Clinical phase III trial to compare Treosulfan-based conditioning therapy with Busulfan-based reduced-intensity conditioning (RIC) prior to allogeneic haematopoietic stem cell transplantation in patients with AML or MDS considered ineligible to standard conditioning regimens - Clinical Phase III Treosulfan based conditioning vs RIC

Phase 1

• Conditions: Patients with acute myeloid leukaemia (AML)or myelodysplastic syndrome (MDS) considered ineligible to standard conditioning therapies prior to allogenic stem cell transplantation; MedDRA version: 9.1Level: LLTClassification code 10000881Term: Acute myeloid leukaemia (in remission); MedDRA version: 9.1Level: LLTClassification code 10028534Term: Myelodysplastic syndrome NOS

• Registration Number: EUCTR2008-002356-18-IT
• Lead Sponsor: MEDAC RESEARCH

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2008-11-19
• Last Update Posted: 7 January 2019

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 545

Inclusion Criteria

1. Patients with acute myeloid leukemia acc. to WHO, 2001 (AML in complete remission at transplant, i.e. blast counts < 5 % in bone marrow) or myelodysplastic syndrome acc. to WHO, 2001 (MDS with blast counts < 20 % in bone marrow during disease history) indicated for allogeneic haematopoietic progenitor cell transplantation but considered to be at increased risk for standard conditioning therapies according to the following criteria: - patients aged &#8805; 50 years at transplant and / or - patients with a HCT-CI score> 2 [acc. to Sorror et al,, 2005]. 2. Availability of an HLA-identical sibling donor (MRD) or HLAidentical unrelated donor (MUD). Donor selection is based on molecular high-resolution typing (4 digits) of class Il alleles of the DRB1 and DQB1 gene loci and molecular (at least) low-resolution typing (2 digits) of class I alleles (i.e., antigens) of the HLA-A, B and C gene loci. In case, no class I and class II completely identical donor (10 out of 10 gene loci) can be identified, one antigen disparity (class I) and/or one allele disparity (class Il) between patient and donor are acceptable. Conversely, disparity of two antigens (irrespective of the involved gene loci) cannot be accepted. These definitions for the required degree of histocompatibility apply to the selection of related as well as unrelated donors. 3. Adult patients of both gender, age 18 - 70 years. 4. Karnofsky Index &#8805; 60%. 5. Written informed consent. 6. Men capable of reproduction and women of childbearing potential must be willing to consent using a highly effective metod of birth control such as condoms, implants, injectables, combined oral contraceptives, IUDs, sexual abstinence or vasectomised partner while on treatment and for at least 6 mounths thereafter.
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

1. Patients with acute promyelocytic leukaemia with t(15;17)(q22;q12) in CR1. 2. Patients considered contra-indicated for allogeneic HSCT due to severe concomitant Illness (within three weeks prior to scheduled day -6): - patients with severe renal impairment like patients on dialysis or prior renal transplantation or S-creatinine> 3.0 x ULN or calculated creatinine-clearance < 60 ml/min; -patients with severe pulmonary impairment, DLCO/or FEV1 <50 % or severe dyspnoea at rest or requiring oxygen supply; - patients with severe cardiac impairment diagnosed by echocardiography and LVEF <40 % ; - patients with severe hepatic impairment indicated by hyperbilirubinaemia > 3 x ULN or ALT/AST> 5 x ULN . 3. Active malignant involvement of the CNS. 4. HIV-positivity, active non-controlled infectious disease under treatment (no decrease of CRP or PCT) including active viral liver infection. 5. Previous allogeneic HSCT. 6. Pleural effusion or ascites> 1.0 L. 7. Pregnancy or Lactation. 8 Known hypersensitivity to treosulfan, busulfan and/or related ingredients. 9. Participation in another experimental drug trial within 4 weeks prior to day - 6 of the protocol. 10. Non-cooperative behavior or non-compliance. 11. Psychiatric diseases or conditions that might compromise the ability to give informed consent.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified