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

NCT02053064 · Status: COMPLETED · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 4

Last updated 2017-06-20

No results posted yet for this study

Summary

P2-SAF-301 is an open-label interventional study without administration of investigational product, evaluating the long-term safety and tolerability of intracerebral SAF-301 previously administered to 4 patients with Sanfilippo type A syndrome.

The primary objective is to collect additional safety and tolerability data on intracerebral SAF-301 previously administered to 4 patients with Sanfilippo type A syndrome.

The secondary objective is to further collect data to assess the effects of SAF-301 on neurological and psychological status, and potential biological markers.

Conditions

  • Mucopolysaccharidosis Type III A
  • Sanfilippo Disease Type A

Interventions

GENETIC

SAF-301

Sponsors & Collaborators

  • LYSOGENE

    lead INDUSTRY

Principal Investigators

  • Kumaran DEIVA, Dr · AP-HP Hôpital Bicêtre

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2013-05-31
Primary Completion
2017-05-31
Completion
2017-06-30

Countries

  • France

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT02053064 on ClinicalTrials.gov