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Intracerebral Gene Therapy for Sanfilippo Type A Syndrome

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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

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.


Condition or disease Intervention/treatment Phase
Mucopolysaccharidosis Type III A Sanfilippo Disease Type A Genetic: SAF-301 Phase 1 Phase 2

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 4 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: 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.
Study Start Date : August 2011
Actual Primary Completion Date : May 2013
Actual Study Completion Date : May 2013


Arm Intervention/treatment
Experimental: SAF-301 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.




Primary Outcome Measures :
  1. 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)


Secondary Outcome Measures :
  1. 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



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Ages Eligible for Study:   18 Months to 6 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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®

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01474343


Locations
France
Hôpital Bicêtre - Assistance Publique des Hôpitaux de Paris
Le Kremlin Bicêtre, France, 94275
Hôpital Necker, Assistance Publique des Hôpitaux de Paris
Paris, France, 75015
Sponsors and Collaborators
LYSOGENE

Additional Information:
Responsible Party: LYSOGENE
ClinicalTrials.gov Identifier: NCT01474343     History of Changes
Other Study ID Numbers: P1-SAF-301
First Posted: November 18, 2011    Key Record Dates
Last Update Posted: May 6, 2014
Last Verified: May 2014

Keywords provided by LYSOGENE:
Neurodegenerative Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Metabolic Diseases
Gene Therapy
Adenovirus Associated Vector

Additional relevant MeSH terms:
Mucopolysaccharidoses
Mucopolysaccharidosis III
Carbohydrate Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Lysosomal Storage Diseases
Mucinoses
Connective Tissue Diseases
Metabolic Diseases