Safety and Efficacy of Gabapentin for Neuropathic Pain in Fabry Disease

NCT ID: NCT01588314

Last Updated: 2019-05-29

Basic Information

• Recruitment Status: WITHDRAWN
• Clinical Phase: PHASE2
• Study Classification: INTERVENTIONAL
• Study Start Date: 2012-04-30
• Study Completion Date: 2014-06-30

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.

Conditions

Fabry Disease; Neuropathic Pain

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: CROSSOVER
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

gabapentin

Group Type: ACTIVE_COMPARATOR

Gabapentin

Intervention Type: DRUG

gabapentin 100 mg capsules

placebo

Group Type: PLACEBO_COMPARATOR

placebo

Intervention Type: DRUG

compounded placebo capsules

Interventions

Gabapentin

gabapentin 100 mg capsules

Intervention Type: DRUG

placebo

compounded placebo capsules

Intervention Type: DRUG

Other Intervention Names

Neurontin

Eligibility Criteria

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

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Genzyme, a Sanofi Company

INDUSTRY

Sponsor Role: collaborator

University of Minnesota

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Jeanine R. Jarnes, PharmD

Role: PRINCIPAL_INVESTIGATOR

University of Minnesota, Fairview

Locations

University of Minnesota, Fariview

Minneapolis, Minnesota, United States

Countries

United States

Other Identifiers

1112M07943

Identifier Type: -

Identifier Source: org_study_id