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

June 19, 2017 updated by: LYSOGENE

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.

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

4

Phase

  • Phase 2
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

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

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: SAF-301
Genetic: SAF-301

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Checking of adverse events
Time Frame: until 60 months after SAF-301 administration
until 60 months after SAF-301 administration

Secondary Outcome Measures

Outcome Measure
Time Frame
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
5 years after the injection of SAF-301
Information on changes in potential biomarkers of the disease
Time Frame: 5 years after the injection of SAF-301
5 years after the injection of SAF-301
Information to further evaluation of immune response
Time Frame: 5 years after the injection of SAF-301
5 years after the injection of SAF-301

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

LYSOGENE

Investigators

  • Principal Investigator: Kumaran DEIVA, Dr, AP-HP Hôpital Bicêtre

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

May 1, 2013

Primary Completion (Actual)

May 1, 2017

Study Completion (Actual)

June 1, 2017

Study Registration Dates

First Submitted

January 16, 2014

First Submitted That Met QC Criteria

January 31, 2014

First Posted (Estimate)

February 3, 2014

Study Record Updates

Last Update Posted (Actual)

June 20, 2017

Last Update Submitted That Met QC Criteria

June 19, 2017

Last Verified

June 1, 2017

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • P2-SAF-301

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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