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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

Tracking Information
First Submitted Date  ICMJE July 14, 2020
First Posted Date  ICMJE July 17, 2020
Last Update Posted Date July 27, 2020
Estimated Study Start Date  ICMJE July 2020
Estimated Primary Completion Date March 2021   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: July 14, 2020)
  • 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
  • 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
  • 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
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE Not Provided
Original Secondary Outcome Measures  ICMJE Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Phase 2a Study of IW-6463 in Adults Diagnosed With MELAS
Official Title  ICMJE A Phase 2a Safety, Tolerability, Pharmacokinetic, and Pharmacodynamic Study in Individuals With Mitochondrial Encephalomyopathy, Lactic Acidosis, and Stroke-like Episodes (MELAS)
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
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE 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
Condition  ICMJE MELAS
Intervention  ICMJE Drug: IW-6463 Tablets
IW-6463 tablets administered orally (daily)
Study Arms  ICMJE Experimental: IW-6463
Intervention: Drug: IW-6463 Tablets
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Not yet recruiting
Estimated Enrollment  ICMJE
 (submitted: July 14, 2020)
20
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE March 2021
Estimated Primary Completion Date March 2021   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Paul Miller +1 857 338 3355 PMiller@cyclerion.com
Listed Location Countries  ICMJE Not Provided
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT04475549
Other Study ID Numbers  ICMJE C6463-201
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party Cyclerion Therapeutics
Study Sponsor  ICMJE Cyclerion Therapeutics
Collaborators  ICMJE Children's Hospital of Philadelphia
Investigators  ICMJE
Study Director: Chad Glasser, PharmD Cyclerion Therapeutics, Inc.
PRS Account Cyclerion Therapeutics
Verification Date July 2020

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP