Optimizing Haploidentical Aplastic Anemia Transplantation (BMT CTN 1502) (CHAMP)
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|ClinicalTrials.gov Identifier: NCT02918292|
Recruitment Status : Recruiting
First Posted : September 28, 2016
Last Update Posted : August 17, 2018
|Condition or disease||Intervention/treatment||Phase|
|Severe Aplastic Anemia||Drug: Antithymocyte Globulin (ATG) Drug: Fludarabine Drug: Cyclophosphamide Radiation: Total Body Irradiation (TBI) Procedure: Haplo HSCT Drug: Tacrolimus Drug: Mycophenolate mofetil (MMF) Drug: G-CSF||Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||30 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Optimizing Haploidentical Aplastic Anemia Transplantation (CHAMP) (BMT CTN #1502)|
|Actual Study Start Date :||July 3, 2017|
|Estimated Primary Completion Date :||February 2021|
|Estimated Study Completion Date :||February 2022|
Experimental: Haplo Bone Marrow HSCT
Patients will be treated with a preparative regimen of Antithymocyte Globulin (ATG) (4.5 mg/kg), fludarabine (150 mg/m^2), cyclophosphamide (29 mg/kg), and low dose total body irradiation (TBI) (200 cGy) before undergoing the haplo HSCT. GVHD prophylaxis will be with post-HSCT cyclophosphamide (100 mg/kg), tacrolimus, and mycophenolate mofetil (MMF). G-CSF will be administered post-transplant.
Drug: Antithymocyte Globulin (ATG)
Administration of ATG will be 0.5 mg/kg IV on Day -9 over 6 hours and 2 mg/kg IV on Days -8 and -7 over 4 hours.
Other Name: Thymoglobulin®, rATG
Fludarabine dose will be 30 mg/m^2 IV daily for 5 days from Day -6 to Day -2.
Other Name: Fludara®
Cyclophosphamide dose will be 14.5 mg/kg IV daily for 2 days (Day -6 to Day -5) prior to transplantation and 50 mg/kg IV daily for 2 days (Day +3 to Day +4) after transplantation.
Other Name: Cytoxan®
Radiation: Total Body Irradiation (TBI)
TBI is to be delivered in a single dose of 200 cGy on Day -1.
Procedure: Haplo HSCT
Eligible patients without a fully matched related or unrelated donor available will undergo haploidentical bone marrow transplant.
Tacrolimus should be started on Day +5 and administered to maintain a level of 10-15 ng/mL.
Other Name: Prograf®
Drug: Mycophenolate mofetil (MMF)
MMF dose will be 15 mg/kg PO three times a day (TID) up to 1 gm TID (or IV equivalent) starting on Day +5.
Other Name: Cellcept®
G-CSF will be given IV or SQ starting on Day +5 at 5 mcg/kg/day until ANC is > 1500 for 3 days.
Other Name: Filgrastim, Neupogen®
- Overall Survival (OS) [ Time Frame: 1 year ]OS is defined as the time interval from date of transplant to death or to last follow-up, whichever occurs first.
- Neutrophil Recovery [ Time Frame: Day 28 and 56 ]Neutrophil recovery is achieving an absolute neutrophil count (ANC) > 0.5 x10^9/L for three consecutive measurements on different days, with the first of the three days being defined as the day of neutrophil engraftment.
- Platelet Recovery [ Time Frame: Day 28 and 56 ]Platelet recovery is defined by achieving a platelet count > 20 x 10^9/L with no platelet transfusions in the preceding seven days. The first day of the sustained platelet count will be defined as the day of platelet engraftment.
- Alive with Sustained Engraftment [ Time Frame: 1 year ]Being alive and engrafted is defined as not having experienced death, primary graft failure, or secondary graft failure. Donor cell engraftment is defined as donor chimerism greater than or equal to 5% on or after Day 56 after transplantation. Chimerism may be evaluated in whole blood or blood cell fractions, including CD3 and CD33 or CD15 fractions. For this protocol, lineage-specific, myeloid, and T cell chimerisms are required.
- Incidences of Graft Failure [ Time Frame: 1 year ]Primary graft failure is defined by the lack of neutrophil engraftment by Day +56 post-HSCT or failure to achieve at least 5% donor chimerism (whole blood or marrow) on any measurements up to and including Day +56. For this protocol, lineage-specific, myeloid, and T cell chimerisms are required. Myeloid engraftment might not proceed at the same rate as T cell engraftment. If myeloid has greater than or equal to 5% donor, even if T cell compartment does not, this is not considered primary graft failure. Secondary graft failure is defined by initial neutrophil engraftment (ANC greater than or equal to 0.5 x 108/L measured for 3 consecutive measurements on different days) followed by sustained subsequent decline in ANC to less than 0.5 x 10^9/L for three consecutive measurements on different days or initial whole blood or marrow donor chimerism greater than or equal to 5%, but then declining to less than 5% on subsequent measurements or second infusion/transplant given for graft failure.
- Alive with autologous recovery [ Time Frame: 1 year ]Autologous recovery is defined as ANC > 0.5 x 109/L and transfusion independence but with < 5% donor chimerism (whole blood or marrow).
- Incidences of graft-vs-host-disease (GVHD) [ Time Frame: 1 year ]Acute and chronic GVHD are graded according to the BMT CTN Manual of Procedures (MOP).
- Immune Reconstitution [ Time Frame: Days 100, 180, and 365 ]Quantitative assessments of peripheral blood CD4, CD19, and CD56 positive lymphocytes will be done through flow cytometric analysis.
- Incidences of Infection [ Time Frame: 1 year ]Number of participants that experienced at least one infection.
- Health Related Quality of Life (HR-QoL) [ Time Frame: 1 year ]HR-QoL will be measured using patient reported surveys.
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): NCT02918292
|Contact: Alyssa Ramirezemail@example.com|
|Contact: Adam Mendizabal, PhDfirstname.lastname@example.org|
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|Study Director:||Mary Horowitz, MD, MS||Center for International Blood and Marrow Transplant Research|