Small Fiber NeuropathyPhase 2NCT03304522
A Study to Evaluate the Efficacy and Safety of VX-150 in Treating Subjects With Pain Caused by Small Fiber Neuropathy
A Phase 2 study testing VX-150 in people with Small Fiber Neuropathy, run by Vertex Pharmaceuticals Incorporated at 34 sites in 4 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 80 years
- Conditions the study is for
- Small Fiber Neuropathy
- What is being tested?
- VX-150
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 89 participants
- Where
- 34 sitesin 4 countries
- Study dates
- 20 Sep 2017to 8 Nov 2018
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Show all 9Show fewer
- 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.
Trial locations and contacts
34 sites in 4 countries.
34 sites in 4 countries
United States31 sites
Albany Medical Center- Neurology Group
Albany, New York, 12208
University of New Mexico Hospital
Albuquerque, New Mexico, 87131
The Johns Hopkins Hospital
Baltimore, Maryland, 21287
Sutter Health - Alta Bates Summit Medical Center - The Jordan Research & Education Institute
Berkeley, California, 94705
University of North Carolina School of Medicine
Chapel Hill, North Carolina, 27514
Neurology Diagnostics, Inc
Dayton, Ohio, 45459
Duke Neurological Disorders Clinic
Durham, North Carolina, 27710
Blue Sky Neurology
Englewood, Colorado, 80113
Neuropain Medical Center
Fresno, California, 93710
University of Iowa Hospitals and Clinics
Iowa City, Iowa, 52242
University of Kansas Medical Center (KUMC)
Kansas City, Kansas, 66160
University of California San Diego
La Jolla, California, 92093
Dartmouth-Hitchcock Medical Center (DHMC)
Lebanon, New Hampshire, 03756
Robert Wood Johnson Medical School
New Brunswick, New Jersey, 08901
The Mount Sinai Hospital
New York, New York, 10029
Columbia University Medical Center
New York, New York, 10032
SDS Clinical Trials, Inc.
Orange, California, 92868
Bioclinica Research - Orlando
Orlando, Florida, 32806
International Clinical Research Institute (ICRI)
Overland Park, Kansas, 66210
Xenoscience Inc. - 21st Century Neurology
Phoenix, Arizona, 85004
Phoenix Neurological Associates, Ltd.
Phoenix, Arizona, 85251
Stanford University School of Medicine
Redwood City, California, 94063
Carilion Clinic Neurology
Roanoke, Virginia, 24013
University of Rochester
Rochester, New York, 14618
University of Washington
Seattle, Washington, 98105
River Cities Clinical Research Center
Shreveport, Louisiana, 71105
Southern Illinois University (SIU) School of Medicine
Springfield, Illinois, 62702
Washington University School of Medicine in St. Louis
St Louis, Missouri, 63110
Infinity Clinical Research
Sunrise, Florida, 33351
The Richter Clinic for Neurology and Neuro-Psychiatry
Tulsa, Oklahoma, 74104
Carolinas Pain Institute
Winston-Salem, North Carolina, 27103
About this study
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
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
- Being tested
- VX-150, Placebo
- Medical subject headings
- Small Fiber Neuropathy
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
VX-150Drug
Participants received VX-150 1250 milligrams (mg) once daily (qd) orally for 6 weeks.
PlaceboDrug
Participants received placebo matched to VX-150 for 6 weeks.
| Study group | Role | What they receive |
|---|---|---|
| VX-150 | Experimental | Not described Receives: Drug: VX-150 |
| Placebo | Placebo comparator | Not described Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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
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. Percentage of participants >= 30% reduction in the weekly average of daily pain intensity on the 11-Point NRS were reported.
Measured: From Baseline at Week 6
Percentage of Participants With >=50% Reduction in the 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. Percentage of participants >= 50% reduction in the weekly average of daily pain intensity on the 11-Point NRS were reported.
Measured: From Baseline at Week 6
Change in the Daily Sleep Interference Scale (DSIS)
Pain-associated sleep interference was assessed using DSIS, based on an 11-point scale (where 0 signified none: pain does not interfere with sleep and 10 signified severe: pain completely interferes with sleep, unable to sleep). Higher score indicates greater pain associated sleep interference.
