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Safety Tolerability and Efficacy Study of Cabaletta to Treat Oculopharyngeal Muscular Dystrophy (OPMD) Patients

NCT02015481 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 25

Last updated 2017-10-17

Study results available
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Summary

The Purpose of this study is to assess the Safety, Tolerability and Efficacy of Intravenous Cabaletta® in Oculopharyngeal Muscular Dystrophy (OPMD) Patients.

Conditions

  • Oculopharyngeal Muscular Dystrophy

Interventions

DRUG

Cabaletta

Sponsors & Collaborators

  • Bioblast Pharma Ltd.

    lead INDUSTRY

Principal Investigators

  • Yoseph Caraco, M.D · Hadassah Medical Organization

  • Bernard Brais, MD · McGill University

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

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

Timeline & Regulatory

Start
2014-02-28
Primary Completion
2016-04-30
Completion
2016-04-30

Countries

  • United States
  • Canada
  • Israel

Study Locations

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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 NCT02015481 on ClinicalTrials.gov