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

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Xenon Pharmaceuticals Inc.
ClinicalTrials.gov Identifier:
NCT01486446
First received: December 2, 2011
Last updated: March 10, 2014
Last verified: March 2014
Results First Received: January 21, 2014  
Study Type: Interventional
Study Design: Allocation: Randomized;   Endpoint Classification: Safety/Efficacy Study;   Intervention Model: Parallel Assignment;   Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Conditions: Primary Erythromelalgia
Inherited Erythromelalgia
Interventions: Drug: XPF-002
Drug: Placebo

  Participant Flow
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Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
No text entered.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
No text entered.

Reporting Groups
  Description
XPF-002 XEN402 8% w/w ointment for topical application, applied to the skin twice daily for 14 or 21 days
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.

Participant Flow:   Overall Study
    XPF-002     Placebo  
STARTED     7     1  
COMPLETED     6     1  
NOT COMPLETED     1     0  
Adverse Event                 1                 0  



  Baseline Characteristics
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Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
No text entered.

Reporting Groups
  Description
XPF-002 XEN402 8% w/w ointment for topical application, applied to the skin twice daily for 14 or 21 days
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.
Total Total of all reporting groups

Baseline Measures
    XPF-002     Placebo     Total  
Number of Participants  
[units: participants]
  7     1     8  
Age  
[units: participants]
     
<=18 years     0     0     0  
Between 18 and 65 years     6     0     6  
>=65 years     1     1     2  
Age  
[units: years]
Mean ± Standard Deviation
  39.7  ± 19.3     77  ± NA [1]   44.4  ± 22.2  
Gender  
[units: participants]
     
Female     6     1     7  
Male     1     0     1  
Region of Enrollment  
[units: participants]
     
United States     7     1     8  
[1] Only 1 placebo subject, therefore no SD.



  Outcome Measures
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1.  Primary:   Average Daily Use of Cooling for Erythromelalgia-Related Pain in Treatment Period 2   [ Time Frame: 14-21 Days ]

2.  Other Pre-specified:   Average Cooling Duration (Minutes Per Day) for EM-related Pain in Treatment Period 2   [ Time Frame: 14-21 Days ]

3.  Other Pre-specified:   Time to Exit (Minutes) From Standard Heat Inductions in Treatment Period 1, Compared to Baseline   [ Time Frame: Baseline to Day 5 ]

4.  Other Pre-specified:   Time to Exit (Minutes) From Standard Heat Inductions in Treatment Period 2, Compared to Baseline   [ Time Frame: Baseline and 14 or 21 days ]

5.  Other Pre-specified:   Time to Moderate Pain (Minutes) During Standard Heat Inductions in Treatment Period 1, Compared to Baseline   [ Time Frame: Baseline to Day 5 ]

6.  Other Pre-specified:   Time to Moderate Pain (Minutes) During Standard Heat Inductions in Treatment Period 2, Compared to Baseline   [ Time Frame: Baseline and 14 or 21 days ]

7.  Other Pre-specified:   Daily Pain (Maximum Pain Intensity) in Treatment Period 2, Compared to Baseline   [ Time Frame: Baseline to 14 or 21 days ]

8.  Other Pre-specified:   Sleep Interference Due to Pain in Treatment Period 2, Compared to Baseline   [ Time Frame: Baseline to 14 or 21 days ]


  Serious Adverse Events


  Other Adverse Events


  More Information
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Certain Agreements:  
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
The agreement is:
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is less than or equal to 60 days. The sponsor cannot require changes to the communication and cannot extend the embargo.
unchecked Other disclosure agreement that restricts the right of the PI to discuss or publish trial results after the trial is completed.


Limitations and Caveats
Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data

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:  
Name/Title: Dr. Y Paul Goldberg
Organization: Xenon Pharmaceuticals Inc.
phone: 604 484 3300
e-mail: pgoldberg@xenon-pharma.com


No publications provided


Responsible Party: Xenon Pharmaceuticals Inc.
ClinicalTrials.gov Identifier: NCT01486446     History of Changes
Other Study ID Numbers: XPF-002-202
Study First Received: December 2, 2011
Results First Received: January 21, 2014
Last Updated: March 10, 2014
Health Authority: United States: Food and Drug Administration