Apply to Trial

Compensation: Varies

Number of Visits: 12

Duration: 8 weeks

24 Participants Needed

Oxaloacetate for Myasthenia Gravis

Overseen ByAndrew J Heim

Age: 18+

Sex: Any

Trial Phase: Phase 1

Sponsor: University of Kansas Medical Center

Disqualifiers: Chronic degenerative, Severe psychiatric, Neurologic, others

Trial Summary

Will I have to stop taking my current medications?

You can continue taking your current medications as long as there have been no dose changes recently: 60 days for most immunosuppressive drugs and 30 days for prednisone. No changes in your Myasthenia Gravis medications are planned during the study.

How does the drug oxaloacetate differ from other treatments for myasthenia gravis?

Oxaloacetate is unique because it is not a standard treatment for myasthenia gravis, which typically involves drugs that enhance neuromuscular transmission or suppress the immune system. Unlike traditional treatments, oxaloacetate may offer a novel approach, potentially targeting different pathways or mechanisms in the body.¹ ² ³ ⁴ ⁵

What is the purpose of this trial?

This trial is testing if oxaloacetate can help people with Myasthenia Gravis. Patients will take different doses of oxaloacetate over a period of time. The goal is to see if oxaloacetate can improve muscle function by boosting energy production in cells.

Research Team

Dr. Mazen M Dimachkie, MD - Kansas City ...

Mazen Dimachkie

Principal Investigator

University of Kansas Medical Center

Eligibility Criteria

This trial is for adults with Myasthenia Gravis (MG) grades 2 to 4A, who can commit to the study schedule and follow-up visits. They must have stable medication doses for at least 30-60 days and cannot be pregnant or breastfeeding without effective birth control. Those with severe psychiatric disorders, recent thymectomy, or participation in another study within the last three months are excluded.

Inclusion Criteria

Patients must be willing to give written informed consent before participating in this study. A copy of the signed consent must be kept in the patient's medical record.

My myasthenia gravis is classified as mild, moderate, or severe.

I am willing to complete the study and attend all follow-up visits.

See 4 more

Exclusion Criteria

My condition has been severe but stable for the last 6 months.

I have a chronic illness that causes weakness or fatigue, but it's not myasthenia gravis.

I have not had any major illnesses in the last 6 months.

See 4 more

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive either oxaloacetate or placebo for 8 weeks

8 weeks

Visits at weeks 2, 4, 6, 8

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Visits at weeks 10 and 12

Treatment Details

Interventions

Oxaloacetate
Placebo

Trial Overview The trial tests oxaloacetate's effect on MG over a period of eight weeks: four weeks taking the drug and four weeks on placebo. Participants are divided into three groups receiving different doses to assess safety and potential benefits against muscle weakness caused by MG.

Participant Groups

2Treatment groups

Active Control

Placebo Group

Group I: OxaloacetateActive Control1 Intervention

Oxaloacetate (OAA) is a four-carbon molecule involved in many metabolic pathways, including gluconeogenesis, citric acid cycle, glyoxylate cycle, urea cycle, and amino acid metabolism. In the glyoxylate and citric acid cycles, oxaloacetate is formed as the result of the catalysis by malate dehydrogenase. Subjects will take 1000mg OAA BID for 8 weeks.

Group II: PlaceboPlacebo Group1 Intervention

Subjects will take 1000mg Placebo BID for 8 weeks.

Find a Clinic Near You

Who Is Running the Clinical Trial?

University of Kansas Medical Center

Lead Sponsor

Trials

527

Recruited

181,000+

Terra Biological LLC

Industry Sponsor

Trials

Recruited

320+

Findings from Research

Acetylcholinesterase inhibitors remain the first-line treatment for myasthenia gravis (MG), but their effectiveness can diminish over time, leading to the need for additional therapies.

Immunosuppressive treatments, particularly steroids and adjunctive therapies like azathioprine and mycophenolate mofetil, are crucial for managing MG symptoms, though they come with potential side effects such as renal toxicity and hypertension.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

[Actual aspects of myasthenia gravis treatment].Czapliński, A., Radziwiłł, AJ., Steck, AJ.[2006]

In myasthenia gravis (MG), treatment strategies are tailored to disease severity, with acetylcholinesterase inhibitors being effective for mild cases, while more severe cases often require immunosuppressive treatments like azathioprine or glucocorticosteroids, which have been established through observational studies.

For severe and treatment-resistant MG, options like intravenous immunoglobulins and plasmapheresis are recommended, and surgical interventions such as thymectomy are advised for nonthymoma patients, significantly reducing perioperative risks when combined with immunosuppressive therapy.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Progress in the treatment of myasthenia gravis.Gold, R., Hohlfeld, R., Toyka, KV.[2021]

In a study of 990 myasthenia gravis patients, 8.5% were identified as drug-refractory, meaning their condition did not improve with corticosteroids and multiple immunosuppressive agents, highlighting a significant challenge in treatment.

Among drug-refractory patients, only 42.9% achieved remission or minimal symptoms after a mean follow-up of 9.8 years, compared to 79.8% of non-drug-refractory patients, indicating a need for new, more effective treatments for this group.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Drug-refractory myasthenia gravis: Clinical characteristics, treatments, and outcome.Cortés-Vicente, E., Álvarez-Velasco, R., Pla-Junca, F., et al.[2022]