Clinical Trial Results:
A Phase 2, Randomized, Double-blind, Placebo-controlled, 6-Week, Parallel-design Study of the Efficacy and Safety of VX-150 in Treating Subjects With Pain Caused by Small Fiber Neuropathy
Summary
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EudraCT number |
2017-001042-10 |
Trial protocol |
DE NL IT |
Global end of trial date |
08 Nov 2018
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Results information
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Results version number |
v1 |
This version publication date |
27 Nov 2019
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First version publication date |
27 Nov 2019
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Other versions |
v2 |
Trial Information
Subject Disposition
Baseline Characteristics
End Points
Adverse Events
More Information
Subject Disposition
Baseline Characteristics
End Points
Adverse Events
More Information
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Trial identification
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Sponsor protocol code |
VX16-150-102
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Additional study identifiers
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ISRCTN number |
- | ||
US NCT number |
NCT03304522 | ||
WHO universal trial number (UTN) |
- | ||
Sponsors
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Sponsor organisation name |
Vertex Pharmaceuticals Incorporated
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Sponsor organisation address |
50 Northern Avenue, Boston, Massachusetts, United States,
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Public contact |
Medical Monitor, Vertex Pharmaceuticals Incorporated, +1 617 341 6777, medicalinfo@vrtx.com
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Scientific contact |
Medical Monitor, Vertex Pharmaceuticals Incorporated, +1 617 341 6777, medicalinfo@vrtx.com
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Paediatric regulatory details
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Is trial part of an agreed paediatric investigation plan (PIP) |
No
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Does article 45 of REGULATION (EC) No 1901/2006 apply to this trial? |
No
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Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial? |
No
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Results analysis stage
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Analysis stage |
Final
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Date of interim/final analysis |
05 Dec 2018
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Is this the analysis of the primary completion data? |
Yes
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Primary completion date |
12 Oct 2018
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Global end of trial reached? |
Yes
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Global end of trial date |
08 Nov 2018
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Was the trial ended prematurely? |
No
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General information about the trial
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Main objective of the trial |
To evaluate the efficacy of VX-150 for the treatment of pain caused by small fiber neuropathy.
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Protection of trial subjects |
The study was conducted in accordance with the ethical principles stated in the Declaration of Helsinki and the International Council on Harmonization (ICH) Guideline for Good Clinical Practice (GCP).
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Background therapy |
- | ||
Evidence for comparator |
- | ||
Actual start date of recruitment |
20 Sep 2017
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Long term follow-up planned |
No
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Independent data monitoring committee (IDMC) involvement? |
No
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Population of trial subjects
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Number of subjects enrolled per country |
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Country: Number of subjects enrolled |
Italy: 10
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Country: Number of subjects enrolled |
Netherlands: 6
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Country: Number of subjects enrolled |
United States: 71
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Country: Number of subjects enrolled |
Germany: 2
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Worldwide total number of subjects |
89
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EEA total number of subjects |
18
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Number of subjects enrolled per age group |
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In utero |
0
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Preterm newborn - gestational age < 37 wk |
0
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Newborns (0-27 days) |
0
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Infants and toddlers (28 days-23 months) |
0
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Children (2-11 years) |
0
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Adolescents (12-17 years) |
0
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Adults (18-64 years) |
66
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From 65 to 84 years |
23
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85 years and over |
0
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Recruitment
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Recruitment details |
- | ||||||||||||||||||||||||
Pre-assignment
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Screening details |
A total of 89 subjects were enrolled and randomized in the study. | ||||||||||||||||||||||||
Period 1
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Period 1 title |
Overall Trial (overall period)
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Is this the baseline period? |
Yes | ||||||||||||||||||||||||
Allocation method |
Randomised - controlled
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Blinding used |
Double blind | ||||||||||||||||||||||||
Roles blinded |
Subject, Investigator, Carer, Assessor | ||||||||||||||||||||||||
Arms
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Are arms mutually exclusive |
Yes
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Arm title
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VX-150 | ||||||||||||||||||||||||
Arm description |
Subjects received VX-150 once daily for 6 weeks. | ||||||||||||||||||||||||
Arm type |
Experimental | ||||||||||||||||||||||||
Investigational medicinal product name |
VX-150
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Investigational medicinal product code |
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Other name |
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Pharmaceutical forms |
Capsule
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Routes of administration |
Oral use
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Dosage and administration details |
Subjects received VX-150 once daily.
