Trial · NCT04641962
A Study to Evaluate ASP0367 in Participants With Primary Mitochondrial Myopathy
NCT04641962, Phase 2, Terminated, Astellas Pharma Inc; results first posted 2025-06-17
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. A Study to Evaluate ASP0367 in Participants With Primary Mitochondrial Myopathy. Trials, record NCT04641962. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04641962
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Study to Evaluate ASP0367 in Participants With Primary Mitochondrial Myopathy
- NCT number
- NCT04641962
- Status
- Terminated
- Phases
- Phase 2
- Enrollment
- 34 participants, actual
- Lead sponsor
- Astellas Pharma Inc
- Conditions
- Primary Mitochondrial Myopathy
- Interventions
- Bocidelpar, Placebo
- First posted
- 2020-11-24
- Last updated
- 2025-07-03
- Results first posted
- 2025-06-17
- Limitations and caveats
The study was terminated due to futility after protocol version 9.0 was introduced. Some participants received treatment for up to 52 weeks, resulting in efficacy reported for up to 24 weeks and safety for up to 52 weeks.
Source ClinicalTrials.gov
Registered outcomes9
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Change From Baseline in Distanced Walked During a 6 Minute Walk Test (6MWT). | Baseline, week 24 | 6MWT assessed functional capacity and endurance by measuring the distance walked in 6 minutes. This test helped to assess exercise tolerance, physical fitness, disease progression, and treatment effectiveness in individuals with myopathy. The total distance walked by a participant, as well as distance per minute was calculated by rounding to the nearest meter. |
| Primary | Number of Participants With Treatment Emergent Adverse Events | From first dose up to week 52 | An Adverse event (AE) was any untoward medical occurrence in a participant administered a study drug, and which dint have to have a causal relationship with this treatment. An AE could therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom or disease (new or exacerbated) temporally associated with the use of a medicinal product, whether or not considered related to the medicinal product. TEAE was defined as an AE observed after starting administration of the study drug through 28 days after the last dose. |
| Primary | Number of Participants With Suicidal Ideation and/ or Behavior as Assessed by Columbia-Suicide Severity Rating Scale (C-SSRS) | From the first dose up to week 52 | The C-SSRS was a questionnaire used for suicide risk assessment. Affirmative or negative responses were provided to items 1 to 5 for suicidal ideation (1. Wish to be dead, 2. Non-specific active suicidal thoughts, 3. Active suicidal ideation with any methods \[not plan\] without intent to act, 4. Active suicidal ideation with some intent to act, without specific plan, 5. Active suicidal ideation with specific plan and intent) and items 6 to 10 for suicide behavior (6. Preparatory acts or behavior, 7. Aborted attempt, 8. Interrupted attempt, 9. Actual attempt, 10. Completed suicide). The overall data was reported. |
| Secondary | Change From Baseline in Quality of Life in Neurological Disorders (Neuro-QoL) Short Form Fatigue Score | Baseline, week 24 | The Neuro-QoL Short Form Fatigue score was an 8-item self- assessment questionnaire evaluating the perception of fatigue and its impact in daily life activities. Participants had five response options for each item: 1=Never, 2=Rarely, 3=Sometimes, 4=Often, 5=Always. The questionnaire was scored by adding the response to each of the item and then transformed into T-scores based on the scoring manual with a score range of minimum=29.5, maximum=74.1. T-score distributions rescaled raw scores into standardized scores with a mean of 50 and a SD of 10. Higher scores indicated a greater level of fatigue. |
| Secondary | Change From Baseline in Neuro-QoL Short Form Lower Extremity Function (Mobility) Scores | Baseline, week 24 | The Neuro-QoL Short Form Lower Extremity Function (Mobility) score was an 8-item self- assessment questionnaire evaluating the functioning of one's lower extremities. Participants responded to questions on a 1-5 scale. Participants had five response options for each item: 1=Unable to do,2=With much difficulty,3=With some difficulty,4=With a little difficulty, 5=Without any difficult. The questionnaire was scored by adding the response to each of the item and then transformed into T-scores based on the scoring manual with a score range of minimum=16.5, maximum=58.6. T-score distributions rescaled raw scores into standardized scores with a mean of 50 and a SD of 10. Higher scores indicated better function. |
