Trial Outcomes & Findings for Phase 2a Study of IW-6463 in Adults Diagnosed With Mitochondrial Encephalomyopathy, Lactic Acidosis, and Stroke-like Episodes (MELAS) (NCT NCT04475549)
NCT ID: NCT04475549
Last Updated: 2024-08-29
Results Overview
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.
Recruitment status
TERMINATED
Study phase
PHASE2
Target enrollment
8 participants
Primary outcome timeframe
From first dose date to Day 43 (±4)
Results posted on
2024-08-29
Participant Flow
Participant milestones
| Measure |
IW-6463
Open-label IW-6463 15 mg once daily (QD), with possibility to dose reduce to 10 mg.
|
|---|---|
|
Overall Study
STARTED
|
8
|
|
Overall Study
COMPLETED
|
8
|
|
Overall Study
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Phase 2a Study of IW-6463 in Adults Diagnosed With Mitochondrial Encephalomyopathy, Lactic Acidosis, and Stroke-like Episodes (MELAS)
Baseline characteristics by cohort
| Measure |
IW-6463
n=8 Participants
Open-label IW-6463 15 mg QD, with possibility to dose reduce to 10 mg.
|
|---|---|
|
Age, Continuous
|
32.8 years
STANDARD_DEVIATION 12.41 • n=5 Participants
|
|
Sex: Female, Male
Female
|
5 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
3 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
8 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
6 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
1 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: From first dose date to Day 43 (±4)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.
Outcome measures
| Measure |
IW-6463
n=8 Participants
Open-label IW-6463 15 mg QD, with possibility to dose reduce to 10 mg.
|
|---|---|
|
Number of Participants With Study Drug Dose Reductions or Discontinuations Due to ≥ 1 Treatment Emergent Adverse Event (TEAE)
Dose reduction due to TEAE
|
1 Participants
|
|
Number of Participants With Study Drug Dose Reductions or Discontinuations Due to ≥ 1 Treatment Emergent Adverse Event (TEAE)
Study drug discontinuation due to TEAE
|
0 Participants
|
PRIMARY outcome
Timeframe: From first dose date to Day 43 (±4)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).
Outcome measures
| Measure |
IW-6463
n=8 Participants
Open-label IW-6463 15 mg QD, with possibility to dose reduce to 10 mg.
|
|---|---|
|
Number of Participants Who Experienced ≥1 AE, TEAE, Serious AE (SAE), or TEAE of Special Interest (AESI)
Any AE
|
8 Participants
|
|
Number of Participants Who Experienced ≥1 AE, TEAE, Serious AE (SAE), or TEAE of Special Interest (AESI)
Any pretreatment AE
|
1 Participants
|
|
Number of Participants Who Experienced ≥1 AE, TEAE, Serious AE (SAE), or TEAE of Special Interest (AESI)
Any TEAE
|
8 Participants
|
|
Number of Participants Who Experienced ≥1 AE, TEAE, Serious AE (SAE), or TEAE of Special Interest (AESI)
Any severe TEAE
|
0 Participants
|
|
Number of Participants Who Experienced ≥1 AE, TEAE, Serious AE (SAE), or TEAE of Special Interest (AESI)
Any study drug-related TEAE
|
4 Participants
|
|
Number of Participants Who Experienced ≥1 AE, TEAE, Serious AE (SAE), or TEAE of Special Interest (AESI)
Any SAE
|
0 Participants
|
|
Number of Participants Who Experienced ≥1 AE, TEAE, Serious AE (SAE), or TEAE of Special Interest (AESI)
Any TEAE leading to study drug discontinuation
|
0 Participants
|
|
Number of Participants Who Experienced ≥1 AE, TEAE, Serious AE (SAE), or TEAE of Special Interest (AESI)
Any TEAE leading to death
|
0 Participants
|
|
Number of Participants Who Experienced ≥1 AE, TEAE, Serious AE (SAE), or TEAE of Special Interest (AESI)
Any treatment-emergent AESI
|
4 Participants
|
Adverse Events
IW-6463
Serious events: 0 serious events
Other events: 8 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
IW-6463
n=8 participants at risk
Open-label IW-6463 15 mg QD, with possibility to dose reduce to 10 mg.
|
|---|---|
|
Nervous system disorders
Cerebrovascular accident
|
12.5%
1/8 • From signing of informed consent (all-cause mortality) or first dose date (adverse events) to Day 43 (±4).
|
|
Nervous system disorders
Disturbance in attention
|
12.5%
1/8 • From signing of informed consent (all-cause mortality) or first dose date (adverse events) to Day 43 (±4).
|
|
Nervous system disorders
Dizziness
|
12.5%
1/8 • From signing of informed consent (all-cause mortality) or first dose date (adverse events) to Day 43 (±4).
|
|
Nervous system disorders
Dysarthria
|
12.5%
1/8 • From signing of informed consent (all-cause mortality) or first dose date (adverse events) to Day 43 (±4).
|
|
Nervous system disorders
Headache
|
37.5%
3/8 • From signing of informed consent (all-cause mortality) or first dose date (adverse events) to Day 43 (±4).
|
|
Nervous system disorders
Hypoaesthesia
|
12.5%
1/8 • From signing of informed consent (all-cause mortality) or first dose date (adverse events) to Day 43 (±4).
|
|
Gastrointestinal disorders
Abdominal pain upper
|
12.5%
1/8 • From signing of informed consent (all-cause mortality) or first dose date (adverse events) to Day 43 (±4).
