Table 5.

Active and Open Trials for Hematopoietic Stem Cell Transplant in Sickle Cell Disease

ClinicalTrials.gov Identifier	Title	Status	Regimen	Donor Source	Age	N	Start Date	Study Objective	Location
NCT03279094	Haploidentical Transplantation With Pre-Transplant Immunosuppressive Therapy for Patients With SCD	Recruiting	Myeloablative	Haploidentical	1–30	15	October 2017	To evaluate safety and toxicity of 2 cycles of pre-transplant immunosuppressive therapy followed by myeloablative preparative regimen and allogeneic HSCT from a haploidentical donor	USA, California City of Hope Medical Center
NCT02678143	A Pilot Study of Nonmyeloablative Conditioning for Mismatched HSCT for Severe SCD	Recruiting	Non-myeloablative	Haploidentical, Mismatched unrelated	>19	20	April 26, 2016	To evaluate safety and feasibility of non-myeloablative conditioning in haploidentical or one antigen mismatch unrelated HSCT for adult patients with severe SCD	USA, Missouri Washington University
NCT02867800	Abatacept for GVHD Prophylaxis After HSCT for Pediatric SCD	Recruiting	Reduced Intensity	Unrelated	3–20	10	July 2016	To assess tolerability of the costimulation blocking agent abatacept (CTLA4-Ig) when added to standard GVHD prophylaxis	USA and Canada, multicenter
NCT03298399	MSCs for Haploidentical HSCT for SCD	Recruiting	NR	Haploidentical	12–40	18	September 1, 2017	To determine safety, tolerability, and effects on engraftment and GVHD of autologous, BM derived MSCs in patients with SCD undergoing haploidentical HSCT	USA, Georgia Children’s Healthcare of Atlanta, Emory
NCT03214354	Nonmyeloablative SCT in Children With SCD and a Major ABO-Incompatible Matched Sibling Donor (Sickle-MAID)	Recruiting	Non-myeloablative	Matched related	1–19	12	July 5, 2017	To evaluate safety and efficacy of nonmyeloablative conditioning for allogeneic HSCT in pediatric patients with SCD who have a matched related major ABO-incompatible donor	Canada, Alberta Alberta Children’s Hospital
NCT01499888	Ph I/II Study of Allogeneic SCT for Clinically Aggressive SCD	Recruiting	Non-myeloablative	Matched related	16–60	15	November 11, 2011	To determine engraftment and transplant related morbidity and mortality after non-myeloablative allogeneic HSCT using immune-suppressive agents and low-dose TBI without standard chemotherapy in patients with aggressive SCD	USA, Illinois University of Illinois at Chicago
NCT02435901	HSCT For Patients with High Risk Hemoglobinopathies Using Reduced Intensity	Recruiting	Reduced Intensity	Related or unrelated BM, matched or mismatched BM	1–21	19	December 2008	To evaluate use of RIC regimen in patients with high risk hemoglobinopathy SCD and B-Thalassemia Major in combination with standard immunosuppressive medications, followed by a routine SCT	USA, New York Cohen Children’s Medical Center of New York
NCT03121001	Study of HLA-SCT to Treat Clinically Aggressive SCD	Recruiting	Reduced Intensity	Haploidentical	16–60	50	April 28, 2017	To determine engraftment at Day +60 following HLA-haploidentical HSCT protocol using immunosuppressive agents and low-dose TBI for conditioning and post-transplant cyclophosphamide	USA, Illinois University of Illinois at Chicago
NCT02165007	Haploidentical HSCT	Recruiting	Reduced Intensity	Haploidentical, CD34+ selected graft	Up to age 22	15	January 2015	To assess safety and toxicity of RIC haploidentical HSCT using CD34+ selected graft	USA, D.C. Children’s National Medical Center
NCT02105766	Nonmyeloablative PB Mobilized HPCT for SCD and Beta-thalassemia in People with Higher Risk of Transplant Failure	Recruiting	Non-myeloablative	HLA-matched	16–80	162	April 1, 2014	To see if low dose radiation (300 rads), oral cyclophosphamide, pentostatin, and sirolimus help a body to better accept donor stem cells	USA, Maryland National Institutes of Health
NCT02038478	Allograft for SCD and Thalassemia	Recruiting	Non-myeloablative	HLA-matched	18–45	50	January 2014	To determine safety and therapeutic potential of a nonmyeloablative PB Mobilized HPCT	USA, Texas UT Southwestern Medical Center
NCT03077542	Nonmyeloablative Haploidentical PB Mobilized HPCT for SCD	Recruiting	Non-myeloablative	Haploidentical	2–80	84	April 6, 2017	To evaluate safety, efficacy, and tolerance of a nonmyeloablative Haploidentical PB Mobilized HPCT for SCD	USA, Maryland National Institutes of Health
NCT02766465	BMT vs Standard of Care in Patients with Severe SCD (BMT CTN 1503) (STRIDE2)	Recruiting	Myeloablative	HLA-matched related or unrelated donor	15–40	200	November 2016	To compare BMT to standard care	USA, multicenter
NCT01917708	BMT Abatacept for Non-Malignant Diseases	Recruiting	Reduced Intensity	Unrelated adult donor (marrow or PBSC) or an unrelated CB unit	Up to age 21	20	January 2014	To assess the tolerability of abatacept when combined with CsA and MMF as GVHD prophylaxis	USA, Georgia Children’s Healthcare of Atlanta
