Trial · NCT03124459
Study of ACE-083 in Patients With Charcot-Marie-Tooth Disease
NCT03124459, Phase 2, Terminated, Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway, NJ USA; results first posted 2021-07-28
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ConductScience Library. Study of ACE-083 in Patients With Charcot-Marie-Tooth Disease. Trials, record NCT03124459. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03124459
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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 Charcot-Marie-Tooth Disease
- NCT number
- NCT03124459
- Status
- Terminated
- Phases
- Phase 2
- Enrollment
- 63 participants, actual
- Lead sponsor
- Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway, NJ USA
- Conditions
- Charcot-Marie-Tooth Disease
- Interventions
- ACE-083, Placebo
- First posted
- 2017-04-21
- Last updated
- 2022-09-26
- Results first posted
- 2021-07-28
- Limitations and caveats
The study was terminated because the statistically significant increase in total muscle volume (TMV) failed to translate to statistically significant improvements in functional or disease-related quality of life secondary endpoints in the study.
Source ClinicalTrials.gov
Registered outcomes11
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Part 1: Frequency of Adverse Events | From initiation of treatment (Study Day 1) to end of follow-up period for Part 1 (Study Day 141). | Number of subjects with at least one adverse event related to treatment intervention from Part 1 of this study. Since this outcome measure was only pre-specified for Part 1, only data from the Part 1 participants is reported. |
| Primary | Part 2: Percent Change in Muscle Volume to the End of the Double-blind Placebo-controlled Portion of the Study. | From initiation of treatment (Study Day 1) to end of follow-up period of the double-blind placebo-controlled portion of the study (Study Day 190). | The percent change from baseline in volume of injected muscle, by MRI compared to the Day 190 Assessment is reported. The pre-specified timepoint for this outcome was only for data collected from participants to the end of the double-blind placebo controlled portion of the study, therefore data for the open-label arm of the study is not reported. |
| Secondary | Part 2: Absolute Change in Amount of Intramuscular Fat Tissue to the End of the Double-blind Placebo-controlled Portion of the Study | From initiation of treatment (Study Day 1) to end of follow-up period of the double-blind placebo-controlled portion of the study (Study Day 190). | The absolute change from baseline in intramuscular fat fraction of the injected muscle, by MRI compared to Day 190 Assessment is reported. The pre-specified timepoint for this outcome was only for data collected from participants to the end of the double-blind placebo controlled portion of the study, therefore data for the open-label arm of the study is not reported. |
| Secondary | Part 2: Percent Change in Muscle Strength to the End of the Double-blind Placebo-controlled Portion of the Study | From initiation of treatment (Study Day 1) to end of follow-up period of the double-blind placebo-controlled portion of the study (Study Day 190). | Percent change from baseline in strength of the injected muscle, by Quantitative Muscle Testing (QMT) compared to Day 190 Assessment is reported. The pre-specified timepoint for this outcome was only for data collected from participants to the end of the double-blind placebo controlled portion of the study, therefore data for the open-label arm of the study is not reported. |
| Secondary | Part 2: Percent Change in Muscle Function - Walk/Run Time to the End of the Double-blind Placebo-controlled Portion of the Study | From initiation of treatment (Study Day 1) to end of follow-up period of the double-blind placebo-controlled portion of the study (Study Day 190). | The percent change from baseline in functional assessments, as measured by 10-meter walk/run time when compared to Day 190 Assessment is reported. The pre-specified timepoint for this outcome was only for data collected from participants to the end of the double-blind placebo controlled portion of the study, therefore data for the open-label arm of the study is not reported. |
| Secondary | Part 2: Percent Change in Muscle Function - Walk Distance Assessed at the End of the Double-blind Placebo-controlled Portion of the Study | From initiation of treatment (Study Day 1) to end of follow-up period for the double-blind placebo-controlled portion of the study (Study Day 190). | Percent change from baseline in functional assessments, as measured by 6-minute walk distance when compared to Day 190 Assessment, is reported. The pre-specified timepoint for this outcome was only for data collected from participants to the end of the double-blind placebo controlled portion of the study, therefore data for the open-label arm of the study is not reported. |
| Secondary | Part 2: Change in Balance and Fall Risk at the End of the Double-blind Placebo-controlled Portion of the Study. | From initiation of treatment (Study Day 1) to end of follow-up period of the double-blind placebo-controlled portion of the study (Study Day 190). | Change from baseline in static and dynamic balance, as measured by the Berg Balance Scale, a 14-item scoring system to assess balance and fall risk in adults. The Berg balance scale is used to objectively determine a patient's ability (or inability) to safely balance during a series of predetermined tasks. It is a 14 item list with each item consisting of a five-point ordinal scale ranging from 0 to 4, with 0 indicating the lowest level of function and 4 the highest level of function. Total scores are used for reporting, with a range of 0-56, with higher scores mean a better demonstration of function. A score of 56 indicates functional balance. A score of \< 45 indicates individuals may be at greater risk of falling. The pre-specified timepoints for reporting are baseline Berg scale score and Day 190 score, therefore data for the open-label arm of the study is not reported. |
| Secondary | Part 2: Percent Change in Balance and Fall Risk From Baseline to the End of the Double-blind Placebo-controlled Portion of the Study | From initiation of treatment (Study Day 1) to end of follow-up period of the double-blind placebo-controlled portion of the study (Study Day 190). | Percent change was calculated for the difference from baseline and Day 190 Assessment scores on the Berg Balance Scale. The Berg Balance Scale, is a 14-item scoring system to assess balance and fall risk in adults. The Berg balance scale is used to objectively determine a patient's ability (or inability) to safely balance during a series of predetermined tasks. It is a 14 item list with each item consisting of a five-point ordinal scale ranging from 0 to 4, with 0 indicating the lowest level of function and 4 the highest level of function. Total scores are used for reporting, with a range of 0-56, with higher scores mean a better demonstration of function. A score of 56 indicates functional balance. A score of \< 45 indicates individuals may be at greater risk of falling. The pre-specified timepoints for reporting are baseline Berg scale score and Day 190 score, therefore data for the open-label arm of the study is not reported. |
| Secondary | Part 2: Change in Clinical Examination Score From Baseline to End of the Double-blind Placebo-controlled Portion of the Study | From initiation of treatment (Study Day 1) to end of follow-up period of the double-blind placebo-controlled portion of the study (Study Day 190). | Change from baseline in the Charcot-Marie-Tooth (CMT) Examination Score, version 2 (CMTES2), a composite scoring system to assess sensory and motor impairment in subjects with CMT. The total score is a subset of the following items from the CMT neuropathy score instrument: Sensory symptoms, Motor symptoms (legs), Motor symptoms (arms), Pinprick Sensibility, Vibration, Strength (legs), and Strength (arms). Each individual item is assessed using a rating from 0 to 4 inclusive. The range of CMTES2 scores is from 0 to 28 inclusive. A higher score means a greater degree of symptom severity. The Baseline score and score on the Day 190 Assessment are reported. The pre-specified timepoints for reporting are baseline Berg scale score and Day 190 score, therefore data for the open-label arm of the study is not reported. |
| Secondary | Part 2: Percent Change in Clinical Examination Score in Baseline to End of the Double-blind Placebo-controlled Portion of the Study | From initiation of treatment (Study Day 1) to end of follow-up period of the double-blind placebo-controlled portion of the study (Study Day 190). | Percent change was calculated for the difference in the Charcot-Marie-Tooth (CMT) Examination Score (CMTES2) from baseline and Day 190 Assessment scores. The pre-specified timepoints for reporting are baseline Berg scale score and Day 190 score, therefore data for the open-label arm of the study is not reported. |
1 to 10 of 11Source ClinicalTrials.gov
See all 11 registered outcomesOutcomes11
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Part 1: Frequency of Adverse Events | From initiation of treatment (Study Day 1) to end of follow-up period for Part 1 (Study Day 141). | The Safety Set consists of all patients enrolled in the study who have received at least 1 dose of study drug. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Part 2: Percent Change in Muscle Volume to the End of the Double-blind Placebo-controlled Portion of the Study. | From initiation of treatment (Study Day 1) to end of follow-up period of the double-blind placebo-controlled portion of the study (Study Day 190). | Per Protocol Set: All participants enrolled/randomized in the study who received at least 1 dose of the study drug (includes placebo) with no major protocol violations | percent change | LEAST_SQUARES_MEAN |
