Clinical trial NCT01588314: Safety and Efficacy of Gabapentin for Neuropathic Pain in Fabry Disease
Official title: Placebo Controlled Trial Evaluating Gabapentin for the Treatment of Small Fiber Neuropathic Pain in Patients With Fabry Disease
Study type: INTERVENTIONAL
Overall status: WITHDRAWN
Phase: PHASE2
Conditions: Fabry Disease
Neuropathic Pain
Keywords: Fabry disease
neuropathic pain
gabapentin
Lead sponsor: University of Minnesota
Sponsor class: OTHER
Collaborators: name: Genzyme, a Sanofi Company
First posted: 2012-04-30
Enrollment: 0
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: CROSSOVER
Primary purpose: TREATMENT

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.

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

Primary outcomes:
measure: average reduction in hydrocodone-acetaminophen use
timeFrame: assessed at the end of the study
Secondary outcomes:
measure: Number and type of adverse events
timeFrame: assessed at the end of the study
measure: Pain levels
timeFrame: assessed at the end of the study
measure: Define therapeutic level for gabapentin
timeFrame: assessed at the end of the study
Other outcomes:
Locations:
city: Minneapolis
country: United States
facility: University of Minnesota, Fariview
state: Minnesota
Study officials:
affiliation: University of Minnesota, Fairview
name: Jeanine R. Jarnes, PharmD
role: PRINCIPAL_INVESTIGATOR
References: