Fabry Disease, Neuropathic Pain
Conditions
Keywords
Fabry disease, neuropathic pain, gabapentin
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.
Interventions
DRUGGabapentin
gabapentin 100 mg capsules
DRUGplacebo
compounded placebo capsules
Sponsors
Genzyme, a Sanofi Company
University of Minnesota
Study design
Allocation
RANDOMIZED
Intervention model
CROSSOVER
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| average reduction in hydrocodone-acetaminophen use | assessed at the end of the study |
Secondary
| Measure | 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 |
Countries
United States
Outcome results
None listed