Date: 2017-05-30
Type of
information: Completion of patient enrollment
phase: observational
Announcement: completion of patient enrollment
Company: Lysogene (France)
Product:
Action
mechanism:
Disease: mucopolysaccharidosis type IIIA (Sanfilippo B syndrome)
Therapeutic
area: Rare diseases - Genetic diseases
Country: Brazil, France, Germany, The Netherlands, UK
Trial
details:
- This is a multicenter, multinational, longitudinal, observational study in children aged up to and including 9 years, who have been diagnosed with MPS IIIA. The study will detail the natural course of MPS IIIA via standardized clinical, biochemical, neurocognitive, developmental, behavioral and imaging measures. This information is anticipated to inform the design and interpretation of future interventional studies. (NCT02746341)
Latest
news:
- • On May 30, 2017, Lysogene announced the completion of enrollment in its “Sanfilippo A Multinational Observational Study” (SAMOS). SAMOS is particularly important as there is currently no validated biomarker in MPS IIIA that reflects CNS disease progression and response to future therapy. SAMOS has therefore been designed to evaluate clinical change in untreated MPS IIIA patients. As agreed with the regulatory authorities, this international multi-center study is to function as a non-concurrent control group for the upcoming Lysogene Phase II/III pivotal gene therapy trial, scheduled to start during the first quarter of 2018.
- In designing SAMOS, Lysogene established the first international neurologist and neuropsychologist MPS
IIIA expert group. This group determined that the most scientifically rigorous and relevant primary endpoint was cognitive assessment using the Bayley Scales of Infant and Toddler Development, 3rd edition (BSID-III). The Vineland Adaptive Behavior Scale, 2nd edition (VABS-II) was determined to be the most appropriate as a secondary endpoint measure.
- • On March 28th, 2017, Lysogene announced baseline data from its Sanfilippo A Multi-national Observational Study (SAMOS). Data from this first international pivotal observational study was the topic of a poster presentation made at the 13th Annual WORLDSymposium™ in San Diego, Calif. SAMOS has been designed to evaluate the clinical progression in untreated MPS IIIA patients. As agreed with the regulatory authorities, this study is to function as a non-concurrent control for the upcoming Lysogene phase II/III pivotal gene therapy trial.
Is
general: Yes
