Haploidentical Bone Marrow Transplantation in Sickle Cell Patients
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|ClinicalTrials.gov Identifier: NCT03263559|
Recruitment Status : Recruiting
First Posted : August 28, 2017
Last Update Posted : February 6, 2018
|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.Drug: Hydroxyurea
HU will be given daily at 30mg/kg from Day -70 through Day -10.
Other Names:Drug: Rabbit-ATG
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: ThymoglobulinDrug: Thiotepa
Thiotepa will be given at 10mg/kg on Day -7
Other Name: ChemoDrug: Fludarabine
Fludarabine will be given at 30mg/m2 from Day -6 to Day -2
Other Name: FludaraDrug: Cyclophosphamide
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: TBIDrug: Mesna
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: Brianne Allisonemail@example.com|
|Contact: Adam Mendizabal, PhDfirstname.lastname@example.org|
|United States, Alabama|
|University of Alabama at Birmingham||Recruiting|
|Birmingham, Alabama, United States, 35294|
|Contact: Hilary Haines, MD email@example.com|
|United States, California|
|UCSF Benioff Children's Hospital Oakland||Recruiting|
|Oakland, California, United States, 94609|
|Contact: Mark C. Walters, MD 925-783-5070 firstname.lastname@example.org|
|United States, Florida|
|University of Florida College of Medicine||Recruiting|
|Gainesville, Florida, United States, 32610|
|Contact: Lucien Black email@example.com|
|Nicklaus Children's Hospital/University of Miami Children's Hospital||Recruiting|
|Miami, Florida, United States, 33155|
|Contact: Kamar Godder firstname.lastname@example.org|
|H. Lee Moffitt Cancer Center||Recruiting|
|Tampa, Florida, United States, 33612|
|Contact: Michael L. Nieder, MD 813-745-2191 email@example.com|
|United States, Georgia|
|Atlanta, Georgia, United States, 30342|
|Contact: Melhem Solh, MD 404-255-1930 firstname.lastname@example.org|
|United States, Missouri|
|Washington University, St. Louis||Recruiting|
|Saint Louis, Missouri, United States, 63110|
|Contact: Shalini Shenoy email@example.com|
|United States, New Jersey|
|Hackensack University Medical Center||Recruiting|
|Hackensack, New Jersey, United States, 07601|
|Contact: Jennifer Krajewski firstname.lastname@example.org|
|United States, New York|
|Northwell Health/Monter Cancer Center||Recruiting|
|Lake Success, New York, United States, 11042|
|Contact: Ruthee-Lu Bayer email@example.com|
|United States, Texas|
|Texas Transplant Institute||Recruiting|
|San Antonio, Texas, United States, 78229|
|Contact: Paul J. Shaughnessy, MD 210-575-6904 Paul.Shaughnessy@MHShealth.com|
|Study Director:||Mary Horowitz, MD||Center for International Blood and Marrow Transplant Research|