Patient Assisted Intervention for Neuropathy: Comparison of Treatment in Real Life Situations
- Conditions
- Cryptogenic Sensory Polyneuropathy
- Interventions
- Registration Number
- NCT02260388
- Lead Sponsor
- University of Kansas Medical Center
- Brief Summary
The purpose of this large comparative effectiveness study led by Richard J. Barohn, MD, of the University of Kansas Medical Center, is to learn about the safety and effectiveness of nortriptyline, duloxetine, pregabalin and mexiletine in treating cryptogenic sensory polyneuropathy (CSPN).
- Detailed Description
The goal of this research project is to find the best drug for the treatment of pain in patients with CSPN. While the pharmaceutical industry has focused attention on drugs for treating diabetic sensory neuropathy (DSPN), and two drugs are now FDA approved, there have not been any prospective trials in CSPN. And, because there are no studies with CSPN patients, insurance carriers often reject authorizing prescriptions for some drugs for patients with CSPN.
There are four drugs that will be tested in this study: nortriptyline, duloxetine, pregabalin and mexiletine. These drugs are not approved by the FDA for the treatment of CSPN and are considered "investigational" in this study.
There are two periods in this study: Screening/Baseline and Study Drug. During the Screening/Baseline period the researchers will determine eligibility for potential subjects. During the second period, eligible patients who consented to participate will take the study drug. Participants will be randomized to receive one of the four drugs in this study. Participants will know which drug they are taking. Participants will not be allowed to switch groups and receive a different drug during the study.
This study uses an adaptive study design. This means the study can enroll less participants and provide better conclusions. The study design allows the researchers the ability to make changes to the approach of the study or to stop the study early if there are strong results.
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 402
- Diagnosis of cryptogenic sensory polyneuropathy.
- Likert Pain Score of greater than or equal to 4.
- Must not currently be on nortriptyline, duloxetine, pregabalin or mexiletine or similar class of medication for at least 7 days from baseline study visit.
- Any medical condition or current medication that would prevent them from taking either nortriptyline, duloxetine, pregabalin or mexiletine.
- Unable to give consent.
- Unable or not willing to comply with the study.
- Other causes for polyneuropathy.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Nortriptyline Nortriptyline Nortriptyline - 25 mg daily for 1 week at bedtime, then 50 mg daily at bedtime for 1 week, then 75 mg daily at bedtime for the remainder of the study. Mexiletine Mexiletine Mexiletine - 200 mg at bedtime for 1 week, then 200 mg 2 times per day for 1 week, then 200 mg 3 times per day for the remainder of the study. Duloxetine Duloxetine Duloxetine - 20 mg daily for 1 week, then 40 mg daily for 1 week, then 60 mg daily for the remainder of the study. Pregabalin Pregabalin Pregabalin - 100 mg at bedtime for 1 week, then 100 mg 2 times per day for 1 week, then 100 mg 3 times per day for the remainder of the study.
- Primary Outcome Measures
Name Time Method Co-Primary Measures: Percent of Patients With at Least a 50% Decrease in Likert Pain Scale From Baseline to Week 12 Follow Up and Percent of Patients That Quit 12 weeks The final outcome of the study is a combination of two endpoints, efficacy and quit or treatment discontinuation rates. The first endpoint was a patient responder-defined measure of efficacy. A patient was deemed efficacious if a 50% or more reduction was observed in the Likert pain-scale from the baseline visit to the 12 week visit (i.e. 6 at baseline to 3 or less at week 12). The second endpoint was the observed percentage of patients who discontinued treatment prior to the last follow up visit for any reason or were lost to follow up. The utility function, which combines efficacy and quit rates, was used to drive the adaptive randomization, stopping criteria, and final analysis conclusions.
- Secondary Outcome Measures
Name Time Method SF12 Health Composite Scores 12 weeks SF-12v2® Health Survey Standard The Optum™ SF-12v2® Health Survey is a shorter version of the SF-36v2® Health Survey that uses just 12 questions to measure functional health and well-being from the patient's point of view.
Survey provides psychometrically-based physical component summary (PCS) and mental component summary (MCS) scores.
Scores are calibrated so that 50 is the average score or norm, standard deviation = 10.
Higher scores indicate better health for both mental and physical component summary scores.PROMIS Pain Interference Short Form v1.0 8a T Score 12 weeks Higher scores for pain interference represents worse outcome (more pain interference) T-score metric: 50 is the mean of a relevant reference population and 10 is the standard deviation (SD) of that population.
