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A Phase 2 Study to Evaluate the Dose and Pharmacodynamic Efficacy of Sialic Acid-Extended Release (SA-ER) Tablets in Patients With GNE Myopathy or Hereditary Inclusion Body Myopathy

NCT01517880 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 46

Last updated 2016-06-16

No results posted yet for this study

Summary

GNE myopathy or hereditary inclusion body myopathy (HIBM) is a severe progressive metabolic myopathy caused by a defect in the biosynthetic pathway for sialic acid (SA).

Conditions

  • GNE Myopathy
  • Hereditary Inclusion Body Myopathy

Interventions

DRUG

Sialic Acid Extended Release (SA-ER)

SA-ER will be administered in doses of 3000mg per day or 6000mg per day

DRUG

Placebo

Subjects will be randomized to the placebo arm for the first 24 weeks of the study. Then, subjects in this arm will be re-randomized into either the 3,000 mg per day or 6,000 mg per day arm for the remaining 24 weeks of the study (total study duration 48 weeks).

Sponsors & Collaborators

Principal Investigators

  • Alan Pestronk, MD · Washington University School of Medicine

  • Perry Shieh, MD · University of California, Los Angeles

  • Yoseph Caraco, MD · Hadassah University Hospital

  • Heather Lau, MD · NYU Langone Health

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
PARALLEL

Eligibility

Min Age
18 Years
Max Age
65 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2012-05-31
Primary Completion
2013-11-30
Completion
2013-11-30

Countries

  • United States
  • Israel

Study Locations

More Related Trials

Entities

Drugs
Companies

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 NCT01517880 on ClinicalTrials.gov