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Treatment of Oculopharyngeal Muscular Dystrophy With Trehalose

NCT04226924 · Status: WITHDRAWN · Phase: PHASE2 · Type: INTERVENTIONAL

Last updated 2020-01-13

No results posted yet for this study

Summary

BB-OPMD-202 is a randomized, double-blind, placebo-controlled study of IV trehalose for treatment of OPMD. The study includes a 4-week screening period, a 24-week blinded treatment period during which patients will receive weekly infusions of trehalose or placebo, followed by a 24-week open-label extension period during which all patients will receive weekly infusions of trehalose. Patients will undergo a safety follow-up assessment 4 weeks after their last treatment.

Conditions

  • Oculopharyngeal Muscular Dystrophy

Interventions

DRUG

Trehalose

90 mg/ml trehalose solution for IV infusion

Sponsors & Collaborators

  • Bioblast Pharma Ltd.

    lead INDUSTRY

Principal Investigators

  • Bernard Brais, MD · McGill University

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
CROSSOVER

Eligibility

Min Age
50 Years
Max Age
70 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2017-06-15
Primary Completion
2018-02-15
Completion
2018-08-15

Countries

  • Canada

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 NCT04226924 on ClinicalTrials.gov