MiniMUD Study - Unrelated Reduced Intensity Conditioning With Treosulfan® for Allogeneic Stem Cell Transplantation in Patients With Hematological Malignancies

Study Overview
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Brief Summary
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In this study, treosulfan is evaluated for conditioning in allogenic stem cell transplantation. The procedure and the follow-up are the same as in standard allogenic transplant. The donor is unrelated (identical HLA). The graft is haematological peripheral blood stem cell. The conditioning with reduced intensity is: fludarabine (from day -6 to day -2), treosulfan (from day -6 to day -4) and thymoglobuline (from day -2 to day -1).

Official Title
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Unrelated Reduced Intensity Conditioning With Treosulfan® for Allogeneic Stem Cell Transplantation in Patients With Hematological Malignancies

Conditions
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Hematological Malignancies, Allogeneic Transplantation

Intervention / Treatment
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* Drug: treosulfan


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: AGE: >= 18 years and <= 65 years Patients with a too high transplant-related mortality (TRM) after standard transplantation (multiple myeloma, chronic lymphoid leukemia, non Hodgkin's lymphoma, myelodysplasia) Patients with visceral contra-indication for standard transplantation: cardiac: myocardiopathy; forced expiratory volume (FEV) < 50%; respiratory: abnormal carbon monoxide diffusing capacity (DLCO); renal: creatinine clearance < 50ml/min; hepatic: transaminases and bilirubin > 2 upper normal limit; infectious: controlled fungal infection. Karnofsky score >= 70% Unrelated donor HLA identical (ABC, DRB1; DQB1) Signed informed consent Diagnosis : Chronic myelogenous leukemia (CML): In first chronic phase, resistant to interferon with or without aracytine or refractory or resistant to Glivec In complete response (CR) or in 2nd partial response (PR) after being in blastic phase Multiple myeloma (MM): Relapse after autograft if the therapeutic response was evaluated to 50% Non-Hodgkin's lymphoma (NHL): Mantle cell lymphoma after first relapse but in case of chemosensitivity ≥ 50% except for high grade lymphoma In 2nd CR or PR chemosensitive in response ≥ 50% after autograft Chronic lymphocytic leukemia (CLL): In 2nd CR or PR or in response ≥ 50% after autograft or in 2nd relapse after 2 lines of treatment but in case of chemosensitivity ≥ 50% Acute myeloid leukemia (AML): In 2nd CR or in 1st CR for high risk criteria [high risk criteria defined by: LAM 7; leukocytes > 30,000/mm3; chromosomal abnormalities: t(6,9); abnormalities of 11q23, 17p, 11q, 20q, 21q, -5, del(5q), -7/del7q, del 9q et inv 3q] Acute lymphoblastic leukemia (ALL): In 2nd CR or in 1st CR if high risk criteria patients who are defined by chromosomal abnormalities t(9,22); t(1,19); t(4,11); abnormalities of 11q23 Myelodysplastic syndromes (MDS): Patients without prior chemotherapy, with intermediate or high International Prognostic Scoring System (IPSS) score and blast cells < 1% in bone marrow (BM) CR or PR after chemotherapy for patients with 20 to 30% of blast cells in BM Secondary AML patients with a response to chemotherapy (< 30% blasts in BM and < 5% of blast cells in blood) For all: Adequate contraception in female patients of child bearing potential

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 65 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Intervention/Treatment |
| --- |
|Drug: treosulfan|nan|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Overall survival at 1 year |  |  | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Engraftment evaluation |  |  | 
| Acute and chronic graft-versus-host disease incidence and severity |  |  | 
| Response rate and survival without progression |  |  | 
| Evaluation of conditioning and transplant toxicity |  |  | 
| Chimerism evaluation |  |  | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Allogenic stem cell transplantation, Treosulfan, Haematological malignancies, allogeneic stem cell transplantation in patients with hematological malignancies