Trial Outcomes & Findings for A Study to Evaluate the Efficacy and Safety of VX-150 in Treating Subjects With Pain Caused by Small Fiber Neuropathy (NCT NCT03304522)

NCT ID: NCT03304522

Last Updated: 2021-11-15

Results Overview

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.

• Recruitment status: COMPLETED
• Study phase: PHASE2
• Target enrollment: 89 participants
• Primary outcome timeframe: From Baseline at Week 6
• Results posted on: 2021-11-15

Participant Flow

A total of 89 participants were enrolled and randomized in the study.

Participant milestones

Measure	VX-150 Participants received VX-150 1250 milligram (mg) once daily (qd) for 6 weeks.	Placebo Participants received placebo matched to VX-150 for 6 weeks.
Overall Study STARTED	46	43
Overall Study COMPLETED	45	35
Overall Study NOT COMPLETED	1	8

Reasons for withdrawal

Measure	VX-150 Participants received VX-150 1250 milligram (mg) once daily (qd) for 6 weeks.	Placebo Participants received placebo matched to VX-150 for 6 weeks.
Overall Study Adverse Event	0	4
Overall Study Withdrawal of consent (due to lack of efficacy)	1	1
Overall Study Withdrawal of consent (for other reason)	0	2
Overall Study Other	0	1

Baseline Characteristics

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

Baseline characteristics by cohort

Measure	VX-150 n=46 Participants Participants received VX-150 1250 mg qd for 6 weeks.	Placebo n=43 Participants Participants received placebo matched to VX-150 for 6 weeks.	Total n=89 Participants Total of all reporting groups
Age, Continuous	55.1 years STANDARD_DEVIATION 12.34 • n=93 Participants	58.1 years STANDARD_DEVIATION 11.86 • n=4 Participants	56.6 years STANDARD_DEVIATION 12.14 • n=27 Participants
Sex: Female, Male Female	22 Participants n=93 Participants	21 Participants n=4 Participants	43 Participants n=27 Participants
Sex: Female, Male Male	24 Participants n=93 Participants	22 Participants n=4 Participants	46 Participants n=27 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	3 Participants n=93 Participants	5 Participants n=4 Participants	8 Participants n=27 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	43 Participants n=93 Participants	38 Participants n=4 Participants	81 Participants n=27 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=93 Participants	0 Participants n=4 Participants	0 Participants n=27 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=93 Participants	0 Participants n=4 Participants	0 Participants n=27 Participants
Race (NIH/OMB) Asian	0 Participants n=93 Participants	1 Participants n=4 Participants	1 Participants n=27 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=93 Participants	0 Participants n=4 Participants	0 Participants n=27 Participants
Race (NIH/OMB) Black or African American	6 Participants n=93 Participants	4 Participants n=4 Participants	10 Participants n=27 Participants
Race (NIH/OMB) White	39 Participants n=93 Participants	34 Participants n=4 Participants	73 Participants n=27 Participants
Race (NIH/OMB) More than one race	1 Participants n=93 Participants	2 Participants n=4 Participants	3 Participants n=27 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=93 Participants	2 Participants n=4 Participants	2 Participants n=27 Participants
Pain Intensity at Baseline on 11-point Numeric Rating Scale (NRS)	6.433 units on a scale STANDARD_DEVIATION 1.440 • n=93 Participants	5.990 units on a scale STANDARD_DEVIATION 1.413 • n=4 Participants	6.219 units on a scale STANDARD_DEVIATION 1.436 • n=27 Participants

PRIMARY outcome

Timeframe: From Baseline at Week 6

Population: Full Analysis Set (FAS) included all randomized participants who received at least 1 dose of study drug.

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.

Outcome measures

Measure	VX-150 n=46 Participants Participants received VX-150 1250 mg qd for 6 weeks.	Placebo n=43 Participants Participants received placebo matched to VX-150 for 6 weeks.
Change in Weekly Average of Daily Pain Intensity on the 11 Point NRS	-2.018 units on a scale Standard Error 0.274	-0.933 units on a scale Standard Error 0.287

SECONDARY outcome

Timeframe: From Baseline at Week 6

Population: FAS. Here, 'overall number of participants analyzed' signifies participants who were evaluable for this outcome measure.

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.

Outcome measures

Measure	VX-150 n=40 Participants Participants received VX-150 1250 mg qd for 6 weeks.	Placebo n=34 Participants Participants received placebo matched to VX-150 for 6 weeks.
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 percentage of participants	26.5 percentage of participants

SECONDARY outcome

Timeframe: From Baseline at Week 6

Population: FAS. Here, 'overall number of participants analyzed' signifies participants who were evaluable for this outcome measure.

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.

Outcome measures

Measure	VX-150 n=40 Participants Participants received VX-150 1250 mg qd for 6 weeks.	Placebo n=34 Participants Participants received placebo matched to VX-150 for 6 weeks.
Percentage of Participants With >=50% Reduction in the Weekly Average of Daily Pain Intensity on the 11-Point NRS	32.5 percentage of participants	17.6 percentage of participants

SECONDARY outcome

Timeframe: From Baseline at Week 6

Population: FAS.

