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

Mesoblast

Status and phase

Completed

Phase 3

Conditions

Grade D Acute Graft Versus Host Disease

Grade B Acute Graft Versus Host Disease

Grade C Acute Graft Versus Host Disease

Treatments

Biological: Remestemcel-L

Study type

Interventional

Funder types

Industry

Identifiers

NCT02652130

MSB-GVHD002

Details and patient eligibility

About

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.

Full 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.

Enrollment

32 patients

Sex

All

Ages

2 months to 17 years old

Volunteers

No Healthy Volunteers

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.