National CounterACT Initiative

Completed

• Conditions: Mitochondrial Alteration

• Registration Number: NCT03597438
• Lead Sponsor: Children's Hospital of Philadelphia

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.

Study Timeline

• Study Start: 2018-05-07
• Study First Submitted: 2018-07-13
• Study First Submitted QC: 2018-07-13
• Study First Posted: 2018-07-24
• Primary Completion: 2023-04-18
• Status Verified: 2024-02
• Study Completion: 2024-02-22
• Last Update Submitted: 2024-02-22
• Last Update Posted: 2024-02-23

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 236

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.

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Attenuation by the succinate prodrug of toxin-induced decrease in mitochondrial respiration.	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 Outcome Measures

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

Trial Locations

Locations (1)

Children's Hospital of Philadelphia; 🇺🇸 Philadelphia, Pennsylvania, United States