Preexisting Humoral Immunity to AAV in Subjects with MPS type III
Research type
Research Study
Full title
A Study of Pre-existing Humoral immunity to Adeno-Associated Vectors\nin Subjects with Mucopolysaccharidosis type III
IRAS ID
266866
Contact name
Suresh Vijay
Contact email
Sponsor organisation
Abeona Therapeutics Inc.
Duration of Study in the UK
0 years, 1 months, 1 days
Research summary
This study consists of a single clinical evaluation (no study drug is administered) in which the child will undergo a medical history review, a physical exam and a blood sample will be collected. The study population consists of children with Mucopolysaccharidosis type III (MPS III) who are potential candidates for the clinical trials ABT-001 and ABT-002 sponsored by Abeona Therapeutics. \nMucopolysaccharidosis III (MPS III or Sanfilippo syndrome) is a group of lysosomal storage diseases with an estimated incidence of 1 in 70,000 births and caused by the deficiency of enzymes involved in the metabolism of the glycosaminoglycan (GAG) called heparan sulphate. MPS III is caused by the build-up of these GAGs in many parts of the body. MPS III is divided into types IIIA, IIIB, IIIC, and IIID, which are distinguished by their genetic cause. These are severe progressive diseases mainly affecting the brain and spinal cord. \nThere is currently no treatment available for the disease. Gene therapy may offer a new treatment therapy option for MPS III. Adeno-associated virus (AAV) is a type of virus that has promising features as a vector (carrier to deliver the genetic material to the cell) for gene therapy. However some individuals may show pre-existing immunity to adeno-associated viruses (AAVs) and these subjects may be less likely to benefit from AAV vector-based therapies. The main purpose of this study is to provide information in regard to a child’s pre-existing immunity against adeno-associated vector serotype 9 (AAV9) by measuring total antibodies against AAV9 in their blood. Identification of children with pre-existing AAV immunity may be an important consideration for the design of these current and future clinical trials.\nThis study will be conducted at up to 10 centres worldwide with 40-50 children taking part. For the UK these patients are not be expected older than 15 years of age.\n
REC name
South Central - Oxford B Research Ethics Committee
REC reference
19/SC/0332
Date of REC Opinion
3 Sep 2019
REC opinion
Further Information Favourable Opinion