A Phase 2a Study of BIIB074 in the Treatment of Erythromelalgia

Overview

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.

Full Title of Study: “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

  • Study Type: Interventional
  • Study Design
    • Allocation: Randomized
    • Intervention Model: Crossover Assignment
    • Primary Purpose: Treatment
    • Masking: Triple (Participant, Care Provider, Investigator)
  • Study Primary Completion Date: December 27, 2016

Detailed Description

This study was previously posted by Convergence Pharmaceuticals, Ltd., which has been acquired by Biogen.

Interventions

  • Drug: BIIB074
    • Administered as specified in the treatment arm
  • Drug: Placebo
    • Matched placebo

Arms, Groups and Cohorts

  • Experimental: Randomized Group 1
    • After two week run-in, BIIB074 three times a day (TID) followed by placebo (TID) after two week washout period
  • Experimental: Randomized Group 2
    • After two week run-in, Placebo three times a day (TID) followed by BIIB074 (TID) after two week washout period

Clinical Trial Outcome Measures

Primary Measures

  • Weekly average severity of paroxysms
    • Time Frame: Day 1 to Week 12
    • 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 Measures

  • 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.”

Participating in This Clinical Trial

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.

Gender Eligibility: All

Minimum Age: 18 Years

Maximum Age: N/A

Are Healthy Volunteers Accepted: No

Investigator Details

  • Lead Sponsor
    • Biogen
  • Provider of Information About this Clinical Study
    • Sponsor
  • Overall Official(s)
    • Medical Director, Study Director, Biogen

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