Trial · NCT02927080
Study of ACE-083 in Patients With Facioscapulohumeral Muscular Dystrophy (FSHD)
NCT02927080, Phase 2, Terminated, Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway, NJ USA; results first posted 2021-04-22
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Study of ACE-083 in Patients With Facioscapulohumeral Muscular Dystrophy (FSHD). Trials, record NCT02927080. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02927080
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Study of ACE-083 in Patients With Facioscapulohumeral Muscular Dystrophy (FSHD)
- NCT number
- NCT02927080
- Status
- Terminated
- Phases
- Phase 2
- Enrollment
- 95 participants, actual
- Lead sponsor
- Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway, NJ USA
- Conditions
- Facioscapulohumeral Muscular Dystrophy
- Interventions
- ACE-083, ACE-083 or placebo
- First posted
- 2016-10-06
- Last updated
- 2022-09-26
- Results first posted
- 2021-04-22
Source ClinicalTrials.gov
Registered outcomes19
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Safety and Tolerability (Incidence of Adverse Events) | From initiation of treatment to Day 106 for Part 1 and Day 190 for Part 2 | The number of participants that had a least one Treatment Emergent Adverse Event for the duration of each of the respective study parts. |
| Primary | Safety and Tolerability (Severity of Adverse Events, Grade 3 or Higher). | From initiation of treatment to Day 106 for Part 1 and Day 190 for Part 2 | The number of participants that had a least one Treatment Emergent Adverse Event with CTCAE Grade 3 or Higher for the duration of each of the respective study parts. |
| Primary | Safety and Tolerability (Severity of Adverse Events- Dose Interruption, Reduction and/or Drug Withdrawal) | From initiation of treatment to Day 106 for Part 1 and Day 190 for Part 2 | The number of participants that had a least one Treatment Emergent Adverse Event that led to dose interruption, dose reduction and/or drug withdrawn. |
| Primary | Total Muscle Volume (TMV) of the Treated Muscle in Patients With FSHD Administered ACE-083 or Placebo During Part 2 (Randomized, Controlled Portion) | Time Frame: From initiation of treatment to Study Visit Day 190 | Total Muscle Volume (TMV)of the treated muscle in Patients with FSHD administered ACE-083 or placebo During Part 2 (randomized, controlled portion) from Baseline to Day 190. Total Muscle volume was measured by Magnetic Resonance Imaging (MRI). MRI was performed bilaterally on the tibialis anterior and the biceps brachii on Day 1, Day Day 43, Day 106, and Day 190. Baseline and Day 190 total muscle volume, means and standard deviations are reported. |
| Primary | Percent Change of Total Muscle Volume (TMV) of the Treated Muscle in Patients With FSHD Administered ACE-083 or Placebo During Part 2 (Randomized, Controlled Portion) | Time Frame: From initiation of treatment to Study Visit Day 190 | Percent Change of Total Muscle Volume (TMV)of the treated muscle in Patients with FSHD administered ACE-083 or placebo During Part 2 (randomized, controlled portion) from Baseline to Day 190. Total Muscle volume was measured by Magnetic Resonance Imaging (MRI). MRI was performed bilaterally on the tibialis anterior and the biceps brachii on Day 1, Day Day 43, Day 106, and Day 190. Percent Change from Baseline to Day 190 total muscle volume, mean and standard deviation is reported. |
| Secondary | Percent Change in Total Muscle Volume (TMV) in Muscle in Patients With FSHD Administered ACE-083 During Part 1 (Open-label, Dose-escalation Portion) | Time Frame: From initiation of treatment to Study Visit Day 106 | Percent Change in Total Muscle Volume (TMV) in muscle in patients with FSHD administered ACE-083 During 1 (open-label, dose-escalation portion) from Baseline to Day 106. Total Muscle volume was measured by Magnetic Resonance Imaging (MRI). MRI was performed bilaterally on the tibialis anterior and the biceps brachii on Day 1, Day Day 43, and Day 106, change from Baseline and Day 106 reported. |
