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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

Tracking Information
First Submitted Date  ICMJE September 18, 2017
First Posted Date  ICMJE October 9, 2017
Last Update Posted Date December 10, 2018
Actual Study Start Date  ICMJE September 25, 2017
Actual Primary Completion Date October 12, 2018   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: October 3, 2017)
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.
Original Primary Outcome Measures  ICMJE Same as current
Change History Complete list of historical versions of study NCT03304522 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: October 3, 2017)
  • 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
  • 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
  • Change from baseline in the Daily Sleep Interference Scale (DSIS) [ Time Frame: from baseline at Week 6 ]
    Change in DSIS will be reported.
  • 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
  • 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.
  • 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 ]
  • 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 ]
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE A Study to Evaluate the Efficacy and Safety of VX-150 in Treating Subjects With Pain Caused by Small Fiber Neuropathy
Official Title  ICMJE 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
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
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Condition  ICMJE Small Fiber Neuropathy
Intervention  ICMJE
  • Drug: VX-150
    1250 mg total dose administered as 250-mg capsules orally
  • Drug: Placebo
    Matching Placebo
Study Arms  ICMJE
  • Experimental: VX-150
    Intervention: Drug: VX-150
  • Active Comparator: Placebo
    Intervention: Drug: Placebo
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: September 28, 2018)
89
Original Estimated Enrollment  ICMJE
 (submitted: October 3, 2017)
114
Actual Study Completion Date  ICMJE November 8, 2018
Actual Primary Completion Date October 12, 2018   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.

Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years to 80 Years   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Germany,   Italy,   Netherlands,   United States
Removed Location Countries Canada
 
Administrative Information
NCT Number  ICMJE NCT03304522
Other Study ID Numbers  ICMJE VX16-150-102
2017-001042-10 ( EudraCT Number )
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Vertex Pharmaceuticals Incorporated
Study Sponsor  ICMJE Vertex Pharmaceuticals Incorporated
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account Vertex Pharmaceuticals Incorporated
Verification Date December 2018

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP