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| Sponsored by: |
St. Jude Children's Research Hospital |
|---|---|
| Information provided by: | St. Jude Children's Research Hospital |
| ClinicalTrials.gov Identifier: | NCT00186823 |
Purpose
Blood and marrow stem cell transplant has improved the outcome for patients with high-risk hematologic malignancies. However, most patients do not have an appropriate HLA (immune type) matched sibling donor available and/or are unable to identify an acceptable unrelated HLA matched donor through the registries in a timely manner. Another option is haploidentical transplant using a partially matched family member donor.
Although haploidentical transplant has proven curative in many patients, this procedure has been hindered by significant complications, primarily regimen-related toxicity including graft versus host disease (GVHD) and infection due to delayed immune reconstitution. These can, in part, be due to certain white blood cells in the graft called T cells. GVHD happens when the donor T cells recognize the body tissues of the patient (the host) are different and attack these cells. Although too many T cells increase the possibility of GVHD, too few may cause the recipient's immune system to reconstitute slowly or the graft to fail to grow, leaving the patient at high-risk for significant infection.
This research project will investigate the use of particular pre-transplant conditioning regimen (chemotherapy, antibodies and total body irradiation) followed by a stem cell infusion from a "mismatched" family member donor. Once these stem cells are obtained they will be highly purified in an effort to remove T cells using the investigational CliniMACS stem cell selection device. The primary goal of this study will be to determine the rate of neutrophil and platelet engraftment, as well as the degree and rate of immune reconstitution in the first 100 days posttransplant for patients who receive this study treatment. Researchers will also study ways to decrease complications that may occur with a transplant from a genetically mismatched family donor.
| Condition | Intervention | Phase |
|---|---|---|
|
Leukemia, Acute Lymphocytic (ALL) Leukemia, Myeloid, Acute(AML) Leukemia, Myeloid, Chronic(CML) Juvenile Myelomonocytic Leukemia(JMML) Hemoglobinuria, Paroxysmal Nocturnal (PNH) Lymphoma, Non-Hodgkin (NHL) Myelodysplastic Syndrome (MDS) |
Device: Miltenyi Biotec CliniMACS Procedure: Stem Cell Transplantation Drug: TBI, systemic chemotherapy and antibodies as follows: |
Phase III |
| Study Type: | Interventional |
| Study Design: | Treatment, Non-Randomized, Open Label, Uncontrolled, Single Group Assignment, Safety/Efficacy Study |
| Official Title: | Haploidentical Stem Cell Transplantation Utilizing Purified CD34+ Hematopoietic Cells for Patients With Hematologic Malignancies |
| Enrollment: | 57 |
| Study Start Date: | March 2002 |
| Estimated Study Completion Date: | May 2009 |
| Primary Completion Date: | September 2005 (Final data collection date for primary outcome measure) |
| Arms | Assigned Interventions |
|---|---|
| 1 |
Device: Miltenyi Biotec CliniMACS
A stem cell selection device.
Procedure: Stem Cell Transplantation
An infusion of HLA mismatched family member donor stem cells processed through the use of the investigational Miltenyi Biotec CliniMACS device.
Drug: TBI, systemic chemotherapy and antibodies as follows:
Transplant recipients received a myeloablative conditioning regimen consisting of total body irradiation, thiotepa, cyclophosphamide, ATG, and OKT3. Rituximab was provided prior to transplant for PTLPD prophylaxis. In addition to T cell depletion of the donor product, cyclosporine was administered for GVHD prophylaxis.
|
Secondary outcome evaluations for this clinical study include the following:
Originally this study began as a randomized comparison of two methods of stem cell selection utilizing the CliniMACS device. Both arms provided a purified product of CD34+ hematopoietic cells that was depleted of T-lymphocytes. One arm utilized a positive selection methodology using an anti-CD34 antibody and the other arm utilized negative selection with the anti-CD3 antibody OKT-3. There were no toxicities noted that would have required the study to be interrupted early, however, due to low accrual, it was decided to redesign the study. The new design focused on the standard arm utilizing negative selection, and all subsequent participants were treated in that manner. The patients who were treated on the positive selection arm are continuing to be monitored as specified in the original protocol, but will be reported in a descriptive manner only. The primary and secondary objectives of the current version of this study will be answered only by those patients receiving a haploidentical stem cell transplant utilizing a negative selection methodology.
Eligibility| Ages Eligible for Study: | 2 Years to 21 Years |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
Inclusion Criteria:
At least 2 and less than or equal to 21 years of age
Exclusion Criteria:
Contacts and Locations| United States, Tennessee | |
| St. Jude Children's Research Hospital | |
| Memphis, Tennessee, United States, 38105 | |
| Principal Investigator: | Gregory A. Hale, M.D. | St. Jude Children's Research Hospital |
More Information
| Responsible Party: | St. Jude Children's Research Hospital ( G. Hale, MD, Principal Investigator ) |
| Study ID Numbers: | HAPSCT |
| Study First Received: | September 9, 2005 |
| Last Updated: | March 19, 2008 |
| ClinicalTrials.gov Identifier: | NCT00186823 |
| Health Authority: | United States: Food and Drug Administration |
|
Haploidentical stem cell transplant Allogeneic stem cell transplant Mismatched family member stem cell donor transplant Bone marrow transplantHigh risk hematologic malignancies |
|
Juvenile myelomonocytic leukemia Leukemia, Lymphoid Cyclosporine Precancerous Conditions Hematologic Neoplasms Chronic myelomonocytic leukemia Cyclophosphamide Leukemia, Myeloid, Acute Cyclosporins Muromonab-CD3 Leukemia Signs and Symptoms Preleukemia Urologic Diseases Hemoglobinuria, Paroxysmal |
Lymphoma Immunoglobulins Myelodysplastic syndromes Immunoproliferative Disorders Precursor Cell Lymphoblastic Leukemia-Lymphoma Rituximab Urination Disorders Hematologic Diseases Leukemia, Myelomonocytic, Chronic Myelodysplasia Myelodysplastic Syndromes Myeloproliferative Disorders Anemia Anemia, Hemolytic Leukemia, Myelomonocytic, Juvenile |
|
Urological Manifestations Neoplasms Neoplasms by Site Neoplasms by Histologic Type Immune System Diseases |
