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Clinical Trial Report
Gene Therapy Trial Report
Summary
Gene Therapy with Modified Autologous Hematopoietic Stem Cells for Patients with Mucopolysaccharidosis Type II
NCTID
NCT05665166
(View at clinicaltrials.gov)
Description
MPS II is a genetic disorder that affects boys. Boys with MPS II are missing a working enzyme known as iduronate-2-sulfatase (IDS) which is needed to break down long sugar chains in the body. When this enzyme is missing, these sugars build up to excess causing damage, and stop organs such as the brain from working properly. Children with MPS II often have progressive symptoms such as developmental delay and physical problems. The only approved treatment for MPS II is enzyme replacement therapy. This involves a regular infusion of the missing enzyme into the blood stream. But this treatment only helps some symptoms and cannot help problems in the brain. This study will be the first in human clinical trial to check whether using a gene therapy in children with MPS II is safe and is able to provide enough enzyme to help with disease symptoms. Gene therapy involves changing the genetic information that makes up a person, by taking a correct version of the gene that is needed to make the working IDS enzyme and putting it back into the body. This means that the body can then make the missing enzyme itself. The good thing with this therapy is that the body should be able to make this enzyme forever. To make sure the therapy is safe and working patients will be closely followed for 2 years.
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Development Status
Inactive
Indication
Mucopolysaccharidosis II
Disease Ontology Term
DOID:12799
Compound Name
AVR-RD-05
Compound Description
Autologous CD34+ HSCs transduced ex vivo with CD11B LV encoding human IDS tagged with ApoEII
Sponsor
University of Manchester
Funder Type
Other
Recruitment Status
Recruiting
Enrollment Count
5
Results Posted
Not Available
Therapy Information
Target Gene/Variant
IDS
Therapy Type
Gene transfer
Therapy Route
Ex-vivo
Mechanism of Action
Functional gene replacement
Route of Administration
Intravenous
Drug Product Type
Autologous cells
Target Tissue/Cell
CD34+ cells
Delivery System
Viral transduction
Vector Type
LV
Editor Type
Dose 1
Transduced CD34+ cells
Dose 2
Dose 3
Dose 4
Dose 5
Study Record Dates
Current Stage
Phase1, Phase2
Submit Date
2022-12-07
Completion Date
2027-09
Last Update
2024-10-02
Participation Criteria
Eligible Age
3 Months - 22 Months
Standard Ages
Child
Sexes Eligible for Study
MALE
Locations
No.of Trial Sites
1
Locations
United Kingdom
Regulatory Information
Has US IND
False
FDA Designations
Orphan Drug Designation, Rare Pediatric Disease Designation
Recent Updates
AVROBIO supported this IIT, Program development was halted in July 2023
Resources/Links
News and Press Releases
SEC Form 10-K: AVROBIO, Inc. FY2021
Preclinical Publications
Brain-targeted stem cell gene therapy corrects mucopolysaccharidosis type II via multiple mechanisms
Sustained long-term disease correction in a murine model of MPSII following stem cell gene therapy
Establishment of the Effectiveness of Early Versus Late Stem Cell Gene Therapy in Mucopolysaccharidosis II for Treating Central Versus Peripheral Disease
Protocol
Design and validation of a GMP stem cell manufacturing protocol for MPSII hematopoietic stem cell gene therapy