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

Vertex Pharmaceuticals Incorporated34 sites in 4 countries89 target enrollmentSeptember 20, 2017

ConditionsSmall Fiber Neuropathy

InterventionsPlacebo VX-150

DrugsVX-150 Placebo

Overview

Phase: Phase 2
Intervention: Placebo
Conditions: Small Fiber Neuropathy
Sponsor: Vertex Pharmaceuticals Incorporated
Enrollment: 89
Locations: 34
Primary Endpoint: Change in Weekly Average of Daily Pain Intensity on the 11 Point NRS
Status: Completed
Last Updated: 4 years ago

Overview Investigators Eligibility Criteria Arms & Interventions Outcomes Sites Similar Trials

Overview

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.

Registry

clinicaltrials.gov

Start Date

September 20, 2017

End Date

November 8, 2018

Last Updated

4 years ago

Study Type

Interventional

Study Design

Parallel

Sex

All

Investigators

Sponsor

Vertex Pharmaceuticals Incorporated

Responsible Party

Sponsor

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.

Arms & Interventions

Placebo

Intervention: Placebo

VX-150

Intervention: VX-150

Outcomes

Primary Outcomes

Change in Weekly Average of Daily Pain Intensity on the 11 Point NRS

Time Frame: 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 Outcomes

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)
Change in the Daily Sleep Interference Scale (DSIS)(From Baseline at Week 6)
Percentage of Participants With >=50% Reduction in the Weekly Average of Daily Pain Intensity on the 11-Point NRS(From Baseline at Week 6)
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)
Change in Pain Intensity on the 11-Point NRS(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(Day 1 up to Week 10)