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

NCT ID: NCT02757989

Last Updated: 2024-08-02

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

NA

Total Enrollment

79 participants

Study Classification

INTERVENTIONAL

Study Start Date

2016-05-31

Study Completion Date

2024-06-07

Brief Summary

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

Detailed Description

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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.

Conditions

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MDS

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Patients with donor

Patients with a matched donor (8/8 at molecular level unrelated donor or matched sibling)

Group Type EXPERIMENTAL

transplantation

Intervention Type OTHER

allogeneic hematopoietic stem cell transplantation in patients with donor

Patients without donor

Patients without a matched donor

Group Type NO_INTERVENTION

No interventions assigned to this group

Interventions

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transplantation

allogeneic hematopoietic stem cell transplantation in patients with donor

Intervention Type OTHER

Eligibility Criteria

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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
Minimum Eligible Age

18 Years

Maximum Eligible Age

69 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Novartis

INDUSTRY

Sponsor Role collaborator

Neovii Biotech

INDUSTRY

Sponsor Role collaborator

Groupe Francophone des Myelodysplasies

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Marie Robin, MD

Role: PRINCIPAL_INVESTIGATOR

Saint-Louis Hospital, Paris, France

Locations

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CHU d'Amiens

Amiens, , France

Site Status

CHU d'Angers

Angers, , France

Site Status

Centre hospitalier Victor Dupouy

Argenteuil, , France

Site Status

CHU Jean Minjoz

Besançon, , France

Site Status

Hôpital Avicenne

Bobigny, , France

Site Status

CHU de Haut Lévèque

Bordeaux, , France

Site Status

CHRU Côte de Nacre

Caen, , France

Site Status

CHU Estaing

Clermont-Ferrand, , France

Site Status

CHSF Gilles de Corbeil

Corbeil-Essonnes, , France

Site Status

Hôpital Henri Mondor

Créteil, , France

Site Status

CHU de Grenoble

Grenoble, , France

Site Status

CH Le Mans

Le Mans, , France

Site Status

Hôpital Saint Vincent de Paul

Lille, , France

Site Status

Hôpital Huriez

Lille, , France

Site Status

Hôpital Dupuytren

Limoges, , France

Site Status

Centre hospitalier Lyon Sud

Lyon, , France

Site Status

GHEF, site de Meaux

Meaux, , France

Site Status

CHRU de Montpellier

Montpellier, , France

Site Status

CHU de Nantes

Nantes, , France

Site Status

CHU de Nice

Nice, , France

Site Status

CHU de Nîmes

Nîmes, , France

Site Status

Hôpital Saint Louis

Paris, , France

Site Status

Hôpital Pitié Salpétrière

Paris, , France

Site Status

Hôpital Cochin

Paris, , France

Site Status

Hôpital Necker

Paris, , France

Site Status

CH Joffre

Perpignan, , France

Site Status

CHU de Poitiers

Poitiers, , France

Site Status

CH René Dubos

Pontoise, , France

Site Status

CHU de Reims

Reims, , France

Site Status

Hôpital Pontchaillou

Rennes, , France

Site Status

Centre Henri Becquerel

Rouen, , France

Site Status

Institut Curie

Saint-Cloud, , France

Site Status

Institut de cancérologie Lucien Neuwirth

Saint-Priest-en-Jarez, , France

Site Status

Hôpital civil

Strasbourg, , France

Site Status

IUCT-Oncopole

Toulouse, , France

Site Status

Hôpital Bretonneau

Tours, , France

Site Status

Hôpital de Brabois

Vandœuvre-lès-Nancy, , France

Site Status

Countries

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France

References

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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.

Reference Type DERIVED
PMID: 32661295 (View on PubMed)

Other Identifiers

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2015-A00292-47

Identifier Type: OTHER

Identifier Source: secondary_id

MDS-ALLO-RISK

Identifier Type: -

Identifier Source: org_study_id

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