Status:
TERMINATED
Phase 2a Study of IW-6463 in Adults Diagnosed With Mitochondrial Encephalomyopathy, Lactic Acidosis, and Stroke-like Episodes (MELAS)
Lead Sponsor:
Tisento Therapeutics
Conditions:
MELAS
Eligibility:
All Genders
18+ years
Phase:
PHASE2
Brief Summary
This is a single-arm study to evaluate safety and tolerability of oral IW-6463 in adults diagnosed with MELAS.
Detailed Description
IW-6463 tablets will be orally administered once-daily (QD) for up to 29 days
Eligibility Criteria
Inclusion
- 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
- 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
Key Trial Info
Start Date :
November 13 2020
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
January 23 2022
Estimated Enrollment :
8 Patients enrolled
Trial Details
Trial ID
NCT04475549
Start Date
November 13 2020
End Date
January 23 2022
Last Update
August 29 2024
Active Locations (5)
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1
Children's National Hospital of DC
Washington D.C., District of Columbia, United States, 20010
2
Johns Hopkins University
Baltimore, Maryland, United States, 21205
3
Massachusetts General Hospital
Boston, Massachusetts, United States, 02114
4
Columbia University
New York, New York, United States, 10032