The Children's Hospital University of Illinois
A SAD/MAD to Assess the Safety, Pharmacokinetics and Pharmacodynamics of FT-4202 in Healthy Volunteers and Sickle Cell Disease Patients
FT-4202 is an oral small-molecule agonist of pyruvate kinase red blood cell isozyme (PKR) being developed for the treatment of hemolytic anemias. This initial study will characterize the safety, tolerability and the pharmacokinetics/pharmacodynamics (PK/PD) of a single ascending dose and multiple ascending doses of FT-4202 in the context of Phase 1 studies in healthy volunteers and sickle cell disease patients. The effects of food on the absorption of FT-4202 will also be evaluated in healthy volunteers.
August 25, 2020Integration of mHEALTH Into the Care of Patients With Sickle Cell Disease to Increase Hydroxyurea Utilization
This project proposes to develop, test and evaluate targeted interventions to improve clinical provider prescribing of and patient adherence to hydroxyurea (HU). Using a stepped-wedge design, The investigators will test two innovative interventions utilizing mobile health to address both patients' and providers' needs: 1) an mHealth application for patients (InCharge Health app) that includes multi-component features to address the memory, motivation, and knowledge barriers to hydroxyurea use, and 2) an mHealth toolbox application for providers (HU Toolbox app) that addresses clinical knowledge barriers in prescribing and monitoring hydroxyurea use.
August 25, 2020A Safety and Efficacy Study Evaluating CTX001 in Subjects With Severe Sickle Cell Disease
This is a single-arm, open-label, multi-site, single-dose Phase 1/2 study in subjects with severe sickle cell disease (SCD). The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (hHSPCs) using CTX001.
August 25, 2020Dissemination and Implementation of Stroke Prevention Looking at the Care Environment (DISPLACE)
The Dissemination and Implementation of Stroke Prevention Looking at the Care Environment (DISPLACE) study is a multi-center, national, National Heart, Lung and Blood Institute (NHLBI)-funded grant to look at the real-world implementation of stroke prevention guidelines (STOP Protocol) in which transcranial Doppler (TCD), a measure of cerebral blood vessel velocity, is used to screen for stroke risk in children ages 2-16 with sickle cell anemia (SCA). Part 3 of the DISPLACE study is an implementation clinical trial designed to test novel implementation strategies with the goal of improving adherence and implementation of stroke screening. 16 of the lowest scoring implementation rates from DISPLACE Part 1 will participate in DISPLACE Part 3. All original 28 sites from DISPLACE Parts 1 and 2 will receive a patient and provider educational intervention including a re-branding of the TCD as "Sickle Stroke Screen" with a new infographic and educational materials. The 16 sites with moving to Part 3 will be provided a Provider reminder strategy, which is a web based application designed to remind providers of when patients are due for their Sickle Stroke Screen. These 16 sites will be randomized and 8 will be given an additional Patient Communication Strategy. These sites will have a single designed coordinator with whom patients will communicate with about scheduling, rescheduling, and any other questions regarding their Sickle Stroke Screen. Upon completion, data will be analyzed to compare those who have had TCD screenings done appropriately and those who did not as well as the overall effect of the multi level interventions on the changes in TCD rates.
August 25, 2020Sickle Cell Pro-Inflammatory Response to Interval Training Study (SPRINTS)
Recommendations for exercise prescription currently do not exist for individuals with sickle cell anemia (SCA) despite the known impact that SCA-related complications has on physical functioning and fitness. A major barrier to increasing physical activity in SCA is the concern that the well-described inflammatory effects of exercise could precipitate or exacerbate complications such as vaso-occlusive pain or airway bronchoconstriction (i.e. exercise-induced asthma). Although the investigator's preliminary data suggest that increasing physical activity may be beneficial rather than harmful in children with SCA, the pro-inflammatory effects associated with repeated bouts of moderate to vigorous exercise remain poorly understood in this population. The long term goal is to address the safety and health impact of regular exercise in children with SCA. This proposal would help establish the safety of moderate to vigorous intensity exercise in children with SCA and importantly, will inform the design of future clinical trials focused on exercise training as a transformative strategy to improve fitness and overall well-being in this population.