|
|
Gastrointestinal disorders
Constipation
|
12.5%
1/8 • From signing of informed consent (all-cause mortality) or first dose date (adverse events) to Day 43 (±4).
|
|
Gastrointestinal disorders
Diarrhoea
|
12.5%
1/8 • From signing of informed consent (all-cause mortality) or first dose date (adverse events) to Day 43 (±4).
|
|
Gastrointestinal disorders
Dyspepsia
|
12.5%
1/8 • From signing of informed consent (all-cause mortality) or first dose date (adverse events) to Day 43 (±4).
|
|
Gastrointestinal disorders
Frequent bowel movements
|
12.5%
1/8 • From signing of informed consent (all-cause mortality) or first dose date (adverse events) to Day 43 (±4).
|
|
Gastrointestinal disorders
Vomiting
|
12.5%
1/8 • From signing of informed consent (all-cause mortality) or first dose date (adverse events) to Day 43 (±4).
|
|
General disorders
Chills
|
12.5%
1/8 • From signing of informed consent (all-cause mortality) or first dose date (adverse events) to Day 43 (±4).
|
|
General disorders
Feeling abnormal
|
12.5%
1/8 • From signing of informed consent (all-cause mortality) or first dose date (adverse events) to Day 43 (±4).
|
|
General disorders
Impaired healing
|
12.5%
1/8 • From signing of informed consent (all-cause mortality) or first dose date (adverse events) to Day 43 (±4).
|
|
General disorders
Oedema peripheral
|
25.0%
2/8 • From signing of informed consent (all-cause mortality) or first dose date (adverse events) to Day 43 (±4).
|
|
Eye disorders
Blindness transient
|
12.5%
1/8 • From signing of informed consent (all-cause mortality) or first dose date (adverse events) to Day 43 (±4).
|
|
Eye disorders
Vision blurred
|
12.5%
1/8 • From signing of informed consent (all-cause mortality) or first dose date (adverse events) to Day 43 (±4).
|
|
Injury, poisoning and procedural complications
Fall
|
12.5%
1/8 • From signing of informed consent (all-cause mortality) or first dose date (adverse events) to Day 43 (±4).
|
|
Injury, poisoning and procedural complications
Injury
|
12.5%
1/8 • From signing of informed consent (all-cause mortality) or first dose date (adverse events) to Day 43 (±4).
|
|
Investigations
Crystal urine present
|
12.5%
1/8 • From signing of informed consent (all-cause mortality) or first dose date (adverse events) to Day 43 (±4).
|
|
Investigations
Oxygen saturation decreased
|
12.5%
1/8 • From signing of informed consent (all-cause mortality) or first dose date (adverse events) to Day 43 (±4).
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
12.5%
1/8 • From signing of informed consent (all-cause mortality) or first dose date (adverse events) to Day 43 (±4).
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal chest pain
|
12.5%
1/8 • From signing of informed consent (all-cause mortality) or first dose date (adverse events) to Day 43 (±4).
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
12.5%
1/8 • From signing of informed consent (all-cause mortality) or first dose date (adverse events) to Day 43 (±4).
|
|
Psychiatric disorders
Confusional state
|
12.5%
1/8 • From signing of informed consent (all-cause mortality) or first dose date (adverse events) to Day 43 (±4).
|
|
Psychiatric disorders
Sleep disorder
|
12.5%
1/8 • From signing of informed consent (all-cause mortality) or first dose date (adverse events) to Day 43 (±4).
|
|
Vascular disorders
Hypertension
|
12.5%
1/8 • From signing of informed consent (all-cause mortality) or first dose date (adverse events) to Day 43 (±4).
|
|
Vascular disorders
Hypotension
|
12.5%
1/8 • From signing of informed consent (all-cause mortality) or first dose date (adverse events) to Day 43 (±4).
|
|
Vascular disorders
Orthostatic hypotension
|
12.5%
1/8 • From signing of informed consent (all-cause mortality) or first dose date (adverse events) to Day 43 (±4).
|
|
Cardiac disorders
Postural orthostatic tachycardia syndrome
|
12.5%
1/8 • From signing of informed consent (all-cause mortality) or first dose date (adverse events) to Day 43 (±4).
|
|
Hepatobiliary disorders
Cholelithiasis
|
12.5%
1/8 • From signing of informed consent (all-cause mortality) or first dose date (adverse events) to Day 43 (±4).
|
|
Metabolism and nutrition disorders
Dehydration
|
12.5%
1/8 • From signing of informed consent (all-cause mortality) or first dose date (adverse events) to Day 43 (±4).
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Eye naevus
|
12.5%
1/8 • From signing of informed consent (all-cause mortality) or first dose date (adverse events) to Day 43 (±4).
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
|
12.5%
1/8 • From signing of informed consent (all-cause mortality) or first dose date (adverse events) to Day 43 (±4).
|
|
Skin and subcutaneous tissue disorders
Nail growth abnormal
|
12.5%
1/8 • From signing of informed consent (all-cause mortality) or first dose date (adverse events) to Day 43 (±4).
|
|
Skin and subcutaneous tissue disorders
Onychoclasis
|
12.5%
1/8 • From signing of informed consent (all-cause mortality) or first dose date (adverse events) to Day 43 (±4).
|
Additional Information
Cyclerion Clinical Development
Cyclerion Therapeutics, Inc.
Phone: 1-857-327-8778
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee 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.
- Publication restrictions are in place
Restriction type: OTHER