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| ID | Type | Description | Link |
|---|---|---|---|
| 2017-001042-10 | EudraCT Number |
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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| VX-150 | Experimental |
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| Placebo | Placebo Comparator |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| VX-150 | Drug | Participants received VX-150 1250 milligrams (mg) once daily (qd) orally for 6 weeks. |
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| Measure | Description | Time Frame |
|---|---|---|
| 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 |
| Measure | Description | Time Frame |
|---|---|---|
| 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. |
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Inclusion Criteria:
Exclusion Criteria:
Other protocol defined Inclusion/Exclusion criteria may apply.
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Xenoscience Inc. - 21st Century Neurology | Phoenix | Arizona | 85004 | United States | ||
| Phoenix Neurological Associates, Ltd. |
A total of 89 participants were enrolled and randomized in the study.
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| ID | Title | Description |
|---|---|---|
| FG000 | VX-150 | Participants received VX-150 1250 milligram (mg) once daily (qd) for 6 weeks. |
| FG001 | Placebo | Participants received placebo matched to VX-150 for 6 weeks. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | May 25, 2018 | Oct 11, 2021 |
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| Placebo | Drug | Participants received placebo matched to VX-150 for 6 weeks. |
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| 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. | 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. | 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. | 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. | From Baseline at Week 6 |
| Pre-dose Plasma Concentration (Ctrough) of VRT-1207355 and the Metabolite VRT-1268114 | 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. | 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) | Day 1 up to Week 10 |
| Phoenix |
| Arizona |
| 85251 |
| United States |
| Sutter Health - Alta Bates Summit Medical Center - The Jordan Research & Education Institute | Berkeley | California | 94705 | United States |
| Neuropain Medical Center | Fresno | California | 93710 | United States |
| University of California San Diego | La Jolla | California | 92093 | United States |
| SDS Clinical Trials, Inc. | Orange | California | 92868 | United States |
| Stanford University School of Medicine | Redwood City | California | 94063 | United States |
| Blue Sky Neurology | Englewood | Colorado | 80113 | United States |
| Bioclinica Research - Orlando | Orlando | Florida | 32806 | United States |
| Infinity Clinical Research | Sunrise | Florida | 33351 | United States |
| Southern Illinois University (SIU) School of Medicine | Springfield | Illinois | 62702 | United States |
| University of Iowa Hospitals and Clinics | Iowa City | Iowa | 52242 | United States |
| University of Kansas Medical Center (KUMC) | Kansas City | Kansas | 66160 | United States |
| International Clinical Research Institute (ICRI) | Overland Park | Kansas | 66210 | United States |
| River Cities Clinical Research Center | Shreveport | Louisiana | 71105 | United States |
| The Johns Hopkins Hospital | Baltimore | Maryland | 21287 | United States |
| Washington University School of Medicine in St. Louis | St Louis | Missouri | 63110 | United States |
| Dartmouth-Hitchcock Medical Center (DHMC) | Lebanon | New Hampshire | 03756 | United States |
| Robert Wood Johnson Medical School | New Brunswick | New Jersey | 08901 | United States |
| University of New Mexico Hospital | Albuquerque | New Mexico | 87131 | United States |
| Albany Medical Center- Neurology Group | Albany | New York | 12208 | United States |
| The Mount Sinai Hospital | New York | New York | 10029 | United States |
| Columbia University Medical Center | New York | New York | 10032 | United States |
| University of Rochester | Rochester | New York | 14618 | United States |
| University of North Carolina School of Medicine | Chapel Hill | North Carolina | 27514 | United States |
| Duke Neurological Disorders Clinic | Durham | North Carolina | 27710 | United States |
| Carolinas Pain Institute | Winston-Salem | North Carolina | 27103 | United States |
| Neurology Diagnostics, Inc | Dayton | Ohio | 45459 | United States |
| The Richter Clinic for Neurology and Neuro-Psychiatry | Tulsa | Oklahoma | 74104 | United States |
| Carilion Clinic Neurology | Roanoke | Virginia | 24013 | United States |
| University of Washington | Seattle | Washington | 98105 | United States |
| Universitätsklinikum Würzburg | Würzburg | Germany |
| Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta | Milan | Italy |
| Maastricht UMC+ | Maastricht | Netherlands |
| COMPLETED |
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| NOT COMPLETED |
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| ID | Title | Description |
|---|---|---|
| BG000 | VX-150 | Participants received VX-150 1250 mg qd for 6 weeks. |
| BG001 | Placebo | Participants received placebo matched to VX-150 for 6 weeks. |
| BG002 | Total | Total of all reporting groups |
| Units | Counts |
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| Participants |
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| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
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| Age, Continuous | Mean | Standard Deviation | years |
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| Sex: Female, Male | Count of Participants | Participants |
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| Ethnicity (NIH/OMB) | Count of Participants | Participants |
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| Race (NIH/OMB) | Count of Participants | Participants |
