Intracerebral Gene Therapy for Sanfilippo Type A Syndrome

• Register: ClinicalTrials.gov
• Last refreshed on: 19 February 2015
• Main ID: NCT01474343
• Date of registration: 10/11/2011
• Prospective Registration: No
• Primary sponsor: LYSOGENE
• Public title: Intracerebral Gene Therapy for Sanfilippo Type A Syndrome
• Scientific title: An Open-label, Single Arm, Monocentric, Phase I/II Clinical Study of Intracerebral Administration of Adeno-associated Viral Vector Serotype 10 Carrying the Human SGSH and SUMF1 cDNAs for the Treatment of Sanfilippo Type A Syndrome.
• Date of first enrolment: August 2011
• Target sample size: 4
• Recruitment status: Completed
• URL: http://clinicaltrials.gov/show/NCT01474343
• Study type: Interventional
• Study design: Endpoint Classification: Safety Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
• Phase: Phase 1/Phase 2

Countries of recruitment

France

Key inclusion & exclusion 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 French social security or assimilated regimens

- Family understanding the procedure and the informed consent

- Signed informed consent

- Vital laboratory parameters within normal range

Exclusion Criteria:

- Presence of brain atrophy on 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 investigational drug administration

- Intake 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®, Modigraf®, Cellcept®
and Solupred®



Age minimum: 18 Months
Age maximum: 6 Years
Gender: Both

Health Condition(s) or Problem(s) studied

Sanfilippo Disease Type A

Mucopolysaccharidosis Type III A

Intervention(s)

Genetic: SAF-301

Primary Outcome(s)

Tolerance and safety [Time Frame: during the one year follow-up]

Secondary Outcome(s)

To collect data to define exploratory tests that could become evaluation criteria for further clinical phase III efficacy studies [Time Frame: during the one year follow-up]

Secondary ID(s)

P1-SAF-301

Source(s) of Monetary Support

Please refer to primary and secondary sponsors