Inherited erythromelalgia MedDRA version: 12.0 Level: LLT Classification code 10015284 Term: Erythromelalgia
Conditions
Interventions
Product Name: XPF-001
Product Code: XPF-001
Pharmaceutical Form: Capsule, hard
Current Sponsor code: XEN402
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 100-
Pha
Sponsors
Xenon Pharmaceuticals Inc.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: Subjects will include males and females 18 – 75 years of age (inclusive), with a BMI between 19.5 and 34.0 kg/m2 (inclusive) who have a clinical and genetic diagnosis of Inherited Erythromelalgia (IEM); Subjects must (either spontaneously, or using one of the protocol-defined pain induction methods) achieve pain scores of NRS ³4, and/or moderate/severe on the CRS, or an intolerable level of pain irrespective of pain scores; Subjects must be willing to comply with all study procedures, including the stopping use of all medication, including those for pain management between Check-in and Discharge; Subjects must be in general good health and have no contraindications to the IMP, its excipients, or the permitted rescue medication. Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: Subjects with a coexistent source of pain from other conditions that may interfere with the study interpretation; History or evidence of any condition that, in the opinion of the PI, may pose undue risk to the subject; A positive test for HIV, Hepatitis C antibodies, or Hepatitis B surface antigen; Use of any prescription or over the counter (OTC) medication or supplement from Check-in until Discharge (except for rescue medication); Receiving professional psychological support specifically for coping with IEM; Treatment for significant depression within the 6 months prior to Screening; Females who are pregnant, lactating, or who test positive on a serum-based regnancy test at Screening or Check-in; Not currently undertaking adequate measures to prevent a pregnancy throughout the entire study; Clinically significant abnormal laboratory values, ECG, vital signs or physical examination findings at Screening or Check-in; History or presence of alcoholism or alcohol or substance abuse (not including nicotine or caffeine); Presence or history of major psychiatric disturbance and/or substance abuse, or a positive urine drug test at Check-in; Ingestion of any caffeine-containing food or beverages (including chocolate) in excess of the permitted 1 cup of caffeine containing beverage per day, between Day -1 until Discharge; Consumption of alcohol from Check-in until Discharge, or a positive alcohol breath test on Check-in; Consumption of grapefruit or grapefruit containing products within 7 days of Day 1 until Discharge; Smoking more than 3 cigarettes per day (or the equivalent in tobacco or nicotine substitutes) within the 1 week prior to ICheck-in, and the inability to refrain from all nicotine use between Check-in and Discharge; Has taken an investigational drug within the 60 days prior to Day 1; Donation or loss of whole blood or plasma (excluding the volume of blood that will be drawn during the Screening procedures of this study) prior to Day 1 as follows: 50 mL to 499 mL within 30 days, or more than 499 mL within 56 days prior to drug administration; Has previously been enrolled into this study; Study site or Sponsor employee or relative of an employee who is directly involved in the study; Any other reason that would make the subject, in the opinion of the PI or Sponsor, unsuitable for the study.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: - To evaluate the safety and efficacy (onset, duration of relief and overall efficacy) of multiple doses of XPF-001 (400 mg bid) for relief of pain in patients with IEM. - To evaluate the efficacy of multiple doses of XPF-001 (400 mg bid) for relief of vasomotor signs in patient with IEM. - To evaluate the PK profile of XPF-001 and correlate plasma levels of drug to the pharmacodynamic/efficacy endpoints.;Secondary Objective: · QST thresholds to electrical and thermal stimulations · QST central sensitization to electrical and thermal stimulations · TOTPAR-4 and SPID-4 · SPID-6 · TOTPAR-8 and SPID-8 · Time to first perceptible relief (confirmed) · Time to meaningful relief · Mean PID and REL scores at each observation time · Time to rescue medication · Amount of rescue medication per day · Average total daily dose of rescue medication · Duration of relief · Cumulative percentage of subjects taking rescue medication by each time point · Percentage of subjects rescuing at 2, 4, 6, 8, and 12 hours · Global Evaluation (GE) at 6 hours post morning-dose Safety evaluations will include medical history, physical examination, vital signs, ECGs, laboratory tests, and AEs. It is planned that pharmacokinetic variables Cmax, Tmax, AUC0-t, and others parameters of interest, will be evaluated.;Primary end point(s): TOTPAR-6 (Total Pain Relief score of 6 on an 11-point Numerical Rating Scale (NRS), where 0 = no pain at all, 10 = worst pain imaginable). | — |
Countries
Netherlands
Outcome results
None listed