Back to resultsA Study to Evaluate the Efficacy and Safety of VX-150 in Treating Subjects With Pain Caused by Small Fiber Neuropathy
Primary Purpose
Small Fiber Neuropathy
Status
Completed
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
VX-150
Placebo
Sponsored by
About this trial
This is an interventional treatment trial for Small Fiber Neuropathy
Eligibility Criteria
Inclusion Criteria:
- 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
Exclusion Criteria:
- 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.
Sites / Locations
- Xenoscience Inc. - 21st Century Neurology
- Phoenix Neurological Associates, Ltd.
- Sutter Health - Alta Bates Summit Medical Center - The Jordan Research & Education Institute
- Neuropain Medical Center
- University of California San Diego
- SDS Clinical Trials, Inc.
- Stanford University School of Medicine
- Blue Sky Neurology
- Bioclinica Research - Orlando
- Infinity Clinical Research
- Southern Illinois University (SIU) School of Medicine
- University of Iowa Hospitals and Clinics
- University of Kansas Medical Center (KUMC)
- International Clinical Research Institute (ICRI)
- River Cities Clinical Research Center
- The Johns Hopkins Hospital
- Washington University School of Medicine in St. Louis
- Dartmouth-Hitchcock Medical Center (DHMC)
- Robert Wood Johnson Medical School
- University of New Mexico Hospital
- Albany Medical Center- Neurology Group
- The Mount Sinai Hospital
- Columbia University Medical Center
- University of Rochester
- University of North Carolina School of Medicine
- Duke Neurological Disorders Clinic
- Carolinas Pain Institute
- Neurology Diagnostics, Inc
- The Richter Clinic for Neurology and Neuro-Psychiatry
- Carilion Clinic Neurology
- University of Washington
- Universitätsklinikum Würzburg
- Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta
- Maastricht UMC+
Arms of the Study
Arm 1
Arm 2
Arm Type
Experimental
Placebo Comparator
Arm Label
VX-150
Placebo
Arm Description
Outcomes
Primary Outcome Measures
Change in Weekly Average of Daily Pain Intensity on the 11 Point NRS
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
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
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.
Percentage of Participants With >=50% Reduction in the Weekly Average of Daily Pain Intensity on the 11-Point NRS
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.
Change in the Daily Sleep Interference Scale (DSIS)
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 Categorized as Improved on the Patient Global Impression of Change (PGIC) Scale
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
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
Number of Participants With Clinically Meaningful Findings in Columbia Suicide Severity Rating Scale (C-SSRS) Responses
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.
Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
Full Information
NCT ID
NCT03304522
First Posted
September 18, 2017
Last Updated
November 11, 2021
Sponsor
Vertex Pharmaceuticals Incorporated
1. Study Identification
Unique Protocol Identification Number
NCT03304522
Brief Title
A Study to Evaluate the Efficacy and Safety of VX-150 in Treating Subjects With Pain Caused by Small Fiber Neuropathy
Official Title
A Phase 2, Randomized, Double-blind, Placebo-controlled, 6-Week, Parallel-design Study of the Efficacy and Safety of VX-150 in Treating Subjects With Pain Caused by Small Fiber Neuropathy
Study Type
Interventional
2. Study Status
Record Verification Date
November 2021
Overall Recruitment Status
Completed
Study Start Date
September 20, 2017 (Actual)
Primary Completion Date
October 12, 2018 (Actual)
Study Completion Date
November 8, 2018 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Vertex Pharmaceuticals Incorporated
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Small Fiber Neuropathy
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
89 (Actual)
8. Arms, Groups, and Interventions
Arm Title
VX-150
Arm Type
Experimental
Arm Title
Placebo
Arm Type
Placebo Comparator
Intervention Type
Drug
Intervention Name(s)
VX-150
Intervention Description
Participants received VX-150 1250 milligrams (mg) once daily (qd) orally for 6 weeks.
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
Participants received placebo matched to VX-150 for 6 weeks.
Primary Outcome Measure Information:
Title
Change in Weekly Average of Daily Pain Intensity on the 11 Point NRS
Description
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.
Time Frame
From Baseline at Week 6
Secondary Outcome Measure Information:
Title
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
Description
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.
Time Frame
From Baseline at Week 6
Title
Percentage of Participants With >=50% Reduction in the Weekly Average of Daily Pain Intensity on the 11-Point NRS
Description
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.
Time Frame
From Baseline at Week 6
Title
Change in the Daily Sleep Interference Scale (DSIS)
Description
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.
Time Frame
From Baseline at Week 6
Title
Percentage of Participants Categorized as Improved on the Patient Global Impression of Change (PGIC) Scale
Description
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.
Time Frame
At Week 6
Title
Change in Pain Intensity on the 11-Point NRS
Description
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.
Time Frame
From Baseline at Week 6
Title
Pre-dose Plasma Concentration (Ctrough) of VRT-1207355 and the Metabolite VRT-1268114
Time Frame
Pre-dose at Day 7
Title
Number of Participants With Clinically Meaningful Findings in Columbia Suicide Severity Rating Scale (C-SSRS) Responses
Description
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.
Time Frame
Day 1 up to Week 10
Title
Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
Time Frame
Day 1 up to Week 10
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
80 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
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
Exclusion Criteria:
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.
Facility Information:
Facility Name
Xenoscience Inc. - 21st Century Neurology
City
Phoenix
State/Province
Arizona
ZIP/Postal Code
85004
Country
United States
Facility Name
Phoenix Neurological Associates, Ltd.
City
Phoenix
State/Province
Arizona
ZIP/Postal Code
85251
Country
United States
Facility Name
Sutter Health - Alta Bates Summit Medical Center - The Jordan Research & Education Institute
City
Berkeley
State/Province
California
ZIP/Postal Code
94705
Country
United States
Facility Name
Neuropain Medical Center
City
Fresno
State/Province
California
ZIP/Postal Code
93710
Country
United States
Facility Name
University of California San Diego
City
La Jolla
State/Province
California
ZIP/Postal Code
92093
Country
United States
Facility Name
SDS Clinical Trials, Inc.
City
Orange
State/Province
California
ZIP/Postal Code
92868
Country
United States
Facility Name
Stanford University School of Medicine
City
Redwood City
State/Province
California
ZIP/Postal Code
94063
Country
United States
Facility Name
Blue Sky Neurology
City
Englewood
State/Province
Colorado
ZIP/Postal Code
80113
Country
United States
Facility Name
Bioclinica Research - Orlando
City
Orlando
State/Province
Florida
ZIP/Postal Code
32806
Country
United States
Facility Name
Infinity Clinical Research
City
Sunrise
State/Province
Florida
ZIP/Postal Code
33351
Country
United States
Facility Name
Southern Illinois University (SIU) School of Medicine
City
Springfield
State/Province
Illinois
ZIP/Postal Code
62702
Country
United States
Facility Name
University of Iowa Hospitals and Clinics
City
Iowa City
State/Province
Iowa
ZIP/Postal Code
52242
Country
United States
Facility Name
University of Kansas Medical Center (KUMC)
City
Kansas City
State/Province
Kansas
ZIP/Postal Code
66160
Country
United States
Facility Name
International Clinical Research Institute (ICRI)
City
Overland Park
State/Province
Kansas
ZIP/Postal Code
66210
Country
United States
Facility Name
River Cities Clinical Research Center
City
Shreveport
State/Province
Louisiana
ZIP/Postal Code
71105
Country
United States
Facility Name
The Johns Hopkins Hospital
City
Baltimore
State/Province
Maryland
ZIP/Postal Code
21287
Country
United States
Facility Name
Washington University School of Medicine in St. Louis
City
Saint Louis
State/Province
Missouri
ZIP/Postal Code
63110
Country
United States
Facility Name
Dartmouth-Hitchcock Medical Center (DHMC)
City
Lebanon
State/Province
New Hampshire
ZIP/Postal Code
03756
Country
United States
Facility Name
Robert Wood Johnson Medical School
City
New Brunswick
State/Province
New Jersey
ZIP/Postal Code
08901
Country
United States
Facility Name
University of New Mexico Hospital
City
Albuquerque
State/Province
New Mexico
ZIP/Postal Code
87131
Country
United States
Facility Name
Albany Medical Center- Neurology Group
City
Albany
State/Province
New York
ZIP/Postal Code
12208
Country
United States
Facility Name
The Mount Sinai Hospital
City
New York
State/Province
New York
ZIP/Postal Code
10029
Country
United States
Facility Name
Columbia University Medical Center
City
New York
State/Province
New York
ZIP/Postal Code
10032
Country
United States
Facility Name
University of Rochester
City
Rochester
State/Province
New York
ZIP/Postal Code
14618
Country
United States
Facility Name
University of North Carolina School of Medicine
City
Chapel Hill
State/Province
North Carolina
ZIP/Postal Code
27514
Country
United States
Facility Name
Duke Neurological Disorders Clinic
City
Durham
State/Province
North Carolina
ZIP/Postal Code
27710
Country
United States
Facility Name
Carolinas Pain Institute
City
Winston-Salem
State/Province
North Carolina
ZIP/Postal Code
27103
Country
United States
Facility Name
Neurology Diagnostics, Inc
City
Dayton
State/Province
Ohio
ZIP/Postal Code
45459
Country
United States
Facility Name
The Richter Clinic for Neurology and Neuro-Psychiatry
City
Tulsa
State/Province
Oklahoma
ZIP/Postal Code
74104
Country
United States
Facility Name
Carilion Clinic Neurology
City
Roanoke
State/Province
Virginia
ZIP/Postal Code
24013
Country
United States
Facility Name
University of Washington
City
Seattle
State/Province
Washington
ZIP/Postal Code
98105
Country
United States
Facility Name
Universitätsklinikum Würzburg
City
Würzburg
Country
Germany
Facility Name
Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta
City
Milano
Country
Italy
Facility Name
Maastricht UMC+
City
Maastricht
Country
Netherlands
12. IPD Sharing Statement
Learn more about this trial
A Study to Evaluate the Efficacy and Safety of VX-150 in Treating Subjects With Pain Caused by Small Fiber Neuropathy
We'll reach out to this number within 24 hrs