| Primary | Part 2- Frequency of Adverse Events - Presence and Nature of Adverse Events (AE) During Part 2 | The number of participants that had a least one Treatment Emergent Adverse Event during the Part 2 of the study. The pre-specified analysis for this outcome measure was for those participants in the part 2 cohorts of this study therefore, only data of part 2 portion of the study are reported. | The Safety Population consisted of all participants enrolled/randomized in the study who received at least 1 dose of study drug. | Posted | | Count of Participants | | Participants | | From baseline to end of participation of the Part 2 portion of the study | | | | ID | Title | Description |
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| OG000 | Part 2 Cohort 2a | ACE-083 Administered into the TA muscle q4w for up to 24 months (24 doses) for FSHD patients ACE-083: Recombinant fusion protein | | OG001 | Part 2 Cohort 2b | ACE-083 Administered into the BB muscle q4w for up to 24 months (24 doses) for FSHD patients ACE-083: Recombinant fusion protein | | OG002 | Part 2 Cohort 2c | ACE-083 Administered into the TA muscle q4w for up to 24 months (24 doses) for CMT patients ACE-083: Recombinant fusion protein | | OG003 | Part 2 Cohort 3a | ACE-083 Administered into the TA muscle q8w for up to 24 months (12 doses) for FSHD patients ACE-083: Recombinant fusion protein | | OG004 | Part 2 Cohort 3b | ACE-083 Administered into the BB muscle q8w for up to 24 months (12 doses) for FSHD patients ACE-083: Recombinant fusion protein | | OG005 | Part 2 Cohort 3c | ACE-083 Administered into the TA muscle q8w for up to 24 months (12 doses) for CMT patients ACE-083: Recombinant fusion protein |
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| Primary | Part 2: Frequency of Adverse Events - Presence and Nature of Grade 3 or Higher Adverse Events (AE) During the Part 2 of the Study. | The number of participants that had a least one grade 3 or higher Treatment Emergent Adverse Event during Part 2 of the study. The pre-specified analysis for this outcome measure was for those participants in the double-blind, placebo-controlled Part 2 of the study therefore, only data from the part 2 portion of the study are reported. | The Safety Population consisted of all participants enrolled/randomized in the study who received at least 1 dose of study drug. | Posted | | Count of Participants | | Participants | | From baseline to the end of the part 2 portion of the study | | | | ID | Title | Description |
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| OG000 | Part 2 Cohort 2a | ACE-083 Administered into the TA muscle q4w for up to 24 months (24 doses) for FSHD patients ACE-083: Recombinant fusion protein | | OG001 | Part 2 Cohort 2b | ACE-083 Administered into the BB muscle q4w for up to 24 months (24 doses) for FSHD patients ACE-083: Recombinant fusion protein | | OG002 | Part 2 Cohort 2c | ACE-083 Administered into the TA muscle q4w for up to 24 months (24 doses) for CMT patients ACE-083: Recombinant fusion protein |
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| Primary | Part 2: Change in Total Muscle Volume - Percent Change From Baseline to Day 113 in Total Muscle Volume of Injected Muscle by Magnetic Resonance Imaging (MRI) During the Part 2 Portion | The primary pharmacodynamic variable was the difference in mean percent change in total muscle volume (average of left and right side) at the first 6 months of the maintenance phase (Day 169; q4w or q8w) from the total muscle volume (average of left and right sides) at the start of the maintenance phase (or equivalently the end of the loading phase). Due to the early termination of this trial, percent change is only able to be reported as percent change from baseline to Day 113. The pre-specified analysis for this outcome measure was for those participants in the part 2 portion in cohorts with data to Day 113. Therefore, only data from these part 2 arms- 2b, 2c, 3b and 3c of the study are reported. | Per Protocol Set (PPS) consisted of all participants enrolled/randomized in the study, who received at least 1 dose of study drug with no clinical study report-reportable protocol violations and at least 1 post-baseline MRI evaluation. | Posted | | Mean | Standard Error | percent change | | During the Part 2 portion of the study: Baseline to Day 113 | | | | ID | Title | Description |
