Oxaloacetate in Myasthenia Gravis

University of Kansas

Status and phase: Withdrawn; Phase 1

Conditions: Myasthenia Gravis

Treatments: Drug: Placebo; Drug: Oxaloacetate

Study type: Interventional

Funder types: Other; Industry

Identifiers

NCT04965987

MG OAA

Details and patient eligibility

About

This is a phase 1 clinical trial studying whether or not oxaloacetate has a positive effect on patients with Myasthenia Gravis. Patients will be assigned to one of three cohorts which will determine the dose of oxaloacetate they will be given. Subjects will take the study drug for 4 weeks and be on placebo for 4 weeks.

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

1. Patients 18 and older.
2. Patients must have prior or current documentation of MGFA MG grades 2, 3, or 4A generalized MG, according to the MGFA classification system. These grades correspond to mild (2), moderate (3), and severe (4).
3. A total MG-ADL score of greater than 3 at Screening and Baseline with no more than 50% of the total score being due to ocular symptoms.
4. Patient's signs and symptoms should not be better explained by another disease process.
5. Patients must be willing to complete the study and return for follow-up visits.
6. 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.
7. Patients can be on the following drugs as long as there has been no dose change for 60 days: azathioprine, cyclosporine, cyclophosphamide, mycophenolate mofetil, tacrolimus, methotrexate, IVIg or other immunosuppressive drugs.
8. Patients can be on prednisone as long as there has been no dose change for 30 days.
9. No planned changes in MG medications during the study

Exclusion criteria

1. MGFA grade V within 6 months of screening.
2. A history of chronic degenerative, severe psychiatric, or neurologic disorder other than MG that can produce weakness or fatigue.
3. Other major chronic or debilitating illnesses within six months prior to study entry.
4. Female patients who are premenopausal and are: (a) pregnant on the basis of a serum pregnancy test, (b) breast-feeding, or (c) not using an effective method of double barrier (1 hormonal plus 1 barrier method or 2 simultaneous barrier methods) birth control (birth control pills, male condom, female condom, intrauterine device, Norplant, tubal ligation, or other sterilization procedures).
5. Thymectomy in the previous three months.
6. History of severe reactions to OAA
7. Participation in a research study within the last 3 months

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Double Blind

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0 participants in 2 patient groups, including a placebo group

Oxaloacetate

Active Comparator group

Description:

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.

Treatment:

Drug: Oxaloacetate

Placebo

Placebo Comparator group

Description:

Subjects will take 1000mg Placebo BID for 8 weeks.

Treatment:

Drug: Placebo