Primary or secondary myelofibrosis after essential thrombocytemia or polycyhemia vera MedDRA version: 14.0 Level: PT Classification code 10028537 Term: Myelofibrosis System Organ Class: 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Conditions
Interventions
Trade Name: TEPADINA
Pharmaceutical Form: Powder for solution for injection
INN or Proposed INN: Thiotepa
Concentration unit: mg/g milligram(s)/gram
Concentration number: 15-
Trade Name: TEPADINA
Pha
Sponsors
GITMO GRUPPO ITALIANO TRAPIANTO DI MIDOLLO OSSEO,CELLULE STAMINALI EMOPOIETICHE E TERAPIA CELLULARE - ONLUS
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: • Age = 18 = 70 years • Primary or secondary myelofibrosis after essential thrombocytemia or polycyhemia vera • One of the following unfavourable prognostic factors: - Hb 25x106/L - > 1% circulating blasts in the peripheral blood - constitutional symptoms • PS (Karnofsky)= 60% • HCT-CI = 5 • Written informed consent Are the trial subjects under 18? no Number of subjects for this age range: 0 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 40 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 20
Exclusion criteria
Exclusion criteria: • = 20% blasts in the peripheral blood and/or in bone marrow • Positive serologic markers for human immunodeficiency virus (HIV) • Acute hepatitis B virus (HBV) or acute hepatis C virus (HCV) infection • Severe irreversible renal, hepatic , pulmonary or cardiac disease , such as: - total bilirubin, SGOT or SGPT > 5 the upper normal limit - Left ventricular ejection fraction < 30% - Clearance creatinine < 30 ml/min - DLCO < 30% and/or receiving supplementary oxygen • Pregnancy or lactation • Patients not agreeing to take adequate contraceptive measures during the study • Psychiatric disease • Any active , uncontrolled infection 7.3 Donors: Inclusion criteria • Age = 18 < 65 years • HLA-identical sibling donor by high resolution DNA-based HLA-A, -B, -C , -DRB1 typing. • HLA-identical unrelated donor by high resolution DNA-based HLA-A, -B, -C , -DRB1 typing . One allele mismatched (class I) can be accepted for recipients up to 60 years .
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The primary endpoint for this study is to compare Progression Free Survival (PFS) of two different RIC regimens for allogeneic stem cell transplantation in myelofibrosis. PFS is defined as the time from the date of randomization to the date of the first documented disease progression or relapse (according to the International Working Group Consensus Criteria, appendix B) or death due to any cause. Patients who have neither progressed nor died at the time of study completion or who are lost to follow-up are censored at the data of the last follow up for progression of disease for this study.;Secondary Objective: • Non relapse mortality (NRM) is defined as death due to any other cause than progression of malignancy after allogeneic stem cell transplantation. • Overall survival is defined as the time between randomization and the date of death due to any cause or the last date the patient was known to be alive (censored observation) at the date of the data cut-off for the final analysis • Rate of clinical hematological and histological responses (according to IWG consensus criteria, appendix B) • Rate of molecular remissions in patients having a molecular marker (according to IWG consensus criteria, appendix B) • Cumulative incidence of engrafment. The day of engrafment is defined as the first 3 consecutive days on which the blood granulocyte count rises to 0.5 x 109/L • Incidence of acute graft versus host disease • Incidence of chronic graft versus host disease;Primary end point(s): • Progression -free survival at one year;Timepoint(s) of evaluation of this end point: 3 years of patients enrolment plus 1 year of minimum follow-up for last patient enrolled (total 4 years) | — |
Secondary
| Measure | Time frame |
|---|---|
| Timepoint(s) of evaluation of this end point: 3 years of patients enrolment plus 1 year of minimum follow-up for last patient enrolled (total 4 years);Secondary end point(s): • Non relapse mortality at one year • Overall survival • Rate of clinical hematological and histological responses (according to International Working Group consensus criteria) • Rate of molecular remissions in patients having a molecular marker • Engraftment • Incidence of acute graft versus host disease • Incidence of chronic graft versus host disease | — |
Countries
Italy
Contacts
Public Contactcoordinatrice sperimentazioni
gitmo
Outcome results
None listed