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| OG000 | Part 2 Cohort 2b | ACE-083 Administered into the BB muscle q4w for up to 24 months (24 doses) for FSHD patients ACE-083: Recombinant fusion protein | | OG001 | Part 2 Cohort 2c | ACE-083 Administered into the TA muscle q4w for up to 24 months (24 doses) for CMT patients ACE-083: Recombinant fusion protein |
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| Secondary | Part 2: Change in Muscle Function. Absolute Change From Baseline in Functional Assessment for Tibialis Anterior (TA) Muscle:10-meter Walk/Run During Part 2 of the Study | Timing (in seconds) participants took to complete a 10-meter walk/run distance. Baseline and end of treatment visit values were used to report absolute change from baseline in functional assessment due to early termination of the trial. The pre-specified analysis for this outcome measure was for those participants with data available at baseline and at the end of treatment in part 2 of this study therefore, only data from the part 2 cohorts 2a, 2c and 3a of the study are reported. | Per Protocol Set: All patients enrolled/randomized in the study who have received at least one dose of study drug (includes placebo) with no major protocol violations. | Posted | | Mean | Standard Error | seconds | | Baseline and End of Treatment visit for Part 2 of the study | | | | ID | Title | Description |
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| OG000 | Part 2 Cohort 2a | ACE-083 Administered into the TA muscle q4w for up to 24 months (24 doses) for FSHD patients ACE-083: Recombinant fusion protein | | OG001 | Part 2 Cohort 2c | ACE-083 Administered into the TA muscle q4w for up to 24 months (24 doses) for CMT patients ACE-083: Recombinant fusion protein | | OG002 | Part 2 Cohort 3a |
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| Secondary | Part 2: Change in Muscle Function. Percent Change From Baseline in Functional Assessment for Tibialis Anterior (TA) Muscle:10-meter Walk/Run During Part 2 of the Study | Timing (in seconds) participants took to complete a 10-meter walk/run distance. Baseline and end of treatment visit values were used to report absolute change from baseline in functional assessment due to early termination of the trial. The pre-specified analysis for this outcome measure was for those participants with data available at baseline and at the end of treatment in part 2 of this study therefore, only data from the part 2 cohorts 2a, 2c and 3a of the study are reported. | Per Protocol Set: All patients enrolled/randomized in the study who have received at least one dose of study drug (includes placebo) with no major protocol violations. | Posted | | Mean | Standard Error | percent change | | Baseline and End of Treatment visit for Part 2 of the study | | | | ID | Title | Description |
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| OG000 | Part 2 Cohort 2a | ACE-083 Administered into the TA muscle q4w for up to 24 months (24 doses) for FSHD patients ACE-083: Recombinant fusion protein | | OG001 | Part 2 Cohort 2c | ACE-083 Administered into the TA muscle q4w for up to 24 months (24 doses) for CMT patients ACE-083: Recombinant fusion protein | | OG002 | Part 2 Cohort 3a |
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| Secondary | Part 2: Change in Muscle Function. Absolute Change From Baseline in Functional Assessment for Tibialis Anterior (TA) Muscle: 6-minute Walk Test | During Part 2: for the 6-minute walk test, participants walked at normal pace for six minutes and the distance was measured in meters. Absolute change from baseline to end of treatment in phase 2 were reported since this trial was terminated early. Further, only part 2 cohorts 2a, 2c, and 3a had data to report for this analysis. | Per Protocol Set (PPS) consisted of all participants enrolled/randomized in the study, who received at least 1 dose of study drug with no clinical study report-reportable protocol violations and at least 1 post-baseline MRI evaluation. | Posted | | Mean | Standard Error | meters | | Baseline and End of Treatment Visit during Part 2 of the study | | | | ID | Title | Description |
