Saves the questions and what to expect into your notes, next to the visit they belong to.
Keep this study
Lead
LYSOGENE
The study is interventional, single arm and multi-center. Evolution under treatment will be compared to expected natural evolution based on natural history studies.
Age
0–any
Sex
ALL
Healthy volunteers
Not accepted
You may be eligible if
Documented MPS IIIA diagnosis based on genotyping confirming the SGSH gene mutations
Cognitive DQ score on BSID-III: 50% and above
You may not be if
Homozygous for the S298P mutation or non-classical severe form of MPS IIIA, based on investigator's judgement.
Participation in another gene or cell therapy clinical trial.
Past use of SGSH enzyme replacement therapy for a period exceeding 3 months. A washout period of at least 2 months is required prior to screening.
Current participation in a clinical trial of another investigational medicinal product.
History of bleeding disorder or current use of medications that, in the opinion of the investigator, place them at risk of bleeding following surgery.
Any condition that would contraindicate treatment with immunosuppressants such as tacrolimus, mycophenolate mofetil or steroids.
Clareo Health | Study of AAVrh10-h.SGSH Gene Therapy in Patients With Mucopolysaccharidosis Type IIIA (MPS IIIA)
