Back to resultsLong-term Follow-up of Sanfilippo Type A Patients Treated by Intracerebral SAF-301 Gene Therapy
Primary Purpose
Mucopolysaccharidosis Type III A, Sanfilippo Disease Type A
Status
Completed
Phase
Phase 1
Locations
France
Study Type
Interventional
Intervention
SAF-301
Sponsored by
About this trial
This is an interventional treatment trial for Mucopolysaccharidosis Type III A focused on measuring Neurodegenerative diseases, Nervous systems diseases, Genetic diseases, Inborn, Metabolic diseases, Gene therapy, Adeno Associated Virus (AAV)
Eligibility Criteria
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.
Sites / Locations
- Hôpitaux Universitaires Paris Sud (Bicêtre)
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
SAF-301
Arm Description
Outcomes
Primary Outcome Measures
Checking of adverse events
Secondary Outcome Measures
Information on neurological and cognitive changes based on clinical status, standardized neurocognitive and behavioral assessments
Information on changes in potential biomarkers of the disease
Information to further evaluation of immune response
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT02053064
Brief Title
Long-term Follow-up of Sanfilippo Type A Patients Treated by Intracerebral SAF-301 Gene Therapy
Official 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.
Study Type
Interventional
2. Study Status
Record Verification Date
June 2017
Overall Recruitment Status
Completed
Study Start Date
May 2013 (undefined)
Primary Completion Date
May 2017 (Actual)
Study Completion Date
June 2017 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
LYSOGENE
4. Oversight
Data Monitoring Committee
Yes
5. Study Description
Brief 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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
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)
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1, Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
4 (Actual)
8. Arms, Groups, and Interventions
Arm Title
SAF-301
Arm Type
Experimental
Intervention Type
Genetic
Intervention Name(s)
SAF-301
Primary Outcome Measure Information:
Title
Checking of adverse events
Time Frame
until 60 months after SAF-301 administration
Secondary Outcome Measure Information:
Title
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
Title
Information on changes in potential biomarkers of the disease
Time Frame
5 years after the injection of SAF-301
Title
Information to further evaluation of immune response
Time Frame
5 years after the injection of SAF-301
10. Eligibility
Sex
All
Accepts Healthy Volunteers
No
Eligibility Criteria
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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Kumaran DEIVA, Dr
Organizational Affiliation
AP-HP Hôpital Bicêtre
Official's Role
Principal Investigator
Facility Information:
Facility Name
Hôpitaux Universitaires Paris Sud (Bicêtre)
City
Le Kremlin-Bicêtre
ZIP/Postal Code
94275
Country
France
12. IPD Sharing Statement
Learn more about this trial
Long-term Follow-up of Sanfilippo Type A Patients Treated by Intracerebral SAF-301 Gene Therapy
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