Trials / Recruiting
RecruitingNCT03970096
Graft Versus Host Disease-Reduction Strategies for Donor Blood Stem Cell Transplant Patients With Acute Leukemia or Myelodysplastic Syndrome (MDS)
A Phase II Randomized Controlled Trial Comparing GVHD-Reduction Strategies for Allogeneic Peripheral Blood Transplantation (PBSCT) for Patients With Acute Leukemia or Myelodysplastic Syndrome: Selective Depletion of CD45RA+ Naïve T Cells (TND) vs. Post-Transplantation Cyclophosphamide (PTCy)
- Status
- Recruiting
- Phase
- Phase 2
- Study type
- Interventional
- Enrollment
- 120 (estimated)
- Sponsor
- Fred Hutchinson Cancer Center · Academic / Other
- Sex
- All
- Age
- 1 Year – 60 Years
- Healthy volunteers
- Not accepted
Summary
This phase II trial investigates two strategies and how well they work for the reduction of graft versus host disease in patients with acute leukemia or MDS in remission. Giving chemotherapy and total-body irradiation before a donor peripheral blood stem cell transplant helps stop the growth of cells in the bone marrow, including normal blood-forming cells (stem cells) and cancer cells. It may also stop the patient's immune system from rejecting the donor's stem cells. When the healthy stem cells from a donor are infused into the patient, they may help the patient's bone marrow make stem cells, red blood cells, white blood cells, and platelets. The donated stem cells may also replace the patient's immune cells and help destroy any remaining cancer cells.
Detailed description
OUTLINE: Patients are randomized to 1 of 2 arms (Arms A and C). ARM A: Patients are assigned to 1 of 2 arms. ARM A1 (TBI BASED): Patients undergo total-body irradiation (TBI) twice daily (BID) on days -10 to -7, and receive thiotepa intravenously (IV) over 3 hours on days -6 and -5, fludarabine IV over 30 to 60 minutes on days -6 to -2, tacrolimus (or cyclosporine or sirolimus if toxicities occur) IV continuously starting on day -1, CD34+ enriched CD45RA-depleted donor T-lymphocytes IV on day 0, and methotrexate IV on days 1, 3, 6, and 11. If there is no evidence of grade II-IV acute GVHD on or prior to day 50, tacrolimus (or cyclosporine or sirolimus) is tapered per month for capsules (or per week for liquid). ARM A2 (BUSULFAN BASED): Patients receive fludarabine IV over 30 to 60 minutes on days -6 to -2, busulfan IV over 180 minutes on days -5 to -2, and undergo TBI BID on day -1. Patients also receive tacrolimus (or cyclosporine or sirolimus if toxicities occur) IV continuously starting on day -1, CD34+ enriched CD45RA-depleted donor T-lymphocytes IV on day 0, and methotrexate IV on days 1, 3, 6, and 11. If there is no evidence of grade II-IV acute GVHD on or prior to day 50, tacrolimus (or cyclosporine or sirolimus) is tapered per month for capsules (or per week for liquid). ARM C: Patients are assigned to 1 of 2 arms. ARM C1: Patients undergo TBI BID on days -4 to -2 or -3 to -1, and receive PBSC IV on day 0. Patients also receive cyclophosphamide IV over 1 to 2 hours on days 3 and 4, and tacrolimus (or cyclosporine or sirolimus if toxicities occur) IV continuously starting on day 5 approximately 24 hours after the end of the second dose of cyclophosphamide. If there is no evidence of grade II-IV acute GVHD on or prior to day 50, tacrolimus (or cyclosporine or sirolimus) is tapered per month for capsules (or per week for liquid). ARM C2: Patients receive fludarabine IV over 30 to 60 minutes on days -5 to -2, busulfan IV over 180 minutes on days -5 to -2, PBSC IV on day 0, cyclophosphamide IV over 1 to 2 hours on days 3 and 4, and tacrolimus (or cyclosporine or sirolimus if toxicities occur) IV continuously starting on day 5 approximately 24 hours after the end of the second dose of cyclophosphamide. If there is no evidence of grade II-IV acute GVHD on or prior to day 50, tacrolimus (or cyclosporine or sirolimus) is tapered per month for capsules (or per week for liquid). ARM D: (DISCONTINUED NOVEMBER 2021): Patients are assigned to 1 of 2 arms. ARM D1: Patients undergo TBI BID on days -6 to -4, and receive cyclophosphamide IV over 1 hour on days -3 to -2, tacrolimus (or cyclosporine or sirolimus if toxicities occur) IV continuously starting on day -1, PBSC IV on day 0, and methotrexate IV on days 1, 3, 6, and 11. If there is no evidence of grade II-IV acute GVHD on or prior to day 50, tacrolimus (or cyclosporine or sirolimus) is tapered per month for capsules (or per week for liquid). ARM D2: Patients receive busulfan IV over 180 minutes on days -8 to -5, cyclophosphamide IV over 1 hour on days -3 to -2, tacrolimus (or cyclosporine or sirolimus if toxicities occur) IV continuously starting on day -1, PBSC IV on day 0, and methotrexate IV on days 1, 3, 6, and 11. If there is no evidence of grade II-IV acute GVHD on or prior to day 50, tacrolimus (or cyclosporine or sirolimus) is tapered per month for capsules (or per week for liquid). All patients also undergo bone marrow aspiration/biopsy, echocardiogram (ECHO) or multigated acquisition (MUGA) scan, and collection of blood samples throughout the trial. After completion of study treatment, patients are followed up at days 7, 14, 21, 28, 56, 80, 180, and 270 and at 1, 1.5, and 2 years.
Conditions
- Acute Leukemia
- Acute Lymphoblastic Leukemia
- Acute Myeloid Leukemia
- Myelodysplastic Syndrome
- Myeloproliferative Neoplasm
Interventions
| Type | Name | Description |
|---|---|---|
| RADIATION | Total-Body Irradiation | Undergo TBI |
| DRUG | Thiotepa | Given IV |
| DRUG | Fludarabine | Given IV |
| DRUG | Tacrolimus | Given IV |
| BIOLOGICAL | Allogeneic CD34+-enriched and CD45RA-depleted PBSCs | Given IV |
| DRUG | Methotrexate | Given IV |
| DRUG | Cyclophosphamide | Given IV |
| BIOLOGICAL | Peripheral Blood Stem Cell | Given IV |
| DRUG | Cyclosporine | Given IV |
| DRUG | Sirolimus | Given IV |
| DRUG | Busulfan | Given IV |
| PROCEDURE | Bone Marrow Aspiration and Biopsy | Undergo bone marrow aspiration/biopsy |
| PROCEDURE | Echocardiography | Undergo ECHO |
| PROCEDURE | Multigated Acquisition Scan | Undergo MUGA |
| PROCEDURE | Biospecimen Collection | Undergo blood sample collection |
Timeline
- Start date
- 2019-11-19
- Primary completion
- 2027-12-31
- Completion
- 2029-12-31
- First posted
- 2019-05-31
- Last updated
- 2026-03-12
Locations
3 sites across 1 country: United States
Regulatory
- FDA-regulated drug study
Source: ClinicalTrials.gov record NCT03970096. Inclusion in this directory is not an endorsement.