Intracerebral Gene Therapy for Sanfilippo Type A Syndrome

This study is ongoing, but not recruiting participants.

Sponsor:

Information provided by (Responsible Party):

LYSOGENE

ClinicalTrials.gov Identifier:

NCT01474343

First received: November 10, 2011

Last updated: May 30, 2012

Last verified: May 2012

History of Changes

Tracking Information
First Received Date ^ICMJE	November 10, 2011
Last Updated Date	May 30, 2012
Start Date ^ICMJE	August 2011
Estimated Primary Completion Date	January 2014 (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 ] [ 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)
Original Primary Outcome Measures ^ICMJE	Same as current
Change History	Complete list of historical versions of study NCT01474343 on ClinicalTrials.gov Archive Site
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 ] [ Designated as safety issue: No ] Brain MRI Neurocognitive/behavioral tests Biological markers on blood, urine and CSF
Original Secondary Outcome Measures ^ICMJE	Same as current
Current Other Outcome Measures ^ICMJE	Not Provided
Original Other Outcome Measures ^ICMJE	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	Phase 1 Phase 2
Study Design ^ICMJE	Endpoint Classification: Safety Study Intervention Model: Single Group Assignment Masking: 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 Arm (s)	Not Provided
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	Active, not recruiting
Estimated Enrollment ^ICMJE	4
Estimated Completion Date	January 2014
Estimated Primary Completion Date	January 2014 (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®
Gender	Both
Ages	18 Months to 6 Years
Accepts Healthy Volunteers	No
Contacts ^ICMJE	Contact information is only displayed when the study is recruiting subjects
Location Countries ^ICMJE	France

Administrative Information
NCT Number ^ICMJE	NCT01474343
Other Study ID Numbers ^ICMJE	P1-SAF-301
Has Data Monitoring Committee	Yes
Responsible Party	LYSOGENE
Study Sponsor ^ICMJE	LYSOGENE
Collaborators ^ICMJE	Not Provided
Investigators ^ICMJE	Not Provided
Information Provided By	LYSOGENE
Verification Date	May 2012
^ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP

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