Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu
Trial record 1 of 1 for:    NCT01474343
Previous Study | Return to List | Next Study

Intracerebral Gene Therapy for Sanfilippo Type A Syndrome

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT01474343
Recruitment Status : Completed
First Posted : November 18, 2011
Last Update Posted : May 6, 2014
Sponsor:
Information provided by (Responsible Party):
LYSOGENE

Tracking Information
First Submitted Date  ICMJE November 10, 2011
First Posted Date  ICMJE November 18, 2011
Last Update Posted Date May 6, 2014
Study Start Date  ICMJE August 2011
Actual Primary Completion Date May 2013   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: November 15, 2011)
Tolerance and safety [ Time Frame: during the one year follow-up ]
Measured by
  • adverse events (by type and severity)
  • clinical parameters (fever, seizure, headache, abnormal somnolence or lethargy, any new neurological symptoms),
  • radiological parameters (on MRI, any sign of bleeding after surgery, any hypersignal on T2 weighted images or diffusion images that are not at the points of injection, and any necrotic area evaluated through T1-weighted and diffusion imaging as well as modification of lipids in spectroscopy)
  • biological parameters (in particular anemia, leucopenia, thrombopenia, liver dysfunction)
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: November 15, 2011)
To collect data to define exploratory tests that could become evaluation criteria for further clinical phase III efficacy studies [ Time Frame: during the one year follow-up ]
  • Brain MRI
  • Neurocognitive/behavioral tests
  • Biological markers on blood, urine and CSF
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Intracerebral Gene Therapy for Sanfilippo Type A Syndrome
Official Title  ICMJE An Open-label, Single Arm, Monocentric, Phase I/II Clinical Study of Intracerebral Administration of Adeno-associated Viral Vector Serotype 10 Carrying the Human SGSH and SUMF1 cDNAs for the Treatment of Sanfilippo Type A Syndrome.
Brief Summary

The clinical trial P1-SAF-301 is an open-label, single arm, monocentric, phase I/II clinical study evaluating the tolerance and the safety of intracerebral administration of adeno-associated viral vector serotype 10 carrying the human SGSH and SUMF1 cDNAs for the treatment of Sanfilippo type A syndrome The treatment plan consists on a direct injection of the investigational medicinal product SAF-301 to both sides of the brain through 6 image-guided tracks, with 2 deposits per track, in a single neurosurgical session.

The primary objective is to assess the tolerance and the safety associated to the proposed treatment through a one-year follow up.

The secondary objective is to collect data to define exploratory tests that could become evaluation criteria for further clinical phase III efficacy studies.

Four patients will be included in the clinical trial and will be followed during one year.

The enrollment and the follow-up of the patients will take place at Bicêtre Hospital. The Neurosurgery will be performed at Necker-Enfants Malades Hospital.

Safety will be evaluating on clinical, radiological and biological parameters.

Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Phase 2
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE
  • Mucopolysaccharidosis Type III A
  • Sanfilippo Disease Type A
Intervention  ICMJE Genetic: SAF-301
The treatment plan consists on a direct injection of the investigational medicinal product SAF-301 to both sides of the brain through 6 image-guided tracks, with 2 deposits per track, in a single neurosurgical session.
Study Arms  ICMJE Experimental: SAF-301
Intervention: Genetic: SAF-301
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: November 15, 2011)
4
Original Estimated Enrollment  ICMJE Same as current
Actual Study Completion Date  ICMJE May 2013
Actual Primary Completion Date May 2013   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  • Age: 18 (eighteen) months to end of 6 (six) years
  • Onset of clinical manifestations related to MPSIIIA during the first 5 years of life
  • SGSH activity in peripheral blood cell and / or cultured fibroblast extracts of less than 10% of controls.
  • Patient affiliated to the French social security or assimilated regimens
  • Family understanding the procedure and the informed consent
  • Signed informed consent
  • Vital laboratory parameters within normal range

Exclusion Criteria:

  • Presence of brain atrophy on inclusion MRI judged on a cortico-dural distance of more than 1cm
  • No independent walking (Ability to walk without help)
  • Any condition that would contraindicate permanently anaesthesia
  • Any other permanent medical condition not related to MPSIIIA
  • Any vaccination 1 month before investigational drug administration
  • Intake of aspirin within one month
  • Any medication aiming at modifying the natural course of MPSIIIA given during the 6 months before vector injection
  • Any condition that would contraindicate treatment with Prograf®, Modigraf®, Cellcept® and Solupred®
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Months to 6 Years   (Child)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE France
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT01474343
Other Study ID Numbers  ICMJE P1-SAF-301
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party LYSOGENE
Study Sponsor  ICMJE LYSOGENE
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account LYSOGENE
Verification Date May 2014

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP