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Study terminated due to enrollment challenges.
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This is a single-arm study to evaluate safety and tolerability of oral IW-6463 in adults diagnosed with MELAS.
IW-6463 tablets will be orally administered once-daily (QD) for up to 29 days
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| IW-6463 | Experimental | Open-label IW-6463 15 mg once daily (QD), with possibility to dose reduce to 10 mg. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| IW-6463 Tablets | Drug | IW-6463 tablets administered orally (daily) |
|
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With Study Drug Dose Reductions or Discontinuations Due to ≥ 1 Treatment Emergent Adverse Event (TEAE) | A TEAE is defined as an adverse event (AE) with an onset that occurs between the first dose of study drug and the end of study period. AEs are defined as an untoward medical occurrence that does not necessarily have a causal relationship with study drug treatment. | From first dose date to Day 43 (±4) |
| Number of Participants Who Experienced ≥1 AE, TEAE, Serious AE (SAE), or TEAE of Special Interest (AESI) | AEs are defined as an untoward medical occurrence that does not necessarily have a causal relationship with study drug treatment. A TEAE is defined as an AE with an onset that occurs from the first dose of study drug up until the end of study period. An SAE is an AE that fulfills 1 or more of the following: results in death; is immediately life-threatening; requires inpatient hospitalization or prolongation of existing hospitalization; results in persistent or significant disability or incapacity; results in a congenital abnormality or birth defect; is an important medical event that may jeopardize the participant or may require medical intervention to prevent 1 of the outcomes listed above. Events were categorized as mild, moderate, or severe and as related or unrelated to study drug. AESIs include bleeding events, symptomatic hypotensive events and/or tachycardia, dizziness, syncope, and TEAEs related to change of neurobehaviors (ie, suicidality or euphoria). | From first dose date to Day 43 (±4) |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Chad Glasser, PharmD | Cyclerion Therapeutics, Inc. | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Children's National Hospital of DC | Washington D.C. | District of Columbia | 20010 | United States | ||
| Johns Hopkins University |
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| ID | Title | Description |
|---|---|---|
| FG000 | IW-6463 | Open-label IW-6463 15 mg once daily (QD), with possibility to dose reduce to 10 mg. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Jul 23, 2021 | Jan 26, 2023 |
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Open-label, single-arm study with daily dosing for up to 29 days.
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| Baltimore |
| Maryland |
| 21205 |
| United States |
| Massachusetts General Hospital | Boston | Massachusetts | 02114 | United States |
| Columbia University | New York | New York | 10032 | United States |
| Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | 19104 | United States |
| COMPLETED |
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| NOT COMPLETED |
|
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| ID | Title | Description |
|---|---|---|
| BG000 | IW-6463 | Open-label IW-6463 15 mg QD, with possibility to dose reduce to 10 mg. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | years |
| |||||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||||
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||||
| Race (NIH/OMB) | Count of Participants | Participants |
|
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Number of Participants With Study Drug Dose Reductions or Discontinuations Due to ≥ 1 Treatment Emergent Adverse Event (TEAE) | A TEAE is defined as an adverse event (AE) with an onset that occurs between the first dose of study drug and the end of study period. AEs are defined as an untoward medical occurrence that does not necessarily have a causal relationship with study drug treatment. | Posted | Count of Participants | Participants | No | From first dose date to Day 43 (±4) |
|
|
| |||||||||||||||||||||||||||||||||
| Primary | Number of Participants Who Experienced ≥1 AE, TEAE, Serious AE (SAE), or TEAE of Special Interest (AESI) | AEs are defined as an untoward medical occurrence that does not necessarily have a causal relationship with study drug treatment. A TEAE is defined as an AE with an onset that occurs from the first dose of study drug up until the end of study period. An SAE is an AE that fulfills 1 or more of the following: results in death; is immediately life-threatening; requires inpatient hospitalization or prolongation of existing hospitalization; results in persistent or significant disability or incapacity; results in a congenital abnormality or birth defect; is an important medical event that may jeopardize the participant or may require medical intervention to prevent 1 of the outcomes listed above. Events were categorized as mild, moderate, or severe and as related or unrelated to study drug. AESIs include bleeding events, symptomatic hypotensive events and/or tachycardia, dizziness, syncope, and TEAEs related to change of neurobehaviors (ie, suicidality or euphoria). | Posted | Count of Participants | Participants | From first dose date to Day 43 (±4) |
|
|
From signing of informed consent (all-cause mortality) or first dose date (adverse events) to Day 43 (±4).
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | IW-6463 | Open-label IW-6463 15 mg QD, with possibility to dose reduce to 10 mg. | 0 | 8 | 0 | 8 | 8 | 8 |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Cerebrovascular accident | Nervous system disorders | MedDRA 24.0 | Systematic Assessment | Verbatim term: "metabolic stroke-like event" |
|
| Disturbance in attention | Nervous system disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Dizziness | Nervous system disorders | MedDRA 24.0 | Systematic Assessment |
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| Dysarthria | Nervous system disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Hypoaesthesia | Nervous system disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Abdominal pain upper | Gastrointestinal disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Dyspepsia | Gastrointestinal disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Frequent bowel movements | Gastrointestinal disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Chills | General disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Feeling abnormal | General disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Impaired healing | General disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Oedema peripheral | General disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Blindness transient | Eye disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Vision blurred | Eye disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Fall | Injury, poisoning and procedural complications | MedDRA 24.0 | Systematic Assessment |
| |
| Injury | Injury, poisoning and procedural complications | MedDRA 24.0 | Systematic Assessment |
| |
| Crystal urine present | Investigations | MedDRA 24.0 | Systematic Assessment |
| |
| Oxygen saturation decreased | Investigations | MedDRA 24.0 | Systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Musculoskeletal chest pain | Musculoskeletal and connective tissue disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Myalgia | Musculoskeletal and connective tissue disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Confusional state | Psychiatric disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Sleep disorder | Psychiatric disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Hypertension | Vascular disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Hypotension | Vascular disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Orthostatic hypotension | Vascular disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Postural orthostatic tachycardia syndrome | Cardiac disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Cholelithiasis | Hepatobiliary disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Dehydration | Metabolism and nutrition disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Eye naevus | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 24.0 | Systematic Assessment |
| |
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Nail growth abnormal | Skin and subcutaneous tissue disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Onychoclasis | Skin and subcutaneous tissue disorders | MedDRA 24.0 | Systematic Assessment |
|
PI may publish or disclose the results of the study 24 months after final data lock provided that sponsor can review the publication prior to public release, sponsor can request removal of confidential information of sponsor (not including results of trial), and sponsor can request a publication delay in order to protect potentially patentable information. Furthermore, if a publication committee is developing an initial publication, PI is to delay disclosure until that publication is published.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Cyclerion Clinical Development | Cyclerion Therapeutics, Inc. | 1-857-327-8778 | info@cyclerion.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Mar 21, 2022 | Jan 26, 2023 | SAP_001.pdf |
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| ID | Term |
|---|---|
| D017241 | MELAS Syndrome |
| D028361 | Mitochondrial Diseases |
| ID | Term |
|---|---|
| D017237 | Mitochondrial Encephalomyopathies |
| D017240 | Mitochondrial Myopathies |
| D009135 | Muscular Diseases |
| D009140 | Musculoskeletal Diseases |
| D020739 | Brain Diseases, Metabolic, Inborn |
| D001928 | Brain Diseases, Metabolic |
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D059345 | Cerebral Small Vessel Diseases |
| D002561 | Cerebrovascular Disorders |
| D009468 | Neuromuscular Diseases |
| D014652 | Vascular Diseases |
| D002318 | Cardiovascular Diseases |
| D008661 | Metabolism, Inborn Errors |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
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| Unknown or Not Reported |
|
| Native Hawaiian or Other Pacific Islander |
|
| Black or African American |
|
| White |
|
| More than one race |
|
| Unknown or Not Reported |
|
|