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

Phase 2

Completed

Conditions: Primary Inherited Erythromelalgia

Interventions: Drug: BIIB074
Drug: Placebo

Registration Number: NCT02917187

Lead Sponsor: Biogen

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.

Recruitment & Eligibility

Status: COMPLETED

Sex: All

Target Recruitment: 8

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 & Design

Study Type: INTERVENTIONAL

Study Design: CROSSOVER

Arm && Interventions

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

Primary Outcome Measures

Name	Time	Method
Weekly average severity of paroxysms	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 Outcome Measures

Name	Time	Method
Weekly average and weekly maximum duration of paroxysms	Day 1 to Week 12
Patient Global Impression of Change (PGIC) score	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."
Columbia-Suicide Severity Rating Scale (C-SSRS) assessment	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."
Number of participants with clinically significant vital sign abnormalities	Up to Week 13
Weekly maximum severity of paroxysms	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	Day 1 to Week 12
Weekly average and weekly maximum of daily background pain	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.
Weekly average and weekly maximum number of pain-mitigating activities	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	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	Day 1 to Week 13
Weekly average and weekly maximum of the daily sleep interference scale	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	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 12-lead electrocardiograms (ECGs) abnormalities	Up to Week 13
Number of participants with clinically significant laboratory safety test abnormalities	Up to Week 13