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AAVance pivotal phase gene transfer clinical trial for Mucopolysaccharidosis Type IIIA delivering LYS-SAF302

What is the purpose of this study ?

Lysogene is currently recruiting for its phase II/III clinical trial in MPS IIIA. AAVance is a single-arm trial evaluating the safety and effectiveness of a one-time delivery of a recombinant adeno-associated virus vector rh.10 carrying the N-sulfoglucosamine sulfohydrolase (SGSH) gene. The goal of the therapy is to show improved or stabilized neurodevelopmental status of MPS IIIA patients by allowing the brain cells to secrete the missing enzyme. The trial will enroll 20 patients at eight sites in the U.S. and Europe.

Trial at a glance

ConditionStudy typeClinical sites
MPS IIIAInterventionalFrance, Germany, Netherlands, U.K. and U.S
EnrollmentStart dateEnd dateStudy identifierPhase
20December 2018January Identifier :
Phase II/III

What next :

  • Patients are eligible to participate from a variety of geographical locations.
  • Patients recruited to the trial will need to relocate to one of the clinical trial sites for an initial period of a few weeks. Additional visits to the clinical site, for a day or two, will be planned every 3-6 months. 
  • Face-to-face meetings between families and the investigator at the clinical trial site, will take place before formal inclusion.
  • An information letter and informed consent document, in the parents’ own language, will be given to parents before inclusion in the study.
  • Arrangements for travel to clinical trial sites will be made through MPS PACT. They will provide a safe and independent logistical resource to families of children recruited to the trial. This resource will continue to support families throughout the time their children are on the clinical trial.
  • If you are interested in participating and would like to contact someone, please email:


Long-term Follow-up of MPS IIIA Patients Treated by Intracerebral LYS-SAF301 Gene Therapy

What is the purpose of this study?

This is an open-label study evaluating the long-term safety and tolerability of intracerebral LYS-SAF301 previously administered to 4 patients with Sanfilippo type A syndrome. The primary objective is to collect additional safety and tolerability data on intracerebral LYS-SAF301 previously administered to 4 patients with Sanfilippo type A syndrome. The secondary objective is to further collect data to assess the effects of LYS-SAF301 on neurological and psychological status and potential biological markers

Trial at a glance

ConditionStudy typeClinical sites
MPS IIIALong term follow-upFrance
EnrollmentStart dateEnd dateStudy identifierPhase
4May 2013July Identifier :
Phase I/II



Observational study of patients with Mucopolysaccharidosis Type IIIA

What is the purpose of this study?

Lysogene has begun a multi-national observational study (SAMOS) as it prepares to initiate its pivotal clinical trial for the treatment of Sanfilippo Type A, also known as mucopolysaccharidosis IIIA (MPS IIIA). This is an observational study, so no experimental drug will be given. Patients enrolled will have access to regularly scheduled monitoring and follow-up visits. The information gained from this study will enable optimal trial design, greater understanding of disease progression and better predictions of future therapeutic effects.

Trial at a glance

ConditionStudy typeClinical sites
MPS IIIANon interventionalFrance, UK, Germany, the Netherlands and Brazil.
EnrollmentStart dateEnd dateStudy identifier
Up to 25May 2016May Identifier :

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