NCT04204889

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).

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
18

participants targeted

Target at P25-P50 for phase_1

Timeline
Completed

Started Mar 2020

Typical duration for phase_1

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

December 17, 2019

Completed
2 days until next milestone

First Posted

Study publicly available on registry

December 19, 2019

Completed
3 months until next milestone

Study Start

First participant enrolled

March 17, 2020

Completed
3.4 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

August 2, 2023

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

August 2, 2023

Completed
Last Updated

June 6, 2024

Status Verified

June 1, 2024

Enrollment Period

3.4 years

First QC Date

December 17, 2019

Last Update Submit

June 5, 2024

Conditions

Outcome Measures

Primary Outcomes (1)

  • Dose limiting toxicities (DLT)

    Defined as any serious adverse event related to OAA requiring hospitalization, or any AE related to OAA requiring stopping the medication, including a two fold increase in AST and/or LAT and /or a 1.5 times increase in creatinine level.

    Day 28

Secondary Outcomes (4)

  • Pharmacokinetic testing

    Day 28

  • Mitochondrial Biomarkers

    Day 28

  • Platelet Biomarker

    Day 28

  • MR spectroscopy

    Day 28

Study Arms (1)

Oxaloacetate

EXPERIMENTAL

3+3 dose escalating trial starting with 500mg twice daily orally and ending with 2500mg twice daily.

Drug: Oxaloacetic Acid

Interventions

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.

Also known as: Oxaloacetate
Oxaloacetate

Eligibility Criteria

Age21 Years - 80 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • 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.

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

University of Kansas Medical Center

Kansas City, Kansas, 66160, United States

Location

MeSH Terms

Interventions

Oxaloacetic Acid

Intervention Hierarchy (Ancestors)

OxaloacetatesOxalatesDicarboxylic AcidsAcids, AcyclicCarboxylic AcidsOrganic ChemicalsKeto Acids

Study Officials

  • Omar Jawdat, MD

    University of Kansas Medical Center

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
SPONSOR INVESTIGATOR
PI Title
Principal Investigator

Study Record Dates

First Submitted

December 17, 2019

First Posted

December 19, 2019

Study Start

March 17, 2020

Primary Completion

August 2, 2023

Study Completion

August 2, 2023

Last Updated

June 6, 2024

Record last verified: 2024-06

Locations