Table 3A.

Summary of clinical trials of recruiting and not yet recruiting studies for HLA Haploidentical and Matched Sibling Donor HCT

Recruiting
	Identifier/Location	Study Title	Age Range (years)	Participant Number	Phase	Study Goal
HLA Matched Related D
Pediatrics	NCT03214354/Calgary	A Phase II Pilot Study of Nonmyeloablative Conditioning Hematopoietic Stem Cell Transplantation in Children With Sickle Cell Disease Wh+C7:H14o Have a Matched Related Major ABO-Incompatible Donor (Sickle-AID)	1 Year to 19 Years	12	2	Safety and efficacy of NMA conditioning for matched related major ABO-incompatible donor.
	NCT04018937/Emory	Early HLA Matched Sibling Hematopoietic Stem Cell Transplantation for Children With Sickle Cell Disease: A Sickle Transplant Advocacy and Research Alliance (STAR) Trial	2 Years to 13 Years	58	2	Safety and efficacy of fludarabine, alemtuzumab and melphalan (FAM) conditioning in pediatrics.
	NCT03587272/DC	Minimizing Toxicity in HLA-Identical Related Donor Transplantation for Children With Sickle Cell Disease	2 Years to 25 Years	30	2	Decrease toxicity while achieving a high cure rate for children using alemtuzumab, low dose total-body irradiation, and sirolimus
	NCT01499888/Chicago	Ph I/II Study of Allogeneic SCT for Clinically Aggressive SCD	16 Years to 60 Years	15	1 / 2	Use of immune-suppressive agents and low-dose TBI in patients not candidates for or experienced complications from hydroxyurea therapy.
	NCT02105766/NIH	Nonmyeloablative Peripheral Blood Mobilized Hematopoietic Precursor Cell Transplantation for Sickle Cell Disease and Beta-Thalassemia in Individuals With Higher Risk of Transplant Failure	4 Years and older	162	2	To see if low dose radiation (300 cGy), oral cyclophosphamide, pentostatin, and sirolimus help to better accept donor stem cells.
Children and Adults	NCT03421756/Pittsburgh	A Pilot Study Evaluating the Efficacy of Non-Myeloablative Matched Related Donor Peripheral Blood Stem Cell Transplant in Patients With Severe Sickle Cell Disease	18 Years and older	12	1	MRD allogeneic SCT in adults with severe sickle cell disease using alemtuzumab.
	NCT00061568/NIH	Nonmyeloablative Allogeneic Peripheral Blood Mobilized Hematopoietic Precursor Cell Transplantation For Severe Congenital Anemias Including Sickle Cell Disease (SCD) and Beta-Thalassemia	2 Years and older	150	1/2.	Low dose radiation, alemtuzumab and sirolimus as a strategy to provide clinical remission with a lower risk of GVHD development
	NCT05249452/Amsterdam	Adding Azathioprine/Hydroxyurea Preconditioning to Alemtuzumab/TBI to Reduce Risk of Graft Failure in Matched Sibling Donor Allogeneic HSCT in Adult Sickle Cell Patients	16 Years to 60 Years	20	N/A	Investigate whether 3-month long preconditioning with azathioprine and alemtuzumab/TBI improves disease-free survival and donor chimerism
	Identifier	Study Title	Age range (yr)	Participant Number	Phase	Study Goal
HLA Haploidentical
Pediatric	NCT04362293/St. Jude	Reduced Intensity Related Donor Peripheral Blood Derived Hematopoietic Progenitor Cell Transplantation for Patients With Severe SCD	2 Years to 25 Years	40	2	RIC of alemtuzumab, thiotepa and low dose total body irradiation and pre-conditioning of hydroxyurea and azathioprine to reduce the risk of graft rejection.
	NCT04207320/Chicago	Hematopoietic Stem Cell Transplantation for Patients With Severe Sickle Cell Disease Using Myeloablative Conditioning and αβ+ T-cell Depleted Hematopoietic Stem Cells From Partially Matched Familial Donors	2 Years to 25 Years	38	N/A	Safety of haploidentical HSCT using αβ+ T-cell depletion for children and adolescents with severe SCD.
	NCT03367546/Minnesota	Haploidentical Donor T-cell Replete Allogeneic Hematopoietic Cell Transplant Following Reducing Intensity Conditioning for Patients With Selected High Risk Non-Malignant Disease	up to 25 Years	20	2	Use of T-cell replete RIC Haplo HCT for individuals who lack a suitable HLA-matched sibling donor.
	NCT03279094/City of Hope	A Pilot Study of Pre-transplant Immunosuppressive Therapy for Haploidentical Transplants in Patients With SCD	1 Year to 30 Years	18	1	Haploidentical donors and non-toxic, myeloablative regimen, with the goal of achieving consistent donor chimerism.
	NCT03240731/Paris	BMT HLA Haploidentical After RIC and Prevention of GVHD Based on Post-transplant Cyclophosphamide Administration in Patients With Severe SCD	13 Years to 40 Years	16	2	RIC haploidentical marrow transplants and prevention of GVHD based on cyclophosphamide administration post-transplantation.
	NCT03263559/Wisconsin	Haploidentical BMT in Sickle Cell Patients (BMTCTN1507)	5 Years to 45 Years	80	2	Use of pre-conditioning hydroxyurea with Thymoglobulin/Cyclophosp hamide/Fludarabine/Thiotep a and post-transplant high-dose cyclophosphamide.
	NCT02757885/Emory	Transplantation Using Reduced Intensity Approach for Patients With SCD From Mismatched Family Donors of Bone Marrow (TRANSFORM)	15 Years to 40 Years	15	2	Haploidentical SCT with hydroxyurea, fludarabine, thiotepa, anti-thymocyte globulin, radiation and cyclophosphamide.
	NCT02065596/Cleveland	Hematopoietic Stem Cell Transplant for Sickle Cell Disease	18 Years to 65 Years	25	1/2.	Use of fludarabine to reduce graft rejection.
	NCT03077542/NIH	Nonmyeloablative Haploidentical Peripheral Blood Mobilized Hematopoietic Precursor Cell Transplantation for Sickle Cell Disease	2 Years and older	93	1/2.	Decrease risk of GVHD with low radiation dose, Alemtuzumab, Sirolimus, Cyclophosphamide, and pentostatin.