Study of XPF-001 in the Treatment of Pain From Primary/Inherited Erythromelalgia (IEM)

Phase 2a, Double-blind, Placebo-controlled, 2-period, Crossover Study to Evaluate the Safety, Efficacy and Pharmacokinetics of Multiple Oral Doses of XPF-001 in Patients With Inherited Erythromelalgia.

The purpose of this study is to determine whether XPF-001 is safe and effective in the treatment of pain caused by Inherited Erythromelalgia (IEM).

Study Overview

• Status: Completed
• Conditions: Inherited Erythromelalgia; Primary Erythromelalgia
• Intervention / Treatment: Drug: Placebo; Drug: XPF-001

• Study Type: Interventional
• Enrollment (Actual): 4
• Phase: Phase 2; Phase 1

Contacts and Locations

Study Locations

• Netherlands
  • Nijmegen, Netherlands, 6500 HB
    • Clinical Research Centre at Radboud University Nijmegen Medical Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 75 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

The study requires you to:

• Be18-75 years old
• Have inherited erythromelalgia
• Be experiencing pain caused by erythromelalgia (Moderate pain score 4/10)
• Be generally healthy (apart from your pain)
• Stop taking your usual pain medications for 9 days
• Not be pregnant or breast-feeding

Your role in the study includes:

• An out-patient screening visit
• 9-days/8 nights-in-patient treatment visit at Nijmegen Medical Center (where you will be closely monitored)
• A follow up phone call (after your return home)
• Taking the investigational medication daily
• Recording your pain levels daily during stay at the Medical Center.

Exclusion Criteria:

• Coexistent source of pain from other conditions
• Receiving professional psychological support for dealing with IEM
• Treatment for significant depression within 6 months of screening
• Active HIV, Hepatitis B or C
• Use of prescription or OTC medication between check-in and discharge
• Women who are pregnant, or lactating
• Not currently using adequate contraception
• Alcoholism or alcohol or substance abuse
• Presence or history of major psychiatric disturbance
• Unwilling or unable to comply with all dietary and activity restrictions
• Any other condition or finding that may pose undue risk for participation
• Use of any other investigational drug in the 60 days prior to dosing

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Crossover Assignment
• Masking: Double

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Placebo Comparator: Matching Placebo	Drug: Placebo; Oral capsule
Experimental: XPF-001	Drug: XPF-001; Oral capsule

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Pain intensity difference (PID) in each treatment period based on an 11-point Pain Intensity-NRS (where 0 = No pain and 10 = worst pain you can imagine)	2 days

Collaborators and Investigators

• Sponsor: Xenon Pharmaceuticals Inc.
• Investigators: Principal Investigator: Joost PH Drenth, MD PhD, Radboud University Nijmegen Medical Center

Study record dates

Study Major Dates

• Study Start: April 1, 2010
• Primary Completion (Actual): August 1, 2010
• Study Completion (Actual): September 1, 2010

Study Registration Dates

• First Submitted: March 17, 2010
• First Submitted That Met QC Criteria: March 18, 2010
• First Posted (Estimate): March 22, 2010

Study Record Updates

• Last Update Posted (Estimate): March 16, 2012
• Last Update Submitted That Met QC Criteria: March 14, 2012
• Last Verified: March 1, 2012

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Cardiovascular Diseases; Vascular Diseases; Peripheral Vascular Diseases; Erythromelalgia
• Other Study ID Numbers: XPF-001-202; 2009-015619-42 (EudraCT Number)