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ATB200 + AT2221 for Pompe Disease
Study Summary
This trial is testing if it's safe to give two investigational drugs to people with Pompe disease who are already receiving enzyme replacement therapy.
Timeline
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Side effects data
From 2010 Phase 3 trial • 42 Patients • NCT00319046Trial Design
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Who is running the clinical trial?
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- You have had an allergic reaction to miglustat or similar medications in the past.You have been treated with alglucosidase alfa (Myozyme/Lumizyme) for more than 2 years and are unable to walk without a wheelchair.You have a current autoimmune disease like lupus, scleroderma, or rheumatoid arthritis.People aged 18 to 75, including both men and women.You have been taking a specific enzyme replacement therapy for a certain number of years and are currently receiving it every other week. You must be able to walk a certain distance and have a specific lung function measurement.If you haven't received a specific treatment before, you need to be able to walk a certain distance and have a certain lung function.You have been receiving a specific enzyme replacement therapy for a certain period of time and can walk a certain distance in a test, as well as have a specific lung function measurement.You have been diagnosed with Pompe disease.You have active bronchial asthma, and any decision about participating in the study will be discussed with the Amicus Medical Monitor.
- Group 1: ATB200
- Group 2: ATB200 + AT2221
- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
Frequently Asked Questions
Are there any available positions for participants in this trial?
"Though this study is not currently enrolling patients, it's worth noting that 18 other clinical trials for acid maltase deficiency are ongoing and 6 more are actively recruiting participants."
Does this testing only include young adults, or are elderly patients being accepted too?
"The age criteria to participate in this trial is between 18-75 years old."
How many people are receiving care through this program?
"This study is not actively searching for candidates at this time. The clinical trial was initially posted on 1/1/2016 and was most recently updated on 6/9/2022. If you are seeking other studies, there are presently 18 trials actively enrolling participants with acid maltase deficiency and 6 trials for ATB200 actively searching for participants."
What other similar trials exist for this condition?
"Amicus Therapeutics has sponsored the clinical research for ATB200 since 2016. The first Phase 1 & 2 trial was completed in 2016 with a total of 32 participants. Today, there are 6 active trials being conducted in 24 different countries and 39 cities."
At how many hospitals is this trial taking place?
"There are a dozen medical centres running this clinical trial. These include Rutgers New jersey Medical School in Newark, University of California Irvine in Orange, and Great Falls Clinic, LLP in Great Falls."
Are there any other published papers that mention ATB200?
"ATB200 was first explored in 2016 by the University Children's Hospital Department of Neuropediatrics and Inborn Metabolic Disorders. As of now, there have been 12 completed trials with 6 more active studies. The majority of these ongoing research projects are based out of Newark, New jersey."
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