Back to resultsPhase 2a, Exploratory Study to Evaluate the Safety, Efficacy, Tolerability and Pharmacokinetics of XPF-002 in Patients With Primary/Inherited Erythromelalgia
Primary Purpose
Primary Erythromelalgia, Inherited Erythromelalgia
Status
Completed
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
XPF-002
Placebo
Sponsored by
About this trial
This is an interventional treatment trial for Primary Erythromelalgia focused on measuring IEM, EM, Erythromelalgia, Erythermalgia, Red neuralgia, Peripheral Vascular Disease, Vascular Disease, Cardiovascular Disease
Eligibility Criteria
Inclusion Criteria:
- Must have a Body Mass Index (BMI) of 18-40 kg/m2 (inclusive)
- Have primary or inherited erythromelalgia (IEM)
- Experience flares of pain in your feet or hands caused by erythromelalgia
- Be generally healthy (apart from your pain)
- Stop taking your usual pain medications and certain other medications for up to 11.5 weeks
- Not be pregnant or breast-feeding
- Must be able and willing to provide informed consent and willing to comply with all study procedures and restrictions
Exclusion Criteria:
- Must not be in constant pain (must not continually be in moderate pain, 3/10 or more)
- Coexistent source of pain from other conditions that may interfere with the study interpretation
- HIV, Hepatitis B or C
- Treatment for significant depression within 6 months of Screening
- Not willing to use adequate contraception
- Alcoholism, alcohol or substance abuse
- Presence or history of major psychiatric disturbance
- Any other condition or finding that may pose undue risk for participation
- Use of any other investigational drug in the 30 days prior to dosing
- Donation or loss of whole blood or plasma prior to dosing as follows: 50 mL to 499 mL within 30 days, or more than 499 mL within 56 days
- Employee or relative of an employee who is directly involved in the study
Sites / Locations
- Pinnacle Research Group, LLC.
Arms of the Study
Arm 1
Arm 2
Arm Type
Experimental
Placebo Comparator
Arm Label
XPF-002
Placebo
Arm Description
XEN402 8% w/w ointment for topical application, applied to the skin twice daily for 14 or 21 days
XEN402 0% w/w ointment for topical application. Identical content and appearance to the XPF-002 ointment but without the active medicine. Applied to the skin twice daily for 14 or 21 days.
Outcomes
Primary Outcome Measures
Average Daily Use of Cooling for Erythromelalgia-Related Pain in Treatment Period 2
Using diary cards, subjects recorded the use of all non-pharmacological cooling methods used to relieve their erythromelalgia (EM) pain each day during Treatment Period 2.
A smaller average number of cooling uses each day in Treatment Period 2 indicates that subjects were in less pain/required less use of cooling to relieve their EM pain and vice versa.
Secondary Outcome Measures
Full Information
NCT ID
NCT01486446
First Posted
December 2, 2011
Last Updated
March 10, 2014
Sponsor
Xenon Pharmaceuticals Inc.
1. Study Identification
Unique Protocol Identification Number
NCT01486446
Brief Title
Phase 2a, Exploratory Study to Evaluate the Safety, Efficacy, Tolerability and Pharmacokinetics of XPF-002 in Patients With Primary/Inherited Erythromelalgia
Official Title
Phase 2a, Exploratory, Double-blind, Placebo-controlled Two-part Study to Evaluate the Safety, Efficacy, Tolerability and Pharmacokinetics of Topically Applied XPF-002 (XEN402 8% w/w Ointment) in Patients With Primary/Inherited Erythromelalgia
Study Type
Interventional
2. Study Status
Record Verification Date
March 2014
Overall Recruitment Status
Completed
Study Start Date
December 2011 (undefined)
Primary Completion Date
April 2012 (Actual)
Study Completion Date
May 2012 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Xenon Pharmaceuticals Inc.
4. Oversight
Data Monitoring Committee
No
5. Study Description
Brief Summary
This is a Phase 2a single-center, randomized, double-blind, Placebo-controlled, parallel group study with XPF-002 applied twice daily over 14 or 21 days in patients with Primary/Inherited Erythromelalgia (IEM). The purpose of this study is to determine whether XPF-002 is safe and effective in the treatment of pain caused by IEM.
Detailed Description
Your role in the study would include:
Travelling to the clinic (in Anniston, Alabama, USA) for 3 out-patient visits, each lasting approximately 1 day
Travelling and staying in the clinic for 2 in-patient stays:
For the first in-patient visit you will have to spend 8 days and 7 nights in the clinic
For the second in-patient visit you will have to spend 2 days and 1 night in the clinic You can bring books, laptops and DVDs to the clinic.
If you do not live within driving distance to the clinic you will need to stay in the local area (in Anniston, at a local hotel) for 1 to 2 weeks while you use the ointment, in addition to the in-patient portion of the study.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Primary Erythromelalgia, Inherited Erythromelalgia
Keywords
IEM, EM, Erythromelalgia, Erythermalgia, Red neuralgia, Peripheral Vascular Disease, Vascular Disease, Cardiovascular Disease
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1, Phase 2
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
8 (Actual)
8. Arms, Groups, and Interventions
Arm Title
XPF-002
Arm Type
Experimental
Arm Description
XEN402 8% w/w ointment for topical application, applied to the skin twice daily for 14 or 21 days
Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Description
XEN402 0% w/w ointment for topical application. Identical content and appearance to the XPF-002 ointment but without the active medicine. Applied to the skin twice daily for 14 or 21 days.
Intervention Type
Drug
Intervention Name(s)
XPF-002
Intervention Description
XEN402 8% w/w ointment for topical application, applied to the skin twice daily for 14 or 21 days
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
XEN402 0% w/w ointment for topical application. Identical content and appearance to the XPF-002 ointment but without the active medicine. Applied to the skin twice daily for 14 or 21 days.
Primary Outcome Measure Information:
Title
Average Daily Use of Cooling for Erythromelalgia-Related Pain in Treatment Period 2
Description
Using diary cards, subjects recorded the use of all non-pharmacological cooling methods used to relieve their erythromelalgia (EM) pain each day during Treatment Period 2.
A smaller average number of cooling uses each day in Treatment Period 2 indicates that subjects were in less pain/required less use of cooling to relieve their EM pain and vice versa.
Time Frame
14-21 Days
Other Pre-specified Outcome Measures:
Title
Average Cooling Duration (Minutes Per Day) for EM-related Pain in Treatment Period 2
Description
Using diary cards, subjects recorded the use and duration of all non-pharmacological cooling methods used to relieve their EM pain each day during Treatment Period 2.
A smaller average duration of cooling each day in Treatment Period 2 indicates that subjects were in less pain/required less use of cooling to relieve their EM pain and vice versa.
Time Frame
14-21 Days
Title
Time to Exit (Minutes) From Standard Heat Inductions in Treatment Period 1, Compared to Baseline
Description
A standard heat induction was performed on multiple occasions pre-and post treatment. Subjects placed their feet in front of an electric heater for up to 75 minutes. Subjects rated their pain intensity/severity using numerical and categorical rating scales at fixed intervals before, during and after the heating procedure.
The heating procedure continued until one of the pre-defined stopping criteria were met, or when the 75 minutes were over. Subjects could stop the procedure at any time if pain was intolerable. A stopwatch was used to record the time the heating procedure was stopped. This time is known as "Time to Exit".
A longer Time to Exit compared to Baseline indicates that subjects were able to tolerate the heat for a longer period when on treatment.
Time Frame
Baseline to Day 5
Title
Time to Exit (Minutes) From Standard Heat Inductions in Treatment Period 2, Compared to Baseline
Description
A standard heat induction was performed on multiple occasions pre-and post treatment. Subjects placed their feet in front of an electric heater for up to 75 minutes. Subjects rated their pain intensity/severity using numerical and categorical rating scales at fixed intervals before, during and after the heating procedure.
The heating procedure continued until one of the pre-defined stopping criteria were met, or when the 75 minutes were over. Subjects could stop the procedure at any time if pain was intolerable. A stopwatch was used to record the time the heating procedure was stopped. This time is known as "Time to Exit".
A longer Time to Exit compared to Baseline indicates that subjects were able to tolerate the heat for a longer period when on treatment.
Time Frame
Baseline and 14 or 21 days
Title
Time to Moderate Pain (Minutes) During Standard Heat Inductions in Treatment Period 1, Compared to Baseline
Description
A standard heat induction was performed on multiple occasions pre-and post treatment. Subjects placed their feet in front of an electric heater for up to 75 minutes. Subjects rated their pain intensity/severity using numerical and categorical rating scales at fixed intervals before, during and after the heating procedure.
