Clinical trial NCT03304522: 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
Overall status: COMPLETED
Phase: PHASE2
Conditions: Small Fiber Neuropathy
Lead sponsor: Vertex Pharmaceuticals Incorporated
Sponsor class: INDUSTRY
First posted: 2017-10-09
Enrollment: 89
Sex: ALL
Minimum age (years): 18
Maximum age (years): 80
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.


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.

Primary outcomes:
measure: Change in Weekly Average of Daily Pain Intensity on the 11 Point NRS
timeFrame: From Baseline at Week 6
Secondary outcomes:
measure: 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
timeFrame: From Baseline at Week 6
measure: Percentage of Participants With >=50% Reduction in the Weekly Average of Daily Pain Intensity on the 11-Point NRS
timeFrame: From Baseline at Week 6
measure: Change in the Daily Sleep Interference Scale (DSIS)
timeFrame: From Baseline at Week 6
measure: Percentage of Participants Categorized as Improved on the Patient Global Impression of Change (PGIC) Scale
timeFrame: At Week 6
measure: Change in Pain Intensity on the 11-Point NRS
timeFrame: From Baseline at Week 6
measure: Pre-dose Plasma Concentration (Ctrough) of VRT-1207355 and the Metabolite VRT-1268114
timeFrame: Pre-dose at Day 7
measure: Number of Participants With Clinically Meaningful Findings in Columbia Suicide Severity Rating Scale (C-SSRS) Responses
timeFrame: Day 1 up to Week 10
measure: Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
timeFrame: Day 1 up to Week 10
Other outcomes:
Locations:
city: Phoenix
country: United States
facility: Xenoscience Inc. - 21st Century Neurology
state: Arizona
city: Phoenix
country: United States
facility: Phoenix Neurological Associates, Ltd.
state: Arizona
city: Berkeley
country: United States
facility: Sutter Health - Alta Bates Summit Medical Center - The Jordan Research & Education Institute
state: California
city: Fresno
country: United States
facility: Neuropain Medical Center
state: California
city: La Jolla
country: United States
facility: University of California San Diego
state: California
city: Orange
country: United States
facility: SDS Clinical Trials, Inc.
state: California
city: Redwood City
country: United States
facility: Stanford University School of Medicine
state: California
city: Englewood
country: United States
facility: Blue Sky Neurology
state: Colorado
city: Orlando
country: United States
facility: Bioclinica Research - Orlando
state: Florida
city: Sunrise
country: United States
facility: Infinity Clinical Research
state: Florida
city: Springfield
country: United States
facility: Southern Illinois University (SIU) School of Medicine
state: Illinois
city: Iowa City
country: United States
facility: University of Iowa Hospitals and Clinics
state: Iowa
city: Kansas City
country: United States
facility: University of Kansas Medical Center (KUMC)
state: Kansas
city: Overland Park
country: United States
facility: International Clinical Research Institute (ICRI)
state: Kansas
city: Shreveport
country: United States
facility: River Cities Clinical Research Center
state: Louisiana
city: Baltimore
country: United States
facility: The Johns Hopkins Hospital
state: Maryland
city: Saint Louis
country: United States
facility: Washington University School of Medicine in St. Louis
state: Missouri
city: Lebanon
country: United States
facility: Dartmouth-Hitchcock Medical Center (DHMC)
state: New Hampshire
city: New Brunswick
country: United States
facility: Robert Wood Johnson Medical School
state: New Jersey
city: Albuquerque
country: United States
facility: University of New Mexico Hospital
state: New Mexico
city: Albany
country: United States
facility: Albany Medical Center- Neurology Group
state: New York
city: New York
country: United States
facility: The Mount Sinai Hospital
state: New York
city: New York
country: United States
facility: Columbia University Medical Center
state: New York
city: Rochester
country: United States
facility: University of Rochester
state: New York
city: Chapel Hill
country: United States
facility: University of North Carolina School of Medicine
state: North Carolina
city: Durham
country: United States
facility: Duke Neurological Disorders Clinic
state: North Carolina
city: Winston-Salem
country: United States
facility: Carolinas Pain Institute
state: North Carolina
city: Dayton
country: United States
facility: Neurology Diagnostics, Inc
state: Ohio
city: Tulsa
country: United States
facility: The Richter Clinic for Neurology and Neuro-Psychiatry
state: Oklahoma
city: Roanoke
country: United States
facility: Carilion Clinic Neurology
state: Virginia
city: Seattle
country: United States
facility: University of Washington
state: Washington
city: Würzburg
country: Germany
facility: Universitätsklinikum Würzburg
city: Milano
country: Italy
facility: Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta
city: Maastricht
country: Netherlands
facility: Maastricht UMC+
Study officials:
References: