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Intracerebral Gene Therapy for Sanfilippo Type A Syndrome

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
LYSOGENE
ClinicalTrials.gov Identifier:
NCT01474343
First received: November 10, 2011
Last updated: May 5, 2014
Last verified: May 2014

November 10, 2011
May 5, 2014
August 2011
May 2013   (final data collection date for primary outcome measure)
Tolerance and safety [ Time Frame: during the one year follow-up ] [ Designated as safety issue: Yes ]

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)
Same as current
Complete list of historical versions of study NCT01474343 on ClinicalTrials.gov Archive Site
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 ] [ Designated as safety issue: No ]
  • Brain MRI
  • Neurocognitive/behavioral tests
  • Biological markers on blood, urine and CSF
Same as current
Not Provided
Not Provided
 
Intracerebral Gene Therapy for Sanfilippo Type A Syndrome
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.

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.

Not Provided
Interventional
Phase 1
Phase 2
Endpoint Classification: Safety Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
  • Mucopolysaccharidosis Type III A
  • Sanfilippo Disease Type A
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.
Experimental: SAF-301
Intervention: Genetic: SAF-301
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Completed
4
May 2013
May 2013   (final data collection date for primary outcome measure)

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®
Both
18 Months to 6 Years
No
Contact information is only displayed when the study is recruiting subjects
France
 
NCT01474343
P1-SAF-301
Yes
LYSOGENE
LYSOGENE
Not Provided
Not Provided
LYSOGENE
May 2014

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