The heating procedure continued until one of the pre-defined stopping criteria were met, or when the 75 minutes were over. Subjects could stop the procedure at any time if pain was intolerable.
A stopwatch was used to record the time the subject recorded a pain intensity numerical score of 5 or more (on a scale of 0-10). This time is known as "Time to Moderate Pain".
A longer Time to Moderate Pain compared to Baseline indicates that subjects were able to tolerate the heat for a longer period when on treatment.
Time Frame
Baseline to Day 5
Title
Time to Moderate Pain (Minutes) During Standard Heat Inductions in Treatment Period 2, Compared to Baseline
Description
A standard heat induction was performed on multiple occasions pre-and post treatment. Subjects placed their feet in front of an electric heater for up to 75 minutes. Subjects rated their pain intensity/severity using numerical and categorical rating scales at fixed intervals before, during and after the heating procedure.
The heating procedure continued until one of the pre-defined stopping criteria were met, or when the 75 minutes were over. Subjects could stop the procedure at any time if pain was intolerable.
A stopwatch was used to record the time the subject recorded a pain intensity numerical score of 5 or more (on a scale of 0-10). This time is known as "Time to Moderate Pain".
A longer Time to Moderate Pain compared to Baseline indicates that subjects were able to tolerate the heat for a longer period when on treatment.
Time Frame
Baseline and 14 or 21 days
Title
Daily Pain (Maximum Pain Intensity) in Treatment Period 2, Compared to Baseline
Description
During Baseline and Treatment Period 2, subjects recorded pain scores in their diary cards 3 times each day (upon waking, lunchtime and evening).
On each recording occasion, subjects recorded the maximum pain experienced since the previous recording occasion using an 11 point numerical rating scale of pain intensity, PINRS (where 0 = no pain and 10 = worst pain imaginable).
A lower score compared to Baseline indicates that the subjects experienced less severe pain on treatment than during Baseline.
Time Frame
Baseline to 14 or 21 days
Title
Sleep Interference Due to Pain in Treatment Period 2, Compared to Baseline
Description
During Baseline and Treatment Period 2, subjects recorded sleep interference scores in their diary cards for each night (scores were recorded upon waking).
Sleep Interference due to pain is scored using an 11 point numerical rating scale where 0 = pain does not interfere with sleep and 10 = completely interferes, unable to sleep due to pain.
A lower sleep interference score compared to Baseline indicates that the subjects' pain interfered with sleep less on treatment than during Baseline.
Time Frame
Baseline to 14 or 21 days
10. Eligibility
Sex
All
Minimum Age & Unit of Time
19 Years
Maximum Age & Unit of Time
80 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Must have a Body Mass Index (BMI) of 18-40 kg/m2 (inclusive)
Have primary or inherited erythromelalgia (IEM)
Experience flares of pain in your feet or hands caused by erythromelalgia
Be generally healthy (apart from your pain)
Stop taking your usual pain medications and certain other medications for up to 11.5 weeks
Not be pregnant or breast-feeding
Must be able and willing to provide informed consent and willing to comply with all study procedures and restrictions
Exclusion Criteria:
Must not be in constant pain (must not continually be in moderate pain, 3/10 or more)
Coexistent source of pain from other conditions that may interfere with the study interpretation
HIV, Hepatitis B or C
Treatment for significant depression within 6 months of Screening
Not willing to use adequate contraception
Alcoholism, alcohol or substance abuse
Presence or history of major psychiatric disturbance
Any other condition or finding that may pose undue risk for participation
Use of any other investigational drug in the 30 days prior to dosing
Donation or loss of whole blood or plasma prior to dosing as follows: 50 mL to 499 mL within 30 days, or more than 499 mL within 56 days
Employee or relative of an employee who is directly involved in the study
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Almena L Free, MD
Organizational Affiliation
Pinnacle Research Group LLC.
Official's Role
Principal Investigator
Facility Information:
Facility Name
Pinnacle Research Group, LLC.
City
Anniston
State/Province
Alabama
ZIP/Postal Code
36201
Country
United States
12. IPD Sharing Statement
Learn more about this trial
Phase 2a, Exploratory Study to Evaluate the Safety, Efficacy, Tolerability and Pharmacokinetics of XPF-002 in Patients With Primary/Inherited Erythromelalgia
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