Study to Assess the Safety and Efficacy of Tacrolimus (Prograf Capsule/Injection) and Methotrexate (MTX) Combination Therapy for Prevention of Graft Versus Host Disease (GVHD) in Patients Who Received Peripheral Hematopoietic Stem Cell Transplantation From a Sibling Donor

Phase 4

Completed

• Conditions: Graft Versus Host Disease
• Interventions: Drug: Prograf; Drug: Methotrexate; Drug: Cyclosporine

• Registration Number: NCT02660684
• Lead Sponsor: Astellas Pharma Korea, Inc.

Brief Summary

The objective of this study is to assess the safety and efficacy of Tacrolimus (Prograf capsule, Prograf injection) and Methotrexate combination therapy for GVHD prophylaxis in patients who received peripheral hematopoietic stem cell transplantation from a sibling donor, and to compare with data from a historical control group that administered a conventional Cyclosporine formulation.

Detailed Description

Not available

Study Timeline

• Study Start: 2008-02
• Primary Completion: 2010-02
• Study Completion: 2010-02
• Study First Submitted: 2016-01-18
• Study First Submitted QC: 2016-01-18
• Study First Posted: 2016-01-21
• Status Verified: 2016-02
• Last Update Submitted: 2016-02-29
• Last Update Posted: 2016-03-01

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 39

Inclusion Criteria

• Blood diseases for a standard risk group

  • Primary remission of AML (acute myeloid leukemia)
  • Primary remission of ALL (acute lymphocytic leukemia)
  • Secondary remission of ALL (acute lymphocytic leukemia)
  • SAA (severe aplastic anemia)
  • Chronic stage CML (chronic myeloid leukemia)
  • MDS (myelodysplastic syndrome)
  • Myeloma (multiple myeloma)

• A patient who received peripheral hematopoietic stem cell transplantation from a HLA-matched sibling donor

Exclusion Criteria

• A patient with renal impairment (serum creatinine level ≥ 1.5mg/dl or 130μmol/l, GFR≤ 30%)
• A pregnant or breastfeeding woman
• A woman who is unwilling or unable to practice appropriate contraception during the study
• A patient who is highly likely to experience aggravation during treatment due to active tuberculosis, other hepatic disease, hypertension, heart failure, chronic obstructive respiratory disease, etc.
• A patient with hypersensitivity to tacrolimus

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Prograf + MTX	Methotrexate	-
Cyclosporine + MTX (historical control)	Methotrexate	-
Prograf + MTX	Prograf	-
Cyclosporine + MTX (historical control)	Cyclosporine	-

Primary Outcome Measures

Name	Time	Method
Number of occurrence sites of acute GVHD occurring within 100 days after peripheral hematopoietic stem cell transplantation from a sibling donor	Day 1 up to Day 100 post-transplant
Severity of acute GVHD occurring within 100 days after peripheral hematopoietic stem cell transplantation from a sibling donor	Day 1 up to Day 100 post-transplant	Severity is graded according to the Seattle criteria, between 1+ \~ 4+ depending on the severity of the injury of the skin, liver and digestive system, and the overall grade is classified by including the performance change.
Number of occurrence sites of chronic GVHD occurring after 100 days after peripheral hematopoietic stem cell transplantation from a sibling donor	Day 100 up to 1 year post-transplant
Classification of chronic GVHD occurring after 100 days after peripheral hematopoietic stem cell transplantation from a sibling donor	Day 100 up to 1 year post-transplant	Depending on the extent of occurrence, it is classified as limited (present only locally) and extensive (systemic lesions). Depending on the presence of preceding acute GVHD, it is classified as progressive for a patient with preceding acute GVHD (chronic GVHD following the occurrence of acute GVHD), quiescent (chronic GVHD after relief of acute GVHD symptoms) or otherwise (de novo).
Safety as assessed by adverse events	Day 1 up to 1 year post-transplant
Safety as assessed by laboratory assessments	Day 1 up to 1 year post-transplant	Includes hematology, biochemistry and trough plasma concentration analysis

Secondary Outcome Measures

Name	Time	Method
Percentage of patients alive after peripheral hematopoietic stem cell transplantation from a sibling donor	Day 1 up to 1 year post-transplant
Percentage of patients alive after peripheral hematopoietic stem cell transplantation from a sibling donor in comparison with Cyclosporine in combination with MTX	Day 1 up to 1 year post-transplant