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This study is now recruiting.
The purpose of this research is to investigate study drug IW-6463 in patients with Mitochondrial Encephalomyopathy, Lactic Acidosis, and Stroke-Like Episodes (MELAS). IW-6463 is not approved by the U.S. Food and Drug Administration for any use. The study period will last for approximately 75 days and will include at least 3 to 6 on-site visits at the Children's Hospital of Philadelphia (CHOP), where participants will be provided the study drug and monitored for safety. For more information, please contact Sara Nguyen at email@example.com.
Eligibility & Criteria
The study will require at least 4 to 6 on-site visits; procedures include:
- Medical history review; physical examinations
- Study assessments; review of daily study diary
- Cardiac tests
- Brain imaging
- Blood and urine tests
- A single-time lumbar puncture (spinal tap)
- Administration of study drug