Phase 2a, Exploratory Study to Evaluate the Safety, Efficacy, Tolerability and Pharmacokinetics of XPF-002 in Patients With Primary/Inherited Erythromelalgia

NCT01486446 | Completed Phase 1 Results

• 1 other identifier: XPF-002-202
• Study Type: interventional
• Target: 8
• Locations: 1 country
• Sites: 1

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.

Conditions

Primary Erythromelalgia; Inherited Erythromelalgia

Keywords

IEM; EM; Erythromelalgia; Erythermalgia; Red neuralgia; Peripheral Vascular Disease; Vascular Disease; Cardiovascular Disease

Outcome Measures

Primary Outcomes (1)

• 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

Other Outcomes (7)

• Average Cooling Duration (Minutes Per Day) for EM-related Pain in Treatment Period 2

  14-21 Days

• Time to Exit (Minutes) From Standard Heat Inductions in Treatment Period 1, Compared to Baseline

  Baseline to Day 5

• Time to Exit (Minutes) From Standard Heat Inductions in Treatment Period 2, Compared to Baseline

  Baseline and 14 or 21 days

Study Arms (2)

XPF-002

EXPERIMENTAL

XEN402 8% w/w ointment for topical application, applied to the skin twice daily for 14 or 21 days

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.

Drug: Placebo

Interventions

XPF-002 DRUG

XEN402 8% w/w ointment for topical application, applied to the skin twice daily for 14 or 21 days

XPF-002

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.

Placebo

Eligibility Criteria

• Age: 19 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Xenon Pharmaceuticals Inc.: lead

Study Sites (1)

Pinnacle Research Group, LLC.

Anniston, Alabama, 36201, United States

Location

MeSH Terms

Conditions

Erythromelalgia; Peripheral Vascular Diseases; Vascular Diseases; Cardiovascular Diseases

Limitations and Caveats

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.

Results Point of Contact

• Title: Dr. Y Paul Goldberg
• Organization: Xenon Pharmaceuticals Inc.

pgoldberg@xenon-pharma.com

604 484 3300

Study Officials

• Almena L Free, MD

  Pinnacle Research Group LLC.

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: GT60
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 2, 2011
• First Posted: December 6, 2011
• Study Start: December 1, 2011
• Primary Completion: April 1, 2012
• Study Completion: May 1, 2012
• Last Updated: April 14, 2014
• Results First Posted: April 14, 2014

Record last verified: 2014-03

Locations

US (1)