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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04475549
Recruitment Status : Not yet recruiting
First Posted : July 17, 2020
Last Update Posted : July 27, 2020
Sponsor:
Collaborator:
Children's Hospital of Philadelphia
Information provided by (Responsible Party):
Cyclerion Therapeutics

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

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 20 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)
Estimated Study Start Date : July 2020
Estimated Primary Completion Date : March 2021
Estimated Study Completion Date : March 2021


Arm Intervention/treatment
Experimental: IW-6463 Drug: IW-6463 Tablets
IW-6463 tablets administered orally (daily)




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



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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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 (>2.0 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

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


Contacts
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Contact: Paul Miller +1 857 338 3355 PMiller@cyclerion.com

Sponsors and Collaborators
Cyclerion Therapeutics
Children's Hospital of Philadelphia
Investigators
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Study Director: Chad Glasser, PharmD Cyclerion Therapeutics, Inc.
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Responsible Party: Cyclerion Therapeutics
ClinicalTrials.gov Identifier: NCT04475549    
Other Study ID Numbers: C6463-201
First Posted: July 17, 2020    Key Record Dates
Last Update Posted: July 27, 2020
Last Verified: July 2020
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
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
Metabolic Diseases
Mitochondrial Diseases