Study of AAVrh10-h.SGSH Gene Therapy in Patients With Mucopolysaccharidosis Type IIIA (MPS IIIA)

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

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Recruitment Status

UNKNOWN

Clinical Phase

PHASE2/PHASE3

Total Enrollment

20 participants

Study Classification

INTERVENTIONAL

Study Start Date

2018-12-17

Study Completion Date

2022-03-31

Brief Summary

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MPS IIIA is predominantly a central nervous system disease causing cognitive disability, progressive loss of acquired skills, behavioral and sleep disturbance. LYS-SAF302 is a gene therapy which is intended to deliver a functional copy of the SGSH gene to the brain. This is a phase 2-3 study to assess the efficacy in improving or stabilizing the neurodevelopmental state of MPS IIIA patients.

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Detailed Description

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The study is interventional, single arm and multi-center. Evolution under treatment will be compared to expected natural evolution based on natural history studies.

Conditions

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Mucopolysaccharidosis Type IIIA

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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AAV SGSH gene therapy (LYS-SAF302)

One-time intracerebral administration of adeno-associated viral vector serotype rh10 containing the human N-sulfoglucosamine sulfohydrolase (SGSH) cDNA.

Group Type EXPERIMENTAL

LYS-SAF302

Intervention Type DRUG

Treatment will involve direct injections of the investigational product into both sides of the brain through image-guided tracks, in a single neurosurgical session.

Interventions

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LYS-SAF302

Treatment will involve direct injections of the investigational product into both sides of the brain through image-guided tracks, in a single neurosurgical session.

Intervention Type DRUG

Other Intervention Names

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AAVrh10-h.SGSH

Eligibility Criteria

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Inclusion Criteria

* Documented MPS IIIA diagnosis based on genotyping confirming the SGSH gene mutations
* Cognitive DQ score on BSID-III: 50% and above

Exclusion Criteria

* Homozygous for the S298P mutation or non-classical severe form of MPS IIIA, based on investigator's judgement.
* Participation in another gene or cell therapy clinical trial.
* Past use of SGSH enzyme replacement therapy for a period exceeding 3 months. A washout period of at least 2 months is required prior to screening.
* Current participation in a clinical trial of another investigational medicinal product.
* History of bleeding disorder or current use of medications that, in the opinion of the investigator, place them at risk of bleeding following surgery.
* Any condition that would contraindicate treatment with immunosuppressants such as tacrolimus, mycophenolate mofetil or steroids.

Minimum Eligible Age

6 Months

Eligible Sex

ALL

Accepts Healthy Volunteers

No