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

NCT03304522 | Completed Phase 2 Results DMC FDA Drug

• 2 other identifiers: VX16-150-1022017-001042-10
• Study Type: interventional
• Target: 89
• Locations: 4 countries
• Sites: 34

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.

Conditions

Small Fiber Neuropathy

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (8)

• 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

• Percentage of Participants With >=50% 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 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

Study Arms (2)

VX-150

EXPERIMENTAL

Drug: VX-150

Placebo

PLACEBO COMPARATOR

Drug: Placebo

Interventions

VX-150 DRUG

Participants received VX-150 1250 milligrams (mg) once daily (qd) orally for 6 weeks.

VX-150

Placebo DRUG

Participants received placebo matched to VX-150 for 6 weeks.

Placebo

Eligibility Criteria

• Age: 18 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Vertex Pharmaceuticals Incorporated: lead

Study Sites (34)

Xenoscience Inc. - 21st Century Neurology

Phoenix, Arizona, 85004, United States

Location

Phoenix Neurological Associates, Ltd.

Phoenix, Arizona, 85251, United States

Location

Sutter Health - Alta Bates Summit Medical Center - The Jordan Research & Education Institute

Berkeley, California, 94705, United States

Location

Neuropain Medical Center

Fresno, California, 93710, United States

Location

University of California San Diego

La Jolla, California, 92093, United States

Location

SDS Clinical Trials, Inc.

Orange, California, 92868, United States

Location

Stanford University School of Medicine

Redwood City, California, 94063, United States

Location

Blue Sky Neurology

Englewood, Colorado, 80113, United States

Location

Bioclinica Research - Orlando

Orlando, Florida, 32806, United States

Location

Infinity Clinical Research

Sunrise, Florida, 33351, United States

Location

Southern Illinois University (SIU) School of Medicine

Springfield, Illinois, 62702, United States

Location

University of Iowa Hospitals and Clinics

Iowa City, Iowa, 52242, United States

Location

University of Kansas Medical Center (KUMC)

Kansas City, Kansas, 66160, United States

Location

International Clinical Research Institute (ICRI)

Overland Park, Kansas, 66210, United States

Location

River Cities Clinical Research Center

Shreveport, Louisiana, 71105, United States

Location

The Johns Hopkins Hospital

Baltimore, Maryland, 21287, United States

Location

Washington University School of Medicine in St. Louis

St Louis, Missouri, 63110, United States

Location

Dartmouth-Hitchcock Medical Center (DHMC)

Lebanon, New Hampshire, 03756, United States

Location

Robert Wood Johnson Medical School

New Brunswick, New Jersey, 08901, United States

Location

University of New Mexico Hospital

Albuquerque, New Mexico, 87131, United States

Location

Albany Medical Center- Neurology Group

Albany, New York, 12208, United States

Location

The Mount Sinai Hospital

New York, New York, 10029, United States

Location

Columbia University Medical Center

New York, New York, 10032, United States

Location

University of Rochester

Rochester, New York, 14618, United States

Location

University of North Carolina School of Medicine

Chapel Hill, North Carolina, 27514, United States

Location

Duke Neurological Disorders Clinic

Durham, North Carolina, 27710, United States

Location

Carolinas Pain Institute

Winston-Salem, North Carolina, 27103, United States

Location

Neurology Diagnostics, Inc

Dayton, Ohio, 45459, United States

Location

The Richter Clinic for Neurology and Neuro-Psychiatry

Tulsa, Oklahoma, 74104, United States

Location

Carilion Clinic Neurology

Roanoke, Virginia, 24013, United States

Location

University of Washington

Seattle, Washington, 98105, United States

Location

Universitätsklinikum Würzburg

Würzburg, Germany

Location

Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta

Milan, Italy

Location

Maastricht UMC+

Maastricht, Netherlands

Location

MeSH Terms

Conditions

Small Fiber Neuropathy

Condition Hierarchy (Ancestors)

Peripheral Nervous System Diseases; Neuromuscular Diseases; Nervous System Diseases

Results Point of Contact

• Title: Medical Monitor
• Organization: Vertex Pharmaceuticals Incorporated

medicalinfo@vrtx.com

617-341-6777

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 18, 2017
• First Posted: October 9, 2017
• Study Start: September 20, 2017
• Primary Completion: October 12, 2018
• Study Completion: November 8, 2018
• Last Updated: November 15, 2021
• Results First Posted: November 15, 2021

Record last verified: 2021-11

Locations

NL (1); DE (1); US (31); IT (1)