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Phase 1
Phase I/II Trial of Lentiviral Gene Transfer for SCID-X1 With Low Dose Targeted Busulfan Conditioning
This is a phase I/II open label multi-center study in which patients will receive low dose targeted busulfan followed by infusion of autologous CD34+ selected …
Severe Combined Immunodeficiency, X LinkedGene Therapy
David WilliamsNCT03311503
Protocol For A Research Database For Hematopoietic Stem Cell Transplantation, Other Cellular Therapies and Marrow Toxic Injuries
The primary purpose of the Research Database is to have a comprehensive source of observational data that can be used to study HSC transplantation and cellular…
Autologous Stem Cell TransplantationAllogeneic Stem Cell TransplantationSolid Tumors+4 more
Center for International Blood and Marrow Transplant ResearchNCT01166009
Phase 1
A Phase 1 Study to Evaluate the Safety of KLN-1010, a Novel, In Vivo Gene Therapy to Generate Anti-B Cell Maturation Antigen (Anti-BCMA) Chimeric Antigen Receptor-T Cells (CAR-T) in Patients With Relapsed and Refractory Multiple Myeloma
The goal of this clinical trial is to evaluate the safety, tolerability, and recommended Phase 2 Dose (RP2D) of KLN-1010 in patients with relapsed or refractor…
Multiple Myeloma in RelapseMyeloma MultipleMultiple Myeloma Progression+11 more
Kelonia Therapeutics, Inc.NCT07075185
Phase 1
Safety and Efficacy of AAV9/AP4B1 For Patients With AP4B1-related Hereditary Spastic Paraplegia Type 47 (SPG47): A Phase 1/2 Single-Center, Open-Label Study of Stereotactic Intra-cisterna Magna Administration
Safety and Efficacy of AAV9/AP4B1 For Patients with AP4B1-related Hereditary Spastic Paraplegia Type 47 (SPG47): A Phase 1/2 Single-Center, Open-Label Study of…
HSPHereditary Spastic ParaplegiaHereditary Spastic Paraparesis+10 more
BlackfinBio LtdNCT06948019