NMA Haplo or MUD BMT for Newly Diagnosed Severe Aplastic Anemia
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT02833805|
Recruitment Status : Completed
First Posted : July 14, 2016
Results First Posted : July 12, 2022
Last Update Posted : July 12, 2022
|Condition or disease||Intervention/treatment||Phase|
|Severe Aplastic Anemia Aplastic Anemia Bone Marrow Failure Immunosuppression||Drug: Thymoglobulin Drug: Fludarabine Drug: Cyclophosphamide Radiation: Total body irradiation Drug: Tacrolimus Drug: Mycophenolate mofetil||Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||21 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||A Phase II Trial of Non-Myeloablative (NMA) Conditioning and Transplantation of Partially HLA-Mismatched/Haploidentical Related or Matched Unrelated Donor (MUD) Bone Marrow for Newly Diagnosed Patients With Severe Aplastic Anemia|
|Actual Study Start Date :||September 2016|
|Actual Primary Completion Date :||July 2021|
|Actual Study Completion Date :||July 2021|
Experimental: Bone marrow transplant
Non-myeloablative bone marrow transplant with a Thymoglobulin (ATG), fludarabine (Flu), cyclophosphamide (Cy), total body irradiation (TBI) preparative regimen and post-transplant Cy, mycophenolate mofetil (MMF), and tacrolimus as GVHD prophylaxis.
Day -9: 0.5 mg/kg Days -8 and -7: 2 mg/kg daily
Days -6 through -2: 30 mg/m^2 IV daily
Other Name: Fludara
Days -6 and -5: 14.5 mg/kg IV daily Days 3 and 4: 50 mg/kg IV daily
Radiation: Total body irradiation
Day -1: 200 centigray (cGy) in a single fraction
Other Name: TBI
Start on Day 5 through Day 365
Drug: Mycophenolate mofetil
Days 5 through 35: 15 mg/kg PO three times daily (max 3 g/day)
- Overall Survival and Engraftment at One Year [ Time Frame: 1 year ]Number of enrolled participants who receive BMT, achieve engraftment, and are alive at one year post bone marrow transplant (BMT).
- Overall Survival at One Year [ Time Frame: 1 year ]Number of participants alive at one year after BMT.
- Probability of Neutrophil Recovery as Assessed by the Number of Participants Who Have Recovered Neutrophil Counts [ Time Frame: 1 year ]Probability of neutrophil recovery will be assessed by the number of participants who have recovered neutrophil counts at 1 year (>500 ANC).
- Probability of Platelet Recovery as Assessed by Number of Participants Who Have Recovered Platelet Counts [ Time Frame: 1 year ]Probability of platelet recovery will be assessed by the number of participants who have recovered platelet counts at 1 year.
- Number of Participants Who Experience Primary Graft Failure [ Time Frame: 1 year ]Number of participants who experience primary graft failure by one year after BMT.
- Number of Participants Who Experience Secondary Graft Failure [ Time Frame: 1 year ]Number of participants who experience secondary graft failure by one year after BMT.
- Number of Participants Who Experience Grades II-IV Acute GVHD [ Time Frame: Day 100 ]Number of participants who experience grade II, III, or IV acute GVHD by Day 100.
- Number of Participants Who Experience Grades III-IV Acute GVHD [ Time Frame: Day 100 ]Number of participants who experience grade III or IV acute GVHD by Day 100.
- Number of Participants Who Experience Chronic GVHD [ Time Frame: 2 years ]Number of participants who experience chronic GVHD by two years after BMT.
- Number of Participants With Full Donor Chimerism [ Time Frame: Day 60 ]Number of participants with full donor chimerism at Day 60.
- GVHD-free Relapse-free Survival (GRFS) [ Time Frame: 1 year ]Number of participants alive, without relapse, and without GVHD at 1 year.
- Transplant-related Mortality [ Time Frame: 1 year ]Number of participants deceased for reasons related to BMT at 1 year.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02833805
|United States, Maryland|
|Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins|
|Baltimore, Maryland, United States, 21287|
|Principal Investigator:||Amy E DeZern, MD||Johns Hopkins University|