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

Sponsor
Groupe Francophone des Myelodysplasies (Other)
Overall Status
Active, not recruiting
CT.gov ID
NCT02757989
Collaborator
Novartis (Industry), Neovii Biotech (Industry)
79
Enrollment
37
Locations
2
Arms
96
Anticipated Duration (Months)
2.1
Patients Per Site
0
Patients Per Site Per Month

Study Details

Study Description

Brief Summary

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

Condition or Disease Intervention/Treatment Phase
  • Other: transplantation
 N/A

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.

Study Design

Study Type:
Interventional
Actual Enrollment :
79 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
Anticipated Primary Completion Date :
Jun 1, 2024
Anticipated Study Completion Date :
Jun 1, 2024

Arms and Interventions

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

Outcome Measures

Primary Outcome Measures

  1. overall survival [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 [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 [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 [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 [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 [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 [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 [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

Eligibility Criteria

Criteria

Ages Eligible for Study:
18 Years to 69 Years
Sexes Eligible for Study:
All
Accepts Healthy Volunteers:
No
Inclusion Criteria:

  1. Signed Informed consent

  2. Classical IPSS intermediate 1 or low myelodysplastic syndrome associated with at least one poor prognosis feature:

  3. Intermediate or higher risk revised IPSS

  4. RBC transfusion dependent anemia and failure to 2 or more lines or therapy (including EPO, Lenalidomide or demethylating agent…)

  5. thrombocytopenia < 20 G/L requiring transfusion

  6. neutropenia < 0.5 G/L associated with severe infection (defined as requiring hospitalization)

  7. Patient aged ≥ 18 and < 70 years For young patients, 18-45 years, Fanconi disease and dyskeratosis should be ruled out

  8. 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

  9. Performance status 0-2 on the Eastern Cooperative Oncology Group (ECOG) Scale (At time of screening)

  10. Negative pregnancy and adequate contraception (including in male patients wishing to father), if relevant.

  11. 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

  1. Prior malignancy (except in situ cervix carcinoma, limited basal cell carcinoma, or other tumors if not active during the last 3 years)

  2. MDS with the following causal germline disease : Fanconi anemia, GATA2 related syndromes and telomere disorders

Contacts and Locations

Locations

Site City State Country Postal Code
1 CHU d'Amiens Amiens France 80054
2 CHU d'Angers Angers France 49933
3 Centre hospitalier Victor Dupouy Argenteuil France 95100
4 CHU Jean Minjoz Besançon France 25030
5 Hôpital Avicenne Bobigny France 93009
6 CHU de Haut Lévèque Bordeaux France 33604
7 CHRU Côte de Nacre Caen France 14033
8 CHU Estaing Clermont Ferrand France 63000
9 CHSF Gilles de Corbeil Corbeil-Essonnes France 91106
10 Hôpital Henri Mondor Créteil France 94010
11 CHU de Grenoble Grenoble France 38043
12 CH Le Mans Le Mans France 72037
13 Hôpital Saint Vincent de Paul Lille France 59020
14 Hôpital Huriez Lille France 59037
15 Hôpital Dupuytren Limoges France 87042
16 Centre hospitalier Lyon Sud Lyon France 69495
17 GHEF, site de Meaux Meaux France 77100
18 CHRU de Montpellier Montpellier France 34295
19 CHU de Nantes Nantes France 44093
20 CHU de Nice Nice France 06202
21 CHU de Nîmes Nîmes France 30029
22 Hôpital Saint Louis Paris France 75010
23 Hôpital Pitié Salpétrière Paris France 75013
24 Hôpital Cochin Paris France 75014
25 Hôpital Necker Paris France 75015
26 CH Joffre Perpignan France 66046
27 CHU de Poitiers Poitiers France 86021
28 CH René Dubos Pontoise France 95300
29 CHU de Reims Reims France 51092
30 Hôpital Pontchaillou Rennes France 35033
31 Centre Henri Becquerel Rouen France 76038
32 Institut Curie Saint-Cloud France 92210
33 Institut de cancérologie Lucien Neuwirth Saint-Priest-en-Jarez France 42271
34 Hôpital civil Strasbourg France 67091
35 IUCT-Oncopole Toulouse France 31059
36 Hôpital Bretonneau Tours France 37000
37 Hôpital de Brabois Vandoeuvre les nancy France 54550

Sponsors and Collaborators

  • Groupe Francophone des Myelodysplasies
  • Novartis
  • Neovii Biotech

Investigators

  • Principal Investigator: Marie Robin, MD, Saint-Louis Hospital, Paris, France

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.
Responsible Party:
Groupe Francophone des Myelodysplasies
ClinicalTrials.gov Identifier:
NCT02757989
Other Study ID Numbers:
  • MDS-ALLO-RISK
  • 2015-A00292-47
First Posted:
May 2, 2016
Last Update Posted:
Jul 6, 2022
Last Verified:
Jul 1, 2022
Keywords provided by Groupe Francophone des Myelodysplasies
Low risk MDS
Transplantation
Additional relevant MeSH terms:
Preleukemia
Myelodysplastic Syndromes

Study Results

No Results Posted as of Jul 6, 2022