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A Study of Deflazacort (Emflaza®) in Participants With Limb-Girdle Muscular Dystrophy 2I (LGMD2I)

NCT03783923 · Status: TERMINATED · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 11

Last updated 2022-06-27

Study results available
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Summary

This study is designed to evaluate the safety and efficacy of deflazacort in participants with LGMD2I. Most participants enrolled will have a screening visit and 3 additional visits (after 1, 13, and 26 weeks of treatment).

Conditions

  • Limb-Girdle Muscular Dystrophy

Interventions

DRUG

Deflazacort

Deflazacort tablet will be administered as per the dose and schedule specified in the arm.

Sponsors & Collaborators

Principal Investigators

  • Cristobal Passalacqua, MD · PTC Therapeutics

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2019-10-31
Primary Completion
2021-01-01
Completion
2021-01-01
FDA Drug
Yes

Countries

  • United States
  • Canada
  • Denmark
  • France
  • Germany
  • Norway
  • Russia
  • Sweden

Study Locations

More Related Trials

Entities

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 NCT03783923 on ClinicalTrials.gov