ACTIVE_NOT_RECRUITING

A Study to Test an Oral Medicine, Belumosudil, in Combination With Corticosteroids in Participants at Least 12 Years of Age With Newly Diagnosed Chronic Graft Versus Host Disease.

Conditions

Quick Navigation

Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

This is a parallel, Phase 3, two-arm study for the treatment of newly diagnosed moderate or severe chronic GVHD. The study duration for a participant includes up to 4 weeks for screening; a treatment period until clinically meaningful cGVHD progression (defined as progression requiring addition of new systemic treatment for cGVHD), relapse/recurrence of the underlying disease, participant starts new systemic treatment for cGVHD or experiences an unacceptable toxicity, at the request of the participants or the investigators, or until the end of study is reached, whichever comes first; at least 30 days follow-up of adverse events (AEs) after the last dose until resolution or stabilization, if applicable; and long-term follow-up until death or study close-out, whichever comes first.

Official Title

A Randomized, Double-blind, Multicenter, Phase 3 Study to Evaluate Efficacy and Safety of Belumosudil in Combination With Corticosteroids Versus Placebo in Combination With Corticosteroids in Participants at Least 12 Years of Age With Newly Diagnosed Chronic Graft Versus Host Disease (cGVHD)

Quick Facts

Study Start:2024-01-23

Study Completion:2028-09-29

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:ACTIVE_NOT_RECRUITING

Study ID

NCT06143891

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:12 Years

Sexes Eligible for Study:ALL

Accepts Healthy Volunteers:No

Standard Ages:CHILD, ADULT, OLDER_ADULT

Inclusion Criteria	Exclusion Criteria
* Patients must be at least 12 years of age inclusive, at the time of signing the informed consent * Participants who have undergone allogenic HCT with newly diagnosed moderate to severe cGVHD according to NIH consensus diagnosis and staging criteria (2014) * Participants who require systemic treatment with corticosteroids for cGVHD * Participants who have not received any prior systemic treatment for cGVHD (including ECP) * If participants are receiving other immunosuppressive agents for the prophylaxis or treatment of acute GVHD, the dose should be under the threshold pre-defined in protocol * For adult participants, the body weight should be ≥40 kg. For adolescent participants, the body weight should be ≥30 kg * Contraceptive use by men and women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies. * Participants or their legally authorized representative must be capable of giving signed informed consent	* Any evidence (histologic, cytogenetic, molecular, hematologic, or mixed) of progressive or relapsed underlying disease after the most recent allogeneic HCT * Post-transplant lymphoproliferative disease within 4 weeks prior to randomization * Female participants who are pregnant or breastfeeding * Unable to tolerate a prednisone equivalent dose of corticosteroids ≥ 1 mg/kg/day Prior/concomitant therapy * Participant has had previous exposure to belumosudil. * Received any previous systemic treatment for cGVHD with the following exception: Corticosteroids for cGVHD received within 7 days prior to the planned administration of IMP only if in the interest of participant. * Received any investigational agents, or any investigational device or procedure, or prohibited therapy for this study within 28 days or 5 elimination half-lives prior to randomization, whichever is longer Diagnostic assessments * Karnofsky (if aged ≥16 years)/Lansky (if aged \<16 years) Performance Score of \< 60 * Platelets \<25 x 109/L. Platelet transfusion is not allowed within 3 days before the screening hematological test * Absolute neutrophil count (ANC) \<0.5 x 109/L. The use of granulocyte-colony stimulating factor (G-CSF) is not allowed to reach this level during screening * Estimated Glomerular Filtration Rate (eGFR) \<30 mL/min/1.73 m2 using the MDRD-4 variable formula (if aged ≥18 years) or using the Bedside Schwartz formula (if aged \<18 years) * Aspartate aminotransferase (AST) and/or alanine aminotransferase (ALT) \>3 x ULN without liver cGVHD or\>5 × ULN with liver) cGVHD * Total bilirubin \>1.5 × (ULN) (\>3 × ULN if Gilbert syndrome) * Participant has forced expiratory volume in 1 second (FEV1) of predicted ≤39% or has lung score of 3 according to NIH consensus diagnostic and staging criteria (2014) * History or other evidence of severe illness or any other conditions that would make the participant, in the opinion of the Investigator, unsuitable for the study (such as malabsorption syndromes, poorly controlled psychiatric disease or coronary artery disease) * Known history of human immunodeficiency virus (HIV) * Active viral disease including hepatitis B virus (HBV) or hepatitis C virus (HCV) * Active uncontrolled cytomegalovirus (CMV) and Epstein-Barr virus (EBV) infection. Infections are considered controlled if appropriate therapy has been instituted and, at the time of screening, no signs of infection worsening are present according to Investigator's judgement * Diagnosed or treated for another malignancy other than the underlying disease allogeneic HCT was indicated for, within 3 years prior to randomization with the exception of complete resection of basal cell carcinoma or squamous cell carcinoma of the skin, an in-situ malignancy, or low risk prostate cancer after curative therapy * Unable to swallow tablets * Participant not suitable for participation, whatever the reason, as judged by the Investigator, including medical or clinical conditions, or participants potentially at risk of noncompliance to study procedures * Any active, uncontrolled infections assessed to be clinically significant by the Investigator

