The Children's Hospital University of Illinois
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Northwestern University - Ann and Robert H. Lurie Children's Hospital of Chicago
A Study Evaluating the Efficacy and Safety of the LentiGlobin® BB305 Drug Product in Subjects With Transfusion-Dependent β-Thalassemia
This is a single-arm, multi-site, single-dose, Phase 3 study in approximately 18 subjects ≤50 years of age with transfusion-dependent β-thalassemia (TDT), who have a β0/β0, β0/IVS-I-110, or IVS-I-110/IVS-I-110 genotype. The study will evaluate the efficacy and safety of autologous hematopoietic stem cell transplantation (HSCT) using LentiGlobin BB305 Drug Product.
September 1, 2020Acute GVHD Suppression Using Costimulation Blockade to Expand Non-malignant Transplant (ASCENT)
The ASCENT Trial is a single arm, multi-center, phase II study. The primary objective is to determine the rejection-free, severe graft-versus-host disease (GVHD)-free survival in pediatric patients with serious non-malignant hematologic diseases (NMHD) undergoing mismatched unrelated donor (URD) hematopoietic stem cell transplantation (HSCT) with abatacept added to conventional GVHD prophylaxis. The secondary objective is to characterize the impact of abatacept on infection and the reconstitution of protective immunity to infection. Transplanted patients will be followed for 3 years. Weight-based peripheral blood samples will be drawn longitudinally through two years to evaluate immune reconstitution. The study will enroll 28 pediatric patients with serious NMHD undergoing mismatched URD HSCT. The trial will include two strata, based on donor matching. Stratum 1 (n=14) will be for patients with 7/8 donors and stratum 2 (n=14) will be for those with 8/8 (matched) donors. All participants will receive 8 doses of abatacept (10 mg/kg intravenously on days -1, +5, +14, +28, +56, +84, +112, and +150). Recruitment is expected to last for about 2 years and participants will be followed for up to 3 years. This trial will test the hypothesis that extended abatacept administration (combined with a standard regimen of tacrolimus and mycophenolate mofetil) will effectively prevent acute and chronic GVHD in children and adolescents receiving mismatched URD HSCT, without compromising their engraftment or reconstitution of protective immunity to infection.
September 1, 2020- More Thalassemia – Northwestern University - Ann and Robert H. Lurie Children's Hospital of Chicago posts
Medical College of Wisconsin - Children's Hospital of Wisconsin
Myeloablative Conditioning, Prophylactic Defibrotide and Haplo AlloSCT for Patients With Sickle Cell Disease (NYMC-571)
This is a follow-up trial to NYMC 526 (NCT01461837) to assess the safety, efficacy and toxicity of administering Defibrotide prophylaxis for high-risk sickle cell or beta thalassemia patients undergoing a familial haploidentical allogeneic stem cell transplantation with CD34 enrichment and T-cell addback. This patient population historically has a risk of developing sinusoidal obstructive syndrome (SOS) and Defibrotide has demonstrated efficacy in treatment of SOS. The Funding Source is FDA OOPD.
September 1, 2020Myeloablative Conditioning, Prophylactic Defibrotide and Haplo AlloSCT for Patients With Sickle Cell Disease (NYMC-571)
This is a follow-up trial to NYMC 526 (NCT01461837) to assess the safety, efficacy and toxicity of administering Defibrotide prophylaxis for high-risk sickle cell or beta thalassemia patients undergoing a familial haploidentical allogeneic stem cell transplantation with CD34 enrichment and T-cell addback. This patient population historically has a risk of developing sinusoidal obstructive syndrome (SOS) and Defibrotide has demonstrated efficacy in treatment of SOS. The Funding Source is FDA OOPD.
August 25, 2020- More Thalassemia – Medical College of Wisconsin - Children's Hospital of Wisconsin posts
Rush University Children's Hospital
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University of Chicago - Comer Children's Hospital
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University of Iowa - Stead Family Children's Hospital
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University of Minnesota - Masonic Children's Hospital
Haploidentical Allogeneic Hematopoietic Stem Cell Transplantation (HaploHCT) Following Reduced Intensity Conditioning (RIC) for Selected High Risk Non-Malignant Diseases
This is a Phase II study for the use of T-cell replete reduced intensity conditioning (RIC) haploidentical donor allogeneic hematopoietic cell transplantation (HaploHCT) for individuals with high-risk non-malignant diseases who lack a suitable HLA-matched sibling donor.
September 1, 2020Hematopoietic Stem Cell Transplant for High Risk Hemoglobinopathies
This is a study to collect the outcomes of stem cell transplantation for patients with hematologic diseases other than cancer.
August 25, 2020Haploidentical Allogeneic Hematopoietic Stem Cell Transplantation (HaploHCT) Following Reduced Intensity Conditioning (RIC) for Selected High Risk Non-Malignant Diseases
This is a Phase II study for the use of T-cell replete reduced intensity conditioning (RIC) haploidentical donor allogeneic hematopoietic cell transplantation (HaploHCT) for individuals with high-risk non-malignant diseases who lack a suitable HLA-matched sibling donor.
August 25, 2020- More Thalassemia - University of Minnesota - Masonic Children's Hospital posts
University of Wisconsin - American Family Children's Hospital
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