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Appendix I: Select Treatments Currently Under Development for Sickle Cell Disease
Pages 459-468

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From page 459...
... Appendix I Select Treatments Currently Under Development for Sickle Cell Disease The table begins on the following page.
From page 460...
... Sanguinate Prolong •  hase 2 clinical trial -- P Sanguinate is a carbon https://clinicaltrials. Pharmaceuticals final data collection date monoxide releasing molecule gov/ct2/show/ for primary outcome and oxygen transfer agent NCT02411708 measures was in under clinical development December 2017.
From page 461...
... Gamunex Albert Einstein •  hase I/II clinical trial -- P Intravenous immunoglobulin https://clinicaltrials. (intravenous College of estimated final data reduces neutrophil adhesion gov/ct2/show/ gammaglobulin)
From page 462...
... term=Rivarox data collection date for and endothelial cell aban&cond=sickle+cell outcome measures was in activation, and improve &rank=1 December 2018. microvascular blood flow in patients with SCD during the non-crisis, steady state.
From page 463...
... Gene therapy NCT ID: Boston • Open-label, non- The sickle cell gene therapy https://clinicaltrials. NCT03282656 Children's randomized, single center, clinical trial will involve a gov/ct2/show/ Hospital pilot and feasibility, single single infusion of autologous NCT03282656 arm cohort study of bone marrow-derived CD34+ seven patients -- estimated hematopoietic stem cells final data collection date transduced with a lentiviral for primary outcome vector containing a short measures is in February hairpin RNA targeting 2020.
From page 464...
... CTX001 CRISPR •  hase I/II clinical trial -- P An ex vivo gene-edited https://clinicaltrials. Therapeutics estimated final data cell therapy uses a new gov/ct2/show/ and Vertex collection date for primary technology called CRISPR NCT03745287 Pharmaceuticals outcome measures is (clustered regularly Inc.
From page 465...
... Immunosuppressive Medical Center final data collection date haploidentical hematopoietic gov/ct2/show/ Therapy for for primary outcome stem cell transplant and graft- NCT03279094 Haploidentical measures is February versus-host-disease prevention Transplants 2023. with or without pre-transplant immunosuppressive therapy.
From page 466...
... Conditioning for of Wisconsin estimated final data with Hydroxyurea, rabbit- gov/ct2/show/ Haploidentical collection date for primary ATG, Thiotepa, Fludarabine, NCT03263559 Bone Marrow outcome measures is Cyclophosphamide, Total Transplantation December 2023. Body Irradiation, and Mesna will be administered prior to Haploidentical Bone Marrow Transplantation in Eligible patients with a first degree HLA-haploidentical donor.
From page 467...
... Altemia might treat SCD by decreasing blood cell adhesion, chronic inflammation and red blood cell hemolysis, the factors that lead to reduction in pain episodes, vaso occlusive crises, and organ damage. NOTES: There are also psychosocial behavioral interventions and holistic approaches to treat sickle cell disease (see Chapter 4)


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