Measured: From Baseline at Week 6
Percentage of Participants Categorized as Improved on the Patient Global Impression of Change (PGIC) Scale
PGIC scale evaluated the change in activity limitations, symptoms, emotions, and overall quality of life (QoL) related to the participants painful condition on 7-point scale from 1 (improved) to 7 (worse). Participants were categorized as following: scale from 1 - 2 were categorized as "improved", scale from 3 - 4 as "no change" and scale from 5 - 7 were categorized as "worse". Percentage of participants categorized as improved on PGIC scale at week 6 were reported for this outcome measure.
Measured: At Week 6
Change in 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. Higher score indicates greater level of pain.
Measured: From Baseline at Week 6
Pre-dose Plasma Concentration (Ctrough) of VRT-1207355 and the Metabolite VRT-1268114
Measured: Pre-dose at Day 7
Number of Participants With Clinically Meaningful Findings in Columbia Suicide Severity Rating Scale (C-SSRS) Responses
The C-SSRS is an interview-based rating scale was evaluated through a series of questions about suicidal thoughts and behaviors with the possible answers yes or no. Yes represents a worse outcome. Clinically Meaningfulness of C-SSRS responses were judged by investigator based on answers received from participants.
Measured: Day 1 up to Week 10
Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
Measured: Day 1 up to Week 10
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Oct 2017
- Study start20 Sep 2017
- Primary completion12 Oct 2018
- Study completion8 Nov 2018
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Small Fiber NeuropathyStudies of the same condition, open and under way.
- More trials run by Vertex Pharmaceuticals IncorporatedThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 89 participants (actual)
Sponsor and investigators
- Lead sponsor
- Vertex Pharmaceuticals Incorporated
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03304522
- Sponsor's protocol ID
- VX16-150-102
- Other IDs
- 2017-001042-10, 2017
- Status last verified
- Nov 2021
- First submitted
- 18 Sep 2017
- Last updated
- 15 Nov 2021
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 15 Nov 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT03304522
- Acronym / sponsor ID
- VX16-150-102
- Conditions
- Small Fiber Neuropathy
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 89 participants (actual)
- Lead sponsor
- Vertex Pharmaceuticals IncorporatedIndustry (pharma, biotech, devices)
- Study start
- 20 Sep 2017 (actual)
- Primary completion
- 12 Oct 2018 (actual)
- Study completion
- 8 Nov 2018 (actual)
- First posted
- 9 Oct 2017 (actual)
- Results
- Posted 15 Nov 2021
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| VX-150 | Experimental | Drug: VX-150 |
| Placebo | Placebo comparator | Drug: Placebo |
Who may qualify
- Ages
- 18 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- 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
Key exclusion criteria (9 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| VX-150Participants received VX-150 1250 milligrams (mg) once daily (qd) orally for 6 weeks. | Drug | VX-150 |
| PlaceboParticipants received placebo matched to VX-150 for 6 weeks. | Drug | Placebo |
Primary outcome
Change in Weekly Average of Daily Pain Intensity on the 11 Point NRS
Time frame: From Baseline at Week 6
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.
8 secondary outcomes
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
Time frame: From Baseline at Week 6
Percentage of Participants With >=50% Reduction in the Weekly Average of Daily Pain Intensity on the 11-Point NRS
Time frame: From Baseline at Week 6
Change in the Daily Sleep Interference Scale (DSIS)
Time frame: From Baseline at Week 6
Percentage of Participants Categorized as Improved on the Patient Global Impression of Change (PGIC) Scale
Time frame: At Week 6
Change in Pain Intensity on the 11-Point NRS
Time frame: From Baseline at Week 6
Pre-dose Plasma Concentration (Ctrough) of VRT-1207355 and the Metabolite VRT-1268114
Time frame: Pre-dose at Day 7
Number of Participants With Clinically Meaningful Findings in Columbia Suicide Severity Rating Scale (C-SSRS) Responses
Time frame: Day 1 up to Week 10
Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
Time frame: Day 1 up to Week 10
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Nov 2021.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
34 sites in 4 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing VX-150 in people with Small Fiber Neuropathy, run by Vertex Pharmaceuticals Incorporated at 34 sites in 4 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 15 Nov 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 34 sites in United States, Germany, Italy, Netherlands. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 15 Nov 2021). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in Weekly Average of Daily Pain Intensity on the 11 Point NRS (time frame: From Baseline at Week 6). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 15 Nov 2021.