Clinical Trials /

Allogeneic Hematopoietic Stem Cell Transplantation in Patients With Myelodysplastic Syndrome Low Risk

NCT02757989

Description:

Comparison of survival in patients with or without a matched donor at 36 months

Related Conditions:
  • Myelodysplastic Syndromes
Recruiting Status:

Recruiting

Phase:

N/A

Trial Eligibility

Document

Title

  • Brief Title: Allogeneic Hematopoietic Stem Cell Transplantation in Patients With Myelodysplastic Syndrome Low Risk
  • Official Title: Allogeneic Hematopoietic Stem Cell Transplantation in Patients With Low or Intermediate-1 Myelodysplastic Syndrome: A Prospective Multicenter Phase II Study Based on Donor Availability on Behalf of the GFM & SFGM-TC

Clinical Trial IDs

  • ORG STUDY ID: MDS-ALLO-RISK
  • SECONDARY ID: 2015-A00292-47
  • NCT ID: NCT02757989

Conditions

  • MDS

Purpose

Comparison of survival in patients with or without a matched donor at 36 months

Detailed Description

      Patients with a matched donor (8/8 at molecular level unrelated donor or matched sibling)
      received an allogeneic hematopoietic stem cell transplantation.

      Patients without a matched donor received the best available treatment. All patients will be
      followed at least 36 months or until the end of the study.
    

Trial Arms

NameTypeDescriptionInterventions
Patients with donorExperimentalPatients with a matched donor (8/8 at molecular level unrelated donor or matched sibling)
    Patients without donorNo InterventionPatients without a matched donor

      Eligibility Criteria

              Inclusion Criteria:
      
                1. Signed Informed consent
      
                2. Classical IPSS intermediate 1 or low myelodysplastic syndrome associated with at least
                   one poor prognosis feature:
      
                     1. Intermediate or higher risk revised IPSS
      
                     2. RBC transfusion dependent anemia and failure to 2 or more lines or therapy
                        (including EPO, Lenalidomide or demethylating agent…) with the exception of pure
                        RARS with isolated SF3B1 mutation
      
                     3. thrombocytopenia < 20 G/L requiring transfusion
      
                     4. neutropenia < 0.5 G/L associated with severe infection (defined as requiring
                        hospitalization)
      
                     5. 5q- and lenalidomid failure especially but not only with TP53 mutated
      
                3. Patient aged ≥ 18 and < 70 years For young patients, 18-45 years, Fanconi disease and
                   dyskeratosis should be ruled out
      
                4. Patient for whom a transplantation from a matched donor, (8/8 (HLA A, B, C, DRB1)
                   identical at molecular level)unrelated donor or matched sibling), is considered
                   irrespective of donor availability
      
                5. Performance status 0-2 on the Eastern Cooperative Oncology Group (ECOG) Scale (At time
                   of screening)
      
                6. Negative pregnancy and adequate contraception (including in male patients wishing to
                   father), if relevant.
      
                7. Wash-out of at least 30 days since a previous treatment with Vidaza, Lenalidomide, EPO
                   or any other treatment inducing cytopenias.
      
              Exclusion Criteria:
      
                1. MDS classified according to classical IPSS as intermediate 2 or High risk
      
                2. ARSI with SF3B1 mutation
      
                3. Transformation in Acute myeloid Leukemia (AML)
      
                4. Severe active infection or any other uncontrolled severe condition.
      
                5. Organ dysfunctions including the following
      
                     -  Hepatic : total bilirubin > 2 times upper limit of normal (ULN) (except moderate
                        unconjugated hyperbilirubinemia due to intra medullary hemolysis or Gilbert
                        syndrome) , alanine transaminase (ALT) and aspartate transaminase (AST) > 3xULN
      
                     -  Symptomatic respiratory chronic failure
      
                     -  Symptomatic cardiac failure
      
                     -  Renal clearance < 60ml/min
      
                6. Prior malignancy (except in situ cervix carcinoma, limited basal cell carcinoma, or
                   other tumors if not active during the last 3 years)
      
                7. MDS with the following causal germline disease : Fanconi anemia, GATA2 related
                   syndromes and telomere disorders
            
      Maximum Eligible Age:69 Years
      Minimum Eligible Age:18 Years
      Eligible Gender:All
      Healthy Volunteers:No

      Primary Outcome Measures

      Measure:overall survival
      Time Frame:36 months
      Safety Issue:
      Description:comparison of overall survival in patients with or without a matched donor (8/8 unrelated donor or matched sibling) at 36 months

      Secondary Outcome Measures

      Measure:quality of life
      Time Frame:12, 24 and 36 months
      Safety Issue:
      Description:comparison of quality of life in patients with or without a matched donor, quality of life assessed by questionnaire (EORTC version 3) at inclusion, 12, 24 and 36 months
      Measure:number of patients with complete response at 36 month
      Time Frame:36 months
      Safety Issue:
      Description:comparison between patients with or without a donor for cumulative incidence of complete response at 36 month
      Measure:number of patients with transformation in AML at 36 month
      Time Frame:36 months
      Safety Issue:
      Description:comparison between patients with or without a donor for cumulative incidence of transformation in AML at 36 month
      Measure:proportion of patients with iron overload
      Time Frame:16 months
      Safety Issue:
      Description:proportion of patients with iron overload (Serum Ferritin (SF)>1000 ng/mL or Red Blood Cells transfusion>20) at time of inclusion and at 16 month after inclusion for non-transplanted patients and 12 months post-transplant for transplanted patients
      Measure:evolution of innovative iron markers including Non-transferrin binding iron (NTBI), labile plasmatic Iron (LPI) and Hepcidine
      Time Frame:3 and 16 months
      Safety Issue:
      Description:evolution of innovative iron markers including Non-transferrin binding iron (NTBI), labile plasmatic Iron (LPI) and Hepcidine measured at time of inclusion, at 3 month and 16 month post-inclusion for all patients; In transplanted patients these markers will be measured just before conditioning regimen (J-5), Just before the transplantation (J0), at D7, 30, 100 and 12 month after transplant.
      Measure:efficiency of chelation
      Time Frame:3 and 16 months
      Safety Issue:
      Description:the effect of chelation will be assessed at 3 month after inclusion for all patient and post transplant by measuring Serum ferritin level
      Measure:number of patients with adverse events grade III and IV as assessed by CTCAE v4.0
      Time Frame:36 months
      Safety Issue:
      Description:comparison between patients with or without a donor for number of Grade III and IV toxicities (hematological and non-hematological) recorded according to NCI CTCAE criteria versions 4.0 during the 36 months

      Details

      Phase:N/A
      Primary Purpose:Interventional
      Overall Status:Recruiting
      Lead Sponsor:Groupe Francophone des Myelodysplasies

      Trial Keywords

      • Low risk MDS
      • Transplantation

      Last Updated