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: 11 Aug 2025

Price :

$35 ^*

Note:

Adis is an information provider.
Final gross price and currency may vary according to local VAT and billing address.
Your purchase entitles you to full access to the information contained in our trial profile at the time of purchase.
A link to download a PDF version of the trial profile will be included in your email receipt.

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

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.
03 Feb 2025 According to an Orchard Therapeutics media release, data from this trial will be presented at the 21st Annual WORLDSymposium taking place February 3-7 in San Diego.

If your organization or you do not have a subscription, try one of the following:

Contacting your organization’s admin about adding this content to your AdisInsight subscription
Buying a PDF version of any individual profile
Request a free trial (opens in a new window)

If your organization has a subscription, there are several access options, even while working remotely:

Working within your organization’s network
Login with username/password or try to access via your institution
Persisted access using your organization’s identifier stored in your user browser for 90 days

For assistance, contact us at asktheexpert.adisinsight@springer.com