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

NCT03124459 · Status: TERMINATED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 63

Last updated 2022-09-26

Study results available
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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.

Conditions

  • Charcot-Marie-Tooth Disease

Interventions

DRUG

ACE-083

Part 1 - Recombinant fusion protein. Part 2 - Recombinant fusion protein or buffer solution.

DRUG

Placebo

Recombinant fusion protein or buffer solution

Sponsors & Collaborators

  • Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway, NJ USA

    lead INDUSTRY

Principal Investigators

  • Jay Backstrom, MD · Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway, NJ USA

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
PARALLEL

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2017-07-31
Primary Completion
2020-03-11
Completion
2020-03-11
FDA Drug
Yes

Countries

  • United States

Study Locations

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Entities

Drugs

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03124459 on ClinicalTrials.gov