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.

Outcome measures

Measure	VX-150 n=46 Participants Participants received VX-150 1250 mg qd for 6 weeks.	Placebo n=43 Participants Participants received placebo matched to VX-150 for 6 weeks.
Change in the Daily Sleep Interference Scale (DSIS)	-1.777 units on a scale Standard Error 0.276	-0.665 units on a scale Standard Error 0.289

SECONDARY outcome

Timeframe: At Week 6

Population: FAS. Here, 'overall number of participants analyzed' signifies participants who were evaluable for this outcome measure.

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.

Outcome measures

Measure	VX-150 n=43 Participants Participants received VX-150 1250 mg qd for 6 weeks.	Placebo n=37 Participants Participants received placebo matched to VX-150 for 6 weeks.
Percentage of Participants Categorized as Improved on the Patient Global Impression of Change (PGIC) Scale	39.5 percentage of participants	13.5 percentage of participants

SECONDARY outcome

Timeframe: From Baseline at Week 6

Population: FAS.

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.

Outcome measures

Measure	VX-150 n=46 Participants Participants received VX-150 1250 mg qd for 6 weeks.	Placebo n=43 Participants Participants received placebo matched to VX-150 for 6 weeks.
Change in Pain Intensity on the 11-Point NRS	-1.7 units on a scale Standard Error 0.3	-1.1 units on a scale Standard Error 0.3

SECONDARY outcome

Timeframe: Pre-dose at Day 7

Population: 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.

Outcome measures

Measure	VX-150 n=12 Participants Participants received VX-150 1250 mg qd for 6 weeks.	Placebo Participants received placebo matched to VX-150 for 6 weeks.
Pre-dose Plasma Concentration (Ctrough) of VRT-1207355 and the Metabolite VRT-1268114 VRT-1207355	3.89 microgram per milliliter (mcg/mL) Standard Deviation 2.73	—
Pre-dose Plasma Concentration (Ctrough) of VRT-1207355 and the Metabolite VRT-1268114 VRT-1268114	1.35 microgram per milliliter (mcg/mL) Standard Deviation 0.752	—

SECONDARY outcome

Timeframe: Day 1 up to Week 10

Population: Safety Set included all participants who have received at least 1 dose of study drug.

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.

Outcome measures

Measure	VX-150 n=46 Participants Participants received VX-150 1250 mg qd for 6 weeks.	Placebo n=43 Participants Participants received placebo matched to VX-150 for 6 weeks.
Number of Participants With Clinically Meaningful Findings in Columbia Suicide Severity Rating Scale (C-SSRS) Responses	0 participants	0 participants

SECONDARY outcome

Timeframe: Day 1 up to Week 10

Population: Safety set included all participants who had received at least 1 dose of the study drug.

Outcome measures

Measure	VX-150 n=46 Participants Participants received VX-150 1250 mg qd for 6 weeks.	Placebo n=43 Participants Participants received placebo matched to VX-150 for 6 weeks.
Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) Participants with AEs	29 participants	24 participants
Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) Participants with SAEs	0 participants	3 participants

Adverse Events

VX-150

Serious events: 0 serious events

Other events: 16 other events

Deaths: 0 deaths

Placebo

Serious events: 3 serious events

Other events: 7 other events

Deaths: 0 deaths

Serious adverse events

Measure	VX-150 n=46 participants at risk Participants received VX-150 1250 mg qd for 6 weeks.	Placebo n=43 participants at risk Participants received placebo matched to VX-150 for 6 weeks.
Cardiac disorders Atrial fibrillation	0.00% 0/46 • Day 1 up to Week 10	2.3% 1/43 • Day 1 up to Week 10
Infections and infestations Diverticulitis	0.00% 0/46 • Day 1 up to Week 10	2.3% 1/43 • Day 1 up to Week 10
Injury, poisoning and procedural complications Tendon rupture	0.00% 0/46 • Day 1 up to Week 10	2.3% 1/43 • Day 1 up to Week 10

Other adverse events

Measure	VX-150 n=46 participants at risk Participants received VX-150 1250 mg qd for 6 weeks.	Placebo n=43 participants at risk Participants received placebo matched to VX-150 for 6 weeks.
Gastrointestinal disorders Dry mouth	6.5% 3/46 • Day 1 up to Week 10	2.3% 1/43 • Day 1 up to Week 10
Gastrointestinal disorders Nausea	6.5% 3/46 • Day 1 up to Week 10	2.3% 1/43 • Day 1 up to Week 10
Musculoskeletal and connective tissue disorders Arthralgia	6.5% 3/46 • Day 1 up to Week 10	2.3% 1/43 • Day 1 up to Week 10
Musculoskeletal and connective tissue disorders Muscle spasms	6.5% 3/46 • Day 1 up to Week 10	0.00% 0/43 • Day 1 up to Week 10
Nervous system disorders Headache	23.9% 11/46 • Day 1 up to Week 10	11.6% 5/43 • Day 1 up to Week 10

Additional Information

Medical Monitor

Vertex Pharmaceuticals Incorporated

Phone: 617-341-6777

Email: medicalinfo@vrtx.com

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place