Clinical trial NCT03597438: National CounterACT Initiative
Official title: Mitochondrial Targeted Biofuels as Countermeasures Against Chemical Threats
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Mitochondrial Alteration
Lead sponsor: Children's Hospital of Philadelphia
Sponsor class: OTHER
Collaborators: name: National Institute of Neurological Disorders and Stroke (NINDS)
First posted: 2018-07-24
Enrollment: 236
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: yes
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Patients scheduled for elective surgery will be screened for eligibility. Volunteers who are employees, trainees, and students will be screened for eligibility.

Brief summary: 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.

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

Eligibility criteria: Patient Inclusion Criteria:

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

Patient Exclusion Criteria:

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

Volunteer Inclusion Criteria

1. Males or females \> 18 years of age
2. Employee, trainee, or student informed consent

Volunteer Exclusion Criteria:

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

Primary outcomes:
measure: Attenuation by the succinate prodrug of toxin-induced decrease in mitochondrial respiration.
timeFrame: by end of 2018
Secondary outcomes:
measure: Attenuation of toxin-induced decrease in mitochondrial membrane potential and attenuate lactate production by the succinate prodrug.
timeFrame: by end of 2018
Other outcomes:
Locations:
city: Philadelphia
country: United States
facility: Children's Hospital of Philadelphia
state: Pennsylvania
Study officials:
affiliation: Children's Hospital of Philadelphia
name: Todd J Kilbaugh, MD
role: PRINCIPAL_INVESTIGATOR
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