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

Small Fiber Neuropathy Clinical Trial

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03304522
• Other study ID #: VX16-150-102
• Secondary ID: 2017-001042-10
• Status: Completed
• Phase: Phase 2
• Start date: September 20, 2017
• Est. completion date: November 8, 2018

Study information

• Verified date: November 2021
• Source: Vertex Pharmaceuticals Incorporated
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 89
• Est. completion date: November 8, 2018
• Est. primary completion date: October 12, 2018
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 80 Years

Eligibility

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.

Related Conditions & MeSH terms

• Small Fiber Neuropathy

Intervention

Drug:
• VX-150
Participants received VX-150 1250 milligrams (mg) once daily (qd) orally for 6 weeks.
• Placebo
Participants received placebo matched to VX-150 for 6 weeks.

Locations

Country	Name	City	State
Germany	Universitätsklinikum Würzburg	Würzburg
Italy	Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta	Milano
Netherlands	Maastricht UMC+	Maastricht
United States	Albany Medical Center- Neurology Group	Albany	New York
United States	University of New Mexico Hospital	Albuquerque	New Mexico
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	University of North Carolina School of Medicine	Chapel Hill	North Carolina
United States	Neurology Diagnostics, Inc	Dayton	Ohio
United States	Duke Neurological Disorders Clinic	Durham	North Carolina
United States	Blue Sky Neurology	Englewood	Colorado
United States	Neuropain Medical Center	Fresno	California
United States	University of Iowa Hospitals and Clinics	Iowa City	Iowa
United States	University of Kansas Medical Center (KUMC)	Kansas City	Kansas
United States	University of California San Diego	La Jolla	California
United States	Dartmouth-Hitchcock Medical Center (DHMC)	Lebanon	New Hampshire
United States	Robert Wood Johnson Medical School	New Brunswick	New Jersey
United States	Columbia University Medical Center	New York	New York
United States	The Mount Sinai Hospital	New York	New York
United States	SDS Clinical Trials, Inc.	Orange	California
United States	Bioclinica Research - Orlando	Orlando	Florida
United States	International Clinical Research Institute (ICRI)	Overland Park	Kansas
United States	Phoenix Neurological Associates, Ltd.	Phoenix	Arizona
United States	Xenoscience Inc. - 21st Century Neurology	Phoenix	Arizona
United States	Stanford University School of Medicine	Redwood City	California
United States	Carilion Clinic Neurology	Roanoke	Virginia
United States	University of Rochester	Rochester	New York
United States	Washington University School of Medicine in St. Louis	Saint Louis	Missouri
United States	University of Washington	Seattle	Washington
United States	River Cities Clinical Research Center	Shreveport	Louisiana
United States	Southern Illinois University (SIU) School of Medicine	Springfield	Illinois
United States	Infinity Clinical Research	Sunrise	Florida
United States	The Richter Clinic for Neurology and Neuro-Psychiatry	Tulsa	Oklahoma
United States	Carolinas Pain Institute	Winston-Salem	North Carolina

Sponsors (1)

Lead Sponsor	Collaborator
Vertex Pharmaceuticals Incorporated

Countries where clinical trial is conducted

United States,  Germany,  Italy,  Netherlands, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	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
Secondary	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
Secondary	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
Secondary	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
Secondary	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
Secondary	Pre-dose Plasma Concentration (Ctrough) of VRT-1207355 and the Metabolite VRT-1268114		Pre-dose at Day 7
Secondary	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
Secondary	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