Mucopolysaccharidosis Type III A, Sanfilippo Disease Type A
Conditions
Keywords
Neurodegenerative diseases, Nervous systems diseases, Genetic diseases, Inborn, Metabolic diseases, Gene therapy, Adeno Associated Virus (AAV)
Brief summary
P2-SAF-301 is an open-label interventional study without administration of investigational product, evaluating the long-term safety and tolerability of intracerebral SAF-301 previously administered to 4 patients with Sanfilippo type A syndrome. The primary objective is to collect additional safety and tolerability data on intracerebral SAF-301 previously administered to 4 patients with Sanfilippo type A syndrome. The secondary objective is to further collect data to assess the effects of SAF-301 on neurological and psychological status, and potential biological markers.
Interventions
GENETICSAF-301
Sponsors
LYSOGENE
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Healthy volunteers
No
Inclusion criteria
* Patients having completed the previous phase I/II study (P1-SAF-301), * Family understanding the follow-up procedures and the informed consent, * Patient's parents having signed the informed consent form.
Exclusion criteria
* Participation in any other clinical trial.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Checking of adverse events | until 60 months after SAF-301 administration |
Secondary
| Measure | Time frame |
|---|---|
| Information on neurological and cognitive changes based on clinical status, standardized neurocognitive and behavioral assessments | 5 years after the injection of SAF-301 |
| Information on changes in potential biomarkers of the disease | 5 years after the injection of SAF-301 |
| Information to further evaluation of immune response | 5 years after the injection of SAF-301 |
Countries
France
Outcome results
None listed