A Study to Evaluate the Efficacy and Safety of VX-150 in Treating Subjects With Pain Caused by Small Fiber Neuropathy
- Registration Number
- NCT03304522
- Lead Sponsor
- Vertex Pharmaceuticals Incorporated
- Brief Summary
This is a Phase 2, randomized, double-blind, placebo-controlled, parallel-group, multicenter study evaluating the efficacy of VX-150 for the treatment of pain caused by small fiber neuropathy.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 89
- Body mass index (BMI) of 18.0 to 31.0 kg/m2, inclusive, and a total body weight >50 kg
- Diagnosis of small fiber neuropathy, as per European Federation Neurological Societies (EFNS)/American Academy of Neurology (AAN) guidelines, with pain for at least 3 months prior to screening
- Reduction below the 5th percentile of sex and age-adjusted normal values in epidermal nerve fiber density on punch skin biopsy at the distal site of the leg performed at screening
- Normal nerve conduction studies (NCS), including presence of sural response.
- Average NRS score between ≥4 and ≤9 reported in the daily diary on Days -7 through -1
- History in the past 10 years of malignancy except for squamous cell skin cancer, basal cell skin cancer, and Stage 0 cervical carcinoma in situ
- History of connective tissue disorders, sarcoidosis, Sjögren's syndrome, amyloidosis, Fabry's disease, celiac disease, lyme disease, autoimmune disorders
- A known or clinically suspected infection with human immunodeficiency virus or hepatitis B or C viruses
- Current clinically significant liver or kidney dysfunction
- Current uncontrolled thyroid dysfunction
- A diagnosis of diabetes, HbA1C ≥8% at screening
- History of cardiac dysrhythmias requiring anti-arrhythmia treatment(s)
- Concomitant severe pain conditions which may impair self-assessment of pain due to small fiber neuropathy
Other protocol defined Inclusion/Exclusion criteria may apply.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description VX-150 VX-150 - Placebo Placebo -
- Primary Outcome Measures
Name Time Method Change in Weekly Average of Daily Pain Intensity on the 11 Point NRS From Baseline at Week 6 Pain intensity was evaluated using the 11-point NRS (where 0 signified no pain and 10 signified worst imaginable pain) during the last 24 hours on the NRS each evening. Higher score indicates greater level of pain.
- Secondary Outcome Measures
Name Time Method Percentage of Participants With Greater Than or Equal to (>=) 30 Percent (%) Reduction in the Weekly Average of Daily Pain Intensity on the 11-Point NRS From Baseline at Week 6 Pain intensity was evaluated using the 11-point NRS (where 0 signified no pain and 10 signified worst imaginable pain) during the last 24 hours on the NRS each evening. Higher score indicates greater level of pain. Percentage of participants \>= 30% reduction in the weekly average of daily pain intensity on the 11-Point NRS were reported.
Change in the Daily Sleep Interference Scale (DSIS) From Baseline at Week 6 Pain-associated sleep interference was assessed using DSIS, based on an 11-point scale (where 0 signified none: pain does not interfere with sleep and 10 signified severe: pain completely interferes with sleep, unable to sleep). Higher score indicates greater pain associated sleep interference.
Percentage of Participants With >=50% Reduction in the Weekly Average of Daily Pain Intensity on the 11-Point NRS From Baseline at Week 6 Pain intensity was evaluated using the 11-point NRS (where 0 signified no pain and 10 signified worst imaginable pain) during the last 24 hours on the NRS each evening. Higher score indicates greater level of pain. Percentage of participants \>= 50% reduction in the weekly average of daily pain intensity on the 11-Point NRS were reported.
Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) Day 1 up to Week 10 Percentage of Participants Categorized as Improved on the Patient Global Impression of Change (PGIC) Scale At Week 6 PGIC scale evaluated the change in activity limitations, symptoms, emotions, and overall quality of life (QoL) related to the participants painful condition on 7-point scale from 1 (improved) to 7 (worse). Participants were categorized as following: scale from 1 - 2 were categorized as "improved", scale from 3 - 4 as "no change" and scale from 5 - 7 were categorized as "worse". Percentage of participants categorized as improved on PGIC scale at week 6 were reported for this outcome measure.
