Working...
ClinicalTrials.gov
ClinicalTrials.gov Menu

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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT03304522
Recruitment Status : Completed
First Posted : October 9, 2017
Last Update Posted : December 10, 2018
Sponsor:
Information provided by (Responsible Party):
Vertex Pharmaceuticals Incorporated

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

Condition or disease Intervention/treatment Phase
Small Fiber Neuropathy Drug: VX-150 Drug: Placebo Phase 2

Layout table for study information
Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 89 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
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
Actual Study Start Date : September 25, 2017
Actual Primary Completion Date : October 12, 2018
Actual Study Completion Date : November 8, 2018

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Dietary Fiber

Arm Intervention/treatment
Experimental: VX-150 Drug: VX-150
1250 mg total dose administered as 250-mg capsules orally

Active Comparator: Placebo Drug: Placebo
Matching Placebo




Primary Outcome Measures :
  1. Change from baseline in the weekly average of daily pain intensity on the 11 point numeric rating scale (NRS), as reported in the daily diary [ Time Frame: from baseline at Week 6 ]
    Pain intensity will be evaluated using the 11-point NRS (0: no pain to 10: worst imaginable pain). The NRS will be completed once daily asking subjects about their average pain over the last 24 hours.


Secondary Outcome Measures :
  1. Proportion of subjects with ≥30% reduction in the weekly average of daily pain intensity on the 11-point NRS, as reported in the daily diary [ Time Frame: from baseline at Week 6 ]
    Proportion of subjects with ≥30% reduction in the weekly average of daily pain intensity on the 11-point NRS will be reported

  2. Proportion of subjects with ≥50% reduction in the weekly average of daily pain intensity on the 11-point NRS, as reported in the daily diary [ Time Frame: from baseline at Week 6 ]
    Proportion of subjects with ≥50% reduction in the weekly average of daily pain intensity on the 11-point NRS will be reported

  3. Change from baseline in the Daily Sleep Interference Scale (DSIS) [ Time Frame: from baseline at Week 6 ]
    Change in DSIS will be reported.

  4. Proportion of subjects categorized as improved at Week 6 on the patient global impression of change (PGIC) assessment [ Time Frame: from baseline at Week 6 ]
    Proportion of subjects categorized as improved at Week 6 on PGIC assessment will be reported

  5. Change from baseline in pain intensity on the 11-point NRS, as reported at study visits [ Time Frame: from baseline at Week 6 ]
    Change in pain intensity on the 11-point NRS will be reported.

  6. Plasma PK parameters of VX-150 and its primary metabolite: Ctrough [Concentration at end of dosing interval] [ Time Frame: From Day 7 to Week 6 ]
  7. Safety and tolerability based on the number of subjects with adverse events (AEs) assessed based on the Columbia Suicide Severity Rating Scale (C-SSRS) [ Time Frame: Safety follow-up is being performed 28 days(+/-7 days) after the last dose ]


Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Layout table for eligibility information
Ages Eligible for Study:   18 Years to 80 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.


Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03304522


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

Layout table for additonal information
Responsible Party: Vertex Pharmaceuticals Incorporated
ClinicalTrials.gov Identifier: NCT03304522     History of Changes
Other Study ID Numbers: VX16-150-102
2017-001042-10 ( EudraCT Number )
First Posted: October 9, 2017    Key Record Dates
Last Update Posted: December 10, 2018
Last Verified: December 2018

Layout table for additional information
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:
Layout table for MeSH terms
Small Fiber Neuropathy
Peripheral Nervous System Diseases
Neuromuscular Diseases
Nervous System Diseases