Protein engineering to design better enzyme replacement therapies for Fabry disease

Intelligent De-immunization of Enzyme Replacement Therapies (IDERT)

Registry ID

ISRCTN14092141

Source registry

ISRCTN

Status

Recruiting

Study type

OBSERVATIONAL

Sponsor

University of Edinburgh

Enrollment

30

Start date

2024-08-01

Completion date

2026-12-30

Last update

2026-08-17

Conditions

• Nutritional, Metabolic, Endocrine

Summary

Fabry disease

Detailed description

Collection of blood and urine samples 24-hour non-invasive home measure of blood pressure and blood vessel function (optional)

Interventions

• OTHER: Collection of blood and urine samples 24-hour non-invasive home measure of blood pressure and blood vessel function (optional)

Inclusion criteria

1. Diagnosis of Fabry disease (biochemical and/or genetic diagnosis) 2. Aged 16 years or over 3. Able to give informed consent 4. Not on immunosuppressive medications (e.g., previous transplant) which could impact immunogenicity 5. Never received gene-therapy for Fabry disease 6. No upper age limit

Exclusion criteria

1. On immunosuppressive medication (equivalent to >7.5 mg prednisolone daily) 2. Treated with gene therapy for Fabry Disease 3. Unable to give informed consent

Locations

• , , United Kingdom
• , , Scotland

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View on source registry