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

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ClinicalTrials.gov Identifier: NCT02757989

Recruitment Status : Recruiting

First Posted : May 2, 2016

Last Update Posted : November 3, 2020

See Contacts and Locations

Sponsor:

Collaborators:

Novartis

Neovii Biotech

Information provided by (Responsible Party):

Groupe Francophone des Myelodysplasies

Study Description

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.

Study Design

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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 2021
Estimated Study Completion Date :	June 2022

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Myelodysplastic Syndromes

Genetic and Rare Diseases Information Center resources: Myelodysplastic Syndromes

U.S. FDA Resources

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 :

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 :

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
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
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
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
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.
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
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

Eligibility Criteria

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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:

Signed Informed consent
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)
Patient aged ≥ 18 and < 70 years For young patients, 18-45 years, Fanconi disease and dyskeratosis should be ruled out
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
Performance status 0-2 on the Eastern Cooperative Oncology Group (ECOG) Scale (At time of screening)
Negative pregnancy and adequate contraception (including in male patients wishing to father), if relevant.
Wash-out of at least 30 days since a previous treatment with Vidaza, Lenalidomide, EPO or any other treatment inducing cytopenias.

Exclusion Criteria:

MDS classified according to classical IPSS as intermediate 2 or High risk
Transformation in Acute myeloid Leukemia (AML)
Severe active infection or any other uncontrolled severe condition.
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
Prior malignancy (except in situ cervix carcinoma, limited basal cell carcinoma, or other tumors if not active during the last 3 years)
MDS with the following causal germline disease : Fanconi anemia, GATA2 related syndromes and telomere disorders