| SECONDARY | Part 2: Absolute Change in Amount of Intramuscular Fat Tissue to the End of the Double-blind Placebo-controlled Portion of the Study | From initiation of treatment (Study Day 1) to end of follow-up period of the double-blind placebo-controlled portion of the study (Study Day 190). | Per Protocol Set: All participants enrolled/randomized in the study who received at least 1 dose of the study drug (includes placebo) with no major protocol violations | mm3 | LEAST_SQUARES_MEAN |
| SECONDARY | Part 2: Percent Change in Muscle Strength to the End of the Double-blind Placebo-controlled Portion of the Study | From initiation of treatment (Study Day 1) to end of follow-up period of the double-blind placebo-controlled portion of the study (Study Day 190). | Per Protocol Set: All participants enrolled/randomized in the study who received at least 1 dose of the study drug (includes placebo) with no major protocol violations | percent change | LEAST_SQUARES_MEAN |
| SECONDARY | Part 2: Percent Change in Muscle Function - Walk/Run Time to the End of the Double-blind Placebo-controlled Portion of the Study | From initiation of treatment (Study Day 1) to end of follow-up period of the double-blind placebo-controlled portion of the study (Study Day 190). | Per Protocol Set: All participants enrolled/randomized in the study who received at least 1 dose of the study drug (includes placebo) with no major protocol violations | Percent change | LEAST_SQUARES_MEAN |
| SECONDARY | Part 2: Percent Change in Muscle Function - Walk Distance Assessed at the End of the Double-blind Placebo-controlled Portion of the Study | From initiation of treatment (Study Day 1) to end of follow-up period for the double-blind placebo-controlled portion of the study (Study Day 190). | Per Protocol Set: All participants enrolled/randomized in the study who received at least 1 dose of the study drug (includes placebo) with no major protocol violations | Percent change | LEAST_SQUARES_MEAN |
| SECONDARY | Part 2: Change in Balance and Fall Risk at the End of the Double-blind Placebo-controlled Portion of the Study. | From initiation of treatment (Study Day 1) to end of follow-up period of the double-blind placebo-controlled portion of the study (Study Day 190). | Per Protocol Set: All participants enrolled/randomized in the study who received at least 1 dose of the study drug (includes placebo) with no major protocol violations | score on scale | MEAN |
| SECONDARY | Part 2: Percent Change in Balance and Fall Risk From Baseline to the End of the Double-blind Placebo-controlled Portion of the Study | From initiation of treatment (Study Day 1) to end of follow-up period of the double-blind placebo-controlled portion of the study (Study Day 190). | Per Protocol Set: All participants enrolled/randomized in the study who received at least 1 dose of the study drug (includes placebo) with no major protocol violations | percent change in score on scale | MEAN |
| SECONDARY | Part 2: Change in Clinical Examination Score From Baseline to End of the Double-blind Placebo-controlled Portion of the Study | From initiation of treatment (Study Day 1) to end of follow-up period of the double-blind placebo-controlled portion of the study (Study Day 190). | Per Protocol Set: All participants enrolled/randomized in the study who received at least 1 dose of the study drug (includes placebo) with no major protocol violations | score on scale | MEAN |
| SECONDARY | Part 2: Percent Change in Clinical Examination Score in Baseline to End of the Double-blind Placebo-controlled Portion of the Study | From initiation of treatment (Study Day 1) to end of follow-up period of the double-blind placebo-controlled portion of the study (Study Day 190). | Per Protocol Set: All participants enrolled/randomized in the study who received at least 1 dose of the study drug (includes placebo) with no major protocol violations. | percent change in score on scale | MEAN |
1 to 10 of 11Source ClinicalTrials.gov
See all 11 outcomesResults by group27
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Part 1: Frequency of Adverse Events | Part 1 Cohort 1 (150 mg) | 6 | Participants | ||
| Part 1: Frequency of Adverse Events | Part 1 Cohort 2 (200 mg) | 5 | Participants | ||
| Part 1: Frequency of Adverse Events | Part 1 Cohort 3 (240 mg) | 6 | Participants | ||
| Part 2: Percent Change in Muscle Volume to the End of the Double-blind Placebo-controlled Portion of the Study. | Part 2 (Double-blind Placebo Controlled) Placebo Arm | 2.2 | Standard Error 4.1 | percent change | |
