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

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ClinicalTrials.gov Identifier: NCT04475549
Recruitment Status : Terminated (Study terminated due to enrollment challenges.)
First Posted : July 17, 2020
Last Update Posted : January 25, 2023
Sponsor:
Information provided by (Responsible Party):
Cyclerion Therapeutics

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

Condition or disease Intervention/treatment Phase
MELAS Drug: IW-6463 Tablets Phase 2

Detailed Description:
IW-6463 tablets will be orally administered once-daily (QD) for up to 29 days
Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 8 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Intervention Model Description: Open-label, single-arm study with daily dosing for up to 29 days.
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase 2a Safety, Tolerability, Pharmacokinetic, and Pharmacodynamic Study in Individuals With Mitochondrial Encephalomyopathy, Lactic Acidosis, and Stroke-like Episodes (MELAS)
Actual Study Start Date : November 13, 2020
Actual Primary Completion Date : January 23, 2022
Actual Study Completion Date : January 23, 2022

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: IW-6463
Open-label IW-6463 15 mg once daily (QD), with possibility to dose reduce to 10 mg.
 Drug: IW-6463 Tablets
IW-6463 tablets administered orally (daily)
Other Name: CY6463


Outcome Measures
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Primary Outcome Measures :
  1. Number (or %) of participants who discontinue or dose reduced the study drug due to a treatment-emergent adverse event (TEAE) [ Time Frame: From first dose date to Day 43 (±4) ]
    TEAE is defined as an adverse event with an onset that occurs after receiving the study drug, until the end of study period

  2. Number (or %) of participants who experience at least one treatment-emergent adverse event (TEAE) [ Time Frame: From first dose date to Day 43 (±4) ]
    TEAE is defined as an adverse event with an onset that occurs after receiving the study drug, until the end of study period

  3. Number (or %) of participants who experience at least one treatment-emergent adverse event (TEAE) by severity [ Time Frame: From first dose date to Day 43 (±4) ]
    TEAE is defined as an adverse event with an onset that occurs after receiving the study drug, until the end of study period


Eligibility Criteria
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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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 blood pressure (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
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04475549


Locations
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United States, District of Columbia
Children's National Hospital of DC
Washington, District of Columbia, United States, 20010
United States, Maryland
Johns Hopkins University
Baltimore, Maryland, United States, 21205
United States, Massachusetts
Massachusetts General Hospital
Boston, Massachusetts, United States, 02114
United States, New York
Columbia University
New York, New York, United States, 10032
United States, Pennsylvania
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
Sponsors and Collaborators
Cyclerion Therapeutics
Investigators
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Study Director: Chad Glasser, PharmD Cyclerion Therapeutics, Inc.
More Information
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Responsible Party: Cyclerion Therapeutics
ClinicalTrials.gov Identifier: NCT04475549    
Other Study ID Numbers: C6463-201
CY6463 ( Other Identifier: Cyclerion Therapeutics )
First Posted: July 17, 2020    Key Record Dates
Last Update Posted: January 25, 2023
Last Verified: January 2023
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Cyclerion Therapeutics:
mitochondrial disease
Additional relevant MeSH terms:
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MELAS Syndrome
Mitochondrial Encephalomyopathies
Acidosis
Acidosis, Lactic
Acid-Base Imbalance
Metabolic Diseases
Mitochondrial Myopathies
Muscular Diseases
Musculoskeletal Diseases
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
 Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Cerebral Small Vessel Diseases
Cerebrovascular Disorders
Neuromuscular Diseases
Vascular Diseases
Cardiovascular Diseases
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Mitochondrial Diseases