| Secondary | Change From Baseline in Time to Perform the 5 Times Sit to Stand (5XSTS) Test | Baseline, week 24 | The 5XSTS test was a functional assessment that measured the time taken to stand up from a seated position five times in a row, as quickly as possible. The duration from the time instructor indicated "Go" until the time participant's body touched the chair following the fifth repetition was recorded and reported. |
| Secondary | Number of Participants With Patient's Global Impression of Change (PGIC) Score at Week 24 | Week 24 | The PGIC scale evaluated the participant's symptom and assessed if there had been any improvement or decline in clinical status. The participant rated their perceived change on a 7-point scale, with 1 indicating very much improved, 2 = Much Improved, 3=Minimally Improved, 4= No Change, 5=Minimally Worse, 6=Much Worse and 7 indicating 'very much worse". |
| Secondary | Number of Participants With Change From Baseline in Patient Global Impression of Severity (PGIS) at Week 24 | Baseline, week 24 | PGIS score was a patient-reported measure that reflected the individual's overall perception of the severity of their condition on a Likert scale. PGIS score were calculated as -1= mild change from baseline, -2=moderate change from baseline,-3=severe change from baseline,0= none, 1= mild, 2= moderate and 3= severe. The questionnaire asked the participant to best describe the severity of the participant's most bothersome pre-defined symptom over the past week. |
| Secondary | Change From Baseline in Modified Fatigue Impact Scale (MFIS) | Baseline, week 24 | MFIS was a self-reported questionnaire designed to evaluate the impact of fatigue on physical, cognitive and psychosocial functioning. The MFIS consisted of 21 items scored 0-4 (0=Never, 1=Rarely, 2=Sometimes, 3=Often, and 4=Almost always). The total MFIS score ranged from 0 to 84, with three subscales: Physical range 0-36, Cognitive range 0-40, and Psychosocial range 0-8. Higher scores indicated higher level of fatigue. |
Source ClinicalTrials.gov
Outcomes9
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Change From Baseline in Distanced Walked During a 6 Minute Walk Test (6MWT). | Baseline, week 24 | Full analysis set (FAS) All randomized participants who received at least 1 dose of study drug or placebo and had at least 1 post-baseline efficacy measurement. FAS with available data was analyzed. | Meters | MEAN |
| PRIMARY | Number of Participants With Treatment Emergent Adverse Events | From first dose up to week 52 | SAF | Participants | NUMBER |
| PRIMARY | Number of Participants With Suicidal Ideation and/ or Behavior as Assessed by Columbia-Suicide Severity Rating Scale (C-SSRS) | From the first dose up to week 52 | SAF | Participants | NUMBER |
| SECONDARY | Change From Baseline in Quality of Life in Neurological Disorders (Neuro-QoL) Short Form Fatigue Score | Baseline, week 24 | FAS with available data was analyzed. | T score | MEAN |
| SECONDARY | Change From Baseline in Neuro-QoL Short Form Lower Extremity Function (Mobility) Scores | Baseline, week 24 | FAS with available data were analyzed. | T scoree | MEAN |
| SECONDARY | Change From Baseline in Time to Perform the 5 Times Sit to Stand (5XSTS) Test | Baseline, week 24 | FAS with available participants were analyzed. | Seconds | MEAN |
| SECONDARY | Number of Participants With Patient's Global Impression of Change (PGIC) Score at Week 24 | Week 24 | FAS with available data were analyzed. | participants | NUMBER |
| SECONDARY | Number of Participants With Change From Baseline in Patient Global Impression of Severity (PGIS) at Week 24 | Baseline, week 24 | FAS with available data were analyzed. | Participants | NUMBER |
| SECONDARY | Change From Baseline in Modified Fatigue Impact Scale (MFIS) | Baseline, week 24 | FAS with available data was analyzed. | Score on a Scale | MEAN |
Source ClinicalTrials.gov
Results by group74
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Change From Baseline in Distanced Walked During a 6 Minute Walk Test (6MWT). | DBT Bocidelpar 30 mg | 25.3 | Standard Deviation 21.4 | Meters | |
