Back to resultsA Phase 2a Study of BIIB074 in the Treatment of Erythromelalgia
Primary Purpose
Primary Inherited Erythromelalgia
Status
Completed
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
BIIB074
Placebo
Sponsored by
About this trial
This is an interventional treatment trial for Primary Inherited Erythromelalgia
Eligibility Criteria
Key Inclusion Criteria:
- A diagnosis of primary inherited EM with family history of EM made at least 3 months from initial diagnosis.
- Failed at least one prior treatment for EM (defined as an inadequate response or intolerance to treatment).
- Approved concomitant medications must have been stable for at least 4 weeks prior to day 1.
Key Exclusion Criteria:
- Positive screening Hepatitis B surface antigen or positive Hepatitis C antibody result.
- Received nerve blocks and/or steroid injections for neuropathic pain within 4 weeks prior to Day 1.
- Males whose partner is pregnant.
- Failed at least one prior treatment for EM (defined as an inadequate response or intolerance to treatment).
NOTE: Other protocol defined Inclusion/ Exclusion criteria may apply.
Sites / Locations
- Research Site
- Research SIte
- Research Site
- Research Site
- Research Site
Arms of the Study
Arm 1
Arm 2
Arm Type
Experimental
Experimental
Arm Label
Randomized Group 1
Randomized Group 2
Arm Description
After two week run-in, BIIB074 three times a day (TID) followed by placebo (TID) after two week washout period
After two week run-in, Placebo three times a day (TID) followed by BIIB074 (TID) after two week washout period
Outcomes
Primary Outcome Measures
Weekly average severity of paroxysms
11-point Pain Intensity Numerical Rating Scale (PI-NRS) is used to assess EM paroxysmal pain. PI-NRS is an 11-point pain intensity numerical rating scale, where 0=no pain and 10=worst possible pain.
Weekly average is defined as the total of severity scores during a week divided by the total number of paroxysms during that week.
Secondary Outcome Measures
Weekly maximum severity of paroxysms
The weekly maximum severity is the maximum paroxysm severity recorded by a participant in a given week.
Weekly average and maximum number of paroxysms
Weekly average and weekly maximum duration of paroxysms
Weekly average and weekly maximum of daily background pain
Each participant provides a daily background pain score via a diary that records background daily pain intensity using a numerical rating scale from 0-10 with higher scores indicating worse pain.
Patient Global Impression of Change (PGIC) score
PGIC is a 7-point self-report scale depicting a participant's rating of overall improvement. Participants rate their change as "very much improved," "much improved," "minimally improved," "no change," "minimally worse," "much worse," or "very much worse."
Weekly average and weekly maximum number of pain-mitigating activities
Participant diary is used to record pain mitigating cooling activities that include but are not limited to use of cold water or fan.
Weekly average and weekly maximum duration of pain-mitigating activities
Participant diary is used to record pain mitigating activities that include but are not limited to use of cooling, and breathing/relaxation and mental imagery techniques.
Use of rescue medication
Weekly average and weekly maximum of the daily sleep interference scale
Daily Sleep Interference Scale (DSIS) describes how much EM pain interfered with the participant's sleep with 0 indicating "pain did not interfere with sleep" and 10 indicating "pain completely interfered with sleep".
Weekly average and weekly maximum number of awakenings at night due to EM pain
Number of participants experiencing adverse events (AEs) and serious adverse events (SAEs)
Number of participants with clinically significant vital sign abnormalities
Number of participants with clinically significant 12-lead electrocardiograms (ECGs) abnormalities
Number of participants with clinically significant laboratory safety test abnormalities
Columbia-Suicide Severity Rating Scale (C-SSRS) assessment
C-SSRS is a suicidal ideation rating used to evaluate suicidality. It rates an individual's degree of suicidal ideation on a scale, ranging from "wish to be dead" to "active suicidal ideation with specific plan and intent."
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT02917187
Brief Title
A Phase 2a Study of BIIB074 in the Treatment of Erythromelalgia
Official Title
An Exploratory, Randomized, Double-Blind, Crossover Study to Compare the Efficacy and Safety of BIIB074 Versus Placebo in the Treatment of Primary Inherited Erythromelalgia
Study Type
Interventional
2. Study Status
Record Verification Date
January 2018
Overall Recruitment Status
Completed
Study Start Date
September 9, 2016 (Actual)
Primary Completion Date
December 27, 2016 (Actual)
Study Completion Date
January 5, 2017 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Biogen
4. Oversight
Data Monitoring Committee
No
5. Study Description
Brief Summary
The primary objective of the study is to investigate the efficacy of repeat oral dosing of BIIB074 on paroxysmal pain in participants with Primary Inherited Erythromelalgia (EM). The secondary objective of the study is to investigate the efficacy of repeat oral dosing of BIIB074 on varying additional aspects of pain in participants with EM; and to investigate the safety and tolerability of repeat oral dosing of BIIB074 in participants with EM.
