Plerixafor — Drug Intelligence | 13 EU Trials

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13 Registered trials

4 Recruiting

6 Completed trials

6 Sponsors

6 Countries

Phase III

Phase I (FiH)

Phase III: 7 Phase II: 1 Phase I/II: 1 Phase I (Other): 1 Phase I (FiH): 3

Oversight events:

3 corrective measures across 3 trials

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Indications tested

Sickle cell anaemia 4 Immunodeficiency 1 Pyruvate kinase deficiency anaemia 1

Sponsors using this IMP

Vertex Pharmaceuticals Inc. 6 Fondazione Telethon Ets 3 Orchard Therapeutics (Europe) Limited 2 San Raffaele Hospital 2 Genenta Science S.p.A. 1 Rocket Pharmaceuticals Inc. 1

All trials

13 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 I/II open label study to assess safety, feasibility and efficacy of ex vivo expanded, autologous haematopoietic stem and progenitor … Pending

2024-518972-30-00 Phase I (FiH) 1 country Fondazione Telethon Ets; San Raffaele Hospital
A single arm, open label, multicenter, single-dose, phase 2b clinical study evaluating efficacy and safety of gene therapy using autologous … Pending

2025-522160-32-00 Phase II 1 country Fondazione Telethon Ets; San Raffaele Hospital
A Clinical Study to Evaluate the Use of a Cryopreserved Formulation of OTL-103 in Subjects with WAS. Recruiting

2024-517792-20-00 Phase III 1 country Fondazione Telethon Ets
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 study to investigate the efficacy and safety of OTL-203 in subjects with mucopolysaccharidosis type I, Hurler syndrome (MPS-IH) compared w… Ended

2022-500306-17-00 Phase III 2 countries Orchard Therapeutics (Europe) Limited
Gene therapy study with autologous hematopoietic stem cells for patients affected by MPS-IH Ended

2024-514870-29-00 Phase I (FiH) 1 country Orchard Therapeutics (Europe) Limited
A clinical gene therapy study with hematopoietic stem cells for the treatment, with single dose of Temferon, of patients suffering from meta… Ended

2024-512898-27-00 Phase I/II 1 country Genenta Science S.p.A.
Gene Therapy for Pyruvate Kinase Deficiency (PKD): A Phase I Clinical Trial to Evaluate the Safety of the Infusion of Autologous CD34+ Cells… Ended

2024-511520-13-00 Phase I (Other) 1 country Rocket 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 1–13 of 13 trials

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