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

• Status: Completed
• Trial end date: 2018-11-08
• Target enrollment: 0
• Participant gender: All

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

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Vertex Pharmaceuticals Incorporated

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.