Safety and Efficacy of Gabapentin for Neuropathic Pain in Fabry Disease
This study is currently recruiting participants.
Verified March 2013 by University of Minnesota - Clinical and Translational Science Institute
Sponsor:
University of Minnesota - Clinical and Translational Science Institute
Collaborator:
Genzyme
Information provided by (Responsible Party):
University of Minnesota - Clinical and Translational Science Institute
ClinicalTrials.gov Identifier:
NCT01588314
First received: April 26, 2012
Last updated: March 29, 2013
Last verified: March 2013
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Purpose
Fabry disease is an X-linked lysosomal storage disorder which results from reduced activity of the enzyme α-galactosidase A. Accumulation of globotriaosylceramide (GL-3) 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 exists. Opioid analgesics are often used because of their pharmacokinetic properties. The use of opioids, while effective, 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.
| Condition | Intervention | Phase |
|---|---|---|
|
Fabry Disease Neuropathic Pain |
Drug: Gabapentin Drug: placebo |
Phase 2 |
| Study Type: | Interventional |
| Study Design: | Allocation: Randomized Endpoint Classification: Safety/Efficacy Study Intervention Model: Crossover Assignment Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor) Primary Purpose: Treatment |
| Official Title: | Placebo Controlled Trial Evaluating Gabapentin for the Treatment of Small Fiber Neuropathic Pain in Patients With Fabry Disease |
Resource links provided by NLM:
Genetics Home Reference related topics:
Chanarin-Dorfman syndrome
cholesteryl ester storage disease
Fabry disease
Farber lipogranulomatosis
Schindler disease
succinic semialdehyde dehydrogenase deficiency
MedlinePlus related topics:
Dietary Fiber
U.S. FDA Resources
Further study details as provided by University of Minnesota - Clinical and Translational Science Institute:
Primary Outcome Measures:
- average reduction in hydrocodone-acetaminophen use [ Time Frame: assessed at the end of the study ] [ Designated as safety issue: No ]
Secondary Outcome Measures:
- Number and type of adverse events [ Time Frame: assessed at the end of the study ] [ Designated as safety issue: Yes ]
- Pain levels [ Time Frame: assessed at the end of the study ] [ Designated as safety issue: No ]
- Define therapeutic level for gabapentin [ Time Frame: assessed at the end of the study ] [ Designated as safety issue: No ]
| Estimated Enrollment: | 15 |
| Study Start Date: | April 2012 |
| Estimated Study Completion Date: | December 2015 |
| Estimated Primary Completion Date: | December 2015 (Final data collection date for primary outcome measure) |
| Arms | Assigned Interventions |
|---|---|
| Active Comparator: gabapentin |
Drug: Gabapentin
gabapentin 100 mg capsules
|
| Placebo Comparator: placebo |
Drug: placebo
compounded placebo capsules
|
Eligibility| Ages Eligible for Study: | 18 Years and older |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
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
Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01588314
Contacts
| Contact: Jeanine Utz, PharmD | 612-626-5131 | jutz1@fairview.org |
| Contact: Colleen Doyen, BA | 612-672-5213 | cdoyen1@fairview.org |
Locations
| United States, Minnesota | |
| University of Minnesota, Fariview | Recruiting |
| Minneapolis, Minnesota, United States, 55455 | |
| Contact: Jeanine Utz, PharmD 612-626-5131 jutz1@fairview.org | |
| Sub-Investigator: Chester Whitley, PhD, MD | |
| Principal Investigator: Jeanine Utz, PharmD | |
| Sub-Investigator: Kyle Rudser, PhD | |
Sponsors and Collaborators
University of Minnesota - Clinical and Translational Science Institute
Genzyme
Investigators
| Principal Investigator: | Jeanine Utz, PharmD | University of Minnesota, Fairview |
More Information
No publications provided
| Responsible Party: | University of Minnesota - Clinical and Translational Science Institute |
| ClinicalTrials.gov Identifier: | NCT01588314 History of Changes |
| Other Study ID Numbers: | 1112M07943 |
| Study First Received: | April 26, 2012 |
| Last Updated: | March 29, 2013 |
| Health Authority: | United States: Institutional Review Board |
Keywords provided by University of Minnesota - Clinical and Translational Science Institute:
|
Fabry disease neuropathic pain gabapentin |
Additional relevant MeSH terms:
|
Fabry Disease Lysosomal Storage Diseases, Nervous System Brain Diseases, Metabolic, Inborn Brain Diseases, Metabolic Brain Diseases Central Nervous System Diseases Nervous System Diseases Genetic Diseases, X-Linked Genetic Diseases, Inborn Lysosomal Storage Diseases Metabolic Diseases Lipid Metabolism Disorders Peripheral Nervous System Diseases Neuromuscular Diseases Neuralgia |
Sphingolipidoses Metabolism, Inborn Errors Lipidoses Lipid Metabolism, Inborn Errors Pain Neurologic Manifestations Signs and Symptoms Gabapentin Analgesics Sensory System Agents Peripheral Nervous System Agents Physiological Effects of Drugs Pharmacologic Actions Central Nervous System Agents Therapeutic Uses |
ClinicalTrials.gov processed this record on June 18, 2013