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Trial record 2 of 28 for:    oxaloacetate

Trial of Oxaloacetate in ALS (TOALS)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT04204889
Recruitment Status : Recruiting
First Posted : December 19, 2019
Last Update Posted : December 19, 2019
Clinical Research in ALS and Related Disorders for Therapeutic Development
Rare Diseases Clinical Research Network
Terra Biological LLC
Information provided by (Responsible Party):
Omar Jawdat, University of Kansas Medical Center

Brief Summary:
The purpose of this study is to determine the safety and the maximal tolerated dose of Oxaloacetate (OAA) in patients with Amyotrophic Lateral Sclerosis (ALS).

Condition or disease Intervention/treatment Phase
ALS Drug: Oxaloacetic Acid Phase 1

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 24 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Trial of Oxaloacetate in ALS
Estimated Study Start Date : January 2020
Estimated Primary Completion Date : January 2021
Estimated Study Completion Date : February 2021

Arm Intervention/treatment
Experimental: Oxaloacetate
3+3 dose escalating trial starting with 500mg twice daily orally and ending with 2500mg twice daily.
Drug: Oxaloacetic Acid
The first 3 patients will receive 500 mg twice daily, and subsequent cohorts will increase by 500 mg per dose to a maximum of 2500 mg twice daily.
Other Name: Oxaloacetate

Primary Outcome Measures :
  1. Dose limiting toxicities (DLT) [ Time Frame: Day 28 ]
    Defined as any serious adverse event related to OAA requiring hospitalization, or any AE related to OAA requiring stopping the medication, inculding a two fold increase in AST and/or LAT and /or a 1.5 times increase in creatinine level.

Secondary Outcome Measures :
  1. Pharmacokinetic testing [ Time Frame: Day 28 ]
    OAA Levels

  2. Mitochondrial Biomarkers [ Time Frame: Day 28 ]
    Annexin V/Mitotracker in lymphocytes: assess modification of apoptosis/mitochondrial number. TMRE: Mitochondrial membrane potential. MitoSox: Mitochondrial superoxide. Glutamate-plasma: OAA specific biomarker.

  3. Platelet Biomarker [ Time Frame: Day 28 ]
    The levels of Serum TDP43

  4. MR spectroscopy [ Time Frame: Day 28 ]
    MR spectroscopy of brain glutathione

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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Ages Eligible for Study:   21 Years to 80 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • A clinical diagnosis by a study investigator of laboratory-supported probable, probable, or definite ALS, according to the modified El Escorial criteria[1]
  • Vital capacity (VC) greater or equal to 50% of predicted
  • Diagnosis with ALS within 3 years prior to enrollment
  • If patients are taking riluzole for ALS, they must be on a stable dose for at least thirty days prior to the baseline visit
  • Women of childbearing age must use protection against pregnancy.

Exclusion Criteria:

  • Requirement for tracheotomy ventilation or non-invasive ventilation for > 23 hours per day
  • Diagnosis of other neurodegenerative diseases (e.g., Parkinson disease, Alzheimer disease)
  • Clinically significant history of unstable medical illness (e.g., unstable angina, advanced cancer) over the last 30 days
  • Current pregnancy or lactation
  • Limited mental capacity such that the patient cannot provide written informed consent or comply with evaluation procedures
  • Receipt of any investigational drug within the past 30 days from enrollment

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT04204889

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Contact: Laura Herbelin 913-588-5095
Contact: Collin Gerringer 913-574-0008

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United States, Kansas
University of Kansas Medical Center Recruiting
Kansas City, Kansas, United States, 66160
Sponsors and Collaborators
Omar Jawdat
Clinical Research in ALS and Related Disorders for Therapeutic Development
Rare Diseases Clinical Research Network
Terra Biological LLC
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Principal Investigator: Omar Jawdat, MD University of Kansas Medical Center
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Responsible Party: Omar Jawdat, Omar Jawdat MD, University of Kansas Medical Center Identifier: NCT04204889    
Other Study ID Numbers: TOALS
8009 ( Other Identifier: Rare Diseases Clinical Research Network )
First Posted: December 19, 2019    Key Record Dates
Last Update Posted: December 19, 2019
Last Verified: December 2019

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No