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Study to Compare the Efficacy and Safety of Oral AT1001 and Enzyme Replacement Therapy in Patients With Fabry Disease

NCT01218659 · Status: COMPLETED · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 68

Last updated 2018-11-01

Study results available
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Summary

Study to compare the efficacy and safety of migalastat and enzyme replacement therapy (ERT) in male and female participants with Fabry disease who are currently receiving ERT and who have an alpha galactosidase-A (α Gal-A) mutation that is amenable to migalastat, based on the clinical trial human embryonic kidney cell (HEK) assay.

Conditions

Interventions

DRUG

migalastat hydrochloride

150-mg capsule administered orally QOD

BIOLOGICAL

agalsidase

Agalsidase via intravenous infusion as prescribed by the participant's treating physician and in accordance with the approved prescribing information

Sponsors & Collaborators

  • Amicus Therapeutics

    lead INDUSTRY

Principal Investigators

  • Medical Monitor, Clinical Research · Amicus Therapeutics

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
PARALLEL

Eligibility

Min Age
16 Years
Max Age
74 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2011-09-08
Primary Completion
2014-05-27
Completion
2015-05-28

Countries

  • United States
  • Australia
  • Austria
  • Belgium
  • Brazil
  • Denmark
  • France
  • Italy
  • Japan
  • United Kingdom

Study Locations

More Related Trials

Entities

Diseases

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 NCT01218659 on ClinicalTrials.gov