Overview

Phase 2a Study of IW-6463 in Adults Diagnosed With MELAS

Status:
Recruiting
Trial end date:
2021-11-01
Target enrollment:
0
Participant gender:
All
Summary
This is a single-arm study to evaluate safety and tolerability of oral IW-6463 in adults diagnosed with MELAS
Phase:
Phase 2
Accepts Healthy Volunteers?
No
Details
Lead Sponsor:
Cyclerion Therapeutics
Collaborator:
Children's Hospital of Philadelphia
Criteria
Inclusion Criteria:

1. Prior genetic confirmation of a known mitochondrial disease mutation

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

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

4. 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.

5. 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.

6. Other inclusion criteria per protocol

Exclusion Criteria:

1. Positive pregnancy test at Screening or on Day 1

2. Hypotension defined as systolic BP ≤90 mmHg or diastolic BP ≤60 mmHg at Screening or
predose at Day 1

3. Hypertension defined as systolic BP >160 mmHg or diastolic BP >100 mmHg, at Screening
or predose at Day 1

4. Uncontrolled diabetes

5. Severe gastrointestinal dysmotility as determined by the investigator that may impact
compliance and/or oral drug administration, absorption and exposure.

6. Unable to fast for 3-4 hours after a meal

7. 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.

8. Current or past history of clinically significant cardiomyopathy and/or cardiac
conduction abnormality

9. Used any nicotine-containing products (eg, cigarettes, e-cigarettes, vape pens,
cigars) within 1 month of enrollment

10. Other exclusion criteria per protocol