Primary Inherited Erythromelalgia Clinical Trial
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
| Verified date | January 2018 |
| Source | Biogen |
| Contact | n/a |
| Is FDA regulated | No |
| Health authority | |
| Study type | Interventional |
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.
| Status | Completed |
| Enrollment | 8 |
| Est. completion date | January 5, 2017 |
| Est. primary completion date | December 27, 2016 |
| Accepts healthy volunteers | No |
| Gender | All |
| Age group | 18 Years and older |
| Eligibility |
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. |
| Country | Name | City | State |
|---|---|---|---|
| United States | Research Site | Birmingham | Alabama |
| United States | Research Site | Morgantown | West Virginia |
| United States | Research SIte | New London | Connecticut |
| United States | Research Site | Philadelphia | Pennsylvania |
| United States | Research Site | Plano | Texas |
| Lead Sponsor | Collaborator |
|---|---|
| Biogen |
United States,
| Type | Measure | Description | Time frame | Safety issue |
|---|---|---|---|---|
| Primary | 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 | 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 | |
| Secondary | Weekly average and maximum number of paroxysms | Day 1 to Week 12 | ||
| Secondary | Weekly average and weekly maximum duration of paroxysms | Day 1 to Week 12 | ||
| Secondary | 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 | |
| Secondary | 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 | |
| Secondary | 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 | |
| Secondary | 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 | |
| Secondary | Use of rescue medication | Day 1 to Week 13 | ||
| Secondary | 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 | |
| Secondary | Weekly average and weekly maximum number of awakenings at night due to EM pain | Day 1 to Week 12 | ||
| Secondary | Number of participants experiencing adverse events (AEs) and serious adverse events (SAEs) | Up to Week 13 | ||
| Secondary | Number of participants with clinically significant vital sign abnormalities | Up to Week 13 | ||
| Secondary | Number of participants with clinically significant 12-lead electrocardiograms (ECGs) abnormalities | Up to Week 13 | ||
| Secondary | Number of participants with clinically significant laboratory safety test abnormalities | Up to Week 13 | ||
| Secondary | 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 |