ATB200/AT2221 in adult subjects with late-onset Pompe disease
Research type
Research Study
Full title
A Phase 3 Double-blind Randomized Study to Assess the Efficacy and Safety of Intravenous ATB200 Co-administered With Oral AT2221 in Adult Subjects With Late Onset Pompe Disease Compared With Alglucosidase Alfa/Placebo
IRAS ID
258824
Contact name
Mark Roberts
Contact email
Sponsor organisation
Amicus Therapeutics, Inc.
Eudract number
2018-000755-40
Clinicaltrials.gov Identifier
Duration of Study in the UK
1 years, 8 months, 12 days
Research summary
This research study is a randomised, double-blind, placebo-controlled study to evaluate how safe and effective both study drugs, intravenous ATB200 co-administered with oral AT2221, are compared to the combination of standard of care treatment (alglucosidase alfa) with placebo in patients with Late-Onset Pompe Disease (LOPD).
LOPD is a genetic disorder caused by mutations in the gene encoding human acid a-glucosidase (GAA). GAA is responsible for the breakdown of glycogen. Therefore, the complete absence or reduced functioning of GAA means there is more glycogen within cells. This causes cellular disruption, particularly in the heart, skeletal muscles and diaphragm. Most patients experience difficulty walking, climbing stairs and have progressive limitations in motor functions.
The study drugs being investigated are designed to improve the breakdown of glycogen. ATB200 is the main active ingredient that initiates this breakdown. AT2221 is a pharmacological chaperone, meaning it supports in the delivery of ATB200 to affected cells and protects ATB200 from being broken down in the body, ultimately enhancing its therapeutic effect. The study will be conducted in 90 sites globally. 4 participants are planned to be enrolled in the United Kingdom.
Participants will be required to attend numerous study visits across 1 year where any changes in their motor function, pulmonary function, muscle strength and the 6-Minute Walk test will be measured. Patients will also complete patient reported outcomes to determine any changes in their quality of life. Patients will receive either ATB200/AT2221 or an alglucosidase alfa/placebo every 2 weeks.
After participation in the trial, patients will have the option to be treated with the current standard of care therapy or to enrol into an Open-Label Extension study and continue receiving the investigational treatment.REC name
East Midlands - Nottingham 2 Research Ethics Committee
REC reference
19/EM/0021
Date of REC Opinion
18 Mar 2019
REC opinion
Further Information Favourable Opinion