A Phase II Study on Allogeneic Stem Cell Transplantation in Patients With Active Acute Leukemia
Gandalf-01
A Phase II Multicentre Open-label Study on Allogeneic Stem Cell Transplantation From Unrelated, Cord-blood and Family Haploidentical Donors in Patients With Active Acute Leukemia
1 other identifier
interventional
101
1 country
30
Brief Summary
The experimental treatment consists in the application of a therapeutic strategy of allogeneic transplantation as a potential curative procedure in a population of patients with chemoresistant acute leukemias. Therapeutic intervention, namely the conditioning regimen as well as GVHD prophylaxis, are based on regimens currently in standard use in the context of allogeneic transplantation.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P50-P75 for phase_2
Started Jul 2013
Typical duration for phase_2
30 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
March 14, 2013
CompletedFirst Posted
Study publicly available on registry
March 20, 2013
CompletedStudy Start
First participant enrolled
July 1, 2013
CompletedPrimary Completion
Last participant's last visit for primary outcome
June 30, 2017
CompletedStudy Completion
Last participant's last visit for all outcomes
June 30, 2017
CompletedMarch 10, 2023
March 1, 2023
4 years
March 14, 2013
March 9, 2023
Conditions
Keywords
Outcome Measures
Primary Outcomes (5)
Overall Survival (OS)
The overall survival at 2 years (from time of enrolment) of all patients enrolled in to the study (either transplanted or not). This is the simplest outcome, defined as the probability of survival irrespective of disease state at any point in time. Patients alive at their last follow-up are censored. It is analyzed by the Kaplan-Meier method, Log-Rank Test and parametric or semiparametric survival models.
2 years from time of enrolment
Disease-Free Survival (DFS)
DFS is defined as the probability of being alive free of disease at any point in time. Thus, death or disease relapse are treated as events (1). Patients alive and free of disease at their last follow-up are censored. The statistical methods for the analysis of DFS are the same as for OS (Kaplan-Meier curve, Log-Rank Test and survival models).
2 years from enrolment
Relapse Incidence (RI)
RI is defined as the probability of having had a relapse before time t. Death without experiencing a relapse is a competing event. The correct method of analysis is therefore the estimation of the Cumulative Incidence curve, comparable by the Gray Test and, for the multivariate analysis, the application of the proportional hazard model for the sub-distribution of competing risks, by Fine and Gray. In studying relapse, sometimes the interest is not only in the estimation of the cumulative incidence curve, but also in the estimation of the hazard ratios for comparing groups of patients. It is therefore common to apply also a survival (Cox or parametric) model considering relapse as an event and death without relapse as a censoring (the response time is given by the minimum between time to relapse and time to death without relapse; as usual, a patient who is alive and free of relapse is also censored).
2 years from enrolment
Non-Relapse Mortality (NRM)
It is defined as the probability of dying without previous occurrence of a relapse, which is a competing event. The same indications as for the analysis of RI apply.
2 years from enrolment
Progression-Free Survival (PFS)
It is defined as the probability of being alive with no indication of disease progression (relapse is considered as progression for patients in CR). It is analyzed by KaplanMeier curve, Log-Rank Test and parametric or non-parametric survival models.
2 years from enrolment
Secondary Outcomes (3)
Haematopoietic Recovery
participants will be followed for the duration of hospital stay, an expected average of 30 days
Acute Graft-versus-Host Disease (aGvHD)
from date of transplant to until the date of first event of aCGVD assessed up to 100 days post transplant
Chronic Graft-versus-Host Disease (cGvHD)
from day +100 post transplant to until the date of first event to cGVHD assessed up to 2 years post enrolment
Study Arms (1)
allogenic transplant
EXPERIMENTALThe experimental treatment consists in the application of a therapeutic strategy of allogeneic transplantation as a potential curative procedure in a population of patients with chemoresistant acute leukemias. Therapeutic intervention, namely the conditioning regimen as well as GVHD prophylaxis, are based on regimens currently in standard use in the context of allogeneic transplantation.
