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

Stopped early · Phase 2

Conditions studied: MELAS

In brief

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

Key facts

Study ID

NCT04475549

Run by

Tisento Therapeutics

People needed

8

Starts

2020-11-13

Expected to finish

2022-01-23

Last updated by the study team

2024-08-29

Who can join

Age: 18 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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)
• Women of childbearing potential must have a negative pregnancy test prior to randomization and must agree to use protocol-specified contraception from the Screening Visit through 90 days after the final dose of study drug.
• Male participants must be surgically sterile by vasectomy (conducted ≥60 days before the Screening Visit or confirmed via sperm analysis) or must agree to use protocol-specified contraception and agree to refrain from sperm donation from the Screening Visit through 90 days after the final dose of study drug.
• Other inclusion criteria per protocol

You may not qualify if…

• Positive pregnancy test at Screening or on Day 1
• Hypotension defined as systolic blood pressure (BP) ≤90 mmHg or diastolic BP ≤60 mmHg at Screening or predose at Day 1
• Hypertension defined as systolic BP >160 mmHg or diastolic BP >100 mmHg, at Screening or predose at Day 1
• Uncontrolled diabetes
• Severe gastrointestinal dysmotility as determined by the investigator that may impact compliance and/or oral drug administration, absorption and exposure.
• Unable to fast for 3-4 hours after a meal
• Unable or unwilling to adhere to the study schedule, lifestyle restrictions, assessment requirements or, in the clinical judgment of the investigator, is otherwise not suitable for study participation.
• Current or past history of clinically significant cardiomyopathy and/or cardiac conduction abnormality
• Used any nicotine-containing products (eg, cigarettes, e-cigarettes, vape pens, cigars) within 1 month of enrollment
• Other exclusion criteria per protocol

Where it is running

• Children's National Hospital of DC — Washington D.C., District of Columbia, United States
• Johns Hopkins University — Baltimore, Maryland, United States
• Massachusetts General Hospital — Boston, Massachusetts, United States
• Columbia University — New York, New York, United States
• Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States

Full record on ClinicalTrials.gov

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