Phase 2
N=3
Unrelated Cord Blood Transplant Plus a Haplo-Identical (Half-Matched), T-Cell Depleted Stem Transplant From a Related Donor for Subjects With High Risk Malignancies
Hematologic Malignancy · Myelodysplastic Syndrome (MDS) · Aplastic Anemia
Bottom Line
View on ClinicalTrials.gov: NCT00673114 ↗Enrolled (actual)
3
Serious AEs
66.7%
Results posted
Dec 2013
Primary outcome: Primary: The Number of Participants Reaching Primary Endpoint of Absolute Neutrophil Count (ANC) of 500/uL (Engraftment). — 3 participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- haplo/cord transplant (Biological)
- Age
- Pediatric, Adult
- Sex
- All
- Sponsor
- Joanne Kurtzberg, MD
- Primary completion
- Apr 2012
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY The Number of Participants Reaching Primary Endpoint of Absolute Neutrophil Count (ANC) of 500/uL (Engraftment). |
3 | — |
| SECONDARY 180 Day Survival |
3 | — |
| SECONDARY Non-Relapse Mortality at 180 Days Post Transplant |
— | — |
| SECONDARY Platelet Engraftment (Untransfused and Platelet Count > 50,000) |
3 | — |
| SECONDARY Incidence of Primary and Secondary Graft Failure |
— | — |
| SECONDARY Number of Participants With Acute or Chronic Graft-versus-host Disease (GVHD) |
2 | — |
| SECONDARY Rates of Leukemic Relapse |
1 | — |
| SECONDARY Number of Participants With Donor Cells at 100 Days Post-transplant |
3 | — |
Summary
Subjects will be diagnosed with a hematological malignancy (cancer of the blood), which is unlikely to be cured with conventional non-transplant therapy. The best results of bone marrow transplant are obtained with the donor is a relative that has identical tissue type (HLA-type). These subjects will not have such a donor available but they will have a appropriately matching unrelated umbilical cord blood unit (UCB). However, the cord blood unit does not contain a high enough number of cells and may take longer to engraft (or grow). The purpose of this study is to determine whether the addition of stem cells from a family member to supplement a standard unrelated cord blood transplant is safe and will increase the success of the cord blood transplantation procedure. Subjects enrolled in this study will receive an unrelated cord blood transplant plus a haplo-identical (half-matched), T-cell depleted stem transplant from a related donor. The goal of this study is to determine whether the addition of the related stem cells accelerates bone marrow recovery and improves long-term disease free survival.
Eligibility Criteria
Inclusion Criteria
- Patient Selection Criteria: Patients with high risk or refractory malignancies, myelodysplasia (MDS) or severe aplastic anemia amenable to stem cell transplantation therapy but lacking conventional related or unrelated donors will be eligible for this trial.
- Have a consenting related haplo-identical (3/6 or 4/6) stem cell donor;
- Have an available 3, 4, 5, or 6/6 antigen matching unrelated UCB unit that will deliver a cell dose between 2.0-5.0 x 10e7cells/kg.
- Not have a consenting 6/6 or 5/6 antigen matched related bone marrow donor or genetically matched unrelated BM or adult stem cell donor.
- Patients must be 500, 000/ml) or failure to achieve a complete remission after standard induction therapy.
- All patients with ALL or ANLL in second or subsequent remission.
- Patients with ALL or ANLL in relapse.
- Patients with MDS.
- Patients with CML in any chronic phase, accelerated phase or blast crisis.
- Patients with severe aplastic anemia refractory to medical therapy.
- Patients must not have active CNS disease at the time of study enrollment.
- Patients must have a good performance status (Lansky 80-100%, Karnofsky 50-100%).
- Patients must have adequate function of other organ systems as measured by:
- Creatinine 50 cc/min/m2.
- Hepatic transaminases (ALT/AST) 80% of normal value for age).
- Pulmonary function tests demonstrating FVC and FEV1 of >60% of predicted for age. For adult patients DLCO > 60% of predicted. If patient cannot perform PFTs, clearance by the pediatric or adult pulmonologist will be required.
- Patients must not have uncontrolled infections at the time of cytoreduction.
- Patient, parent, or legal guardian must have given written informed consent according to FDA guidelines.
- Patients may not be pregnant or lactating and must have a current negative pregnancy test.
- Patients must have a minimum life expectancy of at least 3 months.
- Patients must have an available related haplo-identical stem cell donor and an available unrelated cord blood donor delivering between 2 x10e7 cells/kg and 5 x 10e7 cells/kg and matching at a minimum of 3/6 HLA loci.
- Patients must be HIV negative.
- Patients must not be concurrently involved in any other clinical trial that affects engraftment or immune reconstitution (e.g. other hematopoietic growth factors).
- Patients must not have any co-morbid condition which, in the view of the Principal Investigators, renders the patient at too high a risk from treatment complications and regimen related morbidity/mortality.
Data sourced from ClinicalTrials.gov (NCT00673114). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.