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

Lysogene

Status and phase: Unknown; Phase 3; Phase 2

Conditions: Mucopolysaccharidosis Type IIIA

Treatments: Drug: LYS-SAF302

Study type: Interventional

Funder types: Industry

Identifiers

NCT03612869

P4-SAF-302

Details and patient eligibility

About

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.

Full description

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

Enrollment: 20 estimated patients

Sex: All

Ages: 6+ months old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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20 participants in 1 patient group

AAV SGSH gene therapy (LYS-SAF302)

Experimental group

Description:

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

Treatment:

Drug: LYS-SAF302