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

An Exploratory, Randomized, Double-Blind, Crossover Study to Compare the Efficacy and Safety of BIIB074 Versus Placebo in the Treatment of Primary Inherited Erythromelalgia

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.

Study Overview

• Status: Completed
• Conditions: Primary Inherited Erythromelalgia
• Intervention / Treatment: Drug: Placebo; Drug: BIIB074

Detailed Description

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

• Study Type: Interventional
• Enrollment (Actual): 8
• Phase: Phase 2

Contacts and Locations

Study Locations

• United States
  • Alabama
    • Birmingham, Alabama, United States, 35242
      • Research Site
  • Connecticut
    • New London, Connecticut, United States, 06320
      • Research Site
  • Pennsylvania
    • Philadelphia, Pennsylvania, United States, 19152
      • Research Site
  • Texas
    • Plano, Texas, United States, 75075
      • Research Site
  • West Virginia
    • Morgantown, West Virginia, United States, 26505
      • Research Site

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 14 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Crossover Assignment
• Masking: Triple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Randomized Group 1; After two week run-in, BIIB074 three times a day (TID) followed by placebo (TID) after two week washout period	Drug: Placebo; Matched placebo; Drug: BIIB074; Administered as specified in the treatment arm; Other Names: CNV1014802
Experimental: Randomized Group 2; After two week run-in, Placebo three times a day (TID) followed by BIIB074 (TID) after two week washout period	Drug: Placebo; Matched placebo; Drug: BIIB074; Administered as specified in the treatment arm; Other Names: CNV1014802

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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.	Day 1 to Week 12

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Weekly maximum severity of paroxysms	The weekly maximum severity is the maximum paroxysm severity recorded by a participant in a given week.	Day 1 to Week 12
Weekly average and maximum number of paroxysms		Day 1 to Week 12
Weekly average and weekly maximum duration of paroxysms		Day 1 to Week 12
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.	Day 1 to Week 12
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."	Day 1 to Week 12
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.	Day 1 to Week 12
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.	Day 1 to Week 12
Use of rescue medication		Day 1 to Week 13
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".	Day 1 to Week 12
Weekly average and weekly maximum number of awakenings at night due to EM pain		Day 1 to Week 12
Number of participants experiencing adverse events (AEs) and serious adverse events (SAEs)		Up to Week 13
Number of participants with clinically significant vital sign abnormalities		Up to Week 13
Number of participants with clinically significant 12-lead electrocardiograms (ECGs) abnormalities		Up to Week 13
Number of participants with clinically significant laboratory safety test abnormalities		Up to Week 13
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."	Up to Week 13

Collaborators and Investigators

• Sponsor: Biogen

Study record dates

Study Major Dates

• Study Start (Actual): September 9, 2016
• Primary Completion (Actual): December 27, 2016
• Study Completion (Actual): January 5, 2017

Study Registration Dates

• First Submitted: July 19, 2016
• First Submitted That Met QC Criteria: September 26, 2016
• First Posted (Estimate): September 28, 2016

Study Record Updates

• Last Update Posted (Actual): January 10, 2018
• Last Update Submitted That Met QC Criteria: January 5, 2018
• Last Verified: January 1, 2018

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Cardiovascular Diseases; Vascular Diseases; Peripheral Vascular Diseases; Erythromelalgia
• Other Study ID Numbers: 1014802-205