NCT02757885	Transplantation Using Reduced Intensity Approach for Patients with SCD Disease From Mismatched Family Donors of Bone Marrow (TRANSFORM)	Recruiting	Reduced Intensity	Mismatched family donor BM	15–40	15	April 2016	To learn if it is possible and safe to treat persons with severe SCD by HSCT from human leukocyte antigen (HLA) half-matched related donors	USA, Georgia Children’s Healthcare of Atlanta
NCT00061568	Improving the Results of Bone Marrow Transplantation for Patients with Severe Congenital Anemias	Recruiting	Non-myeloablative	HLA-matched	2–80	150	May 23, 2003	Evaluate nonmyeloablative conditioning in adults with severe SCD	USA, Maryland National Institutes of Health
NCT01962415	RIC for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT (HSCT+RIC)	Recruiting	Reduced Intensity	4/6, 5/6 or 6/6 HLA matched related or unrelated UCB or 8/8 or 7/8 HLA matched unrelated donor BM or PB progenitor graft	2mo-35	30	February 4, 2014	To evaluate efficacy of using a RIC regimen with UCBT, double cord UCBT, matched unrelated donor, BM transplant or PBSC transplant in patients with non-malignant disorders	USA, Pennsylvania Children’s Hospital of Pittsburgh of UPMC
NCT01850108	Non-Myeloablative Conditioning and BMT	Recruiting	Non-myeloablative	HLA-matched related donor or Haploidentical	2–70	25	May 2013	To evaluate efficacy of a non-myeloablative regimen for partially HLA-mismatched and HLA-matched BM	USA, Tennessee Vanderbilt-Ingram Cancer Center
NCT01049854	CD34+Selection for Partially Matched Family or Matched Unrelated Adult Donor HSCT	Recruiting	Myeloablative Arm and Reduced Intensity Arm	NR	Up to age 70	35	September 2011	To evaluate efficacy of CD34+ selection for patients with a partially matched or matched unrelated donor	USA, New York New York Medical College
NCT03249831	Non-Myeloablative Conditioning Regimen with Haploidentical T-Cell-Depleted PB Transplant for Patients with Severe SCD	Not yet recruiting	Non-myeloablative	Haploidentical	18–45	6	November 2017	To evaluate safety and feasibility of non-myeloablative conditioning and CD4+ T cell depleted grafts for haploidentical HSCT	USA, California City of Hope Medical Center
NCT00745420	Evaluating the Safety and Effectiveness of BMT in Children with SCD (BMT CTN 0601)	Active, not recruiting	Reduced Intensity	Unrelated BM	3–19	39	August 2008	To evaluate the safety and effectiveness of using BM from unrelated donors in children with severe SCD who receive RIC	USA, multicenter
NCT00977691	Nonmyeloablative Haploidentical PB Mobilized HPCT for Severe Congenital Anemias Including SCD and Beta-Thalassemia	Active, not recruiting	Non-myeloablative	Haploidentical	>2	59	September 9, 2009	To determine if a haploidentical BMT, low-intensity radiation, immunosuppressant drugs, and no chemotherapy will be effective in patients with SCD and Beta-thalassemia. To determine the effectiveness of cyclophosphamide in preventing rejection of the donor cells	USA, Maryland National Institutes of Health
Gene Therapy Trials
NCT02140554	A Study Evaluating the Safety and Efficacy of the LentiGlobin BB305 Drug Product in Severe SCD	Recruiting	NR	Gene Therapy	>18	29	August 2014	To evaluate gene therapy by transplantation of autologous CD34+ stem cells transduced ex vivo with the LentiGlobin BB305 lentiviral vector in subjects with severe SCD	USA, multicenter
NCT03282656	Gene Transfer for SCD	Recruiting	NR	Gene Therapy	3–35	7	November 30, 2017	To evaluate feasibility of HSC gene transfer for SCD using autologous BM derived CD34+ HSCs transduced with a lentiviral vector containing a short-hairpin RNA targeting BCL11a	USA, Massachusetts Boston Children’s Hospital
NCT02186418	Gene Transfer for Patients With SCD	Recruiting	NR	Gene Therapy	18	10	July 2014	To determine whether transfer of a fetal hemoglobin gene using a Gamma Globin Lentivirus Vector into human blood making cells is safe and feasible in patients with SCD	USA and Jamaica, Cincinnati Children’s Hospital Medical Center
NCT02247843	Stem Cell Gene Therapy for Sickle Cell Disease	Recruiting	Cytoreductive	Gene Therapy	>18	6	July 2014	Assess the safety and initial evidence for efficacy of an autologous transplant of βAS3-FB vector transduced BM CD34+ cells for adults with severe SCD	USA, California University of California, Los Angeles
NCT02151526	A Study Evaluating the Efficacy and Safety of LentiGlobin BB305 Drug Product in Beta-Thalassemia Major and SCD	Active, not recruiting	NR	Gene Therapy	5–35	7	July 2013	To evaluate safety and efficacy of the administration of LentiGlobin BB305 Drug Product to subjects with either beta-thalassemia major or severe SCD	Paris, France

BM: bone marrow, BMT: bone marrow transplant, CsA: cyclosporine, HPCT: hematopoietic precursor cell transplantation, HSCT: hematopoietic stem cell transplantation, MMF: mycophenolate mofetil, PB: peripheral blood, PBSC: peripheral blood stem cells, MSC: mesenchymal stromal cells, NR: not reported, RIC: reduced intensity conditioning, SCD: sickle cell disease, TBI: total body irradiation, UCBT: umbilical cord blood transplant