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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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ClinicalTrials.gov Identifier: NCT02652130
Recruitment Status : Completed
First Posted : January 11, 2016
Last Update Posted : October 23, 2018
Sponsor:
Collaborator:
Quintiles, Inc.
Information provided by (Responsible Party):
Mesoblast, Ltd.

Brief Summary:
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
Grades B-D Acute Graft Versus Host Disease

Detailed Description:
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.

Study Type : Observational
Actual Enrollment : 32 participants
Observational Model: Case-Only
Time Perspective: Prospective
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
Actual Study Start Date : October 2015
Actual Primary Completion Date : June 15, 2018
Actual Study Completion Date : June 15, 2018





Primary Outcome Measures :
  1. 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 :
  1. 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 ]
  2. GVHD activity by means of Quality of Life survey to investigational medicinal product (IMP) administered in MSB-GVHD001 [ Time Frame: 180 days ]
  3. Corticosteroid use by subjects able to taper by 50% at each time point. [ Time Frame: from Day 100 to Day 180 ]
  4. Administration of any 2nd line GVHD treatments [ Time Frame: 180 days ]
  5. The number of days to GVHD flares [ Time Frame: 180 days ]
  6. The number of subjects with chronic GVHD [ Time Frame: 180 days ]


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Ages Eligible for Study:   2 Months to 17 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
In-patients in selected hospitals from MSB GVHD001
Criteria

Inclusion Criteria:

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.

Exclusion Criteria:

  • The investigator believes it to be in the best interest of the subject not to participate in the safety follow up study.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02652130


  Show 20 Study Locations
Sponsors and Collaborators
Mesoblast, Ltd.
Quintiles, Inc.
Investigators
Study Director: Donna Skerrett, MD, MS Mesoblast CMO

Responsible Party: Mesoblast, Ltd.
ClinicalTrials.gov Identifier: NCT02652130     History of Changes
Other Study ID Numbers: MSB-GVHD002
First Posted: January 11, 2016    Key Record Dates
Last Update Posted: October 23, 2018
Last Verified: October 2018

Keywords provided by Mesoblast, Ltd.:
GVHD, Graft versus Host Disease

Additional relevant MeSH terms:
Graft vs Host Disease
Immune System Diseases