Haploidentical Bone Marrow Transplantation in Sickle Cell Patients (BMT CTN 1507)
|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: NCT03263559|
Recruitment Status : Recruiting
First Posted : August 28, 2017
Last Update Posted : January 14, 2020
|Condition or disease||Intervention/treatment||Phase|
|Sickle Cell Disease||Procedure: Haploidentical Bone Marrow Transplantation Drug: Hydroxyurea Drug: Rabbit-ATG Drug: Thiotepa Drug: Fludarabine Drug: Cyclophosphamide Radiation: Total Body Irradiation Drug: Mesna||Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||80 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Reduced Intensity Conditioning for Haploidentical Bone Marrow Transplantation in Patients With Symptomatic Sickle Cell Disease|
|Actual Study Start Date :||October 3, 2017|
|Estimated Primary Completion Date :||December 2023|
|Estimated Study Completion Date :||December 2024|
Experimental: Haploidentical Transplantation
A conditioning regimen with Hydroxyurea, rabbit-ATG, Thiotepa, Fludarabine, Cyclophosphamide, Total Body Irradiation, and Mesna will be administered prior to Haploidentical Bone Marrow Transplantation.
Procedure: Haploidentical Bone Marrow Transplantation
Eligible patients with a first degree Human Leukocyte Antigen (HLA)- haploidentical donor will undergo Haploidentical bone marrow transplantation at Day 0 with non T-cell depleted bone marrow. For Graft-vs-Host Disease (GVHD) prophylaxis, patients will be given sirolimus and mycophenolate mofetil beginning on Day +5.
HU will be given daily at 30mg/kg from Day -70 through Day -10.
Rabbit-ATG (rATG) will be given at 0.5mg/kg on Day -9, and at 2.0mg/kg on Day -8 and Day -7.
Other Name: Thymoglobulin
Thiotepa will be given at 10mg/kg on Day -7
Other Name: Chemo
Fludarabine will be given at 30mg/m2 from Day -6 to Day -2
Other Name: Fludara
Cyclophosphamide will be given at 14.5mg/kg on Day -6 and Day -5, and at 50 mg/kg on Days +3 and +4.
Other Name: Cytoxan®
Radiation: Total Body Irradiation
Total Body Irradiation will be given at 200cGy on Day -1
Other Name: TBI
Mesna will be given at 40mg/kg on Days +3 and +4
Other Name: Mesnex
- Two-Year Post-Transplant Event Free Survival (EFS) [ Time Frame: 2 years ]EFS is defined as survival without a qualifying event. Primary graft rejection, secondary graft rejection, second infusion of hematopoietic cells or death will count as events for this endpoint.
- Overall Survival [ Time Frame: 1 and 2 years ]Death from any cause will be the event and patients will be censored at the date of last contact or two years post-transplant whichever comes first.
- One-Year Post-Transplant EFS [ Time Frame: 1 year ]EFS is defined as survival without a qualifying event, Primary graft rejection, secondary graft rejection, second infusion of hematopoietic cells or death will count as events for this endpoint.
- Graft Rejection [ Time Frame: Day 42 ]Graft rejection is defined as not having engraftment on or before Day 42 post-transplant. Engraftment is defined as having greater than or equal to 5% donor cells post-transplant, from any molecular chimerism assessment (e.g., unsorted, myeloid, or T-cell) on a peripheral blood or bone marrow aspirate sample.
- Chimerism [ Time Frame: Days 28, 100, and 180 and at 1 and 2 years ]Characterize donor hematopoietic chimerism in peripheral blood will be assessed.
- Disease Recurrence [ Time Frame: 2 years ]Disease recurrence is defined as the return of sickle erythropoiesis (in the absence of red blood count transfusion, Hb S level > 70%), or primary or secondary graft rejection, as defined above, or second infusion of hematopoietic cells.
- Patient Reported Quality of Life (QoL) [ Time Frame: 1 and 2 years ]Health-Related 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): NCT03263559
|Contact: Dinma Ahaiwefirstname.lastname@example.org|
|Contact: Adam Mendizabal, PhDemail@example.com|
|Study Director:||Mary Horowitz, MD||Center for International Blood and Marrow Transplant Research|