Phase 2
N=89
A Study to Evaluate the Efficacy and Safety of VX-150 in Treating Subjects With Pain Caused by Small Fiber Neuropathy
Small Fiber Neuropathy
Bottom Line
View on ClinicalTrials.gov: NCT03304522 ↗Enrolled (actual)
89
Serious AEs
3.4%
Results posted
Nov 2021
Primary outcome: Primary: Change in Weekly Average of Daily Pain Intensity on the 11 Point NRS — -2.018; -0.933 units on a scale — p=<0.0001
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- VX-150 (Drug); Placebo (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Vertex Pharmaceuticals Incorporated
- Primary completion
- Oct 2018
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Change in Weekly Average of Daily Pain Intensity on the 11 Point NRS |
-2.018; -0.933 | <0.0001 sig |
| 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 |
45.0; 26.5 | — |
| SECONDARY Percentage of Participants With >=50% Reduction in the Weekly Average of Daily Pain Intensity on the 11-Point NRS |
32.5; 17.6 | — |
| SECONDARY Change in the Daily Sleep Interference Scale (DSIS) |
-1.777; -0.665 | <0.0001 sig |
| SECONDARY Percentage of Participants Categorized as Improved on the Patient Global Impression of Change (PGIC) Scale |
39.5; 13.5 | — |
| SECONDARY Change in Pain Intensity on the 11-Point NRS |
-1.7; -1.1 | <0.0001 sig |
| SECONDARY Pre-dose Plasma Concentration (Ctrough) of VRT-1207355 and the Metabolite VRT-1268114 |
3.89; 1.35 | — |
| SECONDARY Number of Participants With Clinically Meaningful Findings in Columbia Suicide Severity Rating Scale (C-SSRS) Responses |
0; 0 | — |
| SECONDARY Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) |
29; 24; 0; 3 | — |
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.
Eligibility 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.
Data sourced from ClinicalTrials.gov (NCT03304522). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.