On the T-score metric: A score of 40 is one SD lower than the mean of the reference population; A score of 60 is one SD higher than the mean of the reference population.PROMIS Fatigue Short Form v1.0 8a 12 Weeks Higher scores for fatigue represents worse outcome (more fatigue). T-score metric: 50 is the mean of a relevant reference population and 10 is the standard deviation (SD) of that population.
On the T-score metric: A score of 40 is one SD lower than the mean of the reference population; A score of 60 is one SD higher than the mean of the reference population.PROMIS Sleep Disturbance Short Form v1.0 8a 12 weeks Higher scores for sleep disturbance represents worse outcome (more sleep disturbance).
T-score metric: 50 is the mean of a relevant reference population and 10 is the standard deviation (SD) of that population.
On the T-score metric: A score of 40 is one SD lower than the mean of the reference population; A score of 60 is one SD higher than the mean of the reference population.
Higher scores equals more of the concept being measured
Trial Locations
- Locations (46)
Cedars-Sinai Medical Center
🇺🇸Los Angeles, California, United States
University of Miami Miller School of Medicine
🇺🇸Miami, Florida, United States
Indiana University
🇺🇸Indianapolis, Indiana, United States
Brigham and Women's Hospital
🇺🇸Boston, Massachusetts, United States
Cleveland Clinic
🇺🇸Cleveland, Ohio, United States
University of Texas Health Science Center at Houton
🇺🇸Houston, Texas, United States
University of Cincinnati
🇺🇸Cincinnati, Ohio, United States
University of California San Francisco
🇺🇸San Francisco, California, United States
Mercy Medical Center - Des Moines
🇺🇸Des Moines, Iowa, United States
Barrow Neurological Institute
🇺🇸Phoenix, Arizona, United States
Phoenix Neurological Associates
🇺🇸Phoenix, Arizona, United States
Colorado Springs Neurological Associates
🇺🇸Colorado Springs, Colorado, United States
University of Colorado Denver Anschutz Campus
🇺🇸Aurora, Colorado, United States
Neurological Services of Orlando Research
🇺🇸Orlando, Florida, United States
University of Florida Health Science Center - Jacksonville
🇺🇸Jacksonville, Florida, United States
NorthShore Neurological Institute
🇺🇸Glenview, Illinois, United States
University of Iowa Hospitals and Clinics
🇺🇸Iowa City, Iowa, United States
Hutchinson Clinic, PA
🇺🇸Hutchinson, Kansas, United States
Norton Neurology Services
🇺🇸Louisville, Kentucky, United States
Henry Ford Hospital
🇺🇸Detroit, Michigan, United States
University of Michigan
🇺🇸Ann Arbor, Michigan, United States
University of Minnesota
🇺🇸Minneapolis, Minnesota, United States
University of Buffalo School of Medicine and Biomedical Sciences
🇺🇸Buffalo, New York, United States
University of Nebraska Medical Center
🇺🇸Omaha, Nebraska, United States
Saint Louis University
🇺🇸Saint Louis, Missouri, United States
Oregon Health & Science University
🇺🇸Portland, Oregon, United States
The Ohio State University Wexner Medical Center
🇺🇸Columbus, Ohio, United States
Mount Sinai Beth Israel
🇺🇸New York, New York, United States
Penn State Medical Center
🇺🇸Hershey, Pennsylvania, United States
Austin Neuromuscular Center
🇺🇸Austin, Texas, United States
Sara Austin, MD, PA
🇺🇸Austin, Texas, United States
Texas Neurology
🇺🇸Dallas, Texas, United States
Seton Brain and Spine Institute
🇺🇸Austin, Texas, United States
University of Texas Southwestern
🇺🇸Dallas, Texas, United States
University of Utah
🇺🇸Salt Lake City, Utah, United States
Grand Medical Clinic
🇺🇸Katy, Texas, United States
Neurology Clinic of Central Texas
🇺🇸New Braunfels, Texas, United States
University of Virginia
🇺🇸Charlottesville, Virginia, United States
University of Texas Health Science Center in San Antonio
🇺🇸San Antonio, Texas, United States
University of Vermont Medical Center
🇺🇸Burlington, Vermont, United States
University of Florida - Gainesville
🇺🇸Gainesville, Florida, United States
California Pacific Medical Center
🇺🇸San Francisco, California, United States
University of South Florida
🇺🇸Tampa, Florida, United States
Toronto General Hospital
🇨🇦Toronto, Ontario, Canada
Spectrum Health System
🇺🇸Grand Rapids, Michigan, United States
University of Kansas Medical Center
🇺🇸Kansas City, Kansas, United States