Safety Follow-up of Treatment With Remestemcel-L in Pediatric Participants Who Have Failed to Respond to Steroid Treatment for Acute GVHD

Phase 3

Completed

• Conditions: Grade B Acute Graft Versus Host Disease; Grade C Acute Graft Versus Host Disease; Grade D Acute Graft Versus Host Disease
• Interventions: Biological: Remestemcel-L

• Registration Number: NCT02652130
• Lead Sponsor: Mesoblast, Inc.

Brief Summary

Ongoing safety assessment follow-up to Protocol MSB-GVHD001 (NCT02336230) of remestemcel-L treatment in pediatric participants with acute graft versus host disease (aGVHD), following allogeneic hematopoietic stem cell transplant (HSCT), that have failed to respond to treatment with systemic corticosteroid therapy.

Detailed Description

This is a safety follow-up study through 180 days of remestemcel-L treatment in participants who took part in MSB-GVHD001. This study will also explore duration of response over time. Participants who took part in in MSB-GVHD001 and received at least one dose of remestemcel-L as outlined in that protocol will be evaluated at baseline (Day 100) and at Days 120, 140, 160 and 180 for safety endpoints. Participants who took part in Protocol MSB-GVHD001 and received the first 8 doses of remestemcel-L as outlined in that protocol will be evaluated at baseline (Day 100) and at Days 120, 140, 160 and 180 after initial remestemcel-L infusion for evidence of duration of response over time.

Study Timeline

• Study Start: 2015-10-28
• Study First Submitted: 2015-11-03
• Study First Submitted QC: 2016-01-07
• Study First Posted: 2016-01-11
• Primary Completion: 2018-06-15
• Study Completion: 2018-06-15
• Results First Submitted: 2021-12-01
• Status Verified: 2022-02
• Results First Submitted QC: 2022-02-18
• Last Update Submitted: 2022-02-18
• Results First Posted: 2022-03-16
• Last Update Posted: 2022-03-16

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 32

Inclusion Criteria

• Participants must have participated in MSB-GVHD001 and have received at least one infusion of remestemcel-L.
• Participant or participant's authorized representative must be capable of providing written informed consent. Assent, if applicable, must also be collected when required by the Institutional Review Board (IRB)/Ethics Committee (EC).
• Female participants of childbearing potential (≥ 10 years of age) must use a medically accepted method of contraception and must agree to continue use of this method for the duration of the study and for the follow-up time period. Acceptable methods of contraception include abstinence, barrier method with spermicide, intrauterine device (IUD), or steroidal contraceptive (oral, transdermal, implanted, and injected) in conjunction with a barrier method.
• The participant must be willing and able to comply with study procedures, remain at the clinic as required during the study period, and return to the clinic for the follow-up evaluation as specified in this protocol.

Read More

Exclusion Criteria

• The investigator believes it to be in the best interest of the participant not to participate in the safety follow-up study.
• Participant has participated or is currently participating in any autologous or allogeneic stem cell or gene therapy study for the treatment of aGVHD. Participants participating in investigative protocols aimed at modification of the transplant graft (such as T cell depletion) or aimed at modification of the conditioning regimen will be allowed in the study.

Read More

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Safety population	Remestemcel-L	All participants who were enrolled and had received at least 1 dose of remestemcel-L in Study MSB-GVHD001.

Primary Outcome Measures

Name	Time	Method
Overall Survival Rate Through Day 180	From Baseline Day 1 in the Study MSB-GVHD001 up to Day 180 in Study MSB-GVHD002 (180 days)	The overall survival rate is defined as the percentage of participants alive at the given time point. OS is defined as the time to death from the start of drug therapy.

Secondary Outcome Measures

Name	Time	Method
Overall Survival Rate at Day 180 for Participants Who Had Overall Response (OR) at Day 28 of Study MSB-GVHD001	From Baseline (Day 1) in the Study MSB-GVHD001 up to Day 180 in the Study MSB-GVHD002 (180 days)	The overall survival rate is defined as the percentage of participants alive at the given time point. OS is defined as the time to death from the start of drug therapy.

Trial Locations

Locations (20)

Children's Hospital Los Angeles; 🇺🇸 Los Angeles, California, United States

CHOC Children's Hospital of Orange County; 🇺🇸 Orange, California, United States

UCSF Benioff Children's Hospital; 🇺🇸 San Francisco, California, United States

Alfred I. duPont Hospital for Children of the Nemours Foundation; 🇺🇸 Wilmington, Delaware, United States

Miami Children's Research Institute; 🇺🇸 Miami, Florida, United States

Ann & Robert H. Lurie Children's Hospital of Chicago; 🇺🇸 Chicago, Illinois, United States

Children's Hospital of Michigan; 🇺🇸 Detroit, Michigan, United States

University of Mississippi Medical Center; 🇺🇸 Jackson, Mississippi, United States

Washington University; 🇺🇸 Saint Louis, Missouri, United States

Columbia University Medical Center; 🇺🇸 New York, New York, United States

Memorial Sloan Kettering Cancer Center; 🇺🇸 New York, New York, United States

The Children's Hospital at Montefiore; 🇺🇸 New York, New York, United States

Duke University Medical Center; 🇺🇸 Durham, North Carolina, United States

Oregon Health & Science University; 🇺🇸 Portland, Oregon, United States

Medical University of South Carolina; 🇺🇸 Charleston, South Carolina, United States

Texas Transplant Institute; 🇺🇸 San Antonio, Texas, United States

Virginia Commonwealth University; 🇺🇸 Richmond, Virginia, United States

Fred Hutchinson Cancer Center; 🇺🇸 Seattle, Washington, United States

Children's Hospital Colorado Center for Cancer/Blood Disorders; 🇺🇸 Aurora, Colorado, United States

Medical College of Wisconsin; 🇺🇸 Milwaukee, Wisconsin, United States