Intracerebral Gene Therapy for Sanfilippo Type A Syndrome

NCT ID: NCT01474343

Last Updated: 2014-05-06

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE1/PHASE2
• Total Enrollment: 4 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2011-08-31
• Study Completion Date: 2013-05-31

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.

Conditions

Mucopolysaccharidosis Type III A; Sanfilippo Disease Type A

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

SAF-301

Group Type: EXPERIMENTAL

SAF-301

Intervention Type: GENETIC

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.

Interventions

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.

Intervention Type: GENETIC

Eligibility 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®

• Minimum Eligible Age: 18 Months
• Maximum Eligible Age: 6 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

LYSOGENE

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

Hôpital Bicêtre - Assistance Publique des Hôpitaux de Paris

Le Kremlin-Bicêtre, , France

Hôpital Necker, Assistance Publique des Hôpitaux de Paris

Paris, , France

Countries

France

Other Identifiers

P1-SAF-301

Identifier Type: -

Identifier Source: org_study_id