| Part 2: Percent Change in Muscle Volume to the End of the Double-blind Placebo-controlled Portion of the Study. | Part 2 (Double-blind Placebo Controlled) ACE-083 Arm | 15.8 | Standard Error 4.3 | percent change | |
| Part 2: Absolute Change in Amount of Intramuscular Fat Tissue to the End of the Double-blind Placebo-controlled Portion of the Study | Part 2 (Double-blind Placebo Controlled) Placebo Arm | 1 | Standard Error 1.8 | mm3 | |
| Part 2: Absolute Change in Amount of Intramuscular Fat Tissue to the End of the Double-blind Placebo-controlled Portion of the Study | Part 2 (Double-blind Placebo Controlled) ACE-083 Arm | -2.1 | Standard Error 1.9 | mm3 | |
| Part 2: Percent Change in Muscle Strength to the End of the Double-blind Placebo-controlled Portion of the Study | Part 2 (Double-blind Placebo Controlled) Placebo Arm | -3.4 | Standard Error 19.8 | percent change | |
| Part 2: Percent Change in Muscle Strength to the End of the Double-blind Placebo-controlled Portion of the Study | Part 2 (Double-blind Placebo Controlled) ACE-083 Arm | 32.0 | Standard Error 19.8 | percent change | |
| Part 2: Percent Change in Muscle Function - Walk/Run Time to the End of the Double-blind Placebo-controlled Portion of the Study | Part 2 (Double-blind Placebo Controlled) Placebo Arm | -10.1 | Standard Error 4.7 | Percent change |
1 to 10 of 27Source ClinicalTrials.gov
See all 27 results by groupParticipant flow54
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Part 1-Open Label Dose Escalation | STARTED | Part 1 Cohort 1 (150 mg) | 6 | |
| Part 1-Open Label Dose Escalation | STARTED | Part 1 Cohort 2 (200 mg) | 6 | |
| Part 1-Open Label Dose Escalation | STARTED | Part 1 Cohort 3 (240 mg) | 6 | |
| Part 1-Open Label Dose Escalation | STARTED | Part 2 (Double-blind Placebo Controlled) Placebo Arm | 0 | |
| Part 1-Open Label Dose Escalation | STARTED | Part 2 (Double-blind Placebo Controlled) ACE-083 Arm | 0 | |
| Part 1-Open Label Dose Escalation | STARTED | Part 2 (Open-label) ACE-083 Arm | 0 | |
| Part 1-Open Label Dose Escalation | COMPLETED | Part 1 Cohort 1 (150 mg) | 6 | |
| Part 1-Open Label Dose Escalation | COMPLETED | Part 1 Cohort 2 (200 mg) | 6 | |
| Part 1-Open Label Dose Escalation | COMPLETED | Part 1 Cohort 3 (240 mg) | 6 | |
| Part 1-Open Label Dose Escalation | COMPLETED | Part 2 (Double-blind Placebo Controlled) Placebo Arm | 0 |
1 to 10 of 54Source ClinicalTrials.gov
See all 54 participant flowAdverse events966
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 | pain in extremity | Musculoskeletal and connective tissue disorders | Part 1 Cohort 1(150mg) | 3 | 6 | |
| other | pain in extremity | Musculoskeletal and connective tissue disorders | Part 1 Cohort 2 (200mg) | 0 | 6 | |
| other | pain in extremity | Musculoskeletal and connective tissue disorders | Part 1 Cohort 3 (250mg) | 2 | 6 | |
| other | pain in extremity | Musculoskeletal and connective tissue disorders | Part 2 (Double-blind Placebo Controlled) Placebo Arm | 4 | 21 | |
| other | pain in extremity | Musculoskeletal and connective tissue disorders | Part 2 (Double-blind Placebo Controlled) ACE-083 Arm | 6 | 23 | |
| other | pain in extremity | Musculoskeletal and connective tissue disorders | Part 2 (Open Label) | 8 | 40 | |
| other | muscle spasm | Musculoskeletal and connective tissue disorders | Part 1 Cohort 1(150mg) | 1 | 6 | |
| other | muscle spasm | Musculoskeletal and connective tissue disorders | Part 1 Cohort 2 (200mg) | 2 | 6 | |
| other | muscle spasm | Musculoskeletal and connective tissue disorders | Part 1 Cohort 3 (250mg) | 1 | 6 | |
| other | muscle spasm | Musculoskeletal and connective tissue disorders | Part 2 (Double-blind Placebo Controlled) Placebo Arm | 1 | 21 |
1 to 10 of 966Source ClinicalTrials.gov
See all 966 adverse eventsAdverse event totals18
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Part 1 Cohort 1(150mg) | 0 | 6 |
| Total, all-cause mortality | Part 1 Cohort 2 (200mg) | 0 | 6 |
| Total, all-cause mortality | Part 1 Cohort 3 (250mg) | 0 | 6 |
| Total, all-cause mortality | Part 2 (Double-blind Placebo Controlled) Placebo Arm | 0 | 21 |
| Total, all-cause mortality | Part 2 (Double-blind Placebo Controlled) ACE-083 Arm | 0 | 23 |
| Total, all-cause mortality | Part 2 (Open Label) | 0 | 40 |
| Total, other adverse events | Part 1 Cohort 1(150mg) | 6 | 6 |
| Total, other adverse events | Part 1 Cohort 2 (200mg) | 6 | 6 |
| Total, other adverse events | Part 1 Cohort 3 (250mg) | 6 | 6 |
| Total, other adverse events | Part 2 (Double-blind Placebo Controlled) Placebo Arm | 19 | 21 |
1 to 10 of 18Source ClinicalTrials.gov
See all 18 adverse event totalsPapers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 35545446 | DERIVED |
Source PubMed
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.
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