Intracerebral Gene Therapy in Children With Sanfilippo Type B Syndrome

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

COMPLETED

Clinical Phase

PHASE1/PHASE2

Total Enrollment

4 participants

Study Classification

INTERVENTIONAL

Study Start Date

2013-09-17

Study Completion Date

2019-11-27

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

This is an open-label, phase I/II study of intra-cerebral administration of adenovirus-associated viral vector containing the human NAGLU cDNA to children suffering from Sanfilippo type B syndrome.

Related Clinical Trials

Explore similar clinical trials based on study characteristics and research focus.

Intracerebral Gene Therapy for Sanfilippo Type A Syndrome

NCT01474343

Mucopolysaccharidosis Type III A Sanfilippo Disease Type A

COMPLETED PHASE1/PHASE2

Long-term Follow-up of Sanfilippo Type A Patients Treated by Intracerebral SAF-301 Gene Therapy

NCT02053064

Mucopolysaccharidosis Type III A Sanfilippo Disease Type A

COMPLETED PHASE1/PHASE2

Safety Study of a Gene Transfer Vector for Children With Late Infantile Neuronal Ceroid Lipofuscinosis

NCT00151216

Batten Disease Late Infantile Neuronal Ceroid Lipofuscinosis

COMPLETED PHASE1

A Open Label Study in Previously Studied, SBC-103 Treatment Naïve MPS IIIB Subjects to Investigate the Safety, Pharmacokinetics, and Pharmacodynamics/Efficacy of SBC-103 Administered Intravenously

NCT02618512

Mucopolysaccharidosis III, Type B (MPS IIIB) Sanfilippo B

TERMINATED PHASE1/PHASE2

Safety Study of a Gene Transfer Vector (Rh.10) for Children With Late Infantile Neuronal Ceroid Lipofuscinosis (LINCL)

NCT01161576

Batten Disease Late-Infantile Neuronal Ceroid Lipofuscinosis

COMPLETED PHASE1

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

This is an open-label, phase I/II study of intra-cerebral administration of adenovirus-associated viral vector containing the human NAGLU cDNA to children suffering from Sanfilippo type B syndrome.

Four patients, 18 months up to the 5th birthday, have been included.

The inclusion period will be 8 to 12 months. The duration of follow-up for each patient is 1 year post-surgery. The duration of the first extension phase is 18 months. The duration of the second extension phase is 36 months. Therefore, the maximum time of the follow-up will be 66 months

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Sanfilippo Syndrome B

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

rAAV2/5-hNAGLU

Each patient will receive 960 µL of vector suspension. The vector suspension will be deposited simultaneously at 16 sites, each deposit containing 2.4x 1011 vg (4x1012 vg in total).

Group Type EXPERIMENTAL

rAAV2/5-hNAGLU

Intervention Type DRUG

one-time brain intraparenchymal gene therapy dose

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

rAAV2/5-hNAGLU

one-time brain intraparenchymal gene therapy dose

Intervention Type DRUG

Other Intervention Names

Discover alternative or legacy names that may be used to describe the listed interventions across different sources.

AAV5

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

Inclusion Criteria

* Age: 18 months up to 60 months (5th birthday);
* Onset of clinical manifestations related to mucopolysaccharidosis type IIIB (MPSIIIB);
* NAGLU activity in peripheral blood cell and/or cultured fibroblast extracts of less than 10% of controls;
* Patient affiliated to, or covered by a French social security regimen, or European patients with European Health Insurance Card;
* Family understanding the procedure and the informed consent;
* Signed informed consent by both parents or legal representative;
* Vital laboratory parameters within normal range.

Exclusion Criteria

* Presence of brain atrophy on baseline MRI judged on a cortico-dural distance of more than 0.6 cm;
* Any condition that would contraindicate general anesthesia;
* Any other permanent medical condition not related to MPSIIIB that could contraindicate the study participation;
* No independent walking (ability to walk without help);
* Any medication aiming at modifying the natural course of MPSIIIB given during the 6 months before vector injection (sleep and mood regulators are accepted);
* Any condition that would contraindicate treatment with Modigraf®, Cellcept® and prednisolone (Solupred® and Solumedrol®).

Minimum Eligible Age

18 Months

Maximum Eligible Age

60 Months

Eligible Sex

ALL

Accepts Healthy Volunteers

No