Primary Inherited ErythromelalgiaPhase 2NCT02917187
A Phase 2a Study of BIIB074 in the Treatment of Erythromelalgia
A Phase 2 study testing BIIB074 in people with Primary Inherited Erythromelalgia, run by Biogen at 5 sites in United States.
Trial 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). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Primary Inherited Erythromelalgia
- What is being tested?
- BIIB074
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 8 participants
- Where
- 5 sitesUnited States
- Study dates
- 9 Sep 2016to 5 Jan 2017
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
5 sites in 1 country.
5 sites in 1 country
United States5 sites
Research Site
Birmingham, Alabama, 35242
Research Site
Morgantown, West Virginia, 26505
Research SIte
New London, Connecticut, 06320
Research Site
Philadelphia, Pennsylvania, 19152
Research Site
Plano, Texas, 75075
About this study
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
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.
Read the detailed description
This study was previously posted by Convergence Pharmaceuticals, Ltd., which has been acquired by Biogen.
- Conditions
- Primary Inherited Erythromelalgia
- Being tested
- BIIB074, Placebo
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
BIIB074Drug
Also called CNV1014802
Administered as specified in the treatment arm
PlaceboDrug
Matched placebo
| Study group | Role | What they receive |
|---|---|---|
| Randomized Group 1 | Experimental | After two week run-in, BIIB074 three times a day (TID) followed by placebo (TID) after two week washout period Receives: Drug: BIIB074; Drug: Placebo |
| Randomized Group 2 | Experimental | After two week run-in, Placebo three times a day (TID) followed by BIIB074 (TID) after two week washout period Receives: Drug: BIIB074; Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: Day 1 to Week 12
Secondary outcomes (15)
Weekly maximum severity of paroxysms
The weekly maximum severity is the maximum paroxysm severity recorded by a participant in a given week.
Measured: Day 1 to Week 12
Weekly average and maximum number of paroxysms
Measured: Day 1 to Week 12
Weekly average and weekly maximum duration of paroxysms
Measured: 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.
Measured: 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."
Measured: 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.
Measured: 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.
Measured: Day 1 to Week 12
Use of rescue medication
Measured: 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".
Measured: Day 1 to Week 12
Weekly average and weekly maximum number of awakenings at night due to EM pain
Measured: Day 1 to Week 12
Number of participants experiencing adverse events (AEs) and serious adverse events (SAEs)
Measured: Up to Week 13
Number of participants with clinically significant vital sign abnormalities
Measured: Up to Week 13
Number of participants with clinically significant 12-lead electrocardiograms (ECGs) abnormalities
Measured: Up to Week 13
Number of participants with clinically significant laboratory safety test abnormalities
Measured: 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."
Measured: Up to Week 13
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Sep 2016 (estimated)
- Study start9 Sep 2016
- Primary completion27 Dec 2016
- Study completion5 Jan 2017
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Primary Inherited ErythromelalgiaStudies of the same condition, open and under way.
- More trials run by BiogenThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 8 participants (actual)
Sponsor and investigators
- Lead sponsor
- Biogen
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Medical Director, Study director, Biogen
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02917187
- Sponsor's protocol ID
- 1014802-205
- Status last verified
- Jan 2018
- First submitted
- 19 Jul 2016
- Last updated
- 10 Jan 2018
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 10 Jan 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT02917187
- Acronym / sponsor ID
- 1014802-205
- Conditions
- Primary Inherited Erythromelalgia
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 8 participants (actual)
- Lead sponsor
- BiogenIndustry (pharma, biotech, devices)
- Study start
- 9 Sep 2016 (actual)
- Primary completion
- 27 Dec 2016 (actual)
- Study completion
- 5 Jan 2017 (actual)
- First posted
- 28 Sep 2016 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Randomized Group 1 | Experimental | Drug: BIIB074, Drug: PlaceboAfter two week run-in, BIIB074 three times a day (TID) followed by placebo (TID) after two week washout period |
| Randomized Group 2 | Experimental | Drug: BIIB074, Drug: PlaceboAfter two week run-in, Placebo three times a day (TID) followed by BIIB074 (TID) after two week washout period |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- 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 (5 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| BIIB074Also: CNV1014802Administered as specified in the treatment arm | Drug | Randomized Group 1, Randomized Group 2 |
| PlaceboMatched placebo | Drug | Randomized Group 1, Randomized Group 2 |
Primary outcome
Weekly average severity of paroxysms
Time frame: 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.
15 secondary outcomes
Weekly maximum severity of paroxysms
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
Time frame: Day 1 to Week 12
Patient Global Impression of Change (PGIC) score
Time frame: Day 1 to Week 12
Weekly average and weekly maximum number of pain-mitigating activities
Time frame: Day 1 to Week 12
Weekly average and weekly maximum duration of pain-mitigating activities
Time frame: Day 1 to Week 12
Use of rescue medication
Time frame: Day 1 to Week 13
Weekly average and weekly maximum of the daily sleep interference scale
Time frame: Day 1 to Week 12
Weekly average and weekly maximum number of awakenings at night due to EM pain
Time frame: Day 1 to Week 12
Number of participants experiencing adverse events (AEs) and serious adverse events (SAEs)
Time frame: Up to Week 13
Number of participants with clinically significant vital sign abnormalities
Time frame: Up to Week 13
Number of participants with clinically significant 12-lead electrocardiograms (ECGs) abnormalities
Time frame: Up to Week 13
Number of participants with clinically significant laboratory safety test abnormalities
Time frame: Up to Week 13
Columbia-Suicide Severity Rating Scale (C-SSRS) assessment
Time frame: Up to Week 13
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Jan 2018.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Medical Director study director
Biogen
5 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing BIIB074 in people with Primary Inherited Erythromelalgia, run by Biogen at 5 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 10 Jan 2018. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Weekly average severity of paroxysms (time frame: Day 1 to Week 12). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 10 Jan 2018.