Trial Profile

ET3-201: Phase 1 Study of Hematopoietic Stem Cell Transplantation (HSCT) Gene Therapy Incorporating a Lentiviral Vector (LV) Encoding a High Expressing Factor VIII Transgene for Treatment of Severe Hemophilia A

Status: Not yet recruiting

Phase of Trial: Phase I

Latest Information Update: 16 Dec 2024

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At a glance

Drugs Gene therapies (Primary) ; Stem cell therapies (Primary) ; Busulfan; Granulocyte colony-stimulating factors; Levetiracetam; Plerixafor
Indications Haemophilia A
Focus Adverse reactions; First in man
Acronyms ET3-201
Sponsors Expression Therapeutics

11 Dec 2024 According to the Expression Therapeutics Media Release, results of the study were published by the New England Journal of Medicine.
11 Dec 2024 According to the Expression Therapeutics Media Release, company presented the results from this study at at the Annual Meeting of the American Society of Hematology (ASH) in San Diego, California.
15 Feb 2024 Planned End Date changed from 1 Apr 2039 to 1 Aug 2039.

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