Trial Profile

Phase I/II Study Evaluating Safety and Efficacy of Autologous Hematopoietic Stem and Progenitor Cells Genetically Modified With IDUA Lentiviral Vector Encoding for the Human α-L-iduronidase Gene for the Treatment of Patients Affected by Mucopolysaccharidosis Type I, Hurler Variant

Status: Active, no longer recruiting

Phase of Trial: Phase I/II

Latest Information Update: 07 Jan 2026

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At a glance

Drugs OTL 203 (Primary) ; Busulfan; Fludarabine; Lenograstim; Plerixafor; Rituximab
Indications Mucopolysaccharidosis I
Focus Adverse reactions; First in man; Proof of concept; Therapeutic Use
Acronyms TigetT10_MPS1H
Sponsors Orchard Therapeutics

09 Dec 2026 Planned primary completion date changed from 1 Mar 2028 to 1 Jan 2028.
27 Jun 2025 Planned End Date changed from 1 Jan 2025 to 1 Mar 2028.
27 Jun 2025 Planned primary completion date changed from 1 Jan 2025 to 1 Mar 2028.

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