Phase 2a Study of IW-6463 in Adults Diagnosed With Mitochondrial Encephalomyopathy, Lactic Acidosis, and Stroke-like Episodes (MELAS)

Recruiting in Palo Alto (17 mi)

+4 other locations

Age: 18+

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 2

Waitlist Available

Sponsor: Cyclerion Therapeutics

No Placebo Group

Prior Safety Data

Trial Summary

What is the purpose of this trial?

This is a single-arm study to evaluate safety and tolerability of oral IW-6463 in adults diagnosed with MELAS.

Eligibility Criteria

Inclusion Criteria

Prior genetic confirmation of a known mitochondrial disease mutation

Neurological features of MELAS (can be based on medical history)

Elevated plasma lactate levels at Screening Visit (≥1.0 mmol/L)

Participant Groups

Group I: IW-6463 Experimental Treatment 1 Intervention

Open-label IW-6463 15 mg once daily (QD), with possibility to dose reduce to 10 mg.

Find a Clinic Near You

Research Locations Nearby Select from list below to view details:

Johns Hopkins University Baltimore, MD

Children's Hospital of Philadelphia Philadelphia, PA

Children's National Hospital of DC Washington, United States

Massachusetts General Hospital Boston, MA

More Trial Locations

Who Is Running the Clinical Trial?

Cyclerion Therapeutics Lead Sponsor

Tisento Therapeutics Lead Sponsor

Children's Hospital of Philadelphia Collaborator

References