Detailed Description
This study was previously posted by Convergence Pharmaceuticals, Ltd., which has been acquired by Biogen.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Primary Inherited Erythromelalgia
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Crossover Assignment
Masking
ParticipantCare ProviderInvestigator
Allocation
Randomized
Enrollment
8 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Randomized Group 1
Arm Type
Experimental
Arm Description
After two week run-in, BIIB074 three times a day (TID) followed by placebo (TID) after two week washout period
Arm Title
Randomized Group 2
Arm Type
Experimental
Arm Description
After two week run-in, Placebo three times a day (TID) followed by BIIB074 (TID) after two week washout period
Intervention Type
Drug
Intervention Name(s)
BIIB074
Other Intervention Name(s)
CNV1014802
Intervention Description
Administered as specified in the treatment arm
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
Matched placebo
Primary Outcome Measure Information:
Title
Weekly average severity of paroxysms
Description
11-point Pain Intensity Numerical Rating Scale (PI-NRS) is used to assess EM paroxysmal pain. PI-NRS is an 11-point pain intensity numerical rating scale, where 0=no pain and 10=worst possible pain.
Weekly average is defined as the total of severity scores during a week divided by the total number of paroxysms during that week.
Time Frame
Day 1 to Week 12
Secondary Outcome Measure Information:
Title
Weekly maximum severity of paroxysms
Description
The weekly maximum severity is the maximum paroxysm severity recorded by a participant in a given week.
Time Frame
Day 1 to Week 12
Title
Weekly average and maximum number of paroxysms
Time Frame
Day 1 to Week 12
Title
Weekly average and weekly maximum duration of paroxysms
Time Frame
Day 1 to Week 12
Title
Weekly average and weekly maximum of daily background pain
Description
Each participant provides a daily background pain score via a diary that records background daily pain intensity using a numerical rating scale from 0-10 with higher scores indicating worse pain.
Time Frame
Day 1 to Week 12
Title
Patient Global Impression of Change (PGIC) score
Description
PGIC is a 7-point self-report scale depicting a participant's rating of overall improvement. Participants rate their change as "very much improved," "much improved," "minimally improved," "no change," "minimally worse," "much worse," or "very much worse."
Time Frame
Day 1 to Week 12
Title
Weekly average and weekly maximum number of pain-mitigating activities
Description
Participant diary is used to record pain mitigating cooling activities that include but are not limited to use of cold water or fan.
Time Frame
Day 1 to Week 12
Title
Weekly average and weekly maximum duration of pain-mitigating activities
Description
Participant diary is used to record pain mitigating activities that include but are not limited to use of cooling, and breathing/relaxation and mental imagery techniques.
Time Frame
Day 1 to Week 12
Title
Use of rescue medication
Time Frame
Day 1 to Week 13
Title
Weekly average and weekly maximum of the daily sleep interference scale
Description
Daily Sleep Interference Scale (DSIS) describes how much EM pain interfered with the participant's sleep with 0 indicating "pain did not interfere with sleep" and 10 indicating "pain completely interfered with sleep".
Time Frame
Day 1 to Week 12
Title
Weekly average and weekly maximum number of awakenings at night due to EM pain
Time Frame
Day 1 to Week 12
Title
Number of participants experiencing adverse events (AEs) and serious adverse events (SAEs)
Time Frame
Up to Week 13
Title
Number of participants with clinically significant vital sign abnormalities
Time Frame
Up to Week 13
Title
Number of participants with clinically significant 12-lead electrocardiograms (ECGs) abnormalities
Time Frame
Up to Week 13
Title
Number of participants with clinically significant laboratory safety test abnormalities
Time Frame
Up to Week 13
Title
Columbia-Suicide Severity Rating Scale (C-SSRS) assessment
Description
C-SSRS is a suicidal ideation rating used to evaluate suicidality. It rates an individual's degree of suicidal ideation on a scale, ranging from "wish to be dead" to "active suicidal ideation with specific plan and intent."
Time Frame
Up to Week 13
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Key Inclusion Criteria:
A diagnosis of primary inherited EM with family history of EM made at least 3 months from initial diagnosis.
Failed at least one prior treatment for EM (defined as an inadequate response or intolerance to treatment).
Approved concomitant medications must have been stable for at least 4 weeks prior to day 1.
Key Exclusion Criteria:
Positive screening Hepatitis B surface antigen or positive Hepatitis C antibody result.
Received nerve blocks and/or steroid injections for neuropathic pain within 4 weeks prior to Day 1.
Males whose partner is pregnant.
Failed at least one prior treatment for EM (defined as an inadequate response or intolerance to treatment).
NOTE: Other protocol defined Inclusion/ Exclusion criteria may apply.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Medical Director
Organizational Affiliation
Biogen
Official's Role
Study Director
Facility Information:
Facility Name
Research Site
City
Birmingham
State/Province
Alabama
ZIP/Postal Code
35242
Country
United States
Facility Name
Research SIte
City
New London
State/Province
Connecticut
ZIP/Postal Code
06320
Country
United States
Facility Name
Research Site
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19152
Country
United States
Facility Name
Research Site
City
Plano
State/Province
Texas
ZIP/Postal Code
75075
Country
United States
Facility Name
Research Site
City
Morgantown
State/Province
West Virginia
ZIP/Postal Code
26505
Country
United States
12. IPD Sharing Statement
Learn more about this trial
A Phase 2a Study of BIIB074 in the Treatment of Erythromelalgia
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