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| Pain Intensity at Baseline on 11-point Numeric Rating Scale (NRS) | Pain intensity was evaluated using the 11-point NRS (where 0 signified no pain and 10 signified worst imaginable pain). Higher score indicates greater level of pain. Baseline score was average of daily pain scores from Day -7 to Day -1. | Mean | Standard Deviation | units on a scale |
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| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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| Primary | 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. | Full Analysis Set (FAS) included all randomized participants who received at least 1 dose of study drug. | Posted | Least Squares Mean | Standard Error | units on a scale | From Baseline at Week 6 |
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| Secondary | 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. | FAS. Here, 'overall number of participants analyzed' signifies participants who were evaluable for this outcome measure. | Posted | Number | percentage of participants | From Baseline at Week 6 |
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| Secondary | 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. | FAS. Here, 'overall number of participants analyzed' signifies participants who were evaluable for this outcome measure. | Posted | Number | percentage of participants | From Baseline at Week 6 |
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| Secondary | 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. | FAS. | Posted | Least Squares Mean | Standard Error | units on a scale | From Baseline at Week 6 |
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| Secondary | 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. | FAS. Here, 'overall number of participants analyzed' signifies participants who were evaluable for this outcome measure. | Posted | Number | percentage of participants | At Week 6 |
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| Secondary | 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. | FAS. | Posted | Least Squares Mean | Standard Error | units on a scale | From Baseline at Week 6 |
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| Secondary | Pre-dose Plasma Concentration (Ctrough) of VRT-1207355 and the Metabolite VRT-1268114 | Pharmacokinetic (PK) set included participants who received at least 1 dose of study drug and for whom the primary PK data was considered to be sufficient and interpretable. | Posted | Mean | Standard Deviation | microgram per milliliter (mcg/mL) | Pre-dose at Day 7 |
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| Secondary | 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. | Safety Set included all participants who have received at least 1 dose of study drug. | Posted | Number | participants | Day 1 up to Week 10 |
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| Secondary | Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) | Safety set included all participants who had received at least 1 dose of the study drug. | Posted | Number | participants | Day 1 up to Week 10 |
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Day 1 up to Week 10
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | VX-150 | Participants received VX-150 1250 mg qd for 6 weeks. | 0 | 46 | 0 | 46 | 16 | 46 |
| EG001 | Placebo | Participants received placebo matched to VX-150 for 6 weeks. | 0 | 43 | 3 | 43 | 7 | 43 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Atrial fibrillation | Cardiac disorders | MedDRA 21.1 | Systematic Assessment |
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| Diverticulitis | Infections and infestations | MedDRA 21.1 | Systematic Assessment |
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| Tendon rupture | Injury, poisoning and procedural complications | MedDRA 21.1 | Systematic Assessment |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
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| Dry mouth | Gastrointestinal disorders | MedDRA 21.1 | Systematic Assessment |
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| Nausea | Gastrointestinal disorders | MedDRA 21.1 | Systematic Assessment |
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| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA 21.1 | Systematic Assessment |
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| Muscle spasms | Musculoskeletal and connective tissue disorders | MedDRA 21.1 | Systematic Assessment |
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| Headache | Nervous system disorders | MedDRA 21.1 | Systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Medical Monitor | Vertex Pharmaceuticals Incorporated | 617-341-6777 | medicalinfo@vrtx.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Nov 6, 2018 | Oct 11, 2021 | SAP_001.pdf |
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| ID | Term |
|---|---|
| D000071075 | Small Fiber Neuropathy |
| ID | Term |
|---|---|
| D010523 | Peripheral Nervous System Diseases |
| D009468 | Neuromuscular Diseases |
| D009422 | Nervous System Diseases |
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| Male |
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| Not Hispanic or Latino |
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| Unknown or Not Reported |
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| Asian |
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| Native Hawaiian or Other Pacific Islander |
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| Black or African American |
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| White |
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| More than one race |
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| Unknown or Not Reported |
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| Title | Denominators | Categories |
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| VRT-1207355 |
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| VRT-1268114 |
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