Skip to main content
OpenTrials
Completed

NCT Number: NCT02053064

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

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.

Completed

Looking for future studies?

Notify Me

Key information

Conditions

Mucopolysaccharidosis Type III A Carbohydrate Metabolism, Inborn Errors Congenital, Hereditary, and Neonatal Diseases and Abnormalities Connective Tissue Diseases Genetic Diseases, Inborn Lysosomal Storage Diseases Metabolic Diseases Metabolism, Inborn Errors Mucinoses Mucopolysaccharidoses Mucopolysaccharidosis III Nervous System Diseases Neurodegenerative Diseases Nutritional and Metabolic Diseases Sanfilippo Disease Type A Skin and Connective Tissue Diseases

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1 / Phase 2

Primary location

Hôpitaux Universitaires Paris Sud (Bicêtre)

Le Kremlin-Bicêtre, 94275, France

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

  • Participation in any other clinical trial.

Treatment and study plan

SAF-301

Genetic

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

Sponsors and collaborators

Lead sponsor

LYSOGENE

Industry

Registry information

Official study title

Long-term Follow-up of Patients With Sanfilippo Type A Syndrome Who Have Previously Been Treated in the P1-SAF-301 Clinical Study Evaluating the Tolerability and Safety of the Intracerebral Administration of SAF-301.

Important dates

Study start
2013
Primary completion
2017
Study completion
2017
First posted
Feb 3, 2014
Registry last updated
Jun 20, 2017

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Related clinical trials

Published trials that share one or more normalized conditions with this study.