Inclusion Criteria

Exclusion Criteria

* Patients must be at least 12 years of age inclusive, at the time of signing the informed consent
* Participants who have undergone allogenic HCT with newly diagnosed moderate to severe cGVHD according to NIH consensus diagnosis and staging criteria (2014)
* Participants who require systemic treatment with corticosteroids for cGVHD
* Participants who have not received any prior systemic treatment for cGVHD (including ECP)
* If participants are receiving other immunosuppressive agents for the prophylaxis or treatment of acute GVHD, the dose should be under the threshold pre-defined in protocol
* For adult participants, the body weight should be ≥40 kg. For adolescent participants, the body weight should be ≥30 kg
* Contraceptive use by men and women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies.
* Participants or their legally authorized representative must be capable of giving signed informed consent

* Any evidence (histologic, cytogenetic, molecular, hematologic, or mixed) of progressive or relapsed underlying disease after the most recent allogeneic HCT
* Post-transplant lymphoproliferative disease within 4 weeks prior to randomization
* Female participants who are pregnant or breastfeeding
* Unable to tolerate a prednisone equivalent dose of corticosteroids ≥ 1 mg/kg/day Prior/concomitant therapy
* Participant has had previous exposure to belumosudil.
* Received any previous systemic treatment for cGVHD with the following exception: Corticosteroids for cGVHD received within 7 days prior to the planned administration of IMP only if in the interest of participant.
* Received any investigational agents, or any investigational device or procedure, or prohibited therapy for this study within 28 days or 5 elimination half-lives prior to randomization, whichever is longer Diagnostic assessments
* Karnofsky (if aged ≥16 years)/Lansky (if aged \<16 years) Performance Score of \< 60
* Platelets \<25 x 109/L. Platelet transfusion is not allowed within 3 days before the screening hematological test
* Absolute neutrophil count (ANC) \<0.5 x 109/L. The use of granulocyte-colony stimulating factor (G-CSF) is not allowed to reach this level during screening
* Estimated Glomerular Filtration Rate (eGFR) \<30 mL/min/1.73 m2 using the MDRD-4 variable formula (if aged ≥18 years) or using the Bedside Schwartz formula (if aged \<18 years)
* Aspartate aminotransferase (AST) and/or alanine aminotransferase (ALT) \>3 x ULN without liver cGVHD or\>5 × ULN with liver) cGVHD
* Total bilirubin \>1.5 × (ULN) (\>3 × ULN if Gilbert syndrome)
* Participant has forced expiratory volume in 1 second (FEV1) of predicted ≤39% or has lung score of 3 according to NIH consensus diagnostic and staging criteria (2014)
* History or other evidence of severe illness or any other conditions that would make the participant, in the opinion of the Investigator, unsuitable for the study (such as malabsorption syndromes, poorly controlled psychiatric disease or coronary artery disease)
* Known history of human immunodeficiency virus (HIV)
* Active viral disease including hepatitis B virus (HBV) or hepatitis C virus (HCV)
* Active uncontrolled cytomegalovirus (CMV) and Epstein-Barr virus (EBV) infection. Infections are considered controlled if appropriate therapy has been instituted and, at the time of screening, no signs of infection worsening are present according to Investigator's judgement
* Diagnosed or treated for another malignancy other than the underlying disease allogeneic HCT was indicated for, within 3 years prior to randomization with the exception of complete resection of basal cell carcinoma or squamous cell carcinoma of the skin, an in-situ malignancy, or low risk prostate cancer after curative therapy
* Unable to swallow tablets
* Participant not suitable for participation, whatever the reason, as judged by the Investigator, including medical or clinical conditions, or participants potentially at risk of noncompliance to study procedures
* Any active, uncontrolled infections assessed to be clinically significant by the Investigator

