A Phase 2 interventional study of VX-150 and Placebo in Small Fiber Neuropathy, sponsored by Vertex Pharmaceuticals Incorporated. Completed at 34 sites in 4 countries. Open to participants aged 18 Years to 80 Years. Per ClinicalTrials.gov, last updated 2021-11-15.
Sponsored by Vertex Pharmaceuticals Incorporated · Phase 2, Interventional, and Treatment
Among 12 studies in Small Fiber Neuropathy with a site in United States — 3 open to participants now, 3 under way, none of them phase 3. Small Fiber Neuropathy · United States.
Among the 12 studies in Small Fiber Neuropathy with a site in United States, this one is the 3rd largest by enrollment of the 11 interventional ones that give a count, is the only one that started in 2017, is one of 6 in phase 2, and was the 5th registered.
50 studies on the registry are indexed under Small Fiber Neuropathy; 12 are open to participants now.
Browse Small Fiber Neuropathy studies →3 of the 12 studies in Small Fiber Neuropathy with a site in United States are open now. Recruiting first, the newest first:
Endeavor Health · Sites in United States
Ages 18+
Brigham and Women's Hospital · Sites in United States
Ages 18+
TriCity Research Center · Sites in United States
Ages 19+
A study can be open while its sites in United States have not opened yet or have stopped. Only those recruiting at a site in United States →
Of the 9 Small Fiber Neuropathy studies recruiting now, 6 set no upper age limit and 2 stop at 75 or younger; none take children under 18; none take healthy volunteers.
This study: 18 Years to 80 Years · all sexes · no healthy volunteers.
The full table on the hub →This tab as a report to print or save as PDF → With an account.
12 studies; the count open now beside each. This study's row is marked.
A study in two phases is counted once above. Listed by phase, each with its combined phases: Phase 1 1 · Phase 2 7
First in 2011; this study’s year is marked. 1 started in 2017.
By drug class, each counting the studies that test any of its medicines:
This study's enrollment of 89 is above the median of 31 across 31 interventional studies indexed under Small Fiber Neuropathy.
Median enrollment 27 across the 11 interventional studies, 400 across the 1 observational. Of the 5 that completed or were terminated, 5 (100%) have results posted.
This tab as a report to print or save as PDF → With an account.
3 of 12 have an industry lead sponsor. The 10 most frequent:
Vertex Pharmaceuticals Incorporated is the lead sponsor of 249 studies on the registry; 17 are open to participants now.
Of its 107 completed or terminated interventional studies of FDA-regulated products, 53 (50%) have results posted.
Vertex Pharmaceuticals Incorporated has 1 study filed under Small Fiber Neuropathy — fewer than ten, so there is no comparison to draw.
3 of the studies in Small Fiber Neuropathy with a site in United States that are under way reach primary completion within a year, 0 of them phase 3. Phase 3 first, then by date:
| Study | Primary completion |
|---|---|
| NCT04759443Brigham and Women's HospitalDetection of Small Fiber Neuropathy Using Skin Properties | 2027-02-01 |
| NCT04153422Endeavor HealthIVIG in the Treatment of Autoimmune Small Fiber Neuropathy With TS-HDS, FGFR-3, or Plexin D1 Antibodies | 2027-08-27 |
| NCT07704931TriCity Research CenterPilot Study to Evaluate Changes in Intra-Epidermal Nerve Fiber Density (IENFD) in Patients With Small-Fiber Neuropathy | 2027-09-30 |
Add all 3 to your calendar (.ics) → With an account.
Among the 12 studies in Small Fiber Neuropathy with a site in United States, from their registry updates:
This tab as a report to print or save as PDF → With an account.
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.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Other protocol defined Inclusion/Exclusion criteria may apply.
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Weight in kilograms divided by height in metres squared. Example: 70 kg and 1.75 m gives 70 ÷ (1.75 × 1.75) ≈ 22.9.
What counts as overweight or obesity differs between countries (some use lower cut-offs), so go by the range the study states.
A biopsy takes a small piece of tissue to examine. “Archival tissue” is a sample already taken, for example at diagnosis, and kept by the hospital.
Whether a stored sample will do depends on when it was taken, how much there is and what the study needs. Ask whether the study accepts your stored sample or needs a new biopsy.
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
A condition in which the immune system attacks the body’s own tissues, such as rheumatoid arthritis, lupus, psoriasis, type 1 diabetes or inflammatory bowel disease.
Immunotherapy studies often leave out active autoimmune disease, and some name exceptions, such as thyroid disease on replacement hormone. Check the exceptions this study lists.
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
A blood test of average blood sugar over the past two to three months, given as a percentage or in mmol/mol. 6.5% (48 mmol/mol) or more is the usual threshold for diabetes, normally confirmed by a second test.
BMI (body mass index)
Weight in kilograms divided by height in metres squared. Example: 70 kg and 1.75 m gives 70 ÷ (1.75 × 1.75) ≈ 22.9.
What counts as overweight or obesity differs between countries (some use lower cut-offs), so go by the range the study states.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Biopsy and archival tissue
A biopsy takes a small piece of tissue to examine. “Archival tissue” is a sample already taken, for example at diagnosis, and kept by the hospital.
Whether a stored sample will do depends on when it was taken, how much there is and what the study needs. Ask whether the study accepts your stored sample or needs a new biopsy.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Cancer (malignancy)
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Autoimmune disease
A condition in which the immune system attacks the body’s own tissues, such as rheumatoid arthritis, lupus, psoriasis, type 1 diabetes or inflammatory bowel disease.
