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Safety and Efficacy of Gabapentin for Neuropathic Pain in Fabry Disease

This study has been withdrawn prior to enrollment.
(No patients were enrolled. No data & no study results to report.)
Sponsor:
Collaborator:
Genzyme, a Sanofi Company
Information provided by (Responsible Party):
University of Minnesota - Clinical and Translational Science Institute
ClinicalTrials.gov Identifier:
NCT01588314
First received: April 26, 2012
Last updated: August 10, 2016
Last verified: August 2016
  Purpose
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.

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:


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 ]

Enrollment: 0
Study Start Date: April 2012
Study Completion Date: June 2014
Primary Completion Date: June 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Active Comparator: gabapentin Drug: Gabapentin
gabapentin 100 mg capsules
Other Name: Neurontin
Placebo Comparator: placebo Drug: placebo
compounded placebo capsules

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

Ages Eligible for Study:   18 Years and older   (Adult, Senior)
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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01588314

Locations
United States, Minnesota
University of Minnesota, Fariview
Minneapolis, Minnesota, United States, 55455
Sponsors and Collaborators
University of Minnesota - Clinical and Translational Science Institute
Genzyme, a Sanofi Company
Investigators
Principal Investigator: Jeanine Utz, PharmD University of Minnesota, Fairview
  More Information

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: August 10, 2016
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
Nervous System Diseases
Peripheral Nervous System Diseases
Neuromuscular Diseases
Lysosomal Storage Diseases, Nervous System
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Cerebral Small Vessel Diseases
Cerebrovascular Disorders
Vascular Diseases
Cardiovascular Diseases
Genetic Diseases, X-Linked
Genetic Diseases, Inborn
Lysosomal Storage Diseases
Metabolic Diseases
Lipid Metabolism Disorders
Neuralgia
Pain
Neurologic Manifestations
Signs and Symptoms
Sphingolipidoses
Metabolism, Inborn Errors
Lipidoses
Lipid Metabolism, Inborn Errors
Gabapentin
gamma-Aminobutyric Acid
Analgesics
Sensory System Agents

ClinicalTrials.gov processed this record on December 08, 2016