Phase 2a Study of IW-6463 in Adults Diagnosed With Mitochondrial Encephalomyopathy, Lactic Acidos… (NCT04475549) | Clinical Trial Compass
TerminatedPhase 2
Phase 2a Study of IW-6463 in Adults Diagnosed With Mitochondrial Encephalomyopathy, Lactic Acidosis, and Stroke-like Episodes (MELAS)
Stopped: Study terminated due to enrollment challenges.
United States8 participantsStarted 2020-11-13
Plain-language summary
This is a single-arm study to evaluate safety and tolerability of oral IW-6463 in adults diagnosed with MELAS.
Who can participate
Age range18 Years
SexALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
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)
✓. 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
Exclusion criteria
✕. 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.
What they're measuring
1
Number of Participants With Study Drug Dose Reductions or Discontinuations Due to ≥ 1 Treatment Emergent Adverse Event (TEAE)
Timeframe: From first dose date to Day 43 (±4)
2
Number of Participants Who Experienced ≥1 AE, TEAE, Serious AE (SAE), or TEAE of Special Interest (AESI)
✕. 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