Immunotherapy studies often leave out active autoimmune disease, and some name exceptions, such as thyroid disease on replacement hormone. Check the exceptions this study lists.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
HIV
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hepatitis B tests
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Clinically significant
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
HbA1c
A blood test of average blood sugar over the past two to three months, given as a percentage or in mmol/mol. 6.5% (48 mmol/mol) or more is the usual threshold for diabetes, normally confirmed by a second test.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Drug: VX-150
Drug: Placebo
Participants received VX-150 1250 milligrams (mg) once daily (qd) orally for 6 weeks.
Participants received placebo matched to VX-150 for 6 weeks.
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.
Time frame: From Baseline at Week 6
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.
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
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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
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
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.
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
| Milestone | VX-150 | Placebo |
|---|---|---|
| Started | 46 | 43 |
| Completed | 45 | 35 |
| Not completed | 1 | 8 |
| Withdrew: Adverse event | 0 | 4 |
| Withdrew: Withdrawal of consent (due to lack of efficacy) | 1 | 1 |
| Withdrew: Withdrawal of consent (for other reason) | 0 | 2 |
| Withdrew: Other | 0 | 1 |
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.
| units on a scale | VX-150 | Placebo |
|---|---|---|
| Change in Weekly Average of Daily Pain Intensity on the 11 Point NRS | -2.018 ± 0.274 | -0.933 ± 0.287 |
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.
| percentage of participants | VX-150 | Placebo |
|---|---|---|
| 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 | 45.0 | 26.5 |
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.
| percentage of participants | VX-150 | Placebo |
|---|---|---|
| Percentage of Participants With >=50% Reduction in the Weekly Average of Daily Pain Intensity on the 11-Point NRS | 32.5 | 17.6 |
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.
| units on a scale | VX-150 | Placebo |
|---|---|---|
| Change in the Daily Sleep Interference Scale (DSIS) | -1.777 ± 0.276 | -0.665 ± 0.289 |
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.
| percentage of participants | VX-150 | Placebo |
|---|---|---|
| Percentage of Participants Categorized as Improved on the Patient Global Impression of Change (PGIC) Scale | 39.5 | 13.5 |
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.
| units on a scale | VX-150 | Placebo |
|---|---|---|
| Change in Pain Intensity on the 11-Point NRS | -1.7 ± 0.3 | -1.1 ± 0.3 |
| microgram per milliliter (mcg/mL) | VX-150 |
|---|---|
| VRT-1207355 | 3.89 ± 2.73 |
| VRT-1268114 | 1.35 ± 0.752 |
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.
| participants | VX-150 | Placebo |
|---|---|---|
| Number of Participants With Clinically Meaningful Findings in Columbia Suicide Severity Rating Scale (C-SSRS) Responses | 0 | 0 |
| participants | VX-150 | Placebo |
|---|---|---|
| Participants with AEs | 29 | 24 |
| Participants with SAEs | 0 | 3 |
Collected over Day 1 up to Week 10. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| VX-150 | 0/46 (0%) | 0/46 (0%) | 16/46 (34.8%) |
| Placebo | 0/43 (0%) | 3/43 (7%) | 7/43 (16.3%) |
| Event | VX-150 | Placebo |
|---|---|---|
| Atrial fibrillationCardiac disorders | 0/46 | 1/43 |
| DiverticulitisInfections and infestations | 0/46 | 1/43 |
| Tendon ruptureInjury, poisoning and procedural complications | 0/46 | 1/43 |
| Event | VX-150 | Placebo |
|---|---|---|
| HeadacheNervous system disorders | 11/46 | 5/43 |
| Dry mouthGastrointestinal disorders | 3/46 | 1/43 |
| NauseaGastrointestinal disorders | 3/46 | 1/43 |
| ArthralgiaMusculoskeletal and connective tissue disorders | 3/46 | 1/43 |
| Muscle spasmsMusculoskeletal and connective tissue disorders | 3/46 | 0/43 |
| Age, Continuous(years) | VX-150 | Placebo | Total |
|---|---|---|---|
| Mean | 55.1 ± 12.34 | 58.1 ± 11.86 | 56.6 ± 12.14 |
| Sex: Female, Male(Participants) | VX-150 | Placebo | Total |
|---|---|---|---|
| Female | 22 | 21 | 43 |
| Male | 24 | 22 | 46 |
| Ethnicity (NIH/OMB)(Participants) | VX-150 | Placebo | Total |
|---|---|---|---|
| Hispanic or Latino | 3 | 5 | 8 |
| Not Hispanic or Latino | 43 | 38 | 81 |
| Unknown or Not Reported | 0 | 0 | 0 |
| Race (NIH/OMB)(Participants) | VX-150 | Placebo | Total |
|---|---|---|---|
| American Indian or Alaska Native | 0 | 0 | 0 |
| Asian | 0 | 1 | 1 |
| Native Hawaiian or Other Pacific Islander | 0 | 0 | 0 |
| Black or African American | 6 | 4 | 10 |
| White | 39 | 34 | 73 |
| More than one race | 1 | 2 | 3 |
| Unknown or Not Reported | 0 | 2 | 2 |
| Pain Intensity at Baseline on 11-point Numeric Rating Scale (NRS)(units on a scale) | VX-150 | Placebo | Total |
|---|---|---|---|
| Mean | 6.433 ± 1.440 | 5.990 ± 1.413 | 6.219 ± 1.436 |
PDFs hosted by the registry, ClinicalTrials.gov.
Ran from Sep 2017 to primary completion in Oct 2018, 12 months.
Results were first posted in Nov 2021, 3 years 1 month after primary completion. A results delay was requested in Oct 2019.
How these figures are worked out, and which studies are left out of them.
This study is completed, as verified in Nov 2021. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: VX-150
Vertex Pharmaceuticals Incorporated →