Your session is about to expire
← Back to Search
Epalrestat for PMM2-CDG
Study Summary
This trialwill test if a drug can help kids with a rare genetic disorder. It's a safe and controlled study.
Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowTimeline
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Trial Design
Find a Location
Who is running the clinical trial?
Media Library
- I have had liver problems, like high enzyme levels or liver stiffness, in the last 6 months.I am between 2 and 17 years old.I haven't used any experimental drugs or biologics recently.Female participants who could become pregnant need to have a negative pregnancy test.My kidney function is impaired based on my age-specific creatinine levels.Your hemoglobin levels are lower than 10 grams per deciliter.If the doctor thinks any other important lab test results are not normal, you may not be able to join the study.I have or might have a congenital disorder of glycosylation.You are allergic to aldose reductase inhibitors.You are allergic to epalrestat.Your blood platelet count is lower than 125 billion per liter.I have been diagnosed with PMM2-CDG based on genetic testing.
- Group 1: Epalrestat
- Group 2: Placebo
- Pivotal Trial - The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.
- 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
Is Epalrestat a new medication or have there been other studies on it?
"One Epalrestat clinical trial is currently underway with more planned in the future. The study taking place right now is a Phase 3 trial. Although most research surrounding Epalrestat takes places in Rochester, Minnesota, there are other locations conducting trials for this medication."
Is this research project seeking more test subjects?
"That is correct, the information on clinicaltrials.gov does show that this study is looking for new participants. The trial was first advertised on November 15th, 2022 and was last updated a month ago on October 25th, 2022. Only 40 patients are needed for this study which will be conducted at a single site."
What are the main goals of this clinical trial?
"The main success metric that will be tracked over the course of this 12-month clinical trial is the Change in ICARS score from baseline to twelve months. Additionally, researchers will also compare changes in antithrombin III activity percentage, BMI percentile, and factor XI activity percentage between study arms."
Does this research only allow people who are under 50 years old to participate?
"The target population for recruitment in this trial are young patients aged 2-17 years old."
How many people are enrolled in this clinical trial at present?
"The information available on clinicaltrials.gov verifies that this study is seeking volunteers. The listing was first posted on November 15th, 2020 and was most recently updated on October 25th, 2020. Currently, the trial is looking for 40 individuals across 1 location."
Which type of patient does this trial seek?
"Eligible participants for this trial must have phosphomannomutase and be aged 2-17. The research team is hoping to enroll around 40 individuals in total."
Has the FDA greenlit Epalrestat for public consumption?
"We feel that Epalrestat is safe to use because it has been tested in multiple Phase 3 trials, meaning that there is both evidence of its efficacy and safety."
Have other researchers looked into this area before?
"Research on epalrestat began in 2022. A small study of 40 patients was conducted that year and sponsored by Maggie's Pearl, LLC. After successful completion of Phase 3 drug approval stage, there is currently one active epalrestat clinical trial being sponsored by the same company."
Share this study with friends
Copy Link
Messenger