Clinical trial NCT02504047: Use of Haploidentical Related Donors for Patients Without Matched Sibling, Unrelated Donor or Cord Blood Units
Official title: Use of Haploidentical Related Donors for Patients Without Matched Sibling, Unrelated Donor or Cord Blood Units
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: NA
Conditions: Hematologic Neoplasms
Keywords: Hematopoietic Stem Cell Transplantation
Lead sponsor: University of Calgary
Sponsor class: OTHER
Collaborators: name: The Canadian Blood and Marrow Transplant Group
First posted: 2015-07-21
Enrollment: 40
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: The administration of high-dose chemotherapy followed by the infusion of blood or bone marrow stem cells (stem cell transplantation) from a matched donor has become standard treatment for patients with high-risk or relapsed hematological cancers. Currently, donors are found for approximately 80% of people who require such treatment, although the chance of finding a donor is much lower in some ethnic communities. In the current study the investigators will offer patients requiring transplantation, but for whom well matched donors cannot be identified either from within the family or on the donor registry, a transplant from a half-matched (haploidentical) family member. A myeloablative conditioning regimen and un-manipulated peripheral blood stem cells will be used. Post-transplant cyclophosphamide, tacrolimus and mycophenolate mofetil will be used to prevent graft versus host disease (GVHD). The primary outcome measure will be 6 month survival free from graft failure, relapse and grade 3-4 acute GVHD. Other outcomes of interest will include the frequency of Cytomegalovirus (CMV) or Epstein-Barr Virus (EBV) requiring treatment, overall survival and progression-free survival.


Eligibility criteria: Inclusion Criteria:

* A hematological malignancy such as acute leukemia or myelodysplastic syndrome requiring hematopoietic stem cell transplantation.
* A haploidentical family member willing to donate.
* Suitable performance status, organ function (as defined locally) and disease status for transplantation.
* Patients with acute leukemia must be in morphological complete remission. Patients with chronic myelogenous leukemia must be in chronic phase and those with lymphoma must have chemosensitive disease (at least partial remission to most recent chemotherapy regimen).
* Given written, informed consent to participate in a clinical trial.

Exclusion Criteria:

* An 8/8 matched sibling donor or matched unrelated donor (based on high-resolution typing as appropriate), available within a time frame acceptable to the treating physician
* Patients with an available single allele or single antigen mismatched donor (7/8 or 9/10) or available cord blood donor can be included at the discretion of the treating team, according to local institutional practice.
* Prior allogeneic transplant
* Any exclusion criteria for allogeneic stem cell transplant, as defined by the program. Examples typically include pregnancy, significant comorbidity, and active uncontrolled infection.

Primary outcomes:
measure: Six month survival, free from relapse, graft failure and grade III/IV acute GVHD
timeFrame: Six months
Secondary outcomes:
measure: Incidence of Grade III/IV Acute Graft Versus Host Disease
timeFrame: Six months
measure: Incidence of Chronic Extensive Graft Versus Host Disease
timeFrame: One year
measure: Cumulative incidence of non-relapse mortality at 1 year
timeFrame: One year
measure: Graft failure rate (ANC < 0.5 and low donor chimerism)
timeFrame: Six months
measure: Overall and Disease Free Survival
timeFrame: One year
measure: Relapse Rate
timeFrame: One year
measure: Patients remaining on immune suppression at 1 year
timeFrame: One year
measure: Incidence of CMV and EBV reactivation requiring treatment
timeFrame: One year
Other outcomes:
Locations:
city: Calgary
country: Canada
facility: Foothills Medical Center / Tom Baker Cancer Center
state: Alberta
city: Winnipeg
country: Canada
facility: Health Sciences Center
state: Manitoba
city: Ottawa
country: Canada
facility: Ottawa General Hospital
state: Ontario
city: Montreal
country: Canada
facility: McGill University Health Centre
state: Quebec
city: Saskatoon
country: Canada
facility: Royal University Hospital
state: Saskatchewan
Study officials:
affiliation: University of Calgary
name: Andrew Daly, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: University of Manitoba
name: Kristjan Paulson, MD
role: PRINCIPAL_INVESTIGATOR
References: