NCT02652130

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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
32

participants targeted

Target at below P25 for phase_3

Timeline
Completed

Started Oct 2015

Typical duration for phase_3

Geographic Reach
1 country

20 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

October 28, 2015

Completed
6 days until next milestone

First Submitted

Initial submission to the registry

November 3, 2015

Completed
2 months until next milestone

First Posted

Study publicly available on registry

January 11, 2016

Completed
2.4 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

June 15, 2018

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

June 15, 2018

Completed
3.8 years until next milestone

Results Posted

Study results publicly available

March 16, 2022

Completed
Last Updated

March 16, 2022

Status Verified

February 1, 2022

Enrollment Period

2.6 years

First QC Date

November 3, 2015

Results QC Date

December 1, 2021

Last Update Submit

February 18, 2022

Conditions

Keywords

GVHD

Outcome Measures

Primary Outcomes (1)

  • Overall Survival Rate Through Day 180

    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.

    From Baseline Day 1 in the Study MSB-GVHD001 up to Day 180 in Study MSB-GVHD002 (180 days)

Secondary Outcomes (1)

  • 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)

Study Arms (1)

Safety population

EXPERIMENTAL

All participants who were enrolled and had received at least 1 dose of remestemcel-L in Study MSB-GVHD001.

Biological: Remestemcel-L

Interventions

Remestemcel-LBIOLOGICAL

No intervention was given in Study MSB-GVHD002 (NCT02652130). It was a safety follow-up trial of remestemcel-L-treated participants from Study MSB-GVHD001.

Safety population

Eligibility Criteria

Age2 Months - 17 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17)

You may qualify if:

  • 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.

You may not qualify if:

  • 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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (20)

Children's Hospital Los Angeles

Los Angeles, California, 90027, United States

Location

CHOC Children's Hospital of Orange County

Orange, California, 92868, United States

Location

UCSF Benioff Children's Hospital

San Francisco, California, 94143, United States

Location

Children's Hospital Colorado Center for Cancer/Blood Disorders

Aurora, Colorado, 80045, United States

Location

Alfred I. duPont Hospital for Children of the Nemours Foundation

Wilmington, Delaware, 19803, United States

Location

Miami Children's Research Institute

Miami, Florida, 33155, United States

Location

Ann & Robert H. Lurie Children's Hospital of Chicago

Chicago, Illinois, 60611, United States

Location

Children's Hospital of Michigan

Detroit, Michigan, 48201, United States

Location

University of Mississippi Medical Center

Jackson, Mississippi, 39216, United States

Location

Washington University

St Louis, Missouri, 63110, United States

Location

Columbia University Medical Center

New York, New York, 10032, United States

Location

Memorial Sloan Kettering Cancer Center

New York, New York, 10174, United States

Location

The Children's Hospital at Montefiore

New York, New York, 10467, United States

Location

Duke University Medical Center

Durham, North Carolina, 27705, United States

Location

Oregon Health & Science University

Portland, Oregon, 97239, United States

Location

Medical University of South Carolina

Charleston, South Carolina, 29425, United States

Location

Texas Transplant Institute

San Antonio, Texas, 78229, United States

Location

Virginia Commonwealth University

Richmond, Virginia, 23284, United States

Location

Fred Hutchinson Cancer Center

Seattle, Washington, 98109, United States

Location

Medical College of Wisconsin

Milwaukee, Wisconsin, 53226, United States

Location

MeSH Terms

Interventions

remestemcel-l

Results Point of Contact

Title
Christopher James, VP, Head of Clinical Operations
Organization
Mesoblast, Inc.

Study Officials

  • Christopher James

    Mesoblast, Inc.

    STUDY DIRECTOR

Publication Agreements

PI is Sponsor Employee
No
Restriction Type
OTHER
Restrictive Agreement
Yes

Study Design

Study Type
interventional
Phase
phase 3
Allocation
NA
Masking
NONE
Purpose
OTHER
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

November 3, 2015

First Posted

January 11, 2016

Study Start

October 28, 2015

Primary Completion

June 15, 2018

Study Completion

June 15, 2018

Last Updated

March 16, 2022

Results First Posted

March 16, 2022

Record last verified: 2022-02

Data Sharing

IPD Sharing
Will not share

Locations