A gene therapy human clinical trial for Sanfilippo B is scheduled to begin in January of 2012 at Pasteur Institute in Paris, France.

Inclusion criteria

1. Age: 18 months to the end of the 5th year
2. Onset of clinical manifestations related to MPSIIIB during the first 5 years of life
3. NaGlu activity in peripheral blood cell and/or cultured fibroblast extracts of less than 10% of controls
4. Patient affiliated to a social security regimen
5. Family understanding the procedure and the informed consent
6. Vital laboratory parameters within normal range

Sanfilippo disease is a rare disease and so the countries of recruitment will depend on the availabilities of the patient at the time of recruitment. There will be one study centre in France. [Note: however, only European patients are being considered for inclusion.]

Participants – exclusion criteria

1. Presence of brain atrophy on pre-inclusion MRI judged on a cortico-dural distance of more than 1 cm
2. Any condition that would contraindicate permanently anaesthesia
3. Any other permanent medical condition not related to MPSIIIB
4. No independent walking (Ability to walk without help)
5. Any vaccination 1 month before vector injection
6. Receipt of aspirin within one month
7. Any medication aiming at modifying the natural course of MPSIIIB given during the 6 months before vector injection

Pasteur Institute

The sponsoring institution’s website is Pasteur Institute