Primary ErythromelalgiaPhase 1/2NCT01486446
Phase 2a, Exploratory Study to Evaluate the Safety, Efficacy, Tolerability and Pharmacokinetics of XPF-002 in Patients With Primary/Inherited Erythromelalgia
A Phase 1/2 study testing XPF-002 in people with Primary Erythromelalgia and Inherited Erythromelalgia, run by Xenon Pharmaceuticals Inc. at 1 site in United States.
Trial 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). Read more about this study
- Who is it for?
- Ages 19 to 80 years
- Conditions the study is for
- Primary Erythromelalgia, Inherited Erythromelalgia
- What is being tested?
- XPF-002
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 8 participants
- Where
- 1 siteUnited States
- Study dates
- Dec 2011to May 2012
- Phase
- Phase 1/2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 19 to 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Show all 11Show fewer
- 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
Trial locations and contacts
1 site in 1 country.
United States1 site
Pinnacle Research Group, LLC.
Anniston, Alabama, 36201
About this study
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
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.
Read the 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.
- Conditions
- Primary Erythromelalgia, Inherited Erythromelalgia
- Being tested
- XPF-002, Placebo
- Keywords
- IEM, EM, Erythromelalgia, Erythermalgia, Red neuralgia, Peripheral Vascular Disease, Vascular Disease, Cardiovascular Disease
- Medical subject headings
- Erythromelalgia, Peripheral Vascular Diseases, Vascular Diseases, Cardiovascular Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
XPF-002Drug
XEN402 8% w/w ointment for topical application, applied to the skin twice daily for 14 or 21 days
PlaceboDrug
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.
| Study group | Role | What they receive |
|---|---|---|
| XPF-002 | Experimental | XEN402 8% w/w ointment for topical application, applied to the skin twice daily for 14 or 21 days Receives: Drug: XPF-002 |
| 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. Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 14-21 Days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Dec 2011 (estimated)
- Study startDec 2011
- Primary completionApr 2012
- Study completionMay 2012
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Primary ErythromelalgiaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 8 participants (actual)
Sponsor and investigators
- Lead sponsor
- Xenon Pharmaceuticals Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Almena L Free, MD, Principal investigator, Pinnacle Research Group LLC.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01486446
- Sponsor's protocol ID
- XPF-002-202
- Status last verified
- Mar 2014
- First submitted
- 2 Dec 2011
- Last updated
- 14 Apr 2014
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 14 Apr 2014. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT01486446
- Acronym / sponsor ID
- XPF-002-202
- Conditions
- Primary Erythromelalgia, Inherited Erythromelalgia
- Keywords
- IEM, EM, Erythromelalgia, Erythermalgia, Red neuralgia, Peripheral Vascular Disease, Vascular Disease, Cardiovascular Disease
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 8 participants (actual)
- Lead sponsor
- Xenon Pharmaceuticals Inc.Industry (pharma, biotech, devices)
- Study start
- Dec 2011
- Primary completion
- Apr 2012 (actual)
- Study completion
- May 2012 (actual)
- First posted
- 6 Dec 2011 (estimated)
- Results
- Posted 14 Apr 2014
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| XPF-002 | Experimental | Drug: XPF-002XEN402 8% w/w ointment for topical application, applied to the skin twice daily for 14 or 21 days |
| Placebo | Placebo comparator | Drug: PlaceboXEN402 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. |
Who may qualify
- Ages
- 19 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- 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
Key exclusion criteria (11 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| XPF-002XEN402 8% w/w ointment for topical application, applied to the skin twice daily for 14 or 21 days | Drug | XPF-002 |
| PlaceboXEN402 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 |
Primary outcome
Average Daily Use of Cooling for Erythromelalgia-Related Pain in Treatment Period 2
Time frame: 14-21 Days
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.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 Apr 2014.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Related trials for Primary Erythromelalgia
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Almena L Free, MD principal investigator
Pinnacle Research Group LLC.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing XPF-002 in people with Primary Erythromelalgia and Inherited Erythromelalgia, run by Xenon Pharmaceuticals Inc. at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 11 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 14 Apr 2014. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 14 Apr 2014). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Average Daily Use of Cooling for Erythromelalgia-Related Pain in Treatment Period 2 (time frame: 14-21 Days). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 14 Apr 2014.