Contacts and Locations

Study Locations (Sites)

University of Arkansas for Medical Sciences-Site Number : 8400019

Little Rock, Arkansas, 72205

United States

City of Hope National Medical Center- Site Number : 8400001

Duarte, California, 91010

United States

University of California San Francisco - Parnassus Heights- Site Number : 8400035

San Francisco, California, 94143

United States

AdventHealth Orlando- Site Number : 8400023

Orlando, Florida, 32803

United States

The Hope Clinic of Emory University- Site Number : 8400020

Decatur, Georgia, 30030

United States

Northwestern University- Site Number : 8400017

Chicago, Illinois, 60611

United States

Indiana University Health University Hospital- Site Number : 8400006

Indianapolis, Indiana, 46202

United States

University of Kentucky Chandler Medical Center- Site Number : 8400024

Lexington, Kentucky, 40536

United States

Johns Hopkins Hospital- Site Number : 8400033

Baltimore, Maryland, 21287

United States

Dana Farber Cancer Institute Site Number : 8400005

Boston, Massachusetts, 02115

United States

Karmanos Cancer Institute - Detroit- Site Number : 8400013

Detroit, Michigan, 48201

United States

UNC Children's Hospital- Site Number : 8400025

Chapel Hill, North Carolina, 27514

United States

Atrium Health Wake Forest Baptist Medical Center - Winston-Salem - Medical Center Boulevard- Site Number : 8400007

Winston-Salem, North Carolina, 27157

United States

Oncology Hematology Care - Kenwood- Site Number : 8400030

Cincinnati, Ohio, 45236

United States

The Ohio State University Comprehensive Cancer Center (OSUCCC) - The James Cancer Hospital and Solove Research Institute- Site Number : 8400026

Columbus, Ohio, 43210

United States

Oregon Health and Science University- Site Number : 8400027

Portland, Oregon, 97239

United States

UPMC Hillman Cancer Center- Site Number : 8400008

Pittsburgh, Pennsylvania, 15232

United States

Sarah Cannon Research Institute Site Number : 8400003

Nashville, Tennessee, 37203

United States

St. David's South Austin Medical Center- Site Number : 8400002

Austin, Texas, 78704

United States

Texas Oncology - Dallas - Worth Street- Site Number : 8400010

Dallas, Texas, 75246

United States

Texas Transplant Institute - Methodist Hospital- Site Number : 8400037

San Antonio, Texas, 78229

United States

University of Virginia Comprehensive Cancer Center-Site Number : 8400031

Charlottesville, Virginia, 22903

United States

Fred Hutchinson Cancer Research Center- Site Number : 8400004

Seattle, Washington, 98109

United States

University of Wisconsin Carbone Cancer Center-Site Number : 8400029

Madison, Wisconsin, 53792

United States

Collaborators and Investigators

Sponsor: Sanofi

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2024-01-23

Study Completion Date2028-09-29

Study Record Updates

Study Start Date2024-01-23

Study Completion Date2028-09-29

Terms related to this study

Additional Relevant MeSH Terms

Chronic Graft Versus Host Disease