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CompletedNCT02053064Updated Jun 20, 2017

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

A Phase 1/2 interventional study of SAF-301 in Mucopolysaccharidosis Type III A and Sanfilippo Disease Type A, sponsored by LYSOGENE. Completed at 1 site in France. Per ClinicalTrials.gov, last updated 2017-06-20.

Sponsored by LYSOGENE · Phase 1/2, Interventional, and Treatment

Phase
Phase 1/2
Study type
Interventional
Enrollment
4
Allocation
Not applicable
Sex
All
01

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.

02

Conditions studied

  • Mucopolysaccharidosis Type III A
  • Sanfilippo Disease Type A

Keywords

  • Neurodegenerative diseases
  • Nervous systems diseases
  • Genetic diseases, Inborn
  • Metabolic diseases
  • Gene therapy
  • Adeno Associated Virus (AAV)
03

In context

Mucopolysaccharidoses

145 studies on the registry are indexed under Mucopolysaccharidoses; 11 are open to participants now.

This study's enrollment of 4 is below the median of 15 across 80 interventional studies indexed under Mucopolysaccharidoses.

Browse Mucopolysaccharidoses studies →

Lead sponsor

LYSOGENE is the lead sponsor of 6 studies on the registry; none are open to participants now.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
Child (0–17), Adult (18–64), Older adult (65+)
Sexes eligible
All
Accepts healthy volunteers
No

Inclusion criteria

  • Patients having completed the previous phase I/II study (P1-SAF-301),
  • Family understanding the follow-up procedures and the informed consent,
  • Patient's parents having signed the informed consent form.

Exclusion criteria

Exclusion Criteria:

  • Participation in any other clinical trial.
05

Study design

Phase
Phase 1 / Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrollment
4 participants (actual)

Study arms

  • Experimental
    SAF-301

    Genetic: SAF-301

Interventions

  • GeneticSAF-301
06

What researchers measure

Primary outcomes

  1. Checking of adverse events

    Time frame: until 60 months after SAF-301 administration

Secondary outcomes

  1. Information on neurological and cognitive changes based on clinical status, standardized neurocognitive and behavioral assessments

    Time frame: 5 years after the injection of SAF-301

  2. Information on changes in potential biomarkers of the disease

    Time frame: 5 years after the injection of SAF-301

  3. Information to further evaluation of immune response

    Time frame: 5 years after the injection of SAF-301

07

Study locations

1 site
  • Hôpitaux Universitaires Paris Sud (Bicêtre)
    Le Kremlin-Bicêtre, 94275, France
08

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Jun 20, 2017, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
09

Registry details

Key details

Study ID
NCT02053064
Lead sponsor
LYSOGENE
Responsible party
Sponsor
First posted
Feb 3, 2014
Start date
May 2013
Primary completion
May 2017
Completion
Jun 2017
Last update
Jun 20, 2017

Study contacts

Kumaran DEIVA, Dr
principal investigator · AP-HP Hôpital Bicêtre

Oversight

Data monitoring committee
Yes
View the source record on ClinicalTrials.gov ↗

Not currently enrolling

This study is completed, as verified in Jun 2017. You cannot join it, but the record below documents what was studied.

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