MELAS Study

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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 nguyens2@chop.edu.

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] chop.edu or 267-426-0225.

Eligibility & Criteria

IRB #:
19-017185
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
Gender:
All
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
Leader
Marni J. Falk

Marni J. Falk, MD