Busulfan

29 trials 16 recruiting LOncology Orphan designation

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29 Registered trials

16 Recruiting

10 Completed trials

21 Sponsors

19 Countries

Phase III

Phase II

Phase III: 19 Phase II/III: 2 Phase II: 3 Phase I/II: 1 Phase IV: 1 Phase I (Other): 1 Phase I (FiH): 2

Oversight events:

1 urgent safety measure 3 corrective measures across 4 trials

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Indications tested

Sickle cell anaemia 4 Myelodysplastic syndrome 3 Neuroblastoma 2 Acute myeloid leukaemia 1 Myelofibrosis 1 Immunodeficiency 1 Pyruvate kinase deficiency anaemia 1

Sponsors using this IMP

Vertex Pharmaceuticals Inc. 6 Centre Hospitalier Universitaire De Nantes 2 Fondazione Gimema Franco Mandelli Onlus 2 Fondazione Telethon Ets 2 Orchard Therapeutics (Europe) Limited 2 Celgene Corp. 1 Genenta Science S.p.A. 1 GPOH gGmbH 1 Institut Gustave Roussy 1 Institut Paoli Calmettes 1 Klinikum Der Landeshauptstadt Stuttgart gKAöR 1 Medical Center - University Of Freiburg 1

All trials — page 2

29 total

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 study comparing current standard therapies with fedratinib for the treatment of myelofibrosis (either diagnosed alone or after polycythemi… Ended

2024-511972-33-00 Phase III 10 countries Celgene Corp.
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 25–29 of 29 trials

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