18 and older, any sex, with Charcot-Marie-Tooth Disease. Patients with the condition only — healthy volunteers not accepted.
Results — posted to ClinicalTrials.gov
Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.
Part 1: Frequency of Adverse EventsPrimary· 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.
Group
Value
95% CI
Part 1 Cohort 1 (150 mg)
6
Part 1 Cohort 2 (200 mg)
5
Part 1 Cohort 3 (240 mg)
6
Part 2: Percent Change in Muscle Volume to the End of the Double-blind Placebo-controlled Portion of the Study.Primary· 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.
Group
Value
95% CI
Part 2 (Double-blind Placebo Controlled) Placebo Arm
2.2
± 4.1
Part 2 (Double-blind Placebo Controlled) ACE-083 Arm
15.8
± 4.3
Part 2: Absolute Change in Amount of Intramuscular Fat Tissue to the End of the Double-blind Placebo-controlled Portion of the StudySecondary· 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.
Group
Value
95% CI
Part 2 (Double-blind Placebo Controlled) Placebo Arm
1
± 1.8
Part 2 (Double-blind Placebo Controlled) ACE-083 Arm
-2.1
± 1.9
Part 2: Percent Change in Muscle Strength to the End of the Double-blind Placebo-controlled Portion of the StudySecondary· 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.
Group
Value
95% CI
Part 2 (Double-blind Placebo Controlled) Placebo Arm
-3.4
± 19.8
Part 2 (Double-blind Placebo Controlled) ACE-083 Arm
32.0
± 19.8
Part 2: Percent Change in Muscle Function - Walk/Run Time to the End of the Double-blind Placebo-controlled Portion of the StudySecondary· 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.
Group
Value
95% CI
Part 2 (Double-blind Placebo Controlled) Placebo Arm
-10.1
± 4.7
Part 2 (Double-blind Placebo Controlled) ACE-083 Arm
-8.2
± 4.7
Part 2: Percent Change in Muscle Function - Walk Distance Assessed at the End of the Double-blind Placebo-controlled Portion of the StudySecondary· 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.
Group
Value
95% CI
Part 2 (Double-blind Placebo Controlled) Placebo Arm
6
± 4
Part 2 (Double-blind Placebo Controlled) ACE-083 Arm
9.1
± 3.8
Part 2: Change in Balance and Fall Risk at the End of the Double-blind Placebo-controlled Portion of the Study.Secondary· 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 f
Baseline
Group
Value
95% CI
Part 2 (Double-blind Placebo Controlled) Placebo Arm
52.8
± 1.99
Part 2 (Double-blind Placebo Controlled) ACE-083 Arm
50.8
± 5.58
Day 190
Group
Value
95% CI
Part 2 (Double-blind Placebo Controlled) Placebo Arm
53
± 2.77
Part 2 (Double-blind Placebo Controlled) ACE-083 Arm
51.4
± 5.6
Part 2: Percent Change in Balance and Fall Risk From Baseline to the End of the Double-blind Placebo-controlled Portion of the StudySecondary· 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
Group
Value
95% CI
Part 2 (Double-blind Placebo Controlled) Placebo Arm
0.4
± 3.59
Part 2 (Double-blind Placebo Controlled) ACE-083 Arm
1.3
± 3.13
Part 2: Change in Clinical Examination Score From Baseline to End of the Double-blind Placebo-controlled Portion of the StudySecondary· 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 Bas
Baseline
Group
Value
95% CI
Part 2 (Double-blind Placebo Controlled) Placebo Arm
10.1
± 3.4
Part 2 (Double-blind Placebo Controlled) ACE-083 Arm
10.9
± 3.67
Day 190
Group
Value
95% CI
Part 2 (Double-blind Placebo Controlled) Placebo Arm
9.5
± 3.02
Part 2 (Double-blind Placebo Controlled) ACE-083 Arm
11.2
± 4.77
Part 2: Percent Change in Clinical Examination Score in Baseline to End of the Double-blind Placebo-controlled Portion of the StudySecondary· 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.
Group
Value
95% CI
Part 2 (Double-blind Placebo Controlled) Placebo Arm
-0.2
± 34.63
Part 2 (Double-blind Placebo Controlled) ACE-083 Arm
2.4
± 24.26
Part 2: Change in Patient-reported Quality of Life From Baseline to the End of the Double-blind Placebo-controlled Portion of the StudySecondary· 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 Charcot-Marie-Tooth Health Index (CMT-HI), a disease-specific, patient-reported health index score 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.
Group
Value
95% CI
Part 2 (Double-blind Placebo Controlled) Placebo Arm
-0.2
± 3.3
Part 2 (Double-blind Placebo Controlled) ACE-083 Arm
-2.2
± 3.1
Adverse events — posted to ClinicalTrials.gov
Time frame: Part 1 adverse events (AEs) were collected from baseline through Day 141. For Part 2, AEs were collected from on or after the first double-blind study drug administration up to the end of the double-blind period (Day 190). For the open-label period, adverse events are defined as AEs that started or worsened in intensity on or after the first open-label study drug administration up to the end of the study(Day 393/end-of-study)..
Reporting threshold: 0%.
Adverse-event reports describe events observed during the trial — not all are caused by the drug.
Part 1 Cohort 1(150mg)
Serious: 0/6 (0%)
Deaths: 0/6
Part 1 Cohort 2 (200mg)
Serious: 0/6 (0%)
Deaths: 0/6
Part 1 Cohort 3 (250mg)
Serious: 1/6 (17%)
Deaths: 0/6
Part 2 (Double-blind Placebo Controlled) Placebo Arm
Serious: 1/21 (5%)
Deaths: 0/21
Part 2 (Double-blind Placebo Controlled) ACE-083 Arm
Serious: 0/23 (0%)
Deaths: 0/23
Part 2 (Open Label)
Serious: 1/40 (3%)
Deaths: 0/40
Serious adverse events (3 terms)
Reaction
System
Part 1 Cohort 1(150mg)
Part 1 Cohort 2 (200mg)
Part 1 Cohort 3 (250mg)
Part 2 (Double-blind Place…
Part 2 (Double-blind Place…
Part 2 (Open Label)
Hypokalaemia
Metabolism and nutrition disorders
—
—
—
—
—
—
Suicide attempt
Psychiatric disorders
—
—
—
—
—
—
Cellulitis
Infections and infestations
—
—
—
—
—
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Other adverse events (158 terms — click to expand)
This is a multicenter, phase 2 study to evaluate the safety, tolerability, pharmacodynamics (PD), efficacy, and pharmacokinetics (PK) of ACE-083 in patients with Charcot-Marie-Tooth Disease Type 1 and Type X (CMT1 and CMTX), to be conducted in two parts. Part 1 is non-randomized, open-label, dose-escalation and Part 2 is randomized, double-blind, and placebo-controlled.
Publications & conference data
8 peer-reviewed publications reference this trial (live from Europe PMC):
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Other Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway, NJ USA trials
Trials by the same sponsor.
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Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway, NJ USA
Last refreshed: 26 September 2022
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT03124459.