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

Biogen8 enrolled

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.

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

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

TRIPLE

Enrollment

Conditions

Primary Inherited Erythromelalgia

Interventions

BIIB074DRUG

Administered as specified in the treatment arm

PlaceboDRUG

Matched placebo

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

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.

Locations (5)

Research Site

Birmingham, Alabama, United States

Research SIte

New London, Connecticut, United States

Research Site

Philadelphia, Pennsylvania, United States

Research Site

Plano, Texas, United States

Outcomes

Primary Outcomes

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.

Time frame: Day 1 to Week 12

Secondary Outcomes

Weekly maximum severity of paroxysms

The weekly maximum severity is the maximum paroxysm severity recorded by a participant in a given week.

Time frame: Day 1 to Week 12

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

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

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."

Time frame: 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.

Data from ClinicalTrials.gov

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