A study to investigate how safe and able to produce an effect in the body study drug VX-150 is in patients with pain that typically begins in the feet or hand
- Conditions
- Pain Caused by Small Fiber NeuropathyMedDRA version: 20.0Level: PTClassification code 10033371Term: PainSystem Organ Class: 10018065 - General disorders and administration site conditionsMedDRA version: 20.1Level: PTClassification code 10073928Term: Small fibre neuropathySystem Organ Class: 10029205 - Nervous system disordersTherapeutic area: Body processes [G] - Bones and nerves physological processes [G11]
- Registration Number
- EUCTR2017-001042-10-IT
- Lead Sponsor
- VERTEX PHARMACEUTICALS INCORPORATED
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 89
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) = 18.0
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 response
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
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 anti-coagulant) 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 nonproliferative diabetic retinopathy)15
• 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
15. Alcohol, analgesic/opioid, and/or illicit drug abuse as defined by the American Psychiatric Association Diagnostic and Statistical Manual of Mental Disorders, Fifth Edition, in the last 12 months before screening, or a positive test for drugs of abuse at screening
• A positive drug screen for a known concomitant medication that is not otherwise exclusionary (e.g., benzodiazepines) will not disqualify subjects; however, marijuana and marijuana derivatives will not be allowed
16. Subject, or close relative of the subject, is the investigator or a subinvestigator, research assistant, pha
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method 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<br>¿ 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 Outcome Measures
Name Time Method