| Secondary | Absolute Change in Fat Fraction (FF) of the Muscle in Patients With FSHD Administered ACE-083 or Placebo During Part 2 (Randomized, Controlled Portion) | Time Frame: From initiation of treatment to Study Visit Day 190 | Absolute change in Fat Fraction (FF) of the muscle in patients with FSHD administered ACE-083 or Placebo During Part 2 (randomized, controlled portion) from Baseline to Day 190. Absolute change in intramuscular fat fraction in the tibialis anterior and biceps brachii were measured by Magnetic Resonance Imaging (MRI). MRI was performed bilaterally on the tibialis anterior and the biceps brachii on Day 1, Day Day 43, Day 106, and Day 190, change from Baseline and Day 190 reported. |
| Secondary | Percent Change From Baseline in Function of Tibialis Anterior, Part 2 (Randomized, Controlled Portion) | From initiation of treatment (Study Day 1) to Study Visit Day 190 | Percent change from baseline in function of Tibialis Anterior during Part 2 assessed by: 6-minute walk test, 10 meter walk/run and 4-stair climb (ascend) |
| Secondary | Percent Change From Baseline in Strength of Biceps Brachii, Part 2, Randomized-controlled | From initiation of treatment (Study Day 1) to Study Visit Day 190 | Elbow flexion strength measured by hand-held dynamometry (quantitative muscle testing), maximum voluntary isometric contraction (MVIC). |
| Secondary | Percent Change From Baseline in Performance of the Upper Limb (PUL) Mid-Level Elbow Dimension, Part 2, Randomized-controlled | From initiation of treatment (Study Day 1) to Study Visit Day 190 | Percent Change from Baseline in Performance of the Upper Limb (PUL) Mid-Level Elbow Dimension, Part 2, Randomized-controlled, PUL from baseline to end of treatment (Day 190). The Performance of the Upper Limb is an assessment specifically designed for patients with Duchenne muscular dystrophy. The measures used in this study was a subset of the assessment. PUL was assessment by measures of high-level of movement (lifting weights of 50g, 200g, 500g and 1000g at shoulder height and above shoulder height) and mid-level movement by performing tasks with and without weights: hand to mouth with and without weights (50, 200g), hand to table, moving weights on table (100g, 200g, 500g and 1000g), lift light and heavy cans, stack light and heavy cans, remove lid from container, tearing paper). |
1 to 10 of 19Source ClinicalTrials.gov
See all 19 registered outcomesOutcomes19
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Safety and Tolerability (Incidence of Adverse Events) | From initiation of treatment to Day 106 for Part 1 and Day 190 for Part 2 | The safety set population is all participants that received at least one dose of ACE-083 or placebo during the study. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Safety and Tolerability (Severity of Adverse Events, Grade 3 or Higher). | From initiation of treatment to Day 106 for Part 1 and Day 190 for Part 2 | The safety set population is all participants that received at least one dose of ACE-083 or placebo during the study. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Safety and Tolerability (Severity of Adverse Events- Dose Interruption, Reduction and/or Drug Withdrawal) | From initiation of treatment to Day 106 for Part 1 and Day 190 for Part 2 | The safety set population is all participants that received at least one dose of ACE-083 or placebo during the study. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Total Muscle Volume (TMV) of the Treated Muscle in Patients With FSHD Administered ACE-083 or Placebo During Part 2 (Randomized, Controlled Portion) | Time Frame: From initiation of treatment to Study Visit Day 190 | The Per Protocol Set: All patients enrolled/randomized in the study who received at least one dose of the study drug (includes placebo) with no major protocol violations. | mm3 | MEAN |
