Pain Caused by Small Fiber Neuropathy MedDRA version: 20.0 Level: PT Classification code 10033371 Term: Pain System Organ Class: 10018065 - General disorders and administration site conditions MedDRA version: 20.0 Level: LLT Classification code 10073937 Term: Small fiber neuropathy System Organ Class: 100000004852
Conditions
Interventions
Product Name: VX-150
Product Code: VX-150
Pharmaceutical Form: Capsule
INN or Proposed INN: -
Current Sponsor code: VX-150
Sponsors
Vertex Pharmaceuticals Incorporated
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Subject will sign and date an informed consent form (ICF) 2. Willing and able to comply with scheduled visits, treatment plan, study restrictions, laboratory tests, contraceptive guidelines, and other study procedures 3. Subjects (male and female) will be between the ages of 18 and 80 years, inclusive. 4. Body mass index (BMI) of =18.0 kg/m2 5. 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 6. 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 or within 6 months of screening 7. Presence of sural nerve responses 8. Average NRS score between =4 and =9 reported in the daily diary on Days -7 through -1 Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 86 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 28
Exclusion criteria
Exclusion criteria: 1. 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 (all 3 with no recurrence for the last 5 years) 2. Exposure to neurotoxic drugs (i.e., chemotherapy) since diagnosis of small fiber neuropathy. Untreated or uncontrolled connective tissue disorders, sarcoidosis, Sjögren’s syndrome, amyloidosis, Fabry’s disease, celiac disease, Lyme disease, autoimmune disorders (i.e., as assessed by anti-nuclear antibodies, rheumatoid factor, sedimentation rate, and/or lupus anticoagulant) including myasthenia gravis and Guillain-Barre syndrome, which in the opinion of the investigator makes the subject unsuitable for inclusion in this study. 3. A known or clinically suspected infection with human immunodeficiency virus or hepatitis B or C viruses 4. Current clinically significant liver or kidney dysfunction 5. Current uncontrolled thyroid dysfunction 6. Subjects with a diagnosis of diabetes who have any 1 of the following criteria: ? - HbA1c =11% at screening - are not stabilized on oral hypoglycemics and/or subcutaneous insulin or diet, in the opinion of the investigator ? - evidence of ulcers or severe nephropathy resulting from their diabetes ? - advanced retinopathy, defined as greater than State 3 (moderate non-proliferative diabetic retinopathy) ? - history of a clinical atherosclerotic event, such as myocardial infarction or stroke 7. History of cardiac dysrhythmias requiring anti-arrhythmia treatment(s); or history or evidence of abnormal ECGs that in the opinion of the investigator or medical monitor would preclude the subject's participation in the study 8. Standard 12-lead ECG demonstrating QTc >450 msec at screening. If QTc exceeds 450 msec, the ECG will be repeated 2 more times, and the average of the 3 QTc values will be used to determine the subject’s eligibility. 9. Concomitant severe pain conditions (i.e., low back pain, radiculopathy, severe bone and musculoskeletal disorders) which may impair self-assessment of pain due to small fiber neuropathy 10. Abnormal laboratory results indicative of any significant medical disease that, in the opinion of the investigator, would preclude the subject's participation in the study 11. Other serious, acute, or chronic medical or psychiatric illness that, in the judgment of the investigator, could compromise subject safety, limit the subject’s ability to complete the study and/or compromise the objectives of the study 12. Female subjects who are pregnant, nursing, or planning to become pregnant during the study or within 90 days after the last study drug dose 13. Male subjects with a female partner who is pregnant, nursing, or planning to become pregnant during the study or within 90 days after the last study drug dose 14. Use of restricted medication or food within the specified duration before the first dose of study drug
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To evaluate the efficacy of VX-150 for the treatment of pain caused by small fiber neuropathy; Secondary Objective: • To evaluate the safety and tolerability of VX-150 • To evaluate the PK of VRT-1207355 and the metabolite VRT-1268114 ;Primary end point(s): Change from baseline in the weekly average of daily pain intensity on the 11-point numeric rating scale (NRS), as reported in the daily diary, at Week 6;Timepoint(s) of evaluation of this end point: Week 6 | — |
Secondary
| Measure | Time frame |
|---|---|
| Timepoint(s) of evaluation of this end point: • At week 6 • Plasma PK parameters at day 7, week 3, week 6 and early termination visit • Safety and tolerability at each visit ; Secondary end point(s): • Proportion of subjects with =30% reduction in the weekly average of daily pain intensity on the 11-point NRS, as reported in the daily diary, at Week 6 • Proportion of subjects with =50% reduction in the weekly average of daily pain intensity on the 11-point NRS, as reported in the daily diary, at Week 6 • Change from baseline in the Daily Sleep Interference Scale (DSIS) at Week 6 • Proportion of subjects categorized as improved at Week 6 on the patient global impression of change (PGIC) assessment • Change from baseline in pain intensity on the 11-point NRS, as reported at study visits, at Week 6 • Plasma PK parameters of VRT-1207355 and the metabolite VRT-1268114 • Safety and tolerability based on the Columbia Suicide Severity Rating Scale (C-SSRS), incidence and type of AEs, changes from baseline in clinically significant laboratory test results, vital signs, and ECGs at each visit | — |
Countries
Canada, Germany, Italy, Netherlands, United States
Contacts
Public ContactClinical Trials and Medical Info
Vertex Pharmaceuticals Incorporated
Outcome results
None listed