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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

Who Do I Contact?

If you are interested in participating in the study or want to learn more please contact our study team at nguyens2 [at] or 267-426-0225.

Eligibility & Criteria

IRB #:
Official Title:
A Phase 2a Safety, Tolerability, Pharmacokinetic, And Pharmacodynamic Study In Individuals With Mitochondrial Encephalomyopathy, Lactic Acidosis, And Stroke-Like Episodes (MELAS)
Study Phase:
Phase II
Eligible Age Range:
18 - 99 Years
Study Categories:

Visit 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