| PRIMARY | Percent Change of Total Muscle Volume (TMV) of the Treated Muscle in Patients With FSHD Administered ACE-083 or Placebo During Part 2 (Randomized, Controlled Portion) | Time Frame: From initiation of treatment to Study Visit Day 190 | The Per Protocol Set: All patients enrolled/randomized in the study who received at least one dose of the study drug (includes placebo) with no major protocol violations. | percent change | MEAN |
| SECONDARY | Percent Change in Total Muscle Volume (TMV) in Muscle in Patients With FSHD Administered ACE-083 During Part 1 (Open-label, Dose-escalation Portion) | Time Frame: From initiation of treatment to Study Visit Day 106 | The Per Protocol Set: All patients enrolled/randomized in the study who received at least one dose of the study drug (includes placebo) with no major protocol violations. | percent change | MEAN |
| SECONDARY | Absolute Change in Fat Fraction (FF) of the Muscle in Patients With FSHD Administered ACE-083 or Placebo During Part 2 (Randomized, Controlled Portion) | Time Frame: From initiation of treatment to Study Visit Day 190 | The Per Protocol Set: All patients enrolled/randomized in the study who received at least one dose of the study drug (includes placebo) with no major protocol violations. | Percent change | LEAST_SQUARES_MEAN |
| SECONDARY | Percent Change From Baseline in Function of Tibialis Anterior, Part 2 (Randomized, Controlled Portion) | From initiation of treatment (Study Day 1) to Study Visit Day 190 | Per Protocol Set: All patients enrolled/randomized in the study who received at least one dose of the study drug (includes placebo) with no major protocol violations. | percent change | LEAST_SQUARES_MEAN |
| SECONDARY | Percent Change From Baseline in Strength of Biceps Brachii, Part 2, Randomized-controlled | From initiation of treatment (Study Day 1) to Study Visit Day 190 | Per Protocol Set: All patients enrolled/randomized in the study who received at least one dose of the study drug (includes placebo) with no major protocol violations. | percent change | LEAST_SQUARES_MEAN |
| SECONDARY | Percent Change From Baseline in Performance of the Upper Limb (PUL) Mid-Level Elbow Dimension, Part 2, Randomized-controlled | From initiation of treatment (Study Day 1) to Study Visit Day 190 | Per Protocol Set: All patients enrolled/randomized in the study who received at least one dose of the study drug (includes placebo) with no major protocol violations. | percent change | LEAST_SQUARES_MEAN |
1 to 10 of 19Source ClinicalTrials.gov
See all 19 outcomesResults by group102
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Safety and Tolerability (Incidence of Adverse Events) | ACE-083 (Part 1, Cohort 1a) Tibialis Anterior (TA) 150 mg | 6 | Participants | ||
| Safety and Tolerability (Incidence of Adverse Events) | ACE-083 (Part 1, Cohort 2a) Tibialis Anterior (TA) 200 mg | 6 | Participants | ||
| Safety and Tolerability (Incidence of Adverse Events) | ACE-083 (Part 1, Cohort 3a) Tibialis Anterior (TA) 200 mg | 5 | Participants | ||
| Safety and Tolerability (Incidence of Adverse Events) | ACE-083 (Part 1, Cohort 1b) Biceps Brachii (BB) 150 mg | 6 | Participants | ||
| Safety and Tolerability (Incidence of Adverse Events) | ACE-083 (Part 1, Cohort 2b) Biceps Brachii (BB) 200 mg | 7 | Participants | ||
| Safety and Tolerability (Incidence of Adverse Events) | ACE-083 (Part 1, Cohort 3b) Biceps Brachii (BB) 240 mg | 6 | Participants | ||
| Safety and Tolerability (Incidence of Adverse Events) | Placebo (Part 2, DB-PC) Tibialis Anterior (TA) | 12 | Participants | ||
| Safety and Tolerability (Incidence of Adverse Events) | ACE-083 (Part 2, DB-PC) Tibialis Anterior (TA) 240 mg | 14 | Participants | ||
