Safety and Efficacy of Gabapentin for Neuropathic Pain in Fabry Disease

Phase 2

Withdrawn

• Conditions: Neuropathic Pain; Fabry Disease
• Interventions: Drug: placebo; Drug: Gabapentin

• Registration Number: NCT01588314
• Lead Sponsor: University of Minnesota

Brief Summary

The purpose of this study is to determine efficacy of gabapentin vs. placebo at controlling peripheral neuropathic pain in patients with Fabry disease, and reducing their use of opioid analgesics. The investigators are conducting a randomized, double-blind, placebo controlled, single center, cross-over study. The primary endpoint is percent reduction in patients' use of hydrocodone-acetaminophen.

Detailed Description

Fabry disease is an X-linked lysosomal storage disorder which results from reduced activity of the enzyme α-galactosidase A. This reduced enzyme activity results in accumulation of globotriaosylceramide (GL-3), which causes disturbances in multiple organ systems. Patients often complain of acroparesthesia, caused by small-fiber neuropathy. Limited information is available regarding effective treatments for small-fiber neuropathic pain in Fabry disease, and no standard-of-care has yet been established. Opioid analgesics are often used because of their pharmacokinetic properties. While effective, the use of opioids has complications such as constipation, physical dependence and addiction. The purpose of this study is to determine efficacy of gabapentin vs. placebo at controlling peripheral neuropathic pain and reducing the use of opioid analgesics. The investigators are conducting a randomized, double-blind, placebo controlled, single center, cross-over study. The primary endpoint is percent reduction in hydrocodone-acetaminophen use.

Study Timeline

• Study Start: 2012-04
• Study First Submitted: 2012-04-26
• Study First Submitted QC: 2012-04-27
• Study First Posted: 2012-04-30
• Primary Completion: 2014-06
• Study Completion: 2014-06
• Status Verified: 2019-05
• Last Update Submitted: 2019-05-24
• Last Update Posted: 2019-05-29

Recruitment & Eligibility

• Status: WITHDRAWN
• Sex: All
• Target Recruitment: Not specified

Inclusion Criteria

• diagnosis of Fabry Disease
• age ≥ 18 years of age at study enrollment
• current neuropathic pain at any severity level

Exclusion Criteria

• known sensitivity or allergy to study drug
• history of illicit drug use
• pregnancy
• suicidal thoughts at study enrollment as assess by the C-SSRS

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: CROSSOVER

Arm && Interventions

Group	Intervention	Description
placebo	placebo	-
gabapentin	Gabapentin	-

Primary Outcome Measures

Name	Time	Method
average reduction in hydrocodone-acetaminophen use	assessed at the end of the study

Secondary Outcome Measures

Name	Time	Method
Pain levels	assessed at the end of the study
Number and type of adverse events	assessed at the end of the study
Define therapeutic level for gabapentin	assessed at the end of the study

Trial Locations

Locations (1)

University of Minnesota, Fariview; 🇺🇸 Minneapolis, Minnesota, United States