Change in Pain Intensity on the 11-Point NRS From Baseline at Week 6 Pain intensity was evaluated using the 11-point NRS (where 0 signified no pain and 10 signified worst imaginable pain) during the last 24 hours on the NRS each evening. Higher score indicates greater level of pain. Higher score indicates greater level of pain.
Pre-dose Plasma Concentration (Ctrough) of VRT-1207355 and the Metabolite VRT-1268114 Pre-dose at Day 7 Number of Participants With Clinically Meaningful Findings in Columbia Suicide Severity Rating Scale (C-SSRS) Responses Day 1 up to Week 10 The C-SSRS is an interview-based rating scale was evaluated through a series of questions about suicidal thoughts and behaviors with the possible answers yes or no. Yes represents a worse outcome. Clinically Meaningfulness of C-SSRS responses were judged by investigator based on answers received from participants.
Trial Locations
- Locations (34)
Duke Neurological Disorders Clinic
🇺🇸Durham, North Carolina, United States
University of Washington
🇺🇸Seattle, Washington, United States
Xenoscience Inc. - 21st Century Neurology
🇺🇸Phoenix, Arizona, United States
Phoenix Neurological Associates, Ltd.
🇺🇸Phoenix, Arizona, United States
The Johns Hopkins Hospital
🇺🇸Baltimore, Maryland, United States
Sutter Health - Alta Bates Summit Medical Center - The Jordan Research & Education Institute
🇺🇸Berkeley, California, United States
Maastricht UMC+
🇳🇱Maastricht, Netherlands
Southern Illinois University (SIU) School of Medicine
🇺🇸Springfield, Illinois, United States
Stanford University School of Medicine
🇺🇸Redwood City, California, United States
The Mount Sinai Hospital
🇺🇸New York, New York, United States
River Cities Clinical Research Center
🇺🇸Shreveport, Louisiana, United States
University of Rochester
🇺🇸Rochester, New York, United States
Albany Medical Center- Neurology Group
🇺🇸Albany, New York, United States
Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta
🇮🇹Milano, Italy
University of California San Diego
🇺🇸La Jolla, California, United States
Neuropain Medical Center
🇺🇸Fresno, California, United States
Blue Sky Neurology
🇺🇸Englewood, Colorado, United States
SDS Clinical Trials, Inc.
🇺🇸Orange, California, United States
Bioclinica Research - Orlando
🇺🇸Orlando, Florida, United States
Infinity Clinical Research
🇺🇸Sunrise, Florida, United States
International Clinical Research Institute (ICRI)
🇺🇸Overland Park, Kansas, United States
Washington University School of Medicine in St. Louis
🇺🇸Saint Louis, Missouri, United States
Dartmouth-Hitchcock Medical Center (DHMC)
🇺🇸Lebanon, New Hampshire, United States
Columbia University Medical Center
🇺🇸New York, New York, United States
University of New Mexico Hospital
🇺🇸Albuquerque, New Mexico, United States
The Richter Clinic for Neurology and Neuro-Psychiatry
🇺🇸Tulsa, Oklahoma, United States
Carolinas Pain Institute
🇺🇸Winston-Salem, North Carolina, United States
Universitätsklinikum Würzburg
🇩🇪Würzburg, Germany
Neurology Diagnostics, Inc
🇺🇸Dayton, Ohio, United States
University of North Carolina School of Medicine
🇺🇸Chapel Hill, North Carolina, United States
Robert Wood Johnson Medical School
🇺🇸New Brunswick, New Jersey, United States
Carilion Clinic Neurology
🇺🇸Roanoke, Virginia, United States
University of Iowa Hospitals and Clinics
🇺🇸Iowa City, Iowa, United States
University of Kansas Medical Center (KUMC)
🇺🇸Kansas City, Kansas, United States