| Safety and Tolerability (Incidence of Adverse Events) | Placebo (Part 2, DB-PC) Biceps Brachii (BB) | 13 | Participants | ||
| Safety and Tolerability (Incidence of Adverse Events) | ACE-083 (Part 2, DB-PC) Biceps Brachii (BB) 240 mg | 14 | Participants |
1 to 10 of 102Source ClinicalTrials.gov
See all 102 results by groupParticipant flow40
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | ACE-083 (Part 1, Cohort 1a) Tibialis Anterior (TA) Unilateral | 6 | |
| Overall Study | STARTED | ACE-083 (Part 1, Cohort 2a) Tibialis Anterior (TA) Unilateral | 6 | |
| Overall Study | STARTED | ACE-083 (Part 1, Cohort 3a) Tibialis Anterior (TA) Bilateral | 6 | |
| Overall Study | STARTED | ACE-083 (Part 1, Cohort 1b) Biceps Brachii (BB) Unilateral | 6 | |
| Overall Study | STARTED | ACE-083 (Part 1, Cohort 2b) Biceps Brachii (BB) Unilateral | 7 | |
| Overall Study | STARTED | ACE-083 (Part 1, Cohort 3b) Biceps Brachii (BB) Unilateral | 6 | |
| Overall Study | STARTED | Placebo (Part 2, DB-PC) Tibialis Anterior (TA) | 15 | |
| Overall Study | STARTED | ACE-083 (Part 2, DB-PC) Tibialis Anterior (TA) 240 mg | 14 | |
| Overall Study | STARTED | Placebo (Part 2, DB-PC) Biceps Brachii (BB) | 15 | |
| Overall Study | STARTED | ACE-083 (Part 2, DB-PC) Biceps Brachii (BB) 240 mg | 14 |
1 to 10 of 40Source ClinicalTrials.gov
See all 40 participant flowAdverse events1,992
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 pain | General disorders | ACE-083 (Part 1, Cohort 1a ) Tibialis Anterior (TA) 150mg | 5 | 6 | |
| other | Injection site pain | General disorders | ACE-083 (Part 1, Cohort 2a ) Tibialis Anterior (TA) 200mg | 4 | 6 | |
| other | Injection site pain | General disorders | ACE-083 (Part 1, Cohort 3a ) Tibialis Anterior (TA) 200mg | 3 | 6 | |
| other | Injection site pain | General disorders | ACE-083 (Part 1, Cohort 1b ) Biceps Brachii (BB) 150 mg | 1 | 6 | |
| other | Injection site pain | General disorders | ACE-083 (Part 1, Cohort 2b ) Biceps Brachii (BB) 200 mg | 4 | 7 | |
| other | Injection site pain | General disorders | ACE-083 (Part 1, Cohort 3b ) Biceps Brachii (BB) 240 mg | 3 | 6 | |
| other | Injection site pain | General disorders | Placebo (Part 2, DB-PC) Tibialis Anterior (TA) | 4 | 15 | |
| other | Injection site pain | General disorders | ACE-083 (Part 2, DB-PC) Tibialis Anterior (TA) 240 mg | 5 | 14 | |
| other | Injection site pain | General disorders | Placebo (Part 2, DB-PC) Bicpes Brachii (BB) | 6 | 15 | |
| other | Injection site pain | General disorders | ACE-083 (Part 2, DB-PC) Biceps Brachii (BB) 240 mg | 6 | 14 |
1 to 10 of 1,992Source ClinicalTrials.gov
See all 1,992 adverse eventsAdverse event totals36
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | ACE-083 (Part 1, Cohort 1a ) Tibialis Anterior (TA) 150mg | 0 | 6 |
| Total, all-cause mortality | ACE-083 (Part 1, Cohort 2a ) Tibialis Anterior (TA) 200mg | 0 | 6 |
| Total, all-cause mortality | ACE-083 (Part 1, Cohort 3a ) Tibialis Anterior (TA) 200mg | 0 | 6 |
| Total, all-cause mortality | ACE-083 (Part 1, Cohort 1b ) Biceps Brachii (BB) 150 mg | 0 | 6 |
| Total, all-cause mortality | ACE-083 (Part 1, Cohort 2b ) Biceps Brachii (BB) 200 mg | 0 | 7 |
| Total, all-cause mortality | ACE-083 (Part 1, Cohort 3b ) Biceps Brachii (BB) 240 mg | 0 | 6 |
| Total, all-cause mortality | Placebo (Part 2, DB-PC) Tibialis Anterior (TA) | 0 | 15 |
| Total, all-cause mortality | ACE-083 (Part 2, DB-PC) Tibialis Anterior (TA) 240 mg | 0 | 14 |
| Total, all-cause mortality | Placebo (Part 2, DB-PC) Bicpes Brachii (BB) | 0 | 15 |
| Total, all-cause mortality | ACE-083 (Part 2, DB-PC) Biceps Brachii (BB) 240 mg | 0 | 14 |
1 to 10 of 36Source ClinicalTrials.gov
See all 36 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/NCT02927080
GET https://conductscience.org/api/trials/v1/NCT02927080/results?page=2