Clinical trial NCT02260388: Patient Assisted Intervention for Neuropathy: Comparison of Treatment in Real Life Situations
Official title: Patient Assisted Intervention for Neuropathy: Comparison of Treatment in Real Life Situations (PAIN-CONTRoLS)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE4
Conditions: Cryptogenic Sensory Polyneuropathy
Keywords: CSPN
neuropathy
pain
pain management
Lead sponsor: University of Kansas Medical Center
Sponsor class: OTHER
Collaborators: name: Patient-Centered Outcomes Research Institute
First posted: 2014-10-09
Enrollment: 402
Sex: ALL
Minimum age (years): 30
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.

Eligibility criteria: 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.

Primary outcomes:
measure: 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
timeFrame: 12 weeks
Secondary outcomes:
measure: SF12 Health Composite Scores
timeFrame: 12 weeks
measure: PROMIS Pain Interference Short Form v1.0 8a T Score
timeFrame: 12 weeks
measure: PROMIS Fatigue Short Form v1.0 8a
timeFrame: 12 Weeks
measure: PROMIS Sleep Disturbance Short Form v1.0 8a
timeFrame: 12 weeks
Other outcomes:
Locations:
city: Phoenix
country: United States
facility: Barrow Neurological Institute
state: Arizona
city: Phoenix
country: United States
facility: Phoenix Neurological Associates
state: Arizona
city: Los Angeles
country: United States
facility: Cedars-Sinai Medical Center
state: California
city: San Francisco
country: United States
facility: California Pacific Medical Center
state: California
city: San Francisco
country: United States
facility: University of California San Francisco
state: California
city: Aurora
country: United States
facility: University of Colorado Denver Anschutz Campus
state: Colorado
city: Colorado Springs
country: United States
facility: Colorado Springs Neurological Associates
state: Colorado
city: Gainesville
country: United States
facility: University of Florida - Gainesville
state: Florida
city: Jacksonville
country: United States
facility: University of Florida Health Science Center - Jacksonville
state: Florida
city: Miami
country: United States
facility: University of Miami Miller School of Medicine
state: Florida
city: Orlando
country: United States
facility: Neurological Services of Orlando Research
state: Florida
city: Tampa
country: United States
facility: University of South Florida
state: Florida
city: Glenview
country: United States
facility: NorthShore Neurological Institute
state: Illinois
city: Indianapolis
country: United States
facility: Indiana University
state: Indiana
city: Des Moines
country: United States
facility: Mercy Medical Center - Des Moines
state: Iowa
city: Iowa City
country: United States
facility: University of Iowa Hospitals and Clinics
state: Iowa
city: Hutchinson
country: United States
facility: Hutchinson Clinic, PA
state: Kansas
city: Kansas City
country: United States
facility: University of Kansas Medical Center
state: Kansas
city: Louisville
country: United States
facility: Norton Neurology Services
state: Kentucky
city: Boston
country: United States
facility: Brigham and Women's Hospital
state: Massachusetts
city: Ann Arbor
country: United States
facility: University of Michigan
state: Michigan
city: Detroit
country: United States
facility: Henry Ford Hospital
state: Michigan
city: Grand Rapids
country: United States
facility: Spectrum Health System
state: Michigan
city: Minneapolis
country: United States
facility: University of Minnesota
state: Minnesota
city: Saint Louis
country: United States
facility: Saint Louis University
state: Missouri
city: Omaha
country: United States
facility: University of Nebraska Medical Center
state: Nebraska
city: Buffalo
country: United States
facility: University of Buffalo School of Medicine and Biomedical Sciences
state: New York
city: New York
country: United States
facility: Mount Sinai Beth Israel
state: New York
city: Cincinnati
country: United States
facility: University of Cincinnati
state: Ohio
city: Cleveland
country: United States
facility: Cleveland Clinic
state: Ohio
city: Columbus
country: United States
facility: The Ohio State University Wexner Medical Center
state: Ohio
city: Portland
country: United States
facility: Oregon Health & Science University
state: Oregon
city: Hershey
country: United States
facility: Penn State Medical Center
state: Pennsylvania
city: Austin
country: United States
facility: Austin Neuromuscular Center
state: Texas
city: Austin
country: United States
facility: Sara Austin, MD, PA
state: Texas
city: Austin
country: United States
facility: Seton Brain and Spine Institute
state: Texas
city: Dallas
country: United States
facility: Texas Neurology
state: Texas
city: Dallas
country: United States
facility: University of Texas Southwestern
state: Texas
city: Houston
country: United States
facility: University of Texas Health Science Center at Houton
state: Texas
city: Katy
country: United States
facility: Grand Medical Clinic
state: Texas
city: New Braunfels
country: United States
facility: Neurology Clinic of Central Texas
state: Texas
city: San Antonio
country: United States
facility: University of Texas Health Science Center in San Antonio
state: Texas
city: Salt Lake City
country: United States
facility: University of Utah
state: Utah
city: Burlington
country: United States
facility: University of Vermont Medical Center
state: Vermont
city: Charlottesville
country: United States
facility: University of Virginia
state: Virginia
city: Toronto
country: Canada
facility: Toronto General Hospital
state: Ontario
Study officials:
affiliation: University of Kansas Medical Center
name: Richard Barohn, MD
role: PRINCIPAL_INVESTIGATOR
References:
citation: 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.
citation: 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.