Trial · NCT03943290
Extension Study to Evaluate the Long-Term Effects of ACE-083 in Patients With Facioscapulohumeral Muscular Dystrophy (FSHD) and Charcot-Marie Tooth (CMT) Disease Types 1 and X (CMT1 and CMTX)
NCT03943290, Phase 2, Terminated, Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway, NJ USA; results first posted 2021-06-02
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Extension Study to Evaluate the Long-Term Effects of ACE-083 in Patients With Facioscapulohumeral Muscular Dystrophy (FSHD) and Charcot-Marie Tooth (CMT) Disease Types 1 and X (CMT1 and CMTX). Trials, record NCT03943290. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03943290
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Extension Study to Evaluate the Long-Term Effects of ACE-083 in Patients With Facioscapulohumeral Muscular Dystrophy (FSHD) and Charcot-Marie Tooth (CMT) Disease Types 1 and X (CMT1 and CMTX)
- NCT number
- NCT03943290
- Status
- Terminated
- Phases
- Phase 2
- Enrollment
- 62 participants, actual
- Lead sponsor
- Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway, NJ USA
- Conditions
- Facioscapulohumeral Muscular Dystrophy, Charcot-Marie-Tooth Disease
- Interventions
- ACE-083
- First posted
- 2019-05-09
- Last updated
- 2022-09-26
- Results first posted
- 2021-06-02
- Limitations and caveats
In this open-label, multicenter, phase 2 extension study in participants with FSHD and CMT (types 1 and X), treatment with ACE-083 q4w or q8w was generally safe and well tolerated. Participants were discontinued from this study when their respective parent studies were terminated by the sponsor, and as such, this study was also terminated. Due to premature termination of this study, insufficient efficacy data were obtained to enable meaningful efficacy analyses.
Source ClinicalTrials.gov
Registered outcomes23
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Part 2- Frequency of Adverse Events - Presence and Nature of Adverse Events (AE) During Part 2 | From baseline to end of participation of the Part 2 portion of the study | 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. |
| 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. | From baseline to the end of the part 2 portion 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. |
| 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 | During the Part 2 portion of the study: Baseline to Day 113 | 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. |
| 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 | Baseline and End of Treatment visit for 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. |
| 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 | Baseline and End of Treatment visit for 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. |
| Secondary | Part 2: Change in Muscle Function. Absolute Change From Baseline in Functional Assessment for Tibialis Anterior (TA) Muscle: 6-minute Walk Test | Baseline and End of Treatment Visit during Part 2 of the study | 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. |
| Secondary | Part 2: Change in Muscle Function - Percent Change From Baseline in Functional Assessment for Tibialis Anterior (TA) Muscle in 6-minute Walk Test | Baseline and End of Treatment visit during Part 2 of the study | 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. |
| 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) | Baseline, End of Treatment visit during Part 2 | 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). |
| 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) | Baseline, End of Treatment visit during Part 2 | 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). |
| 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) | Baseline, End of Treatment visit during Part 2 | 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). |
1 to 10 of 23Source ClinicalTrials.gov
See all 23 registered outcomesOutcomes23
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Part 2- Frequency of Adverse Events - Presence and Nature of Adverse Events (AE) During Part 2 | From baseline to end of participation of the Part 2 portion of the study | The Safety Population consisted of all participants enrolled/randomized in the study who received at least 1 dose of study drug. | Participants | COUNT_OF_PARTICIPANTS |
| 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. | From baseline to the end of the part 2 portion of the study | The Safety Population consisted of all participants enrolled/randomized in the study who received at least 1 dose of study drug. | Participants | COUNT_OF_PARTICIPANTS |
| 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 | During the Part 2 portion of the study: 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 and at least 1 post-baseline MRI evaluation. | percent change | MEAN |
| 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 | Baseline and End of Treatment visit for Part 2 of the study | 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. | seconds | MEAN |
| 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 | Baseline and End of Treatment visit for Part 2 of the study | 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. | percent change | MEAN |