Interventions
Allogenic transplant using either a Marrow Unrelated Donor or a Cord Blood unit or a family Haploidentical donor. The conditioning regimen in standard use is: Thiotepa (Tepadina) i.v. 5 mg/kg/daily (total dose 10 mg/kg) day -7 and -6; Busulfan (Busilvex) i.v. 3,2 mg/kg/day (total dose 9,6 mg/kg) as a single daily dose day -5, -4, -3; Fludarabine i.v. 50 mg/m2 (total dose150 mg/m2) day -5, -4, -3. Primary antifungal prophylaxis is Micafungin 50 mg/die i.v. (1 mg/kg if \<40 kg) day 0 to engraftment. After engraftment continue antifungal prophylaxis according to local practice.
Eligibility Criteria
You may qualify if:
- Diagnosis of Primary induction Failure or chemoresistant relapse in Acute Myeloid Leukemia (AML) or Acute Lymphoblastic Leukemia (ALL) patients
- Activation of an alternative donor search by the Italian Bone Marrow Donor Registry (IBMDR)
- Age \>=18\<=70
- Unavailability of a HLA-matched related donor (MRD)
- Performance status: ECOG\<=3
- Written and signed informed consent
- Life expectancy not severely limited by concomitant illness.
You may not qualify if:
- Previous allogeneic transplant (autologous transplant is accepted)
- Positive pregnancy test
- Any active, uncontrolled infection.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (30)
Azienda Ospedaliera SS Antonio e Biagio
Alessandria, Italy
Policlinico
Bari, Italy
Divisione di Ematologia - Ospedali Papa Giovanni XXIII
Bergamo, Italy
Ospedale San Orsola
Bologna, Italy
Ospedale Regionale Generale- Divisione Ematologia
Bolzano, Italy
Ospedale Binaghi
Cagliari, Italy
Ospedale Oncologico Businco
Cagliari, Italy
Ospedale Ferrarotto - Ematologia
Catania, Italy
S.C. Ematologia - Azienda Ospedaliera S. Croce e Carle
Cuneo, Italy
Cattedra di Ematologia - Azienda Ospedaliera di Careggi
Florence, Italy
Ospedale Policlinico San Martino - IST
Genova, Italy
Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico
Milan, Italy
Ospedale Niguarda Ca' Grande
Milan, Italy
Ospedale San Raffaele
Milan, Italy
Divisione Ematologia - Azienda Ospedaliera Universitaria - Policlinico -
Modena, Italy
Cattedra di Medicina Interna ed Ematologia - Ospedale S. Gerardo de' i Tintori - Università degli Studi di Milano
Monza, Italy
AO Ospedali Riuniti Villa Sofia - Cervello
Palermo, Italy
Dipartimento Oncologico La Maddalena
Palermo, Italy
Fondazione IRCCS San Matteo
Pavia, 27100, Italy
Dip. di Ematologia - Unità di Terapia Intensiva Ematologica per il Trapianto Emopoietico - Ospedale Civile di Pescara
Pescara, Italy
Ospedale G. Da Saliceto di Piacenza
Piacenza, Italy
Cattedra di Ematologia - Policlinico
Roma, Italy
Policlinico A. Gemelli
Roma, Italy
Policlinico Universitario Tor Vergata
Roma, Italy
U.O. di Ematologia e Trapianti di Midollo Osseo - Azienda Osp. S. Camillo-Forlanini / Padiglione Morgagni
Roma, Italy
Ematologia e Centro Trapianti Midollo Osseo - Ospedale IRCCS Casa Sollievo della Sofferenza
San Giovanni Rotondo, Italy
Ospedale San Giuseppe Moscato
Taranto, Italy
Azienda ospedaliera Città della Salute e della Scienza
Torino, Italy
Centro Trapianti Metropolitano
Torino, Italy
A.O. Santa Maria della Misericordia
Udine, Italy
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Study Officials
- PRINCIPAL INVESTIGATOR
Fabio Ciceri, MD
Ospedale San Raffaele
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- NA
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- OTHER
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
March 14, 2013
First Posted
March 20, 2013
Study Start
July 1, 2013
Primary Completion
June 30, 2017
Study Completion
June 30, 2017
Last Updated
March 10, 2023
Record last verified: 2023-03