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Arm title
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Placebo | ||||||||||||||||||||||||
Arm description |
Subjects received placebo matched to VX-150 once daily for 6 weeks. | ||||||||||||||||||||||||
Arm type |
Placebo | ||||||||||||||||||||||||
Investigational medicinal product name |
Placebo (matched to VX-150)
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Investigational medicinal product code |
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Other name |
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Pharmaceutical forms |
Capsule
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Routes of administration |
Oral use
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Dosage and administration details |
Subjects received placebo matched to VX-150 once daily.
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Baseline characteristics reporting groups
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Reporting group title |
VX-150
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Reporting group description |
Subjects received VX-150 once daily for 6 weeks. | ||||||||||||||||||||||||||||||||||||||||||||
Reporting group title |
Placebo
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Reporting group description |
Subjects received placebo matched to VX-150 once daily for 6 weeks. | ||||||||||||||||||||||||||||||||||||||||||||
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End points reporting groups
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Reporting group title |
VX-150
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Reporting group description |
Subjects received VX-150 once daily for 6 weeks. | ||
Reporting group title |
Placebo
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Reporting group description |
Subjects received placebo matched to VX-150 once daily for 6 weeks. |
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End point title |
Change From Baseline in Weekly Average of Daily Pain Intensity on the 11 Point Numeric Rating Scale (NRS) Score | ||||||||||||
End point description |
Pain intensity was evaluated using the 11-point NRS (where 0 signified no pain and 10 signified worst imaginable pain).
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End point type |
Primary
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End point timeframe |
At Week 6
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Statistical analysis title |
Statistical Analysis | ||||||||||||
Comparison groups |
VX-150 v Placebo
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Number of subjects included in analysis |
89
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Analysis specification |
Pre-specified
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Analysis type |
superiority | ||||||||||||
Method |
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Parameter type |
LS Mean Difference | ||||||||||||
Point estimate |
-1.085
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Confidence interval |
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level |
95% | ||||||||||||
sides |
2-sided
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lower limit |
-1.876 | ||||||||||||
upper limit |
-0.293 |
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Adverse events information
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Timeframe for reporting adverse events |
From first dose of study drug up to safety follow-up (up to 10 weeks)
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Assessment type |
Systematic | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Dictionary used for adverse event reporting
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Dictionary name |
MedDRA | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Dictionary version |
21.1
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Reporting groups
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Reporting group title |
VX-150
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Reporting group description |
Subjects received VX-150 once daily for 6 weeks. | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Reporting group title |
Placebo
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Reporting group description |
Subjects received placebo matched to VX-150 once daily for 6 weeks. | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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Frequency threshold for reporting non-serious adverse events: 5% | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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Substantial protocol amendments (globally) |
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Were there any global substantial amendments to the protocol? Yes | |||
Date |
Amendment |
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21 Apr 2017 |
Amended inclusion criteria to clarify the study population and Body Mass Index (BMI) |
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19 May 2017 |
Amended the dose limit of VX-150 |
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11 Aug 2017 |
Allowed inclusion of women of childbearing potential; updated the upper limit of BMI and the visit requirements |
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13 Apr 2018 |
Updated inclusion and exclusion criteria to facilitate subject recruitment |
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25 May 2018 |
The protocol was amended to enable timely assessment of study data to support continued development |
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Interruptions (globally) |
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Were there any global interruptions to the trial? No | |||
Limitations and caveats |
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Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data. | |||
None reported |