National CounterACT Initiative



Status:Recruiting
Healthy:No
Age Range:Any
Updated:3/29/2019
Start Date:May 7, 2018
End Date:December 31, 2019
Contact:Todd J Kilbaugh, MD
Email:kilbaugh@email.chop.edu
Phone:267-426-7131

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Mitochondrial Targeted Biofuels as Countermeasures Against Chemical Threats

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.

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

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. Prior enrollment in this study.

2. Known primary mitochondrial disorder.

3. Use of an investigational drug within 30 days prior to enrollment.

4. 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.
We found this trial at
1
site
South 34th Street
Philadelphia, Pennsylvania 19104
 215-590-1000
Children's Hospital of Philadelphia Since its start in 1855 as the nation's first hospital devoted...
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from
Philadelphia, PA
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