August 25, 2020- More Sickle Cell Anemia – The Children's Hospital University of Illinois posts
Northwestern University - Ann and Robert H. Lurie Children's Hospital of Chicago
Acute 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, 2020Shared-Decision Making for Hydroxyurea (ENGAGE-HU)
The goal of the study is to understand how best to help parents of young children with sickle cell disease and their clinicians have a shared discussion about hydroxyurea (one that takes into account medical evidence and parent values and preferences). The study will compare two methods to help clinicians facilitate this—a clinician pocket guide and a clinician hydroxyurea shared decision making toolkit—in a group of parents of children ages 0-5 with sickle cell disease. The investigators hope that both methods lead to parents reaching a high-quality, well-informed decision. In addition, the team hopes to demonstrate that parents who experience a shared decision will have lower anxiety and decisional uncertainty. The researchers also expect these parents to be more likely to choose hydroxyurea and that their children will have less pain, fewer hospitalizations, better developmental outcomes, and higher quality of life. The project team hopes to show that the toolkit method is easy for clinicians to use and gives parents the support needed to make an informed decision.
September 1, 2020Abatacept for GVHD Prophylaxis After Hematopoietic Stem Cell Transplantation for Pediatric Sickle Cell Disease
To assess the tolerability of the costimulation blocking agent abatacept (CTLA4-Ig) when added to the standard graft versus host disease (GVHD) prophylaxis regimen of a calcineurin inhibitor and methotrexate in patients receiving early alemtuzumab followed by fludarabine, thiotepa, melphalan, and alemtuzumab for conditioning.
September 1, 2020Minimizing Toxicity in HLA-identical Related Donor Transplantation for Children With Sickle Cell Disease (SUN)
This multisite prospective study seeks to determine if HLA-identical sibling donor transplantation using alemtuzumab, low dose total-body irradiation, and sirolimus (Sickle transplant Using a Nonmyeloablative approach, "SUN") can decrease the toxicity of transplant while achieving a high cure rate for children with sickle cell disease (SCD).
September 1, 2020Sickle Cell Pro-Inflammatory Response to Interval Training Study (SPRINTS)
Recommendations for exercise prescription currently do not exist for individuals with sickle cell anemia (SCA) despite the known impact that SCA-related complications has on physical functioning and fitness. A major barrier to increasing physical activity in SCA is the concern that the well-described inflammatory effects of exercise could precipitate or exacerbate complications such as vaso-occlusive pain or airway bronchoconstriction (i.e. exercise-induced asthma). Although the investigator's preliminary data suggest that increasing physical activity may be beneficial rather than harmful in children with SCA, the pro-inflammatory effects associated with repeated bouts of moderate to vigorous exercise remain poorly understood in this population. The long term goal is to address the safety and health impact of regular exercise in children with SCA. This proposal would help establish the safety of moderate to vigorous intensity exercise in children with SCA and importantly, will inform the design of future clinical trials focused on exercise training as a transformative strategy to improve fitness and overall well-being in this population.
August 25, 2020- More Sickle Cell Anemia – Northwestern University - Ann and Robert H. Lurie Children's Hospital of Chicago posts
Medical College of Wisconsin - Children's Hospital of Wisconsin
Haplo T-Cell Depleted Transplantation in High-Risk Sickle Cell Disease (HaploSCD)
This study is being done to determine the safety and outcome (long-term control) of a high-dose chemotherapy regimen followed by an infusion of CD34 selected (immune cells) stem cells from a partially matched adult family member donor, called haploidentical stem cell transplantation, in high-risk sickle cell disease patients.
August 25, 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 Sickle Cell Anemia – 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
Haploidentical Hematopoietic Stem Cell Transplantation (HSCT) for Patients With Severe Sickle Cell Disease
The purpose of this study is to develop a safe and curative stem cell transplant approach to treating sickle cell disease by assessing the safety of haploidentical hematopoietic stem cell transplantation using αβ+ T-cell depletion for children and adolescents with severe sickle cell disease (SCD).
August 25, 2020- More Sickle Cell Anemia – University of Chicago - Comer Children's Hospital posts
University of Iowa - Stead Family Children's Hospital
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University of Minnesota - Masonic Children's Hospital
Hematopoietic 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, 2020A Study Evaluating Gene Therapy With BB305 Lentiviral Vector in Sickle Cell Disease
This is a non-randomized, open-label, multi-site, single-dose, Phase 3 study in approximately 35 adults and pediatric subjects ≥2 and ≤50 years of age with sickle cell disease (SCD). The study will evaluate hematopoietic stem cell (HSC) transplantation (HSCT) with LentiGlobin BB305 Drug Product for SCD.
August 25, 2020- More Sickle Cell Anemia - University of Minnesota - Masonic Children's Hospital posts
University of Wisconsin - American Family Children's Hospital
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