French biopharmaceutical firm Lysogene has completed patient enrolment for its Sanfilippo A multi-national observational study (SAMOS) to treat mucopolysaccharidosis type IIIA (MPS IIIA), also known as Sanfilippo A.
Designed to examine clinical changes in untreated patients, the international multi-centre trial enrolled a total of 23 patients across five countries.
The SAMOS trial is being conducted as a non-concurrent control group for a Phase II/III gene therapy trial, scheduled to begin in the first quarter of next year.
The firm’s gene therapy candidate for MPS IIIA includes an AAV vector serotype rh.10 that carries gene coding for N-Sulfoglucosamine Sulfohydrolase (SGSH).
Lysogene chief patient access officer Samantha Parker said: “Enrolment of the 23 children from five countries has been rapid, reflecting the strong interest from the key opinion leaders running our clinical sites and the network of patient associations to address the significant unmet needs in MPS IIIA.”
An international neurologist and neuropsychologist MPS IIIA expert group has been established by the firm to assess the trial.
The group established that cognitive assessment was the primary endpoint, measured using the Bayley scales of infant and toddler development, third edition (BSID-III), while the appropriate secondary endpoint measure was the Vineland adaptive behaviour scale, second edition (VABS-II).
Prior data from SAMOS showed progressive intellectual decline, hyperactivity and behaviour changes during cognitive age assessment with BSID-III in the first 15 patients aged three to eight.
A Phase I/II clinical trial and a five-year follow-up had been successfully completed by Lysogene in four MPS IIIA patients, with the results showing no adverse events during the treatment period.