Safety and Tolerability of SB-913 Gene Transfer in Subjects with MPSII
Research type
Research Study
Full title
A Phase I, Multicenter, Open-Label, Single-Dose, Dose-Ranging Study to Assess the Safety and Tolerability of SB-913, a rAAV2/6-based Gene Transfer in Subjects with Mucopolysaccharidosis II (MPSII)
IRAS ID
249183
Contact name
Maureen Cleary
Contact email
Sponsor organisation
Sangamo Therapeutics, Inc.
Eudract number
2018-000192-33
Clinicaltrials.gov Identifier
Clinicaltrials.gov Identifier
17006, BB-IND
Duration of Study in the UK
4 years, 1 months, 19 days
Research summary
This study is being done to see if there is a new way to possibly treat MPS II(Mucopolysaccharidosis II or Hunter syndrome). MPS II is a rare disease that people are born with due to lack of an important enzyme called IDS. IDS is necessary for the body to breakdown certain sugar molecules which are part of the body’s natural metabolism. When IDS is missing or not working properly, people develop the symptoms of MPS II including damage to the heart, lungs, bones, and sometimes the brain.
This study will test to see if doctors can use a process called “gene therapy” to treat MPS II. Genes are made of DNA and are present inside every cell in your body. Genes are responsible for determining things like hair colour, eye colour, and height, etc. A missing or broken gene can cause a disease like MPS II. When people have MPS II, they are either missing the gene that makes IDS or the gene makes a version of IDS that doesn’t work.
Gene therapy is a treatment that tries to replace the broken or missing gene with a working copy. The gene therapy study drug has a code name called SB-913. In this study, up to 23 study participants with MPS II aged 5 and older will receive SB- 913. The study will observe the study participants for 3 years. The study will determine if SB-913 is a safe and effective treatment to deliver the IDS gene and provide benefit for people with MPS II.REC name
North East - York Research Ethics Committee
REC reference
18/NE/0221
Date of REC Opinion
17 Aug 2018
REC opinion
Further Information Favourable Opinion