Found 13 matching trials
BEACON: A Study Evaluating the Safety and Efficacy of BEAM-101 in Patients With Severe Sickle Cell Disease PHASE1 This is an open-label, single-arm, multicenter, Phase 1/2 study evaluating ...
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Active Not Recruiting
Location
University of Alabama at Birmingham
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A Clinical Study Evaluating the Safety and Efficacy of CS-101 in Treating Subjects With ฮฒ-thalassemia EARLY_PHASE1 The goal of this open label, single-arm clinical study is to learn about th...
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The First Affiliated Hospital of Guangxi Medical University
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Growth and Development-related Outcomes in Children With Transfusion-dependent Beta-thalassemia After Gene Therapy N/A The investigate will conduct a cohort study to compare the growth and devel...
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Regenerative Medicine Center
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Base-Edited Hematopoietic Stem/Progenitor Cell X-Linked Severe Combined Immunodeficiency Gene Therapy PHASE1 Background:
X-linked severe combined immunodeficiency (XSCID) is a rare in...
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Enrolling By Invitation
Location
National Institutes of Health Clinical Center
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Internet-Based Program With or Without Telephone-Based Problem-Solving Training in Helping Long-Term Survivors of Hematopoietic Stem Cell Transplant Cope With Late Complications PHASE3 RATIONALE: A personalized Internet-based program may help improve fatigue, ...
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Fred Hutchinson Cancer Research Center/University of Washington Cancer Consortium
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A Long-term Follow-up Study in Participants Who Received CS-101 N/A This is a study to evaluate the long-term safety and efficacy of CS-101 in ...
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Enrolling By Invitation
Location
The First Affiliated Hospital of Guangxi Medical University
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Study of Safety and Efficacy of Genome-edited Hematopoietic Stem and Progenitor Cells in Sickle Cell Disease (SCD) PHASE1 This study evaluated a genome-edited, autologous, hematopoietic stem and pr...
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University of Chicago
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Base-edited Autologous Hematopoietic Stem Cell Transplantation in Treating Patients With ฮฒ-thalassemia Major EARLY_PHASE1 The goal of this open label, single-arm clinical study is to learn about th...
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Children's Hospital of Fudan University
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Base Editing for Mutation Repair in Hematopoietic Stem & Progenitor Cells for X-Linked Chronic Granulomatous Disease PHASE1 Background:
Chronic granulomatous disease (CGD) is a rare immune disorder ...
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National Institutes of Health Clinical Center
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Base-Edited Hematopoietic Stem/Progenitor Cell Gene Therapy for Treatment of CXCR4-WHIM PHASE1 Background:
Warts, hypogammaglobulinemia, infections and myelokathexis syn...
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National Institutes of Health Clinical Center
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