Contacts and Locations

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 37 study locations

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France
CHU d'Amiens	Recruiting
Amiens, France, 80054
Contact: Amandine Charbonnier, MD +33 3 22 45 59 14 charbonnier.amandine@chu-amiens.fr
Principal Investigator: Amandine Charbonnier, MD
CHU d'Angers	Recruiting
Angers, France, 49933
Contact: Sylvain Thépot, MD +33 2 41 35 44 66 sylvain.thepot@chu-angers.fr
Principal Investigator: Sylvain Thépot, MD
Centre hospitalier Victor Dupouy	Not yet recruiting
Argenteuil, France, 95100
Contact: Ahmad Aljijakli, MD +33 1 34 23 22 39 ahmad.aljijakli@ch-argenteuil.fr
Principal Investigator: Ahmad Aljijakli, MD
CHU Jean Minjoz	Recruiting
Besançon, France, 25030
Contact: Eric Deconinck, MD, PHD +33 3 81 66 83 51 edeconinck@chu-besancon.fr
Principal Investigator: Eric Deconinck, MD, PHD
Principal Investigator: Ana Berceanu, MD
Hôpital Avicenne	Not yet recruiting
Bobigny, France, 93009
Contact: Thorsten Braun, MD +33 1 48 95 70 57 thorsten.braun@aphp.fr
Principal Investigator: Thorsten Braun, MD
CHU de Haut Lévèque	Recruiting
Bordeaux, France, 33604
Contact: Edouard Forcade, MD +33 5 57 65 67 27 edouard.forcade@chu-bordeaux.fr
Principal Investigator: Edouard Forcade, MD
Principal Investigator: Sophie Dimicoli-Salazar, MD
CHRU Côte de Nacre	Recruiting
Caen, France, 14033
Contact: Stéphane Cheze, MD +33 2 31 27 23 60 cheze-s@chu-caen.fr
Principal Investigator: Stéphane Cheze, MD
Principal Investigator: Oumedaly Reman, MD
CHU Estaing	Recruiting
Clermont Ferrand, France, 63000
Contact: Jacques Olivier Bay, MD +33 4 73 75 00 65 jobay@chu-clermontferrand.fr
Principal Investigator: Jacques Olivier Bay, MD
Principal Investigator: Benoit De Renzis, MD
CHSF Gilles de Corbeil	Not yet recruiting
Corbeil-Essonnes, France, 91106
Contact: Stéphanie Haïat, MD +33 1 61 69 61 69 stephanie.haiat@chsf.fr
Principal Investigator: Stéphanie Haïat, MD
Hôpital Henri Mondor	Not yet recruiting
Créteil, France, 94010
Contact: Andrea Toma, MD +33 1 49 81 20 60 andrea.toma@aphp.fr
Principal Investigator: Andrea Toma, MD
CHU de Grenoble	Recruiting
Grenoble, France, 38043
Contact: Martin Carré, MD +33 4 76 76 57 55 mcarre1@chu-grenoble.fr
Principal Investigator: Martin Carré, MD
Principal Investigator: Sophie Park, MD, PHD
CH Le Mans	Not yet recruiting
Le Mans, France, 72037
Contact: Kamel Laribi, MD +33 2 43 43 43 61 klaribi@ch-lemans.fr
Principal Investigator: Kamel Laribi, MD
Hôpital Saint Vincent de Paul	Recruiting
Lille, France, 59020
Contact: Laurent Pascal, MD, PHD +33 3 20 87 45 32 pascal.laurent@ghicl.net
Principal Investigator: Laurent Pascal, MD, PHD
Hôpital Huriez	Recruiting
Lille, France, 59037
Contact: Ibrahim Yakoub-Agha, MD, PHD +33 3 20 44 55 51 ibrahim.yakoub-agha@chru-lille.fr
Principal Investigator: Ibrahim Yakoub-Agha, MD, PHD
Hôpital Dupuytren	Recruiting
Limoges, France, 87042
Contact: Pascal Turlure, MD +33 5 55 05 80 39 pascal.turlure@chu-limoges.fr
Principal Investigator: Marie-Pierre Gourin, MD
Principal Investigator: Pascal Turlure, MD
Centre hospitalier Lyon Sud	Recruiting
Lyon, France, 69495
Contact: Hélène Labussière-Wallet, MD, PHD +33 4 78 86 22 05 helene.labussiere-wallet@chu-lyon.fr
Principal Investigator: Hélène Labussière-Wallet, MD, PHD
Principal Investigator: Eric Wattel, MD, PHD
GHEF, site de Meaux	Not yet recruiting
Meaux, France, 77100
Contact: Loïc Fouillard, MD +33 1 64 35 38 76 lfouillard@ghef.fr
Principal Investigator: Loïc Fouillard, MD
CHRU de Montpellier	Recruiting
Montpellier, France, 34295
Contact: Nathalie Fegueux, MD +33 4 67 33 83 63 n-fegueux@chu-montpellier.fr
Principal Investigator: Nathalie Fegueux, MD
CHU de Nantes	Recruiting
Nantes, France, 44093
Contact: Patrice Chevallier, MD +33 2 40 08 33 33 patrice.chevallier@chu-nantes.fr
Principal Investigator: Patrice Chevallier, MD
Principal Investigator: Pierre Peterlin, MD
CHU de Nice	Recruiting
Nice, France, 06202
Contact: Thomas Cluzeau, MD +33 4 92 03 58 44 cluzeau.t@chu-nice.fr
