Clinical trial NCT02917187: A Phase 2a Study of BIIB074 in the Treatment of Erythromelalgia
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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Primary Inherited Erythromelalgia
Lead sponsor: Biogen
Sponsor class: INDUSTRY
First posted: 2016-09-28
Enrollment: 8
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: CROSSOVER
Primary purpose: TREATMENT

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.

Eligibility criteria: 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.

Primary outcomes:
measure: Weekly average severity of paroxysms
timeFrame: Day 1 to Week 12
Secondary outcomes:
measure: Weekly maximum severity of paroxysms
timeFrame: Day 1 to Week 12
measure: Weekly average and maximum number of paroxysms
timeFrame: Day 1 to Week 12
measure: Weekly average and weekly maximum duration of paroxysms
timeFrame: Day 1 to Week 12
measure: Weekly average and weekly maximum of daily background pain
timeFrame: Day 1 to Week 12
measure: Patient Global Impression of Change (PGIC) score
timeFrame: Day 1 to Week 12
measure: Weekly average and weekly maximum number of pain-mitigating activities
timeFrame: Day 1 to Week 12
measure: Weekly average and weekly maximum duration of pain-mitigating activities
timeFrame: Day 1 to Week 12
measure: Use of rescue medication
timeFrame: Day 1 to Week 13
measure: Weekly average and weekly maximum of the daily sleep interference scale
timeFrame: Day 1 to Week 12
measure: Weekly average and weekly maximum number of awakenings at night due to EM pain
timeFrame: Day 1 to Week 12
measure: Number of participants experiencing adverse events (AEs) and serious adverse events (SAEs)
timeFrame: Up to Week 13
measure: Number of participants with clinically significant vital sign abnormalities
timeFrame: Up to Week 13
measure: Number of participants with clinically significant 12-lead electrocardiograms (ECGs) abnormalities
timeFrame: Up to Week 13
measure: Number of participants with clinically significant laboratory safety test abnormalities
timeFrame: Up to Week 13
measure: Columbia-Suicide Severity Rating Scale (C-SSRS) assessment
timeFrame: Up to Week 13
Other outcomes:
Locations:
city: Birmingham
country: United States
facility: Research Site
state: Alabama
city: New London
country: United States
facility: Research SIte
state: Connecticut
city: Philadelphia
country: United States
facility: Research Site
state: Pennsylvania
city: Plano
country: United States
facility: Research Site
state: Texas
city: Morgantown
country: United States
facility: Research Site
state: West Virginia
Study officials:
affiliation: Biogen
name: Medical Director
role: STUDY_DIRECTOR
References: