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Neuropathic Pain and Fabry Disease

NCT ID: NCT00168974

Last Updated: 2007-11-16

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Total Enrollment

25 participants

Study Classification

OBSERVATIONAL

Study Start Date

2004-01-31

Study Completion Date

2007-11-30

Brief Summary

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Fabry disease is a rare X-linked lysosomal storage disorder. The mutations result in a deficiency of the lysosomal enzyme α-galactosidase causing accumulation of glycosphingolipids in the vascular endothelial cells and many other tissues. An early sign of the disease is painful small fibre neuropathy presenting in two forms: 1. a constant burning sensation in the hand and feet and 2. Fabry crises consisting of attacks of excruciating pain. Given the X-linked inheritance, male patients are severely affected. Recently attention has been drawn to female patients whether they also show signs of nerve involvement.

The purpose of this study is to evaluate the small fibre neuropathy in female Fabry patients. Correlation with X-chromosome inactivation will be attempted. Recombinant human α-galactosidase A is now available for patients. A part of this study is evaluation the long term efficacy of enzyme replacement therapy in female patients with Fabry disease and neuropathy.

Male family members with Fabry disease will be examined.

Detailed Description

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Conditions

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Fabry Disease

Study Design

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Study Time Perspective

PROSPECTIVE

Eligibility Criteria

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Inclusion Criteria

* All adult patients with confirmed Fabry disease

Exclusion Criteria

* Patients who cannot cooperate
* Patients who are unable to understand the purpose
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

Yes

Sponsors

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Danish Pain Research Center

OTHER

Sponsor Role lead

Principal Investigators

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Anette T Moller, MD

Role: PRINCIPAL_INVESTIGATOR

Danish Pain Research Center

Troels S Jensen, MD, PhD

Role: STUDY_CHAIR

Danish Pain Research Center

Locations

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Danish Pain Research Center, Aarhus University Hospital

Aarhus, , Denmark

Site Status

Countries

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Denmark

Other Identifiers

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Fabry2003

Identifier Type: -

Identifier Source: org_study_id