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

Grade B Acute Graft Versus Host Disease Clinical Trial

Official title:

Safety Follow-up Through 180 Days of Treatment With Remestemcel-L in Study MSB-GVHD001 in Pediatric Patients Who Have Failed to Respond to Steroid Treatment for Acute GVHD

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02652130
• Other study ID #: MSB-GVHD002
• Status: Completed
• Phase: Phase 3
• Start date: October 28, 2015
• Est. completion date: June 15, 2018

Study information

• Verified date: February 2022
• Source: Mesoblast, Ltd.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 32
• Est. completion date: June 15, 2018
• Est. primary completion date: June 15, 2018
• Accepts healthy volunteers: No
• Gender: All
• Age group: 2 Months to 17 Years

Eligibility

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.

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.

Related Conditions & MeSH terms

• Grade B Acute Graft Versus Host Disease
• Grade C Acute Graft Versus Host Disease
• Grade D Acute Graft Versus Host Disease
• Graft vs Host Disease

Intervention

Biological:
• Remestemcel-L
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.

Locations

Country	Name	City	State
United States	Children's Hospital Colorado Center for Cancer/Blood Disorders	Aurora	Colorado
United States	Medical University of South Carolina	Charleston	South Carolina
United States	Ann & Robert H. Lurie Children's Hospital of Chicago	Chicago	Illinois
United States	Children's Hospital of Michigan	Detroit	Michigan
United States	Duke University Medical Center	Durham	North Carolina
United States	University of Mississippi Medical Center	Jackson	Mississippi
United States	Children's Hospital Los Angeles	Los Angeles	California
United States	Miami Children's Research Institute	Miami	Florida
United States	Medical College of Wisconsin	Milwaukee	Wisconsin
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	CHOC Children's Hospital of Orange County	Orange	California
United States	Oregon Health & Science University	Portland	Oregon
United States	Virginia Commonwealth University	Richmond	Virginia
United States	Washington University	Saint Louis	Missouri
United States	Texas Transplant Institute	San Antonio	Texas
United States	UCSF Benioff Children's Hospital	San Francisco	California
United States	Fred Hutchinson Cancer Center	Seattle	Washington
United States	Alfred I. duPont Hospital for Children of the Nemours Foundation	Wilmington	Delaware

Sponsors (2)

Lead Sponsor	Collaborator
Mesoblast, Inc.	Quintiles, Inc.

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	Overall Survival Rate at Day 180 for Participants Who Had Overall Response (OR) at Day 28 of Study MSB-GVHD001	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 the Study MSB-GVHD002 (180 days)