Safety and Efficacy of Gabapentin for Neuropathic Pain in Fabry Disease

Study Overview
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Brief Summary
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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
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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.

Official Title
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Placebo Controlled Trial Evaluating Gabapentin for the Treatment of Small Fiber Neuropathic Pain in Patients With Fabry Disease

Conditions
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Fabry Disease, Neuropathic Pain

Intervention / Treatment
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* Drug: Gabapentin
* Drug: placebo


Participation Criteria
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Eligibility Criteria
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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

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: Quadruple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Active Comparator: gabapentin<br> | Drug: Gabapentin<br>* gabapentin 100 mg capsules<br>* Other names: Neurontin;|
| Placebo Comparator: placebo<br> | Drug: placebo<br>* compounded placebo capsules<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| average reduction in hydrocodone-acetaminophen use |  | assessed at the end of the study | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number and type of adverse events |  | assessed at the end of the study | 
| Pain levels |  | assessed at the end of the study | 
| Define therapeutic level for gabapentin |  | assessed at the end of the study | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Fabry disease, neuropathic pain, gabapentin