| SECONDARY | Part 2: Change in Muscle Function. Absolute Change From Baseline in Functional Assessment for Tibialis Anterior (TA) Muscle: 6-minute Walk Test | Baseline and End of Treatment Visit during Part 2 of the study | 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. | meters | MEAN |
| SECONDARY | Part 2: Change in Muscle Function - Percent Change From Baseline in Functional Assessment for Tibialis Anterior (TA) Muscle in 6-minute Walk Test | Baseline and End of Treatment visit during Part 2 of the study | 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. | percent change | MEAN |
| 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) | Baseline, End of Treatment visit during Part 2 | 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. | seconds | MEAN |
| 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) | Baseline, End of Treatment visit during Part 2 | 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. | percent change | MEAN |
| 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) | Baseline, End of Treatment visit during Part 2 | 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. | seconds | MEAN |
1 to 10 of 23Source ClinicalTrials.gov
See all 23 outcomesResults by group60
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Part 2- Frequency of Adverse Events - Presence and Nature of Adverse Events (AE) During Part 2 | Part 2 Cohort 2a | 3 | Participants | ||
| Part 2- Frequency of Adverse Events - Presence and Nature of Adverse Events (AE) During Part 2 | Part 2 Cohort 2b | 4 | Participants | ||
| Part 2- Frequency of Adverse Events - Presence and Nature of Adverse Events (AE) During Part 2 | Part 2 Cohort 2c | 5 | Participants | ||
| Part 2- Frequency of Adverse Events - Presence and Nature of Adverse Events (AE) During Part 2 | Part 2 Cohort 3a | 3 | Participants | ||
| Part 2- Frequency of Adverse Events - Presence and Nature of Adverse Events (AE) During Part 2 | Part 2 Cohort 3b | 4 | Participants | ||
| Part 2- Frequency of Adverse Events - Presence and Nature of Adverse Events (AE) During Part 2 | Part 2 Cohort 3c | 4 | Participants | ||
| Part 2: Frequency of Adverse Events - Presence and Nature of Grade 3 or Higher Adverse Events (AE) During the Part 2 of the Study. | Part 2 Cohort 2a | 0 | Participants | ||
| Part 2: Frequency of Adverse Events - Presence and Nature of Grade 3 or Higher Adverse Events (AE) During the Part 2 of the Study. | Part 2 Cohort 2b | 0 | Participants | ||
| Part 2: Frequency of Adverse Events - Presence and Nature of Grade 3 or Higher Adverse Events (AE) During the Part 2 of the Study. | Part 2 Cohort 2c | 1 | Participants | ||
| Part 2: Frequency of Adverse Events - Presence and Nature of Grade 3 or Higher Adverse Events (AE) During the Part 2 of the Study. | Part 2 Cohort 3a | 0 | Participants |
1 to 10 of 60Source ClinicalTrials.gov
See all 60 results by groupParticipant flow27
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Part 1 Cohort 1a | 7 | |
| Overall Study | STARTED | Part 1 Cohort 1b | 1 | |
| Overall Study | STARTED | Part 1 Cohort 1c | 13 | |
| Overall Study | STARTED | Part 2 Cohort 2a | 6 | |
| Overall Study | STARTED | Part 2 Cohort 2b | 7 | |
| Overall Study | STARTED | Part 2 Cohort 2c | 6 | |
| Overall Study | STARTED | Part 2 Cohort 3a | 6 | |
| Overall Study | STARTED | Part 2 Cohort 3b | 8 | |
| Overall Study | STARTED | Part 2 Cohort 3c | 8 | |
| Overall Study | COMPLETED | Part 1 Cohort 1a | 0 |
1 to 10 of 27Source ClinicalTrials.gov
See all 27 participant flowAdverse events540
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 | injection site bruising | General disorders | Part 1 Cohort 1a | 0 | 7 | |
| other | injection site bruising | General disorders | Part 1 Cohort 1b | 1 | 1 | |
| other | injection site bruising | General disorders | Part 1 Cohort 1c | 2 | 13 | |
| other | injection site bruising | General disorders | Part 2 Cohort 2a | 0 | 6 | |
| other | injection site bruising | General disorders | Part 2 Cohort 2b | 3 | 7 | |
| other | injection site bruising | General disorders | Part 2 Cohort 2c | 0 | 6 | |
| other | injection site bruising | General disorders | Part 2 Cohort 3a | 0 | 6 | |
| other | injection site bruising | General disorders | Part 2 Cohort 3b | 0 | 8 | |
| other | injection site bruising | General disorders | Part 2 Cohort 3c | 0 | 8 | |
| other | injection site discomfort | General disorders | Part 1 Cohort 1a | 0 | 7 |
1 to 10 of 540Source ClinicalTrials.gov
See all 540 adverse eventsAdverse event totals27
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Part 1 Cohort 1a | 0 | 7 |
| Total, all-cause mortality | Part 1 Cohort 1b | 0 | 1 |
| Total, all-cause mortality | Part 1 Cohort 1c | 0 | 13 |
| Total, all-cause mortality | Part 2 Cohort 2a | 0 | 6 |
| Total, all-cause mortality | Part 2 Cohort 2b | 0 | 7 |
| Total, all-cause mortality | Part 2 Cohort 2c | 0 | 6 |
| Total, all-cause mortality | Part 2 Cohort 3a | 0 | 6 |
| Total, all-cause mortality | Part 2 Cohort 3b | 0 | 8 |
| Total, all-cause mortality | Part 2 Cohort 3c | 0 | 8 |
| Total, other adverse events | Part 1 Cohort 1a | 3 | 7 |
1 to 10 of 27Source ClinicalTrials.gov
See all 27 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.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT03943290
GET https://conductscience.org/api/trials/v1/NCT03943290/registered?page=2