Exagamglogene Autotemcel — Drug Intelligence | 7 EU Trials

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7 Registered trials

4 Recruiting

2 Completed trials

1 Sponsors

4 Countries

Phase III

Phase I (FiH)

Phase III: 6 Phase I (FiH): 1

Oversight events:

4 corrective measures across 4 trials

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Indications tested

Sickle cell anaemia 5

Sponsors using this IMP

Vertex Pharmaceuticals Inc. 7

All trials

7 total

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 Pending

2023-503247-34-00 Phase III 2 countries Vertex Pharmaceuticals Inc.
A Phase 3b Study to Evaluate Efficacy and Safety of a Single Dose of Autologous CRISPR Cas9 Modified CD34+ Human Hematopoietic Stem and Prog… Recruiting

2024-514641-12-00 Phase III 2 countries Vertex Pharmaceuticals Inc.
A Phase 3 Study to Evaluate the Safety and Efficacy of a Single Dose of CTX001 in Pediatric Subjects With Severe Sickle Cell Disease Recruiting

2024-513978-22-00 Phase III 2 countries Vertex Pharmaceuticals Inc.
A Phase 3 Study to Evaluate the Safety and Efficacy of a Single Dose of CTX001 in Pediatric Subjects With Transfusion-Dependent β-Thalassemi… Recruiting

2024-513349-35-00 Phase III 2 countries Vertex Pharmaceuticals Inc.
A Long-term Follow-up Study of Subjects with β-Thalassemia or Sickle Cell Disease Treated with Autologous CRISPR-Cas9 Modified Hematopoietic… Recruiting

2024-512654-19-00 Phase III 3 countries Vertex Pharmaceuticals Inc.
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 a… Ended

2024-516067-83-00 Phase I (FiH) 2 countries Vertex Pharmaceuticals Inc.
A Phase 1/2/3 Study of the Safety and Efficacy of a Single Dose of Autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progen… Ended

2024-516894-57-00 Phase III 1 country Vertex Pharmaceuticals Inc.

Showing 1–7 of 7 trials

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