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| OG000 | Part 2 Cohort 2a | ACE-083 Administered into the TA muscle q4w for up to 24 months (24 doses) for FSHD patients ACE-083: Recombinant fusion protein | | OG001 | Part 2 Cohort 2c | ACE-083 Administered into the TA muscle q4w for up to 24 months (24 doses) for CMT patients ACE-083: Recombinant fusion protein | | OG002 | Part 2 Cohort 3a | ACE-083 Administered into the TA muscle q8w for up to 24 months (12 doses) for FSHD patients ACE-083: Recombinant fusion protein |
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| Secondary | Part 2: Change in Muscle Function - Percent Change From Baseline in Functional Assessment for Tibialis Anterior (TA) Muscle in 6-minute Walk Test | During Part 2: for the 6-minute walk test, participants walked at normal pace for six minutes and the distance was measured in meters. Absolute change from baseline to end of treatment in phase 2 were reported since this trial was terminated early. Further, only part 2 cohorts 2a, 2c, and 3c had data to report for this analysis. | Per Protocol Set (PPS) consisted of all participants enrolled/randomized in the study, who received at least 1 dose of study drug with no clinical study report-reportable protocol violations. | Posted | | Mean | Standard Error | percent change | | Baseline and End of Treatment visit during Part 2 of the study | | | | ID | Title | Description |
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| OG000 | Part 2 Cohort 2a | ACE-083 Administered into the TA muscle q4w for up to 24 months (24 doses) for FSHD patients ACE-083: Recombinant fusion protein | | OG001 | Part 2 Cohort 2c | ACE-083 Administered into the TA muscle q4w for up to 24 months (24 doses) for CMT patients ACE-083: Recombinant fusion protein | | OG002 | Part 2 Cohort 3c | ACE-083 Administered into the TA muscle q8w for up to 24 months (12 doses) for FSHD patients ACE-083: Recombinant fusion protein |
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| Secondary | Part 2: Change in Muscle Function. Absolute Change From Baseline in Functional Assessment for Tibialis Anterior (TA) Muscle: 4-stair Climb Up (in Participants With FSHD Only) | During Part 2 in FSHD participants only: This is a measure (in seconds) of participants going up 4 standard stairs. Absolute Change is compared from baseline to the end of treatment visit as this trial was terminated early. As this analysis was pre-specified for FSHD patients only, data is only reported for participants with FSHD (cohorts 2a and 3a). | Per Protocol Set (PPS) consisted of all participants enrolled/randomized in the study, who received at least 1 dose of study drug with no clinical study report-reportable protocol violations. | Posted | | Mean | Standard Error | seconds | | Baseline, End of Treatment visit during Part 2 | | | | ID | Title | Description |
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| OG000 | Part 2 Cohort 2a | ACE-083 Administered into the TA muscle q4w for up to 24 months (24 doses) for FSHD patients ACE-083: Recombinant fusion protein | | OG001 | Part 2 Cohort 3a | ACE-083 Administered into the TA muscle q8w for up to 24 months (12 doses) for FSHD patients ACE-083: Recombinant fusion protein |
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| Secondary | Part 2: Change in Muscle Function - Percent Change From Baseline in Functional Assessment for Tibialis Anterior (TA) Muscle in 4-stair Climb Up (in Participants With FSHD Only) | During Part 2 in FSHD participants only: This is a measure (in seconds) of participants going up 4 standard stairs. Percent Change is compared from baseline to the end of treatment visit as this trial was terminated early. As this analysis was pre-specified for FSHD patients only, data is only reported for participants with FSHD (cohorts 2a and 3a). | Per Protocol Set (PPS) consisted of all participants enrolled/randomized in the study, who received at least 1 dose of study drug with no clinical study report-reportable protocol violations. | Posted | | Mean | Standard Error | percent change | | Baseline, End of Treatment visit during Part 2 | | | | ID | Title | Description |
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| OG000 | Part 2 Cohort 2a | ACE-083 Administered into the TA muscle q4w for up to 24 months (24 doses) for FSHD patients ACE-083: Recombinant fusion protein | | OG001 | Part 2 Cohort 3a | ACE-083 Administered into the TA muscle q8w for up to 24 months (12 doses) for FSHD patients ACE-083: Recombinant fusion protein |
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| Secondary | Part 2: Change in Muscle Function. Absolute Change From Baseline in Functional Assessment for Tibialis Anterior (TA) Muscle: 4-stair Climb Down (in Participants With FSHD Only) | During Part 2 (in FSHD participants only)This is a measure (in seconds) of participants going down 4 standard stairs. Absolute Change is compared from baseline to the end of treatment visit as this trial was terminated early. As this analysis was pre-specified for FSHD patients only, data is only reported for participants with FSHD (cohorts 2a and 3a). | Per Protocol Set (PPS) consisted of all participants enrolled/randomized in the study, who received at least 1 dose of study drug with no clinical study report-reportable protocol violations. | Posted | | Mean | Standard Error | seconds | | Baseline, End of Treatment visit during Part 2 | | | | ID | Title | Description |
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| OG000 | Part 2 Cohort 2a | ACE-083 Administered into the TA muscle q4w for up to 24 months (24 doses) for FSHD patients ACE-083: Recombinant fusion protein | | OG001 | Part 2 Cohort 3a | ACE-083 Administered into the TA muscle q8w for up to 24 months (12 doses) for FSHD patients ACE-083: Recombinant fusion protein |
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| Secondary | Part 2: Change in Muscle Function. Percent Change From Baseline in Functional Assessment for Tibialis Anterior (TA) Muscle: 4-stair Climb Down (in Patients With FSHD Only) | During Part 2 (in FSHD participants only)This is a measure (in seconds) of participants going down 4 standard stairs. Percent Change is compared from baseline to the end of treatment visit as this trial was terminated early. As this analysis was pre-specified for FSHD patients only, data is only reported for participants with FSHD (cohorts 2a and 3a). | Per Protocol Set (PPS) consisted of all participants enrolled/randomized in the study, who received at least 1 dose of study drug with no clinical study report-reportable protocol violations. | Posted | | Mean | Standard Error | percent change | | Baseline, End of Treatment visit during Part 2 | | | | ID | Title | Description |
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| OG000 | Part 2 Cohort 2a | ACE-083 Administered into the TA muscle q4w for up to 24 months (24 doses) for FSHD patients ACE-083: Recombinant fusion protein | | OG001 | Part 2 Cohort 3a | ACE-083 Administered into the TA muscle q8w for up to 24 months (12 doses) for FSHD patients ACE-083: Recombinant fusion protein |
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| Secondary | Part 2: Change in Muscle Function. Absolute Change From Baseline in Functional Assessment for Tibialis Anterior (TA) Muscle: 100-meter Timed Test | During Part 2 this measure is the timing (in seconds) to complete 100-meter walk test. Percent Change is reported from baseline to the end of treatment visit as this trial was terminated early. As this analysis was pre-specified for part 2 only, data is only reported for participants with FSHD and CMT in part 2 with data for both timepoints (cohorts 2a, 2c and 3a). | Per Protocol Set (PPS) consisted of all participants enrolled/randomized in the study, who received at least 1 dose of study drug with no clinical study report-reportable protocol violations. | Posted | | Mean | Standard Error | seconds | | Baseline, End of Treatment visit for Part 2 of the study | | | | ID | Title | Description |
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| OG000 | Part 2 Cohort 2a | ACE-083 Administered into the TA muscle q4w for up to 24 months (24 doses) for FSHD patients ACE-083: Recombinant fusion protein | | OG001 | Part 2 Cohort 2c | ACE-083 Administered into the TA muscle q4w for up to 24 months (24 doses) for CMT patients ACE-083: Recombinant fusion protein | | OG002 | Part 2 Cohort 3a | ACE-083 Administered into the TA muscle q8w for up to 24 months (12 doses) for FSHD patients ACE-083: Recombinant fusion protein |
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| Secondary | Part 2: Change in Muscle Function - Percent Change From Baseline for Tibialis Anterior (TA) Muscle in 100-meter Timed Test | During Part 2 this measure is the timing (in seconds) to complete 100-meter walk test. Percent Change is reported from baseline to the end of treatment visit as this trial was terminated early. As this analysis was pre-specified for part 2 only, data is only reported for participants with FSHD and CMT in part 2 with data for both timepoints (cohorts 2a, 2c and 3a). | Per Protocol Set (PPS) consisted of all participants enrolled/randomized in the study, who received at least 1 dose of study drug with no clinical study report-reportable protocol violations. | Posted | | Mean | Standard Error | percent change | | Baseline, End of Treatment visit, Part 2 | | | | ID | Title | Description |
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| OG000 | Part 2 Cohort 2a | ACE-083 Administered into the TA muscle q4w for up to 24 months (24 doses) for FSHD patients ACE-083: Recombinant fusion protein | | OG001 | Part 2 Cohort 2c | ACE-083 Administered into the TA muscle q4w for up to 24 months (24 doses) for CMT patients ACE-083: Recombinant fusion protein | | OG002 | Part 2 Cohort 3a | ACE-083 Administered into the TA muscle q8w for up to 24 months (12 doses) for FSHD patients ACE-083: Recombinant fusion protein |
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| Secondary | Part 2: Change in Muscle Function. Absolute Change From Baseline in Functional Assessment for Biceps Brachii (BB) Muscle: Mid-level Performance of the Upper Limb (PUL) Test. | Part 2: Performance of upper limb (PUL) - Middle level elbow dimension score- Sum of the 9 items for this particular dimension (Minimum=0, Maximum = 34, with higher scores indicating increased functionality). These include: "Hand(s) to Mouth", "Hand(s) to table from lap", "Move weight on table", "Lifting light cans", "Lifting heavy cans", "Stacking light cans", "Stacking heavy cans", "Remove lid from container", and "Tearing paper". Pre-specified analysis was for absolute change in BB cohorts for FSHD participants only with Baseline and End of Treatment visit data (cohorts 2b and 3b) due to the early termination of the study. Thus, data is only reported for these two cohorts. | Per Protocol Set (PPS) consisted of all participants enrolled/randomized in the study, who received at least 1 dose of study drug with no clinical study report-reportable protocol violations. | Posted | | Mean | Standard Error | units on a scale | | Baseline, End of Treatment visit during Part 2 | | | | ID | Title | Description |
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| OG000 | Part 2 Cohort 2b | ACE-083 Administered into the BB muscle q4w for up to 24 months (24 doses) for FSHD patients ACE-083: Recombinant fusion protein | | OG001 | Part 2 Cohort 3b | ACE-083 Administered into the BB muscle q8w for up to 24 months (12 doses) for FSHD patients ACE-083: Recombinant fusion protein |
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| Secondary | Part 2: Change in Muscle Function - Percent Change From Baseline for Biceps Brachii (BB) Muscle in Mid-level Performance of the Upper Limb (PUL) Test | Part 2: Performance of upper limb (PUL) - Middle level elbow dimension score- Sum of the 9 items for this particular dimension (Minimum=0, Maximum = 34, with higher scores indicating increased functionality). These include: "Hand(s) to Mouth", "Hand(s) to table from lap", "Move weight on table", "Lifting light cans", "Lifting heavy cans", "Stacking light cans", "Stacking heavy cans", "Remove lid from container", and "Tearing paper". Pre-specified analysis was for percent change in BB cohorts for FSHD participants only with Baseline and End of Treatment visit data (cohorts 2b and 3b) due to the early termination of the study. Thus, data is only reported for these two cohorts. | Per Protocol Set (PPS) consisted of all participants enrolled/randomized in the study, who received at least 1 dose of study drug with no clinical study report-reportable protocol violations. | Posted | | Mean | Standard Error | percent change in units on scale | | Baseline, End of Treatment | | | | ID | Title | Description |
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| OG000 | Part 2 Cohort 2b | ACE-083 Administered into the BB muscle q4w for up to 24 months (24 doses) for FSHD patients ACE-083: Recombinant fusion protein | | OG001 | Part 2 Cohort 3b | ACE-083 Administered into the BB muscle q8w for up to 24 months (12 doses) for FSHD patients ACE-083: Recombinant fusion protein |
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| Secondary | Part 2: Change in Muscle Function. Absolute Change From Baseline in Functional Assessment for Biceps Brachii (BB) Muscle: Upper Level Performance of the Upper Limb (PUL) Test. | Performance of upper limb (PUL) -PUL upper level/shoulder dimension- Shoulder domain score which represents the sum of the following 4 items that the patient is asked to do on the preferred side [either right or left side identified by patient to be done on the same side for all scheduled times]: a. Largest weight patient can use to perform shoulder abduction to shoulder height (elbow to shoulder level) b. Largest weight patient can use to perform shoulder abduction above shoulder height (elbow to eye level) c. Largest weight patient can use to perform shoulder flexion to shoulder height (elbow to shoulder level) d. Largest weight patient can use to perform shoulder flexion above shoulder height (elbow to eye level) Each of the 4 components above is scored depending on the highest weight performed where scores are: 0 = Unable; 1 = Able no weights; 2 = 200 g; 3 = 500 g; 4 = 1000 g. The maximum score is 16, with 16 being highest functionality. Reportable data is for Cohort 3b only. | Per Protocol Set (PPS) consisted of all participants enrolled/randomized in the study, who received at least 1 dose of study drug with no clinical study report-reportable protocol violations. | Posted | | Mean | Standard Error | units on a scale | | Baseline, End of Treatment visit during part 2 of the study | | | | ID | Title | Description |
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| OG000 | Part 2 Cohort 3b | ACE-083 Administered into the BB muscle q8w for up to 24 months (12 doses) for FSHD patients ACE-083: Recombinant fusion protein |
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| Secondary | Part 2: Change in Muscle Function. Percent Change From Baseline in Functional Assessment for Biceps Brachii (BB) Muscle: Upper Level Performance of the Upper Limb (PUL) Test. | Performance of upper limb (PUL) -PUL upper level/shoulder dimension- Shoulder domain score which represents the sum of the following 4 items that the patient is asked to do on the preferred side [either right or left side identified by patient to be done on the same side for all scheduled times]: a. Largest weight patient can use to perform shoulder abduction to shoulder height (elbow to shoulder level) b. Largest weight patient can use to perform shoulder abduction above shoulder height (elbow to eye level) c. Largest weight patient can use to perform shoulder flexion to shoulder height (elbow to shoulder level) d. Largest weight patient can use to perform shoulder flexion above shoulder height (elbow to eye level) Each of the 4 components above is scored depending on the highest weight performed where scores are: 0 = Unable; 1 = Able no weights; 2 = 200 g; 3 = 500 g; 4 = 1000 g. The maximum score is 16, with 16 being highest functionality. Reportable data is for Cohort 3b only. | Per Protocol Set (PPS) consisted of all participants enrolled/randomized in the study, who received at least 1 dose of study drug with no clinical study report-reportable protocol violations. | Posted | | Mean | Standard Error | percent change units on a scale | | Baseline, End of Treatment visit during Part 2 | | | | ID | Title | Description |
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| OG000 | Part 2 Cohort 3b | ACE-083 Administered into the BB muscle q8w for up to 24 months (12 doses) for FSHD patients ACE-083: Recombinant fusion protein |
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| Secondary | Part 2: Change in Patient-reported Quality of Life. Absolute Change From Baseline in FSHD-health Index Total Score (FSHD-HI, in Patients With FSHD Only) | The FSHD Health Index (FSHD-HI) is a disease-specific patient reported outcome questionnaire that uses direct patient input to measure disease burden. For this index, the total score is scored from 0 to 100 with 0 representing no disease burden and 100 representing the maximum amount of disease burden in the particular domain. Pre-specified analysis was for FSHD cohorts only with absolute change in total score reported as this trial was terminated early. | Per Protocol Set (PPS) consisted of all participants enrolled/randomized in the study, who received at least 1 dose of study drug with no clinical study report-reportable protocol violations. | Posted | | Mean | Standard Error | units on a scale | | Baseline, End of Treatment visit during part 2 | | | | ID | Title | Description |
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| OG000 | Part 2 Cohort 2a | ACE-083 Administered into the TA muscle q4w for up to 24 months (24 doses) for FSHD patients ACE-083: Recombinant fusion protein | | OG001 | Part 2 Cohort 2b | ACE-083 Administered into the BB muscle q4w for up to 24 months (24 doses) for FSHD patients ACE-083: Recombinant fusion protein | | OG002 | Part 2 Cohort 3a |
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| Secondary | Part2: Change in Patient-reported Quality of Life. Absolute Change From Baseline in CMT Health Index Total Score (CMT-HI, in Patients With CMT Only) | Part 2: The CMT-Health Index (CMT-HI) is a disease-specific patient reported outcome measure designed to measure patient reported disease burden during clinical trials in patients with Charcot-Marie-Tooth Disease. For this index, the total score is scored from 0 to 100 with 0 representing no disease burden and 100 representing the maximum amount of disease burden. Pre-specified analysis was for cohorts of CMT participants only and due to the early termination of this trial, the absolute change is reported for the change from Baseline to Day 113. | Per Protocol Set (PPS) consisted of all participants enrolled/randomized in the study, who received at least 1 dose of study drug with no clinical study report-reportable protocol violations. | Posted | | Mean | Standard Error | units on a scale | | Baseline, Day 113 during Part 2 | | | | ID | Title | Description |
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| OG000 | Part 2 Cohort 2c | ACE-083 Administered into the TA muscle q4w for up to 24 months (24 doses) for CMT patients ACE-083: Recombinant fusion protein | | OG001 | Part 2 Cohort 3c | ACE-083 Administered into the TA muscle q8w for up to 24 months (12 doses) for CMT patients ACE-083: Recombinant fusion protein |
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| Secondary | Part 1: ACE-083 Serum Concentration Samples Part 1-Day1, 24-hour Post-dose | Part1: ACE-083 serum concentration samples were taken on day 1, 24-hours post-dose. PK parameters of ACE-083 were not determined due to limited quantifiable concentration data. Pre-specified analysis was for part 1 cohorts only, therefore, available data is only reported for part 1 cohorts (1a, 1b and 1c). Mean and standard deviation descriptive statistics are being reported for the Day 1, 24-hour post-dose timepoint only, due to early termination of this trial. | The PK Population consisted of all participants who received at least 1 dose of study drug and had sufficient PK samples collected and assayed for PK analysis. | Posted | | Mean | Standard Deviation | ng/mL | | day 1, 24 -hours post-dose in Part 1 | | | | ID | Title | Description |
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| OG000 | Part 1 Cohort 1a | ACE-083 240 mg/muscle administered bilaterally by injection into the TA muscle every 4 weeks for up to 6 doses for FSHD patients ACE-083: Recombinant fusion protein | | OG001 | Part 1 Cohort 1b | ACE-083 240 mg/muscle administered bilaterally by injection into the BB muscle every 4 weeks for up to 6 doses for FSHD patients ACE-083: Recombinant fusion protein | | OG002 | Part 1 Cohort 1c | |
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| Secondary | Part1: ACE-083 Serum Concentration Samples Part 1-Day 85, 24-hour Post-dose | Part1: ACE-083 serum concentration samples were taken on day 85, 24-hours post-dose. PK parameters of ACE-083 were not determined due to limited quantifiable concentration data. Pre-specified analysis was for part 1 cohorts only, therefore, available data is only reported for part 1 cohorts (1a, 1b and 1c). Mean and standard deviation descriptive statistics are being reported for the Day 85, 24-hour post-dose timepoint only, due to early termination of this trial. | The PK Population consisted of all participants who received at least 1 dose of study drug and had sufficient PK samples collected and assayed for PK analysis. | Posted | | Mean | Standard Deviation | ng/mL | | day 85, 24 -hours post-dose in Part 1 | | | | ID | Title | Description |
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| OG000 | Part 1 Cohort 1a | ACE-083 240 mg/muscle administered bilaterally by injection into the TA muscle every 4 weeks for up to 6 doses for FSHD patients ACE-083: Recombinant fusion protein | | OG001 | Part 1 Cohort 1b | ACE-083 240 mg/muscle administered bilaterally by injection into the BB muscle every 4 weeks for up to 6 doses for FSHD patients ACE-083: Recombinant fusion protein | | OG002 | Part 1 Cohort 1c | |
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| Secondary | Part1: Pharmacokinetics Parameter of Time to Maximum Serum Concentration Following Administration (Tmax) | Part 1: ACE-083 concentrations ranged from 20.1 to 238.6 μg/L across FSHD and CMT cohorts with the majority of serum concentrations were below the lower limit of quantification (LLOQ) or just above LLOQ. Further, given the early termination of this trial, there were not enough samples per cohort collected at the pre-specified timeline points to be able to determine Tmax. | The PK Population consisted of all participants who received at least 1 dose of study drug and had sufficient PK samples collected and assayed for PK analysis. | Posted | | | | | | From baseline to End of Treatment in Part 1 | | | | ID | Title | Description |
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| OG000 | Part 1 Cohort 1a | ACE-083 240 mg/muscle administered bilaterally by injection into the TA muscle every 4 weeks for up to 6 doses for FSHD patients ACE-083: Recombinant fusion protein | | OG001 | Part 1 Cohort 1b | ACE-083 240 mg/muscle administered bilaterally by injection into the BB muscle every 4 weeks for up to 6 doses for FSHD patients ACE-083: Recombinant fusion protein | | OG002 | Part 1 Cohort 1c | ACE-083 240 mg/muscle administered bilaterally by injection into the TA muscle every 4 weeks for up to 6 doses for CMT patients ACE-083: Recombinant fusion protein |
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| Secondary | Part1: Pharmacokinetics Parameter of Area Under the Plasma Concentration Versus Time Curve (AUC) | Part 1: ACE-083 concentrations ranged from 20.1 to 238.6 μg/L across FSHD and CMT cohorts with the majority of serum concentrations were below the lower limit of quantification (LLOQ) or just above LLOQ. Further, given the early termination of this trial, there were not enough samples per cohort collected at the pre-specified timeline points to be able to determine AUC. | The PK Population consisted of all participants who received at least 1 dose of study drug and had sufficient PK samples collected and assayed for PK analysis. | Posted | | | | | | From baseline to End of Treatment in Part 1 | | | | ID | Title | Description |
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| OG000 | Part 1 Cohort 1a | ACE-083 240 mg/muscle administered bilaterally by injection into the TA muscle every 4 weeks for up to 6 doses for FSHD patients ACE-083: Recombinant fusion protein | | OG001 | Part 1 Cohort 1b | ACE-083 240 mg/muscle administered bilaterally by injection into the BB muscle every 4 weeks for up to 6 doses for FSHD patients ACE-083: Recombinant fusion protein | | OG002 | Part 1 Cohort 1c | ACE-083 240 mg/muscle administered bilaterally by injection into the TA muscle every 4 weeks for up to 6 doses for CMT patients ACE-083: Recombinant fusion protein |
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