Clinical trial NCT04965987: Oxaloacetate in Myasthenia Gravis
Official title: A Phase I, Double-Blind, Pilot Study of Oxaloacetate in Myasthenia Gravis
Study type: INTERVENTIONAL
Overall status: NOT_YET_RECRUITING
Phase: PHASE1
Conditions: Myasthenia Gravis
Lead sponsor: University of Kansas Medical Center
Sponsor class: OTHER
Collaborators: name: Terra Biological LLC
First posted: 2021-07-16
Enrollment: 24
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.


Eligibility criteria: 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

Primary outcomes:
measure: Safety (Frequency and Severity of Adverse Events)
timeFrame: At weeks 2, 4, 6, 8, 10, and 12
Secondary outcomes:
measure: Myasthenia Gravis-specific Activities of Daily Living Scale (MG-ADL) Score
timeFrame: Change from Week 4 to Week 8
measure: Quantitative Myasthenia Gravis (QMG) Score
timeFrame: Change from Week 4 to Week 8
Other outcomes:
Locations:
city: Kansas City
country: United States
facility: University of Kansas Medical Center
state: Kansas
Study officials:
affiliation: University of Kansas Medical Center
name: Mazen Dimachkie, MD
role: PRINCIPAL_INVESTIGATOR
References: