Safety and Efficacy of Gabapentin for Neuropathic Pain in Fabry Disease

University of Minnesota (UMN)

Status and phase: Withdrawn; Phase 2

Conditions: Fabry Disease; Neuropathic Pain

Treatments: Drug: placebo; Drug: Gabapentin

Study type: Interventional

Funder types: Other; Industry

Identifiers

NCT01588314

1112M07943

Details and patient eligibility

About

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.

Full 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.

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Crossover Assignment

Masking: Quadruple Blind

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0 participants in 2 patient groups, including a placebo group

gabapentin

Active Comparator group

Treatment:

Drug: Gabapentin

placebo

Placebo Comparator group

Treatment:

Drug: placebo