Intracerebral Gene Therapy for Sanfilippo Type A Syndrome
This study is ongoing, but not recruiting participants.
Sponsor:
LYSOGENE
Information provided by (Responsible Party):
LYSOGENE
ClinicalTrials.gov Identifier:
NCT01474343
First received: November 10, 2011
Last updated: May 30, 2012
Last verified: May 2012
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Purpose
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 | Intervention | Phase |
|---|---|---|
|
Mucopolysaccharidosis Type III A Sanfilippo Disease Type A |
Genetic: SAF-301 |
Phase 1 Phase 2 |
| Study Type: | Interventional |
| Study Design: | Endpoint Classification: Safety Study Intervention Model: Single Group Assignment Masking: 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. |
Resource links provided by NLM:
Further study details as provided by LYSOGENE:
Primary Outcome Measures:
- 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)
Secondary Outcome Measures:
- 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
| Estimated Enrollment: | 4 |
| Study Start Date: | August 2011 |
| Estimated Study Completion Date: | January 2014 |
| Estimated Primary Completion Date: | January 2014 (Final data collection date for primary outcome measure) |
Intervention Details:
-
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.
Eligibility| Ages Eligible for Study: | 18 Months to 6 Years |
| Genders Eligible for Study: | Both |
| 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®
Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: 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
More Information
Additional Information:
No publications provided
| Responsible Party: | LYSOGENE |
| ClinicalTrials.gov Identifier: | NCT01474343 History of Changes |
| Other Study ID Numbers: | P1-SAF-301 |
| Study First Received: | November 10, 2011 |
| Last Updated: | May 30, 2012 |
| Health Authority: | France: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis) |
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 |
ClinicalTrials.gov processed this record on May 22, 2013