Sickle Cell Disease Clinical Trials

Trials for Sickle Cell Disease

A Phase 3 Study to Evaluate Efficacy and Safety of a Single Dose of Exa-cel in Subjects With Severe Sickle Cell Disease, βS/βC Genotype

NCT05951205 · WITHDRAWN · Phase 3

St. Jude Autologous Genome Edited Stem Cells For Sickle Cell Disease-1

NCT06506461 · RECRUITING · Phase 1

Clinical Study to Evaluate the Safety and Efficacy of Single Dose Intravenous Infusion of CRISPR/Cas9-edited Autologous CD34 + Hematopoietic Stem/Progenitor Cells (BRL-101) in the Treatment of Severe Sickle Cell Disease

NCT06300723 · ENROLLING BY INVITATION · N/A

Clinical Study on the Safety and Efficacy of a Single Intravenous Dose of CRISPR/Cas9-Edited Autologous CD34+ Hematopoietic Stem/Progenitor Cells (BRL-101) in the Treatment of Sickle Cell Disease

NCT06287086 · NOT YET RECRUITING · N/A

Clinical Study on the Safety and Efficacy of a Single Intravenous Dose of CRISPR/Cas9-Edited Autologous CD34+ Hematopoietic Stem/Progenitor Cells (BRL-101) in the Treatment of Sickle Cell Disease

NCT06287099 · NOT YET RECRUITING · N/A

A Phase I/II Study of Nula-cel in Autologous CD34+ Hematopoietic Stem Cells to Convert HbS to HbA for Treating Severe Sickle Cell Disease

NCT04819841 · RECRUITING · Phase 2

A First-in-patient Phase I/II Clinical Study to Investigate the Safety, Tolerability and Efficacy of Genome-edited Hematopoietic Stem and Progenitor Cells in Subjects With Severe Complications of Sickle Cell Disease

NCT04443907 · TERMINATED · Phase 1

A Phase 1/2/3 Study to Evaluate the Safety and Efficacy of a Single Dose of Autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (CTX001) in Subjects With Severe Sickle Cell Disease

NCT03745287 · COMPLETED · Phase 3

Examining the Knowledge, Attitudes, and Beliefs of Sickle Cell Disease Patients, Parents of Patients With Sickle Cell Disease, and Providers Towards the Integration of CRISPR in Clinical Care

NCT03167450 · COMPLETED · N/A

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