| Change From Baseline in Distanced Walked During a 6 Minute Walk Test (6MWT). | DBT Bocidelpar 75 mg | 28.6 | Standard Deviation 73.3 | Meters | |
| Change From Baseline in Distanced Walked During a 6 Minute Walk Test (6MWT). | DBT: Placebo | 17.7 | Standard Deviation 47.8 | Meters | |
| Number of Participants With Treatment Emergent Adverse Events | DBT Bocidelpar 30 mg | 11 | Participants | ||
| Number of Participants With Treatment Emergent Adverse Events | DBT Bocidelpar 75 mg | 11 | Participants | ||
| Number of Participants With Treatment Emergent Adverse Events | Bocidelpar 30 or 75 mg | 22 | Participants | ||
| Number of Participants With Treatment Emergent Adverse Events | DBT: Placebo | 11 | Participants | ||
| Number of Participants With Suicidal Ideation and/ or Behavior as Assessed by Columbia-Suicide Severity Rating Scale (C-SSRS) | DBT Bocidelpar 30 mg | 0 | Participants | ||
| Number of Participants With Suicidal Ideation and/ or Behavior as Assessed by Columbia-Suicide Severity Rating Scale (C-SSRS) | DBT Bocidelpar 75 mg | 0 | Participants | ||
| Number of Participants With Suicidal Ideation and/ or Behavior as Assessed by Columbia-Suicide Severity Rating Scale (C-SSRS) | Bocidelpar 30 or 75 mg | 0 | Participants |
1 to 10 of 74Source ClinicalTrials.gov
See all 74 results by groupParticipant flow30
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| DBT Period: Up to 52 Weeks | STARTED | DBT: Bocidelpar 30 mg | 12 | |
| DBT Period: Up to 52 Weeks | STARTED | DBT: Bocidelpar 75 mg | 11 | |
| DBT Period: Up to 52 Weeks | STARTED | Bocidelpar 30 mg or 75mg to Open Label Extension Period: Bocidelpar 30 mg | 0 | |
| DBT Period: Up to 52 Weeks | STARTED | DBT: Placebo | 11 | |
| DBT Period: Up to 52 Weeks | STARTED | Placebo to OLE Period: Bocidelpar 30 mg | 0 | |
| DBT Period: Up to 52 Weeks | COMPLETED | DBT: Bocidelpar 30 mg | 5 | |
| DBT Period: Up to 52 Weeks | COMPLETED | DBT: Bocidelpar 75 mg | 5 | |
| DBT Period: Up to 52 Weeks | COMPLETED | Bocidelpar 30 mg or 75mg to Open Label Extension Period: Bocidelpar 30 mg | 0 | |
| DBT Period: Up to 52 Weeks | COMPLETED | DBT: Placebo | 3 | |
| DBT Period: Up to 52 Weeks | COMPLETED | Placebo to OLE Period: Bocidelpar 30 mg | 0 |
1 to 10 of 30Source ClinicalTrials.gov
See all 30 participant flowAdverse events630
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Bundle branch block | Cardiac disorders | DBT Placebo | 0 | 11 | 0 |
| other | Bundle branch block | Cardiac disorders | DBT: Bocidelpar 30 mg | 0 | 12 | 0 |
| other | Bundle branch block | Cardiac disorders | DBT Bocidelpar 75 mg | 0 | 11 | 0 |
| other | Bundle branch block | Cardiac disorders | Placebo to Open-label Extension Period: Bocidelpar 30 mg | 0 | 3 | 0 |
| other | Bundle branch block | Cardiac disorders | Bocidelpar 30 mg or 75 mg to Open-label Extension Period: Bocidelpar 30 mg | 1 | 10 | 1 |
| other | Palpitations | Cardiac disorders | DBT Placebo | 0 | 11 | 0 |
| other | Palpitations | Cardiac disorders | DBT: Bocidelpar 30 mg | 1 | 12 | 1 |
| other | Palpitations | Cardiac disorders | DBT Bocidelpar 75 mg | 0 | 11 | 0 |
| other | Palpitations | Cardiac disorders | Placebo to Open-label Extension Period: Bocidelpar 30 mg | 0 | 3 | 0 |
| other | Palpitations | Cardiac disorders | Bocidelpar 30 mg or 75 mg to Open-label Extension Period: Bocidelpar 30 mg | 0 | 10 | 0 |
1 to 10 of 630Source ClinicalTrials.gov
See all 630 adverse eventsAdverse event totals15
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | DBT Placebo | 0 | 11 |
| Total, all-cause mortality | DBT: Bocidelpar 30 mg | 1 | 12 |
| Total, all-cause mortality | DBT Bocidelpar 75 mg | 0 | 11 |
| Total, all-cause mortality | Placebo to Open-label Extension Period: Bocidelpar 30 mg | 0 | 3 |
| Total, all-cause mortality | Bocidelpar 30 mg or 75 mg to Open-label Extension Period: Bocidelpar 30 mg | 1 | 10 |
| Total, other adverse events | DBT Placebo | 11 | 11 |
| Total, other adverse events | DBT: Bocidelpar 30 mg | 11 | 12 |
| Total, other adverse events | DBT Bocidelpar 75 mg | 11 | 11 |
| Total, other adverse events | Placebo to Open-label Extension Period: Bocidelpar 30 mg | 3 | 3 |
| Total, other adverse events | Bocidelpar 30 mg or 75 mg to Open-label Extension Period: Bocidelpar 30 mg | 10 | 10 |
1 to 10 of 15Source ClinicalTrials.gov
See all 15 adverse event totalsPapers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
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