Trial Profile

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

Status: Discontinued

Phase of Trial: Phase I

Latest Information Update: 14 Feb 2025

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At a glance

Drugs HIX 763 (Primary) ; OTQ-923 (Primary)
Indications Sickle cell anaemia
Focus Adverse reactions; First in man; Pharmacogenomic; Proof of concept; Therapeutic Use
Sponsors Novartis Pharmaceuticals

11 Feb 2025 Status changed from active, no longer recruiting to discontinued due to business reasons and was not a consequence of any safety concern.
18 Dec 2024 Planned End Date changed from 5 Dec 2024 to 17 Jan 2025.
18 Dec 2024 Planned primary completion date changed from 5 Dec 2024 to 16 Jan 2025.

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