Safety Follow-up of Treatment With Remestemcel-L in Pediatric Patients Who Have Failed to Respond to Steroid Treatment for Acute GVHD
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Ongoing safety assessment follow up to Protocol MSB-GVHD 001 of remestemcel-L treatment in pediatric subjects 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.
Condition or disease
Grade B Acute Graft Versus Host DiseaseGrade C Acute Graft Versus Host DiseaseGrade D Acute Graft Versus Host Disease
This is a safety follow-up study through 180 days of remestemcel-L treatment in subjects who participated in MSB-GVHD001. This study will also explore duration of response over time. Subjects who participated 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. Subjects who participated 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.
Safety of remestemcel-L treatment by collecting Serious Adverse Events/Adverse Events/Specific events of interest. [ Time Frame: 180 days ]
Safety of remestemcel-L treatment in subjects who participated in Protocol MSB-GVHD001 By collecting Serious Adverse Events/Adverse Events/Specific events of interest. No additional investigational agent will be administered in this safety follow-up protocol.
Secondary Outcome Measures :
Number of patients who survived at day 180 who received the complete initial therapy through 28 days as outlined in Protocol MSB-GVHD001. [ Time Frame: 180 days ]
GVHD activity by means of Quality of Life survey to investigational medicinal product (IMP) administered in MSB-GVHD001 [ Time Frame: 180 days ]
Corticosteroid use by subjects able to taper by 50% at each time point. [ Time Frame: from Day 100 to Day 180 ]
Administration of any 2nd line GVHD treatments [ Time Frame: 180 days ]
The number of days to GVHD flares [ Time Frame: 180 days ]
The number of subjects with chronic GVHD [ Time Frame: 180 days ]
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Layout table for eligibility information
Ages Eligible for Study:
2 Months to 17 Years (Child)
Sexes Eligible for Study:
Accepts Healthy Volunteers:
In-patients in selected hospitals from MSB GVHD001
Patients are eligible for the study if all of the following criteria are met:
Subjects must have participated in gvhd001 and received at least one infusion of remestemcel L
Subject or subject'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 subjects 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 subject 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.
The investigator believes it to be in the best interest of the subject not to participate in the safety follow up study.