Know Cancer

forgot password

Allogeneic Hematopoietic Cell Transplantation From HLA-matched Donors After Reduced-intensity Conditioning: a Phase II Randomized Study Comparing 2 GVHD Prophylaxis Regimens

Phase 2
16 Years
75 Years
Open (Enrolling)
Graft-Versus-Host Disease, Hematological Malignancies

Thank you

Trial Information

Allogeneic Hematopoietic Cell Transplantation From HLA-matched Donors After Reduced-intensity Conditioning: a Phase II Randomized Study Comparing 2 GVHD Prophylaxis Regimens

Inclusion Criteria:

1. Hematological malignancies confirmed histologically and not rapidly progressing:

- Acute myeloid leukemia (AML) in complete remission (CR) (defined as ≤ 5% marrow
blasts and absence of blasts in the peripheral blood);

- Myelodysplastic syndromes (MDS) with ≤ 5% marrow blasts and absence of blasts in
the peripheral blood;

- Chronic myeloid leukemia (CML) in chronic phase (CP);

- Myeloproliferative neoplasms not in blast crisis and not with extensive marrow

- Acute lymphoid leukemia (ALL)in CR;

- Multiple myeloma not rapidly progressing;

- chronic lymphocytic leukemia (CLL);

- Non-Hodgkin's lymphoma (aggressive NHL should have chemosensitive disease);

- Hodgkin's disease with chemosensitive disease;

2. 10/10 HLA-A, -B, -C, DRB1 and DQBI allele-matched donor fit to/willing to donate

3. Clinical situations:

1. Theoretical indication for a standard allotransplant, but not feasible because:

- Age > 50 yrs;

- Unacceptable end organ performance;

- At the physician's decision;

- Patient's refusal.

2. Indication for a standard auto-transplant: perform mini-allotransplantation 2-6
months after standard autotransplant.

4. Other inclusion criteria:

- Male or female; fertile patients must use a reliable contraception method;

- Age ≤ 75 yrs (children of any age are allowed in the protocol);

- Informed consent given by patient or his/her guardian if of minor age.

Exclusion Criteria:

- Any condition not fulfilling inclusion criteria;

- HIV positive;

- Non-hematological malignancy(ies) (except non-melanoma skin cancer) < 3 years before
nonmyeloablative hematopoietic cell transplantation (HCT);

- Life expectancy severely limited by disease other than malignancy;

- Administration of cytotoxic agent(s) for "cytoreduction" within three weeks prior to
initiating the nonmyeloablative transplant conditioning (Exceptions are hydroxyurea
and imatinib mesylate);

- CNS involvement with disease refractory to intrathecal chemotherapy;

- Terminal organ failure, except for renal failure (dialysis acceptable)

1. Cardiac: Symptomatic coronary artery disease or other cardiac failure requiring
therapy; ejection fraction <35%; uncontrolled arrhythmia, uncontrolled

2. Pulmonary: DLCO < 35% and/or receiving supplementary continuous oxygen;

3. Hepatic: Fulminant liver failure, cirrhosis of the liver with evidence of
portal hypertension, alcoholic hepatitis, esophageal varices, a history of
bleeding esophageal varices, hepatic encephalopathy, uncorrectable hepatic
synthetic dysfunction evinced by prolongation of the prothrombin time, ascites
related to portal hypertension, bacterial or fungal liver abscess, biliary
obstruction, chronic viral hepatitis with total serum bilirubin >3 mg/dL, and
symptomatic biliary disease;

- Uncontrolled infection;

- Karnofsky Performance Score <70%;

- Patient is a fertile man or woman who is unwilling to use contraceptive techniques
during and for 12 months following treatment;

- Patient is a female who is pregnant or breastfeeding;

- Any condition precluding the use of sirolimus or MMF;

- One HLA mismatch with peripheral blood stem cells (PBSC) fit to/willing to donate.

Type of Study:


Study Design:

Allocation: Randomized, Endpoint Classification: Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Prevention

Outcome Measure:

Progression-free survival

Outcome Description:

To compare the 1-year progression-free survival between the 2 prophylactic arms (Tracolimus/Mycophenolate Mofetil and Tracolimus/Sirolimus) in the whole group of patients and separately in those conditioned with Fluradabine/TBI or Fluradabine plus Busulfan and anti-thymocyte globulin.

Outcome Time Frame:

1 year after transplantation

Safety Issue:


Principal Investigator

Frédéric Baron, MD; PhD

Investigator Role:

Principal Investigator

Investigator Affiliation:

University of Liège


Belgium: Federal Agency for Medicinal Products and Health Products

Study ID:




Start Date:

September 2011

Completion Date:

September 2017

Related Keywords:

  • Graft-Versus-Host Disease
  • Hematological Malignancies
  • Allogeneic hematopoeitic cell transplantation
  • Graft-Versus-Host Disease
  • Prophylaxis
  • Reduced-intensity conditioning
  • Immunosuppressive regimen
  • HLA-matched donor
  • Progression free survival
  • Overall survival
  • Neoplasms
  • Graft vs Host Disease
  • Hematologic Neoplasms