NCT02053064

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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
4

participants targeted

Target at below P25 for phase_1

Timeline
Completed

Started May 2013

Longer than P75 for phase_1

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

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Study Timeline

Key milestones and dates

Study Start

First participant enrolled

May 1, 2013

Completed
9 months until next milestone

First Submitted

Initial submission to the registry

January 16, 2014

Completed
18 days until next milestone

First Posted

Study publicly available on registry

February 3, 2014

Completed
3.2 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

May 1, 2017

Completed
1 month until next milestone

Study Completion

Last participant's last visit for all outcomes

June 1, 2017

Completed
Last Updated

June 20, 2017

Status Verified

June 1, 2017

Enrollment Period

4 years

First QC Date

January 16, 2014

Last Update Submit

June 19, 2017

Conditions

Mucopolysaccharidosis Type III ASanfilippo Disease Type A

Keywords

Neurodegenerative diseasesNervous systems diseasesGenetic diseases, InbornMetabolic diseasesGene therapyAdeno Associated Virus (AAV)

Outcome Measures

Primary Outcomes (1)

  • Checking of adverse events

    until 60 months after SAF-301 administration

Secondary Outcomes (3)

  • 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

  • Information to further evaluation of immune response

    5 years after the injection of SAF-301

Study Arms (1)

SAF-301

EXPERIMENTAL
Genetic: SAF-301

Interventions

SAF-301GENETIC
SAF-301

Eligibility Criteria

Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • 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.

You may not qualify if:

  • Participation in any other clinical trial.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Hôpitaux Universitaires Paris Sud (Bicêtre)

Le Kremlin-Bicêtre, 94275, France

Location

MeSH Terms

Conditions

Mucopolysaccharidosis IIINeurodegenerative DiseasesGenetic Diseases, InbornMetabolic Diseases

Condition Hierarchy (Ancestors)

MucopolysaccharidosesCarbohydrate Metabolism, Inborn ErrorsMetabolism, Inborn ErrorsCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesLysosomal Storage DiseasesMucinosesConnective Tissue DiseasesSkin and Connective Tissue DiseasesNutritional and Metabolic DiseasesNervous System Diseases

Study Officials

  • Kumaran DEIVA, Dr

    AP-HP Hôpital Bicêtre

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

January 16, 2014

First Posted

February 3, 2014

Study Start

May 1, 2013

Primary Completion

May 1, 2017

Study Completion

June 1, 2017

Last Updated

June 20, 2017

Record last verified: 2017-06

Locations

FR(1)