Patient Assisted Intervention for Neuropathy: Comparison of Treatment in Real Life Situations

NCT ID: NCT02260388

Last Updated: 2018-07-06

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE4

Total Enrollment

402 participants

Study Classification

INTERVENTIONAL

Study Start Date

2014-10-31

Study Completion Date

2017-09-30

Brief Summary

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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

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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.

Conditions

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Cryptogenic Sensory Polyneuropathy

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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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.

Group Type EXPERIMENTAL

Nortriptyline

Intervention Type DRUG

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.

Group Type EXPERIMENTAL

Duloxetine

Intervention Type DRUG

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.

Group Type EXPERIMENTAL

Pregabalin

Intervention Type DRUG

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.

Group Type EXPERIMENTAL

Mexiletine

Intervention Type DRUG

Interventions

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Nortriptyline

Intervention Type DRUG

Duloxetine

Intervention Type DRUG

Pregabalin

Intervention Type DRUG

Mexiletine

Intervention Type DRUG

Eligibility Criteria

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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.
Minimum Eligible Age

30 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Patient-Centered Outcomes Research Institute

OTHER

Sponsor Role collaborator

University of Kansas Medical Center

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Richard Barohn, MD

Role: PRINCIPAL_INVESTIGATOR

University of Kansas Medical Center

Locations

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Barrow Neurological Institute

Phoenix, Arizona, United States

Site Status

Phoenix Neurological Associates

Phoenix, Arizona, United States

Site Status

Cedars-Sinai Medical Center

Los Angeles, California, United States

Site Status

California Pacific Medical Center

San Francisco, California, United States

Site Status

University of California San Francisco

San Francisco, California, United States

Site Status

University of Colorado Denver Anschutz Campus

Aurora, Colorado, United States

Site Status

Colorado Springs Neurological Associates

Colorado Springs, Colorado, United States

Site Status

University of Florida - Gainesville

Gainesville, Florida, United States

Site Status

University of Florida Health Science Center - Jacksonville

Jacksonville, Florida, United States

Site Status

University of Miami Miller School of Medicine

Miami, Florida, United States

Site Status

Neurological Services of Orlando Research

Orlando, Florida, United States

Site Status

University of South Florida

Tampa, Florida, United States

Site Status

NorthShore Neurological Institute

Glenview, Illinois, United States

Site Status

Indiana University

Indianapolis, Indiana, United States

Site Status

Mercy Medical Center - Des Moines

Des Moines, Iowa, United States

Site Status

University of Iowa Hospitals and Clinics

Iowa City, Iowa, United States

Site Status

Hutchinson Clinic, PA

Hutchinson, Kansas, United States

Site Status

University of Kansas Medical Center

Kansas City, Kansas, United States

Site Status

Norton Neurology Services

Louisville, Kentucky, United States

Site Status

Brigham and Women's Hospital

Boston, Massachusetts, United States

Site Status

University of Michigan

Ann Arbor, Michigan, United States

Site Status

Henry Ford Hospital

Detroit, Michigan, United States

Site Status

Spectrum Health System

Grand Rapids, Michigan, United States

Site Status

University of Minnesota

Minneapolis, Minnesota, United States

Site Status

Saint Louis University

St Louis, Missouri, United States

Site Status

University of Nebraska Medical Center

Omaha, Nebraska, United States

Site Status

University of Buffalo School of Medicine and Biomedical Sciences

Buffalo, New York, United States

Site Status

Mount Sinai Beth Israel

New York, New York, United States

Site Status

University of Cincinnati

Cincinnati, Ohio, United States

Site Status

Cleveland Clinic

Cleveland, Ohio, United States

Site Status

The Ohio State University Wexner Medical Center

Columbus, Ohio, United States

Site Status

Oregon Health & Science University

Portland, Oregon, United States

Site Status

Penn State Medical Center

Hershey, Pennsylvania, United States

Site Status

Austin Neuromuscular Center

Austin, Texas, United States

Site Status

Sara Austin, MD, PA

Austin, Texas, United States

Site Status

Seton Brain and Spine Institute

Austin, Texas, United States

Site Status

Texas Neurology

Dallas, Texas, United States

Site Status

University of Texas Southwestern

Dallas, Texas, United States

Site Status

University of Texas Health Science Center at Houton

Houston, Texas, United States

Site Status

Grand Medical Clinic

Katy, Texas, United States

Site Status

Neurology Clinic of Central Texas

New Braunfels, Texas, United States

Site Status

University of Texas Health Science Center in San Antonio

San Antonio, Texas, United States

Site Status

University of Utah

Salt Lake City, Utah, United States

Site Status

University of Vermont Medical Center

Burlington, Vermont, United States

Site Status

University of Virginia

Charlottesville, Virginia, United States

Site Status

Toronto General Hospital

Toronto, Ontario, Canada

Site Status

Countries

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United States Canada

References

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Barohn RJ, Gajewski B, Pasnoor M, Brown A, Herbelin LL, Kimminau KS, Mudaranthakam DP, Jawdat O, Dimachkie MM; Patient Assisted Intervention for Neuropathy: Comparison of Treatment in Real Life Situations (PAIN-CONTRoLS) Study Team; Iyadurai S, Stino A, Kissel J, Pascuzzi R, Brannagan T, Wicklund M, Ahmed A, Walk D, Smith G, Quan D, Heitzman D, Tobon A, Ladha S, Wolfe G, Pulley M, Hayat G, Li Y, Thaisetthawatkul P, Lewis R, Biliciler S, Sharma K, Salajegheh K, Trivedi J, Mallonee W, Burns T, Jacoby M, Bril V, Vu T, Ramchandren S, Bazant M, Austin S, Karam C, Hussain Y, Kutz C, Twydell P, Scelsa S, Kushlaf H, Wymer J, Hehir M, Kolb N, Ralph J, Barboi A, Verma N, Ahmed M, Memon A, Saperstein D, Lou JS, Swenson A, Cash T. Patient Assisted Intervention for Neuropathy: Comparison of Treatment in Real Life Situations (PAIN-CONTRoLS): Bayesian Adaptive Comparative Effectiveness Randomized Trial. JAMA Neurol. 2021 Jan 1;78(1):68-76. doi: 10.1001/jamaneurol.2020.2590.

Reference Type DERIVED
PMID: 32809014 (View on PubMed)

Brown AR, Gajewski BJ, Aaronson LS, Mudaranthakam DP, Hunt SL, Berry SM, Quintana M, Pasnoor M, Dimachkie MM, Jawdat O, Herbelin L, Barohn RJ. A Bayesian comparative effectiveness trial in action: developing a platform for multisite study adaptive randomization. Trials. 2016 Aug 31;17(1):428. doi: 10.1186/s13063-016-1544-5.

Reference Type DERIVED
PMID: 27577191 (View on PubMed)

Provided Documents

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Document Type: Study Protocol and Statistical Analysis Plan

View Document

Other Identifiers

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PCORI-1306-02496

Identifier Type: OTHER_GRANT

Identifier Source: secondary_id

STUDY00001500

Identifier Type: -

Identifier Source: org_study_id

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