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. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT02833805|
Recruitment Status : Recruiting
First Posted : July 14, 2016
Last Update Posted : May 3, 2017
|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)|
|Estimated Enrollment :||20 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|
|Study Start Date :||August 2016|
|Estimated Primary Completion Date :||August 2020|
|Estimated Study Completion Date :||August 2020|
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 ]Percentage of enrolled participants who receive BMT, achieve engraftment, and are alive at one year post BMT.
- Overall survival at one year [ Time Frame: 1 year ]Percentage of participants alive at one year after BMT.
- Probability of neutrophil recovery [ Time Frame: 1 year ]Percentage of participants who have recovered neutrophil counts at 1 year.
- Probability of platelet recovery [ Time Frame: 1 year ]Percentage of participants who have recovered platelet counts at 1 year.
- Incidence of primary graft failure [ Time Frame: 1 year ]Percentage of participants who experience primary graft failure by one year after BMT.
- Incidence of secondary graft failure [ Time Frame: 1 year ]Percentage of participants who experience secondary graft failure by one year after BMT.
- Incidence of grades II-IV acute GVHD, Day 100 [ Time Frame: Day 100 ]Percentage of participants who experience grade II, III, or IV acute GVHD by Day 100.
- Incidence of grades III-IV acute GVHD, Day 100 [ Time Frame: 1 year ]Percentage of participants who experience grade III or IV acute GVHD by Day 100.
- Incidence of chronic GVHD, one year [ Time Frame: 1 year ]Percentage of participants who experience chronic GVHD by one year after BMT.
- Estimate full donor chimerism [ Time Frame: Day 60 ]Percentage of participants with full donor chimerism at Day 60.
- Estimate GVHD-free relapse-free survival (GRFS) [ Time Frame: 1 year ]Percentage of participants alive, without relapse, and without GVHD at 1 year.
- Estimate transplant-related mortality [ Time Frame: 1 year ]Percentage 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
|Contact: Amy E DeZern, MDfirstname.lastname@example.org|
|United States, Maryland|
|Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins||Recruiting|
|Baltimore, Maryland, United States, 21287|
|Contact: Amy E DeZern, MD 410-502-7208 email@example.com|
|Principal Investigator:||Amy E DeZern, MD||Johns Hopkins University|