 10065, United States

Location

Site 1

Wilmington, North Carolina, 28401, United States

Location

Site 17

Columbus, Ohio, 43210, United States

Location

Site 11

Oklahoma City, Oklahoma, 73104, United States

Location

Site 62

Philadelphia, Pennsylvania, 19107, United States

Location

Site 19

Pittsburgh, Pennsylvania, 15212, United States

Location

Site 71

Pittsburgh, Pennsylvania, 15213, United States

Location

Site 5

Summerville, South Carolina, 29485, United States

Location

Site 51

Dallas, Texas, 75390, United States

Location

Site 3

El Paso, Texas, 79935, United States

Location

Site 65

Houston, Texas, 77079, United States

Location

Site 56

Shavano Park, Texas, 78231, United States

Location

Site 31

Everett, Washington, 98201, United States

Location

Site 47

Vienna, 1090, Austria

Location

Site 27

Sofia, 1431, Bulgaria

Location

Site 70

Halifax, Nova Scotia, B3H-2Y9, Canada

Location

Site 58

Montreal, H2X 0A9, Canada

Location

Site 54

München, 80336, Germany

Location

Site 49

Würzburg, 97080, Germany

Location

Site 30

Jerusalem, 911120, Israel

Location

Site 29

Kfar Saba, 4428164, Israel

Location

Site 28

Petah Tikva, 4941480, Israel

Location

Site 69

Tel Aviv, Israel

Location

Site 43

Ancona, 60030, Italy

Location

Site 15

Messina, 98125, Italy

Location

Site 26

Milan, 20149, Italy

Location

Site 12

Napoli, 80131, Italy

Location

Site 38

Orbassano, 10043, Italy

Location

Site 67

Padua, 35128, Italy

Location

Site 40

Roma, 00161, Italy

Location

Site 16

Roma, 00189, Italy

Location

Site 34

Rotterdam, 3015 AA, Netherlands

Location

Site 77

Bialystok, Poland

Location

Site 37

Chrzanów, 32-500, Poland

Location

Site 59

Krakow, 31- 501, Poland

Location

Site 33

Lublin, 20-412, Poland

Location

Site 66

Bucharest, 010825, Romania

Location

Site 63

Bucharest, 011863, Romania

Location

Site 64

Bucharest, 011863, Romania

Location

Site 73

Iași, 700106, Romania

Location

Site 75

Alicante, 03010, Spain

Location

Site 25

Girona, 17007, Spain

Location

Site 24

Madrid, 28007, Spain

Location

Site 22

Málaga, 29006, Spain

Location

Site 23

Seville, 41013, Spain

Location

Related Publications (1)

• Pivonello R, Munster PN, Terzolo M, Ferrigno R, Simeoli C, Puglisi S, Bali U, Moraitis AG. Glucocorticoid Receptor Antagonism Upregulates Somatostatin Receptor Subtype 2 Expression in ACTH-Producing Neuroendocrine Tumors: New Insight Based on the Selective Glucocorticoid Receptor Modulator Relacorilant. Front Endocrinol (Lausanne). 2022 Jan 4;12:793262. doi: 10.3389/fendo.2021.793262. eCollection 2021.

  PMID: 35058882 DERIVED

MeSH Terms

Conditions

Cushing Syndrome; Pituitary ACTH Hypersecretion; ACTH Syndrome, Ectopic; Diabetes Mellitus, Type 2; Hypertension

Interventions

relacorilant

Condition Hierarchy (Ancestors)

Adrenocortical Hyperfunction; Adrenal Gland Diseases; Endocrine System Diseases; Hyperpituitarism; Pituitary Diseases; Hypothalamic Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Paraneoplastic Endocrine Syndromes; Paraneoplastic Syndromes; Neoplasms; Diabetes Mellitus; Glucose Metabolism Disorders; Metabolic Diseases; Nutritional and Metabolic Diseases; Vascular Diseases; Cardiovascular Diseases

Results Point of Contact

• Title: Medical Director
• Organization: Corcept Therapeutics

info@corcept.com

650-327-3270

Study Officials

• Andreas Moraitis, MD

  Corcept Therapeutics

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 27, 2018
• First Posted: October 5, 2018
• Study Start: November 15, 2018
• Primary Completion: April 8, 2024
• Study Completion: April 15, 2024
• Last Updated: July 16, 2025
• Results First Posted: April 24, 2025

Record last verified: 2025-04

Data Sharing

• IPD Sharing: Will not share

Locations

ES (5); IT (8); DE (2); NL (1); US (32); RO (4); PL (4); AU (1); CA (2); IL (4); BU (1)