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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02757989
Recruitment Status : Recruiting
First Posted : May 2, 2016
Last Update Posted : April 6, 2020
Sponsor:
Collaborators:
Novartis
Neovii Biotech
Information provided by (Responsible Party):
Groupe Francophone des Myelodysplasies

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

Condition or disease Intervention/treatment Phase
MDS Other: transplantation Not Applicable

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.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 105 participants
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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
Actual Study Start Date : May 31, 2016
Estimated Primary Completion Date : June 2020
Estimated Study Completion Date : June 2021

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: Patients with donor
Patients with a matched donor (8/8 at molecular level unrelated donor or matched sibling)
Other: transplantation
allogeneic hematopoietic stem cell transplantation in patients with donor

No Intervention: Patients without donor
Patients without a matched donor



Primary Outcome Measures :
  1. overall survival [ Time Frame: 36 months ]
    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 :
  1. quality of life [ Time Frame: 12, 24 and 36 months ]
    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

  2. number of patients with complete response at 36 month [ Time Frame: 36 months ]
    comparison between patients with or without a donor for cumulative incidence of complete response at 36 month

  3. number of patients with transformation in AML at 36 month [ Time Frame: 36 months ]
    comparison between patients with or without a donor for cumulative incidence of transformation in AML at 36 month

  4. proportion of patients with iron overload [ Time Frame: 16 months ]
    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

  5. evolution of innovative iron markers including Non-transferrin binding iron (NTBI), labile plasmatic Iron (LPI) and Hepcidine [ Time Frame: 3 and 16 months ]
    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.

  6. efficiency of chelation [ Time Frame: 3 and 16 months ]
    the effect of chelation will be assessed at 3 month after inclusion for all patient and post transplant by measuring Serum ferritin level

  7. number of patients with adverse events grade III and IV as assessed by CTCAE v4.0 [ Time Frame: 36 months ]
    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



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Ages Eligible for Study:   18 Years to 69 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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…)
    3. thrombocytopenia < 20 G/L requiring transfusion
    4. neutropenia < 0.5 G/L associated with severe infection (defined as requiring hospitalization)
  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. Transformation in Acute myeloid Leukemia (AML)
  3. Severe active infection or any other uncontrolled severe condition.
  4. 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
  5. Prior malignancy (except in situ cervix carcinoma, limited basal cell carcinoma, or other tumors if not active during the last 3 years)
  6. MDS with the following causal germline disease : Fanconi anemia, GATA2 related syndromes and telomere disorders

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): NCT02757989


Contacts
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Contact: Marie Robin, MD +33 1 42 49 96 60 marie.robin@aphp.fr
Contact: Marie Sébert, MD +33 1 71 20 70 23 marie.sebert@aphp.fr

Locations
Show Show 37 study locations
Sponsors and Collaborators
Groupe Francophone des Myelodysplasies
Novartis
Neovii Biotech
Investigators
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Principal Investigator: Marie Robin, MD Saint-Louis Hospital, Paris, France
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Responsible Party: Groupe Francophone des Myelodysplasies
ClinicalTrials.gov Identifier: NCT02757989    
Other Study ID Numbers: MDS-ALLO-RISK
2015-A00292-47 ( Other Identifier: IDRCB Number )
First Posted: May 2, 2016    Key Record Dates
Last Update Posted: April 6, 2020
Last Verified: April 2020
Keywords provided by Groupe Francophone des Myelodysplasies:
Low risk MDS
Transplantation
Additional relevant MeSH terms:
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Preleukemia
Myelodysplastic Syndromes
Bone Marrow Diseases
Hematologic Diseases
Precancerous Conditions
Neoplasms