Conditioning therapy before haploidentical hematopoietic stem cell transplantation MedDRA version: 22.0 Level: LLT Classification code 10059044 Term: Allogeneic peripheral hematopoietic stem cell transplant System Organ Class: 100000004865
Conditions
Interventions
Trade Name: Trecondi
Pharmaceutical Form: Powder for solution for infusion
INN or Proposed INN: Treosulfan
CAS Number: 299-75-2
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: eq
Sponsors
Medical University of Vienna
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Patients with acute myeloid leukemia (AML) according to WHO 2016 (AML in complete remission at transplant, i.e., blast counts 2 [according to Sorror et al., 2005] 2. Availability of a haploidentical (HLA match =5/10) first- or second-degree related donor. Donor selection is based on molecular high-resolution typing (4 digits) of class II 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. 3. Adult patients of both gender, age 18-70 years 4. Karnofsky Index =60 % 5. Written informed consent 6. Men capable of reproduction and women of childbearing potential must be willing to consent to use a highly effective method of birth control such as condoms, implants, injectables, combined oral contraceptives, IUDs, sexual abstinence, or vasectomized partner while on treatment and for at least 6 months 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 20 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 10
Exclusion criteria
Exclusion criteria: 1. Availability of matched sibling donor eligible for stem cell donation 2. Patients with acute promyelocytic leukemia with t(15;17)(q22;q12) and in CR1 3. Patients considered contraindicated for HSCT due to severe concomitant illness (within 3 weeks before 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 3x ULN or ALT/AST >5 x ULN 4. Active malignant involvement of the CNS 5. HIV-positivity, active non-controlled infectious disease under treatment (no decrease of CRP or PCT) including active viral liver infection 6. Previous allogeneic HSCT 7. Pleural effusion or ascites > 1.0L 8. Pregnancy or lactation 9. Known hypersensitivity to treosulfan, fludarabine, cyclophosphamide and/or related ingredients 10. Participation in another experimental drug trial within 4 weeks before day -6 of the protocol 11. Non-cooperative behavior or non-compliance 12. Psychiatric diseases or conditions that might compromise the ability to give informed consent
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Timepoint(s) of evaluation of this end point: 1 year after haploidentical HSCT;Main Objective: •To assess the efficacy and safety of TreoFC conditioning before haploidentical hematopoietic stem cell transplantation (HSCT) in older or comorbid patients. The primary endpoint is overall survival (OS) 1 year after haploidentical HSCT.;Secondary Objective: •To assess rates of non-relapse mortality (NRM), relapse-free survival (RFS), graft-versus-host disease (GVHD) and relapse-free survival (GRFS), acute GVHD, chronic GVHD, quality of life (QOL), toxicity, engraftment, and chimerism in the study population.;Primary end point(s): Overall survival | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): • Cumulative incidence of relapse • Relapse-free survival (RFS) • Graft-versus-host disease and relapse-free survival (GRFS) • Cumulative incidence of non-relapse mortality (NRM) a • Cumulative incidence of acute graft-versus-host disease (GVHD) a • Cumulative incidence of chronic GVHD • Toxicities according to the current version of the NCI CTCAE • Engraftment • Chimerism ;Timepoint(s) of evaluation of this end point: 1 year after haploidentical HSCT | — |
Countries
Austria
Contacts
Public ContactDepartment of Meidince I / HSCT Uni
Medical University of Vienna
Outcome results
None listed