Clinical Trial: A Phase 2a Study of BIIB074 in the Treatment of Erythromelalgia

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

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

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 Summary: Convergence Pharmaceuticals, Ltd., registered in England, has been acquired and is now an affiliate and subsidiary of Biogen, Inc.
Sponsor: Convergence Pharmaceuticals

Current Primary Outcome: Weekly average severity of paroxysms [ Time Frame: Day 1 to Week 12 ]

Original Primary Outcome: Same as current

Current Secondary Outcome:

• Weekly maximum severity of paroxysms [ Time Frame: Day 1 to Week 12 ]
  The weekly maximum severity is the maximum paroxysm severity recorded by a participant in a given week.
• Weekly average and maximum number of paroxysms [ Time Frame: Day 1 to Week 12 ]
• Weekly average and weekly maximum duration of paroxysms [ Time Frame: Day 1 to Week 12 ]
• Weekly average and weekly maximum of daily background pain [ Time Frame: Day 1 to Week 12 ]
  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 [ Time Frame: Day 1 to Week 12 ]
  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 [ Time Frame: Day 1 to Week 12 ]
  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 [ Time Frame: Day 1 to Week 12 ]
  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 [ Time Frame: Day 1 to Week 13 ]
• Weekly average and weekly maximum of the daily sleep interference scale [ Time Frame: Day 1 to Week 12 ]
  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 [ Time Frame: Day 1 to Week 12 ]
• Number of participants experiencing adverse events (AEs) and serious adverse events (SAEs) [ Time Frame: Up to Week 13 ]
• Number of participants with clinically significant vital sign abnormalities [ Time Frame: Up to Week 13 ]
• Number of participants with clinically significant 12-lead electrocardiograms (ECGs) abnormalities [ Time Frame: Up to Week 13 ]
• Number of participants with clinically significant laboratory safety test abnormalities [ Time Frame: Up to Week 13 ]
• Columbia-Suicide Severity Rating Scale (C-SSRS) assessment [ Time Frame: Up to Week 13 ]
  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."

Original Secondary Outcome:

• Weekly maximum severity of paroxysms [ Time Frame: Day 1 to Week 12 ]
  The weekly maximum severity is the maximum paroxysm severity recorded by a participant in a given week.
• Weekly average and maximum number of paroxysms [ Time Frame: Day 1 to Week 12 ]
• Weekly average and weekly maximum duration of paroxysms [ Time Frame: Day 1 to Week 12 ]
• Weekly average and weekly maximum of daily background pain [ Time Frame: Day 1 to Week 12 ]
  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 [ Time Frame: Day 1 to Week 12 ]
  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 [ Time Frame: Day 1 to Week 12 ]
  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 [ Time Frame: Day 1 to Week 12 ]
• Use of rescue medication [ Time Frame: Day 1 to Week 13 ]
• Weekly average and weekly maximum of the daily sleep interference scale [ Time Frame: Day 1 to Week 12 ]
  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 [ Time Frame: Day 1 to Week 12 ]
• Number of participants experiencing adverse events (AEs) and serious adverse events (SAEs) [ Time Frame: Up to Week 13 ]
• Number of participants with clinically significant vital sign abnormalities [ Time Frame: Up to Week 13 ]
• Number of participants with clinically significant 12-lead electrocardiograms (ECGs) abnormalities [ Time Frame: Up to Week 13 ]
• Number of participants with clinically significant laboratory safety test abnormalities [ Time Frame: Up to Week 13 ]
• Columbia-Suicide Severity Rating Scale (C-SSRS) assessment [ Time Frame: Up to Week 13 ]
  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."

Information By: Convergence Pharmaceuticals

Dates:
Date Received: July 19, 2016
Date Started: September 2016
Date Completion:
Last Updated: January 24, 2017
Last Verified: January 2017