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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| XPF-002 | Experimental | XEN402 8% w/w ointment for topical application, applied to the skin twice daily for 14 or 21 days |
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| Placebo | Placebo Comparator | 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. |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| XPF-002 | Drug | XEN402 8% w/w ointment for topical application, applied to the skin twice daily for 14 or 21 days |
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| Measure | Description | Time Frame |
|---|---|---|
| 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. | 14-21 Days |
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| Measure | Description | Time Frame |
|---|---|---|
| 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. |
Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Almena L Free, MD | Pinnacle Research Group LLC. | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Pinnacle Research Group, LLC. | Anniston | Alabama | 36201 | United States |
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| ID | Title | Description |
|---|---|---|
| FG000 | XPF-002 | XEN402 8% w/w ointment for topical application, applied to the skin twice daily for 14 or 21 days |
| FG001 | Placebo | 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. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
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| Placebo | Drug | 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. |
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| 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. | 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. | 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. | 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. | 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. | 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. | Baseline to 14 or 21 days |
| COMPLETED |
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| NOT COMPLETED |
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| ID | Title | Description |
|---|---|---|
| BG000 | XPF-002 | XEN402 8% w/w ointment for topical application, applied to the skin twice daily for 14 or 21 days |
| BG001 | Placebo | 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. |
| BG002 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
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| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||||
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| Age, Categorical | Count of Participants | Participants |
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| Age, Continuous | Mean | Standard Deviation | years |
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| Sex: Female, Male | Count of Participants | Participants |
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| Region of Enrollment | Number | participants |
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| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Other Pre-specified | 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. | Posted | Mean | Inter-Quartile Range | minutes/day | 14-21 Days |
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| Other Pre-specified | 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. | Posted | Mean | Inter-Quartile Range | Percentage of Baseline | Baseline to Day 5 |
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| Primary | 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. | Posted | Mean | Inter-Quartile Range | cooling uses/day | 14-21 Days |
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| Other Pre-specified | 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. | Posted | Mean | Inter-Quartile Range | Percentage of Baseline | Baseline and 14 or 21 days |
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| Other Pre-specified | 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. | Posted | Mean | Inter-Quartile Range | Percentage of Baseline | Baseline to Day 5 |
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| Other Pre-specified | 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. | Posted | Mean | Inter-Quartile Range | Percentage of Baseline | Baseline and 14 or 21 days |
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| Other Pre-specified | 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. | Posted | Mean | Inter-Quartile Range | Percentage of Baseline | Baseline to 14 or 21 days |
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| Other Pre-specified | 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. | Posted | Mean | Inter-Quartile Range | Percentage of Baseline | Baseline to 14 or 21 days |
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | XPF-002 | XEN402 8% w/w ointment for topical application, applied to the skin twice daily for 14 or 21 days | 1 | 7 | 7 | 7 | ||
| EG001 | Placebo | 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. | 0 | 1 | 1 | 1 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Hospitalization due to worsening erythromelalgia pain | Vascular disorders | Subject was admitted to hospital for treatment of worsening erythromelalgia pain 131 days following their last dose of study drug. |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Headache | Nervous system disorders | MedDRA 15.0 |
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| Paraesthesia | Nervous system disorders | MedDRA 15.0 |
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| Burning sensation | Nervous system disorders | MedDRA 15.0 |
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| Sinus headache | Nervous system disorders | MedDRA 15.0 |
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| Nodule | General disorders | MedDRA 15.0 |
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| Flushing | General disorders | MedDRA 15.0 |
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| Malaise | General disorders | MedDRA 15.0 |
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| Pain | General disorders | MedDRA 15.0 |
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| Nasopharyngitis | Respiratory, thoracic and mediastinal disorders | MedDRA 15.0 |
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| Upper-airway cough syndrome | Respiratory, thoracic and mediastinal disorders | MedDRA 15.0 |
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| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 15.0 |
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| Rhinorrhoea | Respiratory, thoracic and mediastinal disorders | MedDRA 15.0 |
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| Rash | Skin and subcutaneous tissue disorders | MedDRA 15.0 |
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| Erythema | Skin and subcutaneous tissue disorders | MedDRA 15.0 |
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| Rash pruritic | Skin and subcutaneous tissue disorders | MedDRA 15.0 |
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| Skin exfoliation | Skin and subcutaneous tissue disorders | MedDRA 15.0 |
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| Nausea | Gastrointestinal disorders | MedDRA 15.0 |
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| Oopharyngeal pain | Gastrointestinal disorders | MedDRA 15.0 |
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| Tooth infection | Gastrointestinal disorders | MedDRA 15.0 |
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| Flushing | Vascular disorders | MedDRA 15.0 |
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| Cyanosis | Vascular disorders | MedDRA 15.0 |
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| External ear inflammation | Ear and labyrinth disorders | MedDRA 15.0 |
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| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA 15.0 |
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| Muscle spasms | Musculoskeletal and connective tissue disorders | MedDRA 15.0 |
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| Eye inflammation | Eye disorders | MedDRA 15.0 |
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| Urinary tract infection | Renal and urinary disorders | MedDRA 15.0 |
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This was a small trial with many exploratory efficacy measures. There was no primary outcome measure, all measures are "other: pre-specified".
Only 8 subjects were enrolled (1 placebo), the actual number of Adverse Events is used, not incidence.
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Dr. Y Paul Goldberg | Xenon Pharmaceuticals Inc. | 604 484 3300 | pgoldberg@xenon-pharma.com |
| ID | Term |
|---|---|
| D004916 | Erythromelalgia |
| D016491 | Peripheral Vascular Diseases |
| D014652 | Vascular Diseases |
| D002318 | Cardiovascular Diseases |
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| >=65 years |
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| Male |
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