A Study to Evaluate the Efficacy and Safety of VX-150 in Treating Subjects With Pain Caused by Small Fiber Neuropathy

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

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.

Study Overview

• Status: Completed
• Conditions: Small Fiber Neuropathy
• Intervention / Treatment: Drug: VX-150; Drug: Placebo

• Study Type: Interventional
• Enrollment (Actual): 89
• Phase: Phase 2

Contacts and Locations

Study Locations

• Germany
  • Würzburg, Germany
    • Universitatsklinikum Wurzburg
• Italy
  • Milano, Italy
    • Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta
• Netherlands
  • Maastricht, Netherlands
    • Maastricht UMC+
• United States
  • Arizona
    • Phoenix, Arizona, United States, 85004
      • Xenoscience Inc. - 21st Century Neurology
    • Phoenix, Arizona, United States, 85251
      • Phoenix Neurological Associates, Ltd.
  • California
    • Berkeley, California, United States, 94705
      • Sutter Health - Alta Bates Summit Medical Center - The Jordan Research & Education Institute
    • Fresno, California, United States, 93710
      • Neuropain Medical Center
    • La Jolla, California, United States, 92093
      • University of California San Diego
    • Orange, California, United States, 92868
      • SDS Clinical Trials, Inc.
    • Redwood City, California, United States, 94063
      • Stanford University School of Medicine
  • Colorado
    • Englewood, Colorado, United States, 80113
      • Blue Sky Neurology
  • Florida
    • Orlando, Florida, United States, 32806
      • Bioclinica Research - Orlando
    • Sunrise, Florida, United States, 33351
      • Infinity Clinical Research
  • Illinois
    • Springfield, Illinois, United States, 62702
      • Southern Illinois University (SIU) School of Medicine
  • Iowa
    • Iowa City, Iowa, United States, 52242
      • University of Iowa Hospitals and Clinics
  • Kansas
    • Kansas City, Kansas, United States, 66160
      • University of Kansas Medical Center (KUMC)
    • Overland Park, Kansas, United States, 66210
      • International Clinical Research Institute (ICRI)
  • Louisiana
    • Shreveport, Louisiana, United States, 71105
      • River Cities Clinical Research Center
  • Maryland
    • Baltimore, Maryland, United States, 21287
      • The Johns Hopkins Hospital
  • Missouri
    • Saint Louis, Missouri, United States, 63110
      • Washington University School of Medicine in St. Louis
  • New Hampshire
    • Lebanon, New Hampshire, United States, 03756
      • Dartmouth-Hitchcock Medical Center (DHMC)
  • New Jersey
    • New Brunswick, New Jersey, United States, 08901
      • Robert Wood Johnson Medical School
  • New Mexico
    • Albuquerque, New Mexico, United States, 87131
      • University of New Mexico Hospital
  • New York
    • Albany, New York, United States, 12208
      • Albany Medical Center- Neurology Group
    • New York, New York, United States, 10032
      • Columbia University Medical Center
    • New York, New York, United States, 10029
      • The Mount Sinai Hospital
    • Rochester, New York, United States, 14618
      • University of Rochester
  • North Carolina
    • Chapel Hill, North Carolina, United States, 27514
      • University of North Carolina School of Medicine
    • Durham, North Carolina, United States, 27710
      • Duke Neurological Disorders Clinic
    • Winston-Salem, North Carolina, United States, 27103
      • Carolinas Pain Institute
  • Ohio
    • Dayton, Ohio, United States, 45459
      • Neurology Diagnostics, Inc
  • Oklahoma
    • Tulsa, Oklahoma, United States, 74104
      • The Richter Clinic for Neurology and Neuro-Psychiatry
  • Virginia
    • Roanoke, Virginia, United States, 24013
      • Carilion Clinic Neurology
  • Washington
    • Seattle, Washington, United States, 98105
      • University of Washington

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 16 years to 78 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Quadruple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Placebo Comparator: Placebo	Drug: Placebo; Participants received placebo matched to VX-150 for 6 weeks.
Experimental: VX-150	Drug: VX-150; Participants received VX-150 1250 milligrams (mg) once daily (qd) orally for 6 weeks.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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.	From Baseline at Week 6

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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.	From Baseline at Week 6
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.	From Baseline at Week 6
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.	From Baseline at Week 6
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.	At Week 6
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.	From Baseline at Week 6
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	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.	Day 1 up to Week 10
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

Collaborators and Investigators

• Sponsor: Vertex Pharmaceuticals Incorporated

Study record dates

Study Major Dates

• Study Start (Actual): September 20, 2017
• Primary Completion (Actual): October 12, 2018
• Study Completion (Actual): November 8, 2018

Study Registration Dates

• First Submitted: September 18, 2017
• First Submitted That Met QC Criteria: October 3, 2017
• First Posted (Actual): October 9, 2017

Study Record Updates

• Last Update Posted (Actual): November 15, 2021
• Last Update Submitted That Met QC Criteria: November 11, 2021
• Last Verified: November 1, 2021

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Nervous System Diseases; Neuromuscular Diseases; Peripheral Nervous System Diseases; Small Fiber Neuropathy
• Other Study ID Numbers: VX16-150-102; 2017-001042-10 (EudraCT Number)

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No