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NCT03124459 · ClinicalTrials.gov registry record · Phase 2

Study of ACE-083 in Patients With Charcot-Marie-Tooth Disease

A Phase 2 study, sponsored by Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway, NJ USA.

Terminated
Registry status
Phase 2
Development phase
63
Enrollment target

NCT03124459: Clinical Trial Phase 2 study, sponsored by Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway, NJ USA.

NCT03124459 is a Phase 2 study that was terminated before completion, run by Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway, NJ USA. The registered enrollment target is 63 participants, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (53% lower). According to ClinicalTrials.gov, the official US trial registry.

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The verdict

NCT03124459, a Phase 2 study, was terminated before completion, sponsored by Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway, NJ USA.

TERMINATED
Registry status
Phase 2
Development phase
63 participants
Enrollment target

Study Summary

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.

Primary Outcome

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.

Interventions

  • DRUG Placebo
  • DRUG ACE-083

Trial Details

FieldValue
Enrollment Target 63 participants
Start Date 2017-07-31
Est. Completion 2020-03-11
Phase Phase 2

Why NCT03124459 stopped before completion

NCT03124459 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 63 participants, a relatively small participant target, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (53% lower).

The record links to 0 conditions, and to 2 interventions - of which Placebo is the first listed.

NCT03124459 does not publish any study locations in the registry export this page uses.

Frequently Asked Questions

What is clinical trial NCT03124459 about?

NCT03124459 is a clinical study titled "Study of ACE-083 in Patients With Charcot-Marie-Tooth Disease". 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-es...

What is the current status of trial NCT03124459?

This trial is currently terminated. It is a Phase 2 study. The enrollment target is 63 participants. The study started on 2017-07-31. Estimated completion is 2020-03-11.

What interventions are being tested in trial NCT03124459?

The interventions under investigation include: Placebo (DRUG), ACE-083 (DRUG).

Who is sponsoring clinical trial NCT03124459?

This trial is sponsored by Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway, NJ USA, which has 14 total clinical trials registered on ClinicalTrials.gov.

How this trial's enrollment target compares

Where NCT03124459's enrollment target sits among peer trials

63 1009th of 2000 higher than 985 of 2,000 other Phase 2 trials

participants (enrollment target), bucketed by value

Each bar is a band; taller bars hold more other Phase 2 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.

Source ClinicalTrials.gov registry export · 2026-08-08

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Source: ClinicalTrials.gov NCT03124459, the US trial registry maintained by the National Library of Medicine. NCT03124459 (small enrollment · none site footprint · terminated) retrieved and formatted by PlainTrial, see methodology.