Autologous ex-vivo gene modified HSCT in MPSIIIA
Research type
Research Study
Full title
A Phase I-II study of Autologous CD34+ Haematopoietic Stem Cells Transduced ex vivo with CD11B Lentiviral Vector Encoding for Human SGSH in patients with Mucopolysaccharidosis Type IIIA (MPS IIIA, Sanfilippo Syndrome type A).
IRAS ID
265529
Contact name
Robert Wynn
Contact email
Sponsor organisation
University of Manchester
Eudract number
2019-002051-42
Duration of Study in the UK
5 years, 0 months, 1 days
Research summary
Mucopolysaccharidosis Type IIIA is also referred to as MPS IIIA or Sanfilippo Syndrome. This is one of a family of diseases referred to as lysosomal storage diseases. MPS IIIA is an inherited disease that means the person affected is missing a gene which codes for a specific enzyme (heparan-N-sulfatase) . Without this enzyme a waste product builds up in cells in the body causing the symptoms seen in MPS IIIA – delayed development, progressive deteriorating mental status and behavioural problems. Although different treatments have been trialled in the past in this condition, currently there are no known effective treatments for MPS IIIA. In children with other MPS conditions it has been shown that it is possible to provide enzyme to cells in the body by a bone marrow transplant – transplanted cells from a donor, able to make the absent enzyme and secrete to other cells through the blood, however, in MPS IIIA a bone marrow transplant does not seem to deliver enough enzyme to the cells to get rid of the waste product.
The treatment proposed, hopes to deliver increased amounts of enzyme to the cells by genetic manipulaton of the patients own cells. The treatment process can be broken down in to 3 stages: Stem cell collection, chemotherapy conditioning and infusion of gene-modified cells.REC name
London - West London & GTAC Research Ethics Committee
REC reference
19/LO/1175
Date of REC Opinion
25 Oct 2019
REC opinion
Further Information Favourable Opinion