A Phase 1/2 interventional study of XPF-002 and Placebo in Primary Erythromelalgia and Inherited Erythromelalgia, sponsored by Xenon Pharmaceuticals Inc.. Completed at 1 site in United States. Open to participants aged 19 Years to 80 Years. Per ClinicalTrials.gov, last updated 2014-04-14.
Sponsored by Xenon Pharmaceuticals Inc. · Phase 1/2, Interventional, and Treatment
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.
Your role in the study would include:
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.
Xenon Pharmaceuticals Inc. is the lead sponsor of 23 studies on the registry; 8 are open to participants now.
Counted across the registry records on this site, refreshed daily.
Exclusion Criteria:
XEN402 8% w/w ointment for topical application, applied to the skin twice daily for 14 or 21 days
Drug: XPF-002
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.
Drug: Placebo
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.
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.
Time frame: 14-21 Days
Average Cooling Duration (Minutes Per Day) for EM-related Pain in Treatment Period 2
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
Time to Exit (Minutes) From Standard Heat Inductions in Treatment Period 1, Compared to Baseline
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
Time to Exit (Minutes) From Standard Heat Inductions in Treatment Period 2, Compared to Baseline
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
Time to Moderate Pain (Minutes) During Standard Heat Inductions in Treatment Period 1, Compared to Baseline
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
Time to Moderate Pain (Minutes) During Standard Heat Inductions in Treatment Period 2, Compared to Baseline
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
Daily Pain (Maximum Pain Intensity) in Treatment Period 2, Compared to Baseline
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
Sleep Interference Due to Pain in Treatment Period 2, Compared to Baseline
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
| Milestone | XPF-002 | Placebo |
|---|---|---|
| Started | 7 | 1 |
| Completed | 6 | 1 |
| Not completed | 1 | 0 |
| Withdrew: Adverse event | 1 | 0 |
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.
| minutes/day | XPF-002 | Placebo |
|---|---|---|
| Average Cooling Duration (Minutes Per Day) for EM-related Pain in Treatment Period 2 | 7.8 (0 to 13.3) | 86 (NA to NA) |
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.
| Percentage of Baseline | XPF-002 | Placebo |
|---|---|---|
| Time to Exit (Minutes) From Standard Heat Inductions in Treatment Period 1, Compared to Baseline | 138 (78 to 188) | 96 (NA to NA) |
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.
| cooling uses/day | XPF-002 | Placebo |
|---|---|---|
| Average Daily Use of Cooling for Erythromelalgia-Related Pain in Treatment Period 2 | 0.24 (0.03 to 0.27) | 2.4 (NA to NA) |
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.
| Percentage of Baseline | XPF-002 | Placebo |
|---|---|---|
| Time to Exit (Minutes) From Standard Heat Inductions in Treatment Period 2, Compared to Baseline | 100 (92 to 112) | 107 (NA to NA) |
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.
| Percentage of Baseline | XPF-002 | Placebo |
|---|---|---|
| Time to Moderate Pain (Minutes) During Standard Heat Inductions in Treatment Period 1, Compared to Baseline | 137 (74 to 172) | 90 (NA to NA) |
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.
| Percentage of Baseline | XPF-002 | Placebo |
|---|---|---|
| Time to Moderate Pain (Minutes) During Standard Heat Inductions in Treatment Period 2, Compared to Baseline | 103 (91 to 115) | 135 (NA to NA) |
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.
| Percentage of Baseline | XPF-002 | Placebo |
|---|---|---|
| Daily Pain (Maximum Pain Intensity) in Treatment Period 2, Compared to Baseline | 78.5 (75.9 to 94.2) | 76 (NA to NA) |
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.
| Percentage of Baseline | XPF-002 | Placebo |
|---|---|---|
| Sleep Interference Due to Pain in Treatment Period 2, Compared to Baseline | 76.5 (48.8 to 86.9) | 46 (NA to NA) |
Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| XPF-002 | — | 1/7 (14.3%) | 7/7 (100%) |
| Placebo | — | 0/1 (0%) | 1/1 (100%) |
| Event | XPF-002 | Placebo |
|---|---|---|
| Hospitalization due to worsening erythromelalgia painVascular disorders | 1/7 | 0/1 |
| Event | XPF-002 | Placebo |
|---|---|---|
| PainGeneral disorders | 0/7 | 1/1 |
| Skin exfoliationSkin and subcutaneous tissue disorders | 0/7 | 1/1 |
| HeadacheNervous system disorders | 3/7 | 0/1 |
| NasopharyngitisRespiratory, thoracic and mediastinal disorders | 3/7 | 0/1 |
| ParaesthesiaNervous system disorders | 2/7 | 0/1 |
| RashSkin and subcutaneous tissue disorders | 2/7 | 0/1 |
| NauseaGastrointestinal disorders | 2/7 | 0/1 |
| Burning sensationNervous system disorders | 1/7 | 0/1 |
| Sinus headacheNervous system disorders | 1/7 | 0/1 |
| NoduleGeneral disorders | 1/7 | 0/1 |
| Age, Categorical(Participants) | XPF-002 | Placebo | Total |
|---|---|---|---|
| <=18 years | 0 | 0 | 0 |
| Between 18 and 65 years | 6 | 0 | 6 |
| >=65 years | 1 | 1 | 2 |
| Age, Continuous(years) | XPF-002 | Placebo | Total |
|---|---|---|---|
| Mean | 39.7 ± 19.3 | 77 ± NA | 44.4 ± 22.2 |
| Sex: Female, Male(Participants) | XPF-002 | Placebo | Total |
|---|---|---|---|
| Female | 6 | 1 | 7 |
| Male | 1 | 0 | 1 |
| Region of Enrollment(participants) | XPF-002 | Placebo | Total |
|---|---|---|---|
| United States | 7 | 1 | 8 |
This study is completed, as verified in Mar 2014. You cannot join it, but the record below documents what was studied.
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Erythromelalgia
Xenon Pharmaceuticals Inc.