Sanfilippo type A syndrome (also named Mucopolysaccharidosis Type A) MedDRA version: 12.1 Level: LLT Classification code 10056918 Term: Sanfilippo's syndrome
Conditions
Interventions
Product Name: AAVrh.10-hMPSIIIA
Product Code: SAF-301
Pharmaceutical Form: Solution for injection
Sponsors
SANFILIPPO Therapeutics SAS
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: • Age: 18 (eighteen) months to end of 6 (six) years • Onset of clinical manifestations related to MPSIIIA during the first 5 years of life • SGSH activity in peripheral blood cell and/or cultured fibroblast extracts of less than 10% of controls. • Patient affiliated to the social security • Family understanding the procedure and the informed consent • Signed informed consent • Vital laboratory parameters within normal range Are the trial subjects under 18? yes Number of subjects for this age range: F.1.2 Adults (18-64 years) no F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: • Presence of brain atrophy on pre-inclusion MRI judged on a cortico-dural distance of more than 1cm • No independent walking (Ability to walk without help) • Any condition that would contraindicate permanently anaesthesia • Any other permanent medical condition not related to MPSIIIA • Any vaccination 1 month before treatment • Receipt of aspirin within one month • Any medication aiming at modifying the natural course of MPSIIIA given during the 6 months before vector injection • Any condition that would contraindicate treatment with Prograf®, Cellcept® and Solupred®
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To assess the tolerance and safety associated to the proposed treatment through a one-year follow up.;Secondary Objective: To collect data to define exploratory tests that could become evaluation criteria for further clinical phase III efficacy studies (Brain MRI, neuro- and psychological tests and biological markers);Primary end point(s): The safety parameters that will be evaluated are: • Clinical parameters: - during the 15 days after vector injection: fever (>38°C), seizure (anytype), headache (grade 3 or more on the pain scale), abnormal somnolence or lethargy, any new unexpected neurological symptoms - during the one year follow-up: any infectious disease, any new neurological symptom, any liver, spleen or lymph node enlargement, any aphtous lesion, any chronic respiratory symptoms, any abnormal bleeding or paetechia - after the one year protocol: any unexpected neurological or extra-neurological symptoms including malignancies. • Radiological parameters: - on the initial CT scan after surgery: any sign of bleeding - on subsequent MRI: any hypersignal on T2 weighted images or diffusion images that are not at the points of injection, any necrotic area evaluated through T1-weighted and diffusion imaging as well as modification of lipids in spectroscopy • Biological parameters: Parameters listed in sections Haematology and Biochemistry §3.2.2.3. In particular: anemia (X3 normal for transaminases). | — |
Countries
France
Outcome results
None listed