National CounterACT Initiative

The purpose of the study is to determine in vitro effects on mitochondrial function of selected chemical agents in human cells, and assess the capability of a cell-permeable succinate prodrug to attenuate toxic effects The project aims at repurposing this recent pharmaceutical discovery, currently being developed for treatment of toxic exposure, for an expanded indication to treat chemically induced mitochondrial toxicity.

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Key information

Conditions

Mitochondrial Alteration

Sex eligibility

All sexes

Study type

Observational

Primary location

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

About this study

The study duration per subject will be approximately 15 minutes to complete the one time blood collection.

Who can participate

Healthy volunteers accepted: Yes

Only the study team can determine whether someone qualifies for participation.

Patient Inclusion Criteria:

Males or females age greater or equal 2 years of age
Weight greater or equal to 10 kg
Parental/guardian permission (informed consent) and if appropriate, child assent.

Patient Exclusion Criteria:

Known primary mitochondrial disorder.
Use of an investigational drug within 30 days prior to enrollment.
Parents/guardians or subjects who in the opinion of the Investigator, may be non-compliant with study schedules or procedures.

Volunteer Inclusion Criteria

Males or females > 18 years of age
Employee, trainee, or student informed consent

Volunteer Exclusion Criteria:

Prior enrollment in this study.
Known primary mitochondrial disorder.
Use of an investigational drug within 30 days prior to enrollment.

Treatment and study plan

Primary outcomes

Attenuation by the succinate prodrug of toxin-induced decrease in mitochondrial respiration.

Time frame: by end of 2018

Data will be assessed using 2-tailed Student's t-test. Dose-response curves for respiration will be made for each toxin and IC50 calculated. In case of difficulties in demonstrating acute toxicity of chemical agents, dose-titration experiments and long-term incubation (up to 24 hours) will be performed

Secondary outcomes

Attenuation of toxin-induced decrease in mitochondrial membrane potential and attenuate lactate production by the succinate prodrug.

Time frame: by end of 2018

Data will be assessed using 2-tailed Student's t-test. Dose-response curves for respiration will be made for each toxin and IC50 calculated. In case of difficulties in demonstrating acute toxicity of chemical agents, dose-titration experiments and long-term incubation (up to 24 h) will be performed

Sponsors and collaborators

Lead sponsor

Children's Hospital of Philadelphia

Other

Collaborators

National Institute of Neurological Disorders and Stroke (NINDS)

Registry information

Official study title

Mitochondrial Targeted Biofuels as Countermeasures Against Chemical Threats

Important dates

Study start: 2018
Primary completion: 2023
Study completion: 2024
First posted: Jul 24, 2018
Registry last updated: Feb 23, 2024

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Published trials that share one or more normalized conditions with this study.

Completed