Study of VAL-1221 in Patients with Pompe Disease (VAL-1221-201-16)
Research type
Research Study
Full title
A Three-month, Randomized, Parallel, Baseline-controlled, Single and Repeat Dose, Dose-escalation Study of the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Preliminary Efficacy of VAL-1221 Delivered Intravenously (IV) in Ambulatory and Ventilator-free Patients with Late-Onset GSD-II (Pompe Disease)
IRAS ID
218496
Contact name
Deborah Ramsdell
Contact email
Sponsor organisation
Valerion Therapeutics, LLC
Eudract number
2016-004578-16
Duration of Study in the UK
1 years, 6 months, 1 days
Research summary
Pompe disease, also known as glycogen storage disease type II (GSD-II), is a rare inherited disease caused by a deficiency of the enzyme lysosomal acid alpha glucosidase (GAA). The function of GAA is to break down complex sugar molecules called glycogen in a part of the cell called the lysosome. GAA is active in muscle cells and deficiency of GAA damages these cells. In its most severe form, Pompe disease affects young babies who die because of involvement of the heart and breathing muscles. Milder forms can present later in life and do not involve the heart. The main symptoms are difficulty in walking due to weakness of muscles around the pelvis and breathing difficulties due to weakness of diaphragms. Over time, other muscles are also involved, patients become immobile and reliant on a ventilator for breathing.
Pompe disease is treated by enzyme replacement therapy. This involves intravenous (IV) administration of a pharmacological form of the missing GAA enzyme (alglucosidase alfa). This treatment is partially effective, but in most cases does not prevent progression of the disease. One reason for this may be that only a very small amount of alglucosidase alfa which is administered actually finds its way into muscle cells, and even then, it may not access the right part of the cell.
Valerion has developed a new form of GAA called VAL-1221 which has been designed to improve the delivery of functional GAA to muscle cells in Pompe disease patients. VAL-1221-201-16 is an early stage clinical trial which aims to assess the safety, pharmacology and clinical effects of VAL-1221 in adult patients with Pompe disease. It will be the first time VAL-1221 is administered to human subjects. Patients will be randomly assigned to receive either treatment with VAL-1221 or their current standard of care at their routine infusion location.
REC name
London - Surrey Borders Research Ethics Committee
REC reference
17/LO/0627
Date of REC Opinion
9 Jun 2017
REC opinion
Further Information Favourable Opinion