CompletedPHASE1, PHASE2NCT02053064

Long-term Follow-up of Sanfilippo Type A Patients Treated by Intracerebral SAF-301 Gene Therapy

Studying Mucopolysaccharidosis type 3

Last synced from ClinicalTrials.gov May 4, 2026

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Key facts

Sponsor: LYSOGENE
Principal Investigator: Kumaran DEIVA, Dr
AP-HP Hôpital Bicêtre
Intervention: SAF-301(genetic)
Enrollment: 4 enrolled
Eligibility: All sexes
Timeline: 2013 – 2017

Study locations (1)

Hôpitaux Universitaires Paris Sud (Bicêtre), Le Kremlin-Bicêtre, France

Primary source

Recruitment status, site addresses, contacts, and full eligibility criteria can change between syncs. Always verify with the trial team before planning travel or treatment.

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