Principal Investigator: Thomas Cluzeau, MD
CHU de Nîmes	Not yet recruiting
Nîmes, France, 30029
Contact: Stefan Wickenhauser, MD +33 4 66 68 32 31 stefan.wickenhauser@chu-nimes.fr
Principal Investigator: Stefan Wickenhauser, MD
Hôpital Saint Louis	Recruiting
Paris, France, 75010
Contact: Marie Robin, MD +33 1 42 49 96 60 marie.robin@aphp.fr
Principal Investigator: Marie Robin, MD
Principal Investigator: Marie Sébert, MD
Hôpital Pitié Salpétrière	Recruiting
Paris, France, 75013
Contact: Stéphanie Nguyen-Quoc, MD +33 1 42 16 28 25 stephanie.nguyen-quoc@aphp.fr
Principal Investigator: Stéphanie Nguyen-Quoc, MD
Hôpital Cochin	Not yet recruiting
Paris, France, 75014
Contact: Lise Willems, MD +33 1 58 41 26 70 lise.willems@aphp.fr
Principal Investigator: Lise Willems, MD
Hôpital Necker	Recruiting
Paris, France, 75015
Contact: Olivier Hermine, MD, PHD +33 1 44 49 53 68 olivier.hermine@aphp.fr
Principal Investigator: Olivier Hermine, MD, PHD
Principal Investigator: Felipe Suarez, MD
CH Joffre	Not yet recruiting
Perpignan, France, 66046
Contact: Laurence Sanhes, MD +33 4 68 61 64 48 laurence.sanhes@ch-perpignan.fr
Principal Investigator: Laurence Sanhes, MD
CHU de Poitiers	Recruiting
Poitiers, France, 86021
Contact: Natacha Maillard, MD +33 5 48 44 44 44 natacha.maillard@chu-poitiers.fr
Principal Investigator: Jose Miguel Torregrosa Diaz, MD
Principal Investigator: Natacha Maillard, MD
CH René Dubos	Not yet recruiting
Pontoise, France, 95300
Contact: Ioana-Dana Vaida, MD +33 1 30 75 49 28 ioana.vaida@ght-novo.fr
Principal Investigator: Ioana-Dana Vaida, MD
CHU de Reims	Not yet recruiting
Reims, France, 51092
Contact: Chantal Himberlin, MD +33 3 26 78 36 44 chimberlin@chu-reims.fr
Principal Investigator: Chantal Himberlin, MD
Hôpital Pontchaillou	Not yet recruiting
Rennes, France, 35033
Contact: Stanislas Nimubona, MD +33 2 99 28 95 21 stanislas.nimubona@chu-rennes.fr
Principal Investigator: Stanislas Nimubona, MD
Centre Henri Becquerel	Recruiting
Rouen, France, 76038
Contact: Aspasia Stamatoullas, MD +33 2 32 08 22 88 aspasia.stamatoullas@chb.unicancer.fr
Principal Investigator: Aspasia Stamatoullas, MD
Institut Curie	Not yet recruiting
Saint-Cloud, France, 92210
Contact: Jacques Vargaftig, MD +33 1 47 11 15 36 jacques.vargaftig@curie.fr
Principal Investigator: Jacques Vargaftig, MD
Institut de cancérologie Lucien Neuwirth	Not yet recruiting
Saint-Priest-en-Jarez, France, 42271
Contact: Jérôme Cornillon, MD +33 4 77 91 70 60 jerome.cornillon@icloire.fr
Principal Investigator: Jérôme Cornillon, MD
Hôpital civil	Not yet recruiting
Strasbourg, France, 67091
Contact: Shanti Natarajan-Amé, MD +33 3 88 11 67 28 shanti.ame@chru-strasbourg.fr
Principal Investigator: Shanti Natarajan-Amé, MD
IUCT-Oncopole	Recruiting
Toulouse, France, 31059
Contact: Anne Huynh, MD +33 5 31 15 61 91 huynh.anne@iuct-oncopole.fr
Principal Investigator: Anne Huynh, MD
Principal Investigator: Odile Beyne-Rauzy, MD, PHD
Principal Investigator: Christian Recher, MD, PHD
Hôpital Bretonneau	Not yet recruiting
Tours, France, 37000
Contact: Emmanuel Gyan, MD, PHD +33 2 47 25 87 78 e.gyan@chu-tours.fr
Principal Investigator: Emmanuel Gyan, MD, PHD
Hôpital de Brabois	Recruiting
Vandoeuvre les nancy, France, 54550
Contact: Maud D'Aveni, MD +33 3 83 15 30 30 m.daveni-piney@chru-nancy.fr
Principal Investigator: Maud D'Aveni, MD

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

More Information

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

Robin M, Fenaux P. Which lower risk myelodysplastic syndromes should be treated with allogeneic hematopoietic stem cell transplantation? Leukemia. 2020 Oct;34(10):2552-2560. doi: 10.1038/s41375-020-0967-x. Epub 2020 Jul 13. Review.

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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:	November 3, 2020
Last Verified:	November 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

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