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Trial record 3 of 3 for:    vx150

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

This study is currently recruiting participants.
Verified October 2017 by Vertex Pharmaceuticals Incorporated
Sponsor:
ClinicalTrials.gov Identifier:
NCT03304522
First Posted: October 9, 2017
Last Update Posted: October 25, 2017
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. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
Information provided by (Responsible Party):
Vertex Pharmaceuticals Incorporated
  Purpose
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 Intervention Phase
Small Fiber Neuropathy Drug: VX-150 Drug: Placebo Phase 2

Study Type: Interventional
Study Design: 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

Resource links provided by NLM:


Further study details as provided by Vertex Pharmaceuticals Incorporated:

Primary Outcome Measures:
  • 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:
  • 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 ]

Estimated Enrollment: 114
Actual Study Start Date: September 25, 2017
Estimated Study Completion Date: June 19, 2018
Estimated Primary Completion Date: June 19, 2018 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: VX-150 Drug: VX-150
1250 mg total dose administered as 250-mg capsules orally
Active Comparator: Placebo Drug: Placebo
Matching Placebo

  Eligibility

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.


Ages Eligible for Study:   18 Years to 80 Years   (Adult, Senior)
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.

  Contacts and Locations
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


Contacts
Contact: Medical Information 617-341-6777 medicalinfo@vrtx.com

  Show 28 Study Locations
Sponsors and Collaborators
Vertex Pharmaceuticals Incorporated
  More 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 Submitted: September 18, 2017
First Posted: October 9, 2017
Last Update Posted: October 25, 2017
Last Verified: October 2017

Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

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