Oxaloacetate in Myasthenia Gravis

NCT04965987 | Withdrawn Phase 1 FDA Drug

• 1 other identifier: MG OAA
• Study Type: interventional
• Target: N/A
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Myasthenia Gravis

Outcome Measures

Primary Outcomes (1)

• Safety (Frequency and Severity of Adverse Events)

  The primary endpoint of safety will be reported as counts of subjects experiencing adverse events events (including abnormal laboratory results and vital signs). Measures of safety will be reported for the last visit observed for each participant.

  At weeks 2, 4, 6, 8, 10, and 12

Secondary Outcomes (2)

• Myasthenia Gravis-specific Activities of Daily Living Scale (MG-ADL) Score

  Change from Week 4 to Week 8

• Quantitative Myasthenia Gravis (QMG) Score

  Change from Week 4 to Week 8

Study Arms (2)

Oxaloacetate

ACTIVE COMPARATOR

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.

Drug: Oxaloacetate

Placebo

PLACEBO COMPARATOR

Subjects will take 1000mg Placebo BID for 8 weeks.

Drug: Placebo

Interventions

Oxaloacetate DRUG

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. In this reaction, the hydrogen atoms from malate are transferred to NAD+, forming NADH, H+ and oxaloacetate. Oxaloacetate can be converted to citrate with the addition of acetyl-CoA by the enzyme citrate synthase. Oxaloacetate is a critical component in the production of ATP and must be constantly regenerated in order for the citric acid cycle and the electron transport chain to continue

Also known as: CRONaxal

Oxaloacetate

Placebo DRUG

Placebo

Placebo

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Patients 18 and older.
• 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).
• 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.
• Patient's signs and symptoms should not be better explained by another disease process.
• Patients must be willing to complete the study and return for follow-up visits.
• 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.
• 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.
• Patients can be on prednisone as long as there has been no dose change for 30 days.
• No planned changes in MG medications during the study

You may not qualify if:

• MGFA grade V within 6 months of screening.
• A history of chronic degenerative, severe psychiatric, or neurologic disorder other than MG that can produce weakness or fatigue.
• Other major chronic or debilitating illnesses within six months prior to study entry.
• 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).
• Thymectomy in the previous three months.
• History of severe reactions to OAA
• Participation in a research study within the last 3 months

Sponsors & Collaborators

• University of Kansas Medical Center: lead
• Terra Biological LLC: collaborator

Study Sites (1)

University of Kansas Medical Center

Kansas City, Kansas, 66160, United States

Location

MeSH Terms

Conditions

Myasthenia Gravis

Interventions

Oxaloacetic Acid

Condition Hierarchy (Ancestors)

Paraneoplastic Syndromes, Nervous System; Nervous System Neoplasms; Neoplasms by Site; Neoplasms; Paraneoplastic Syndromes; Autoimmune Diseases of the Nervous System; Nervous System Diseases; Neurodegenerative Diseases; Neuromuscular Junction Diseases; Neuromuscular Diseases; Autoimmune Diseases; Immune System Diseases

Intervention Hierarchy (Ancestors)

Oxaloacetates; Oxalates; Dicarboxylic Acids; Acids, Acyclic; Carboxylic Acids; Organic Chemicals; Keto Acids

Study Officials

• Mazen Dimachkie, MD

  University of Kansas Medical Center

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 10, 2021
• First Posted: July 16, 2021
• Study Start: August 1, 2025
• Primary Completion (Estimated): August 1, 2026
• Study Completion (Estimated): December 1, 2026
• Last Updated: July 11, 2025

Record last verified: 2025-07

Locations

US (1)