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Clinical Trial Details — Status: Completed

Administrative data

NCT number NCT02260388
Other study ID # STUDY00001500
Secondary ID PCORI-1306-02496
Status Completed
Phase Phase 4
First received
Last updated
Start date October 2014
Est. completion date September 2017

Study information

Verified date June 2018
Source University of Kansas Medical Center
Contact n/a
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial 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).


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 information / eligibility

Status Completed
Enrollment 402
Est. completion date September 2017
Est. primary completion date September 2017
Accepts healthy volunteers No
Gender All
Age group 30 Years and older
Eligibility Inclusion Criteria:

- 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.

Exclusion Criteria:

- 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


Related Conditions & MeSH terms


Intervention

Drug:
Nortriptyline

Duloxetine

Pregabalin

Mexiletine


Locations

Country Name City State
Canada Toronto General Hospital Toronto Ontario
United States University of Michigan Ann Arbor Michigan
United States University of Colorado Denver Anschutz Campus Aurora Colorado
United States Austin Neuromuscular Center Austin Texas
United States Sara Austin, MD, PA Austin Texas
United States Seton Brain and Spine Institute Austin Texas
United States Brigham and Women's Hospital Boston Massachusetts
United States University of Buffalo School of Medicine and Biomedical Sciences Buffalo New York
United States University of Vermont Medical Center Burlington Vermont
United States University of Virginia Charlottesville Virginia
United States University of Cincinnati Cincinnati Ohio
United States Cleveland Clinic Cleveland Ohio
United States Colorado Springs Neurological Associates Colorado Springs Colorado
United States The Ohio State University Wexner Medical Center Columbus Ohio
United States Texas Neurology Dallas Texas
United States University of Texas Southwestern Dallas Texas
United States Mercy Medical Center - Des Moines Des Moines Iowa
United States Henry Ford Hospital Detroit Michigan
United States University of Florida - Gainesville Gainesville Florida
United States NorthShore Neurological Institute Glenview Illinois
United States Spectrum Health System Grand Rapids Michigan
United States Penn State Medical Center Hershey Pennsylvania
United States University of Texas Health Science Center at Houton Houston Texas
United States Hutchinson Clinic, PA Hutchinson Kansas
United States Indiana University Indianapolis Indiana
United States University of Iowa Hospitals and Clinics Iowa City Iowa
United States University of Florida Health Science Center - Jacksonville Jacksonville Florida
United States University of Kansas Medical Center Kansas City Kansas
United States Grand Medical Clinic Katy Texas
United States Cedars-Sinai Medical Center Los Angeles California
United States Norton Neurology Services Louisville Kentucky
United States University of Miami Miller School of Medicine Miami Florida
United States University of Minnesota Minneapolis Minnesota
United States Neurology Clinic of Central Texas New Braunfels Texas
United States Mount Sinai Beth Israel New York New York
United States University of Nebraska Medical Center Omaha Nebraska
United States Neurological Services of Orlando Research Orlando Florida
United States Barrow Neurological Institute Phoenix Arizona
United States Phoenix Neurological Associates Phoenix Arizona
United States Oregon Health & Science University Portland Oregon
United States Saint Louis University Saint Louis Missouri
United States University of Utah Salt Lake City Utah
United States University of Texas Health Science Center in San Antonio San Antonio Texas
United States California Pacific Medical Center San Francisco California
United States University of California San Francisco San Francisco California
United States University of South Florida Tampa Florida

Sponsors (2)

Lead Sponsor Collaborator
University of Kansas Medical Center Patient-Centered Outcomes Research Institute

Countries where clinical trial is conducted

United States,  Canada, 

Outcome

Type Measure Description Time frame Safety issue
Primary 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 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. 12 weeks
Secondary SF12 Health Composite Scores 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.
12 weeks
Secondary PROMIS Pain Interference Short Form v1.0 8a T Score 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.
12 weeks
Secondary PROMIS Fatigue Short Form v1.0 8a 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.
12 Weeks
Secondary PROMIS Sleep Disturbance Short Form v1.0 8a 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
12 weeks