Long-term Follow-up of Sanfilippo Type A Patients Treated... | NCT02053064

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

NCT02053064

Sponsor

LYSOGENE

Status

Completed

Last Update Posted

Jun 20, 2017Actual

Enrollment

4Actual

Phase

Phase 1Phase 2

Conditions

Mucopolysaccharidosis Type III A
Sanfilippo Disease Type A

Interventions

SAF-301

Countries

France

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

Status Module

Record Verification Date

Jun 2017

Overall Recruitment Status or Expanded Access Status

Completed

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

May 2013

Primary Completion Date

May 2017Actual

Completion Date

Jun 2017Actual

First Submitted Date

Jan 16, 2014

First Submission Date that Met QC Criteria

Jan 31, 2014

First Posted Date

Feb 3, 2014Estimated

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Jun 19, 2017

Last Update Posted Date

Jun 20, 2017Actual

Oversight Module

Has Data Monitoring Committee (DMC)

Yes

Is FDA Regulated Drug

Not provided

Is FDA Regulated Device

Not provided

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

Not provided

FDAAA801 Violation

Not provided

Description Module

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.

Detailed Description

Not provided

Design Module

Study Type

Interventional

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Not provided

Target Follow-Up Duration

Not provided

Phases

Phase 1Phase 2

Interventional Study Design

Allocation

Biospecimen

No data available

No data is available for this block.

Enrollment

4Actual

Label	Type	Description	Intervention Names
SAF-301	Experimental		Genetic: SAF-301

Name	Type	Description	Arm Group Labels	Other Names
SAF-301	Genetic		SAF-301

Outcomes Module

Primary Outcomes

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

Secondary Outcomes

Measure	Description	Time Frame
Information on neurological and cognitive changes based on clinical status, standardized neurocognitive and behavioral assessments		5 years after the injection of SAF-301
Information on changes in potential biomarkers of the disease		5 years after the injection of SAF-301

Other Outcomes

Not provided

Eligibility Module

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.

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

Not provided

Maximum Age

Not provided

Standard Ages

ChildAdultOlder Adult

Study Population

Not provided

Sampling Method

Not provided

Contacts/Locations Module

Central Contacts

Not provided

Overall Officials

Name	Affiliation	Role
Kumaran DEIVA, Dr	AP-HP Hôpital Bicêtre	Principal Investigator

Locations

Facility	Status	City	State	ZIP	Country	Contacts
Hôpitaux Universitaires Paris Sud (Bicêtre)		Le Kremlin-Bicêtre		94275	France

Condition Browse Module

MeSH Terms

ID	Term
D009084	Mucopolysaccharidosis III
D019636	Neurodegenerative Diseases
D030342	Genetic Diseases, Inborn
D008659	Metabolic Diseases

Ancestor Terms

ID	Term
D009083	Mucopolysaccharidoses
D002239	Carbohydrate Metabolism, Inborn Errors
D008661	Metabolism, Inborn Errors
D009358	Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Browse Leaves

Not provided

Browse Branches

Not provided