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Aldose Reductase Inhibitor

Epalrestat for PMM2-CDG

Phase 3
Waitlist Available
Led By Eva Morava-Kozicz, MD, PhD
Research Sponsored by Eva Morava-Kozicz
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Age ≥ 2 and < 18 years
Diagnosis of PMM2-CDG, based on molecularly confirmed biallelic PMM2 pathogenic variants (can be historical diagnosis with lab report on file)
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 9 months
Awards & highlights

Study Summary

This trialwill test if a drug can help kids with a rare genetic disorder. It's a safe and controlled study.

Who is the study for?
This trial is for children aged 2 to under 18 with PMM2-CDG, a rare genetic disorder. They must be able to follow the study plan and not be pregnant or at risk of pregnancy without proper contraception. Kids can't join if they're allergic to epalrestat, have anemia, kidney problems, low platelets, liver issues, other CDGs or are on certain other drugs.Check my eligibility
What is being tested?
The study tests oral Epalrestat against a placebo in kids with PMM2-CDG. It's designed to see if it's safe and tolerable and whether it improves their condition. Participants will randomly receive either the drug or placebo without knowing which one they get.See study design
What are the potential side effects?
While specific side effects for this trial aren't listed, common ones from similar trials may include allergic reactions to medication ingredients and potential impacts on blood sugar levels due to Epalrestat’s mechanism of action.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I am between 2 and 17 years old.
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I have been diagnosed with PMM2-CDG based on genetic testing.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~9 months
This trial's timeline: 3 weeks for screening, Varies for treatment, and 9 months for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Change in Antithrombin III (ATIII)
Change in ICARS
Change in sorbitol (mmol/mol creatinine)
Secondary outcome measures
Change in Nijmegen Pediatric CDG Rating Scale (NPCRS) score
Change of Body Max Index (BMI) percentile
Change of factor XI activity percentage
+3 more

Trial Design

2Treatment groups
Experimental Treatment
Placebo Group
Group I: EpalrestatExperimental Treatment1 Intervention
Epalrestat will be administered orally, 3 times per day (TID) spaced out as evenly as possible over 24 hours in a divided dose starting on Day 1 of the Study.
Group II: PlaceboPlacebo Group1 Intervention
Placebo will be administered orally, 3 times per day (TID) spaced out as evenly as possible over 24 hours in a divided dose starting on Day 1 of the Study.

Find a Location

Who is running the clinical trial?

Eva Morava-KoziczLead Sponsor
3 Previous Clinical Trials
47 Total Patients Enrolled
Maggie's Pearl, LLCLead Sponsor
Eva Morava-Kozicz, MD, PhDPrincipal InvestigatorMayo Clinic
8 Previous Clinical Trials
840 Total Patients Enrolled

Media Library

Epalrestat (Aldose Reductase Inhibitor) Clinical Trial Eligibility Overview. Trial Name: NCT04925960 — Phase 3
Phosphomannomutase 2 Deficiency Research Study Groups: Epalrestat, Placebo
Phosphomannomutase 2 Deficiency Clinical Trial 2023: Epalrestat Highlights & Side Effects. Trial Name: NCT04925960 — Phase 3
Epalrestat (Aldose Reductase Inhibitor) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04925960 — Phase 3

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

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."

Answered by AI

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."

Answered by AI

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."

Answered by AI

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."

Answered by AI

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."

Answered by AI

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."

Answered by AI

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."

Answered by AI

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."

Answered by AI
~17 spots leftby Mar 2025