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BIIB041

Phase 3

Multiple Sclerosis | Small molecule | Neurology |Biogen Inc.|Last Updated: Mar 21, 2017

Success Probability

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Market & Valuation

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Trial Design

RandomizedDouble-BlindPLACEBO_CONTROLLED
Total Trials2
Total Enrollment170

FDA Designations

No designations recorded

Clinical trial landscape

BIIB041 · 4 trials · 6 indications

Phase 3 2Phase 2 1Phase 1 1
NCT01917019A Safety and Efficacy Study of Oral Prolonged-Release Fampridine (BIIB041) in Japanese Participants With Multiple SclerosisMultiple Sclerosis, Remittent Progressive
COMPLETED101 Analytics
NCT01235221Open Label Extension Study to Evaluate the Safety and Tolerability of Oral Fampridine-Sustained Release (SR) in Canadian Participants With Multiple Sclerosis Who Participated in Acorda Extension Trials.Multiple Sclerosis
COMPLETED38 Analytics
PHASE3COMPLETED
A Safety and Efficacy Study of Oral Prolonged-Release Fampridine (BIIB041) in Japanese Participants With Multiple Sclerosis
Multiple Sclerosis, Remittent ProgressiveUnlock trial analytics
PHASE3COMPLETED
Open Label Extension Study to Evaluate the Safety and Tolerability of Oral Fampridine-Sustained Release (SR) in Canadian Participants With Multiple Sclerosis Who Participated in Acorda Extension Trials.
Multiple SclerosisUnlock trial analytics

Study Endpoints

Primary Endpoints

The proportion of participants who show a consistent improvement in walking speed
Part A (Up to 21 Weeks)
Number of participants with Adverse Events (AEs) and Serious Adverse Events (SAEs)
Part B (54 Weeks)
Adverse events (AEs) and serious adverse events (SAEs) as well as Changes in vital signs and clinical laboratory assessments
From Screening (Day 0) to Termination (Month 27)
Change from baseline in self-assessed walking disability as reported on the Multiple Sclerosis Walking Scale-12 (MSWS-12)
Day 1, up to 24 weeks
Change from baseline in static balance as assessed by Berg Balance Scale (BBS)
Day 1, up to 24 weeks
Change from baseline in dynamic balance as assessed by the Timed Up and Go (TUG) scale)
Day 1, up to 24 weeks
Change from baseline in subjective impression of well-being measured by Multiple Sclerosis Impact Scale-29 (MSIS-29)
Day 1, up to 24 weeks
Change from baseline in subjective impression of well-being measured by Euro Quality of Life-5D (EQ-5D)
Day 1, up to 24 weeks
Participant's global impression of change in walking as reported on the Patient Global Impression of Change Scale (PGIC)
Day 1, up to 24 weeks
Summary of Participants with adverse events (AEs) and serious adverse events (SAEs)
Day 1 Up to 26 weeks
Percentage of participants with treatment-emergent adverse events in each ethnic group
Day 1 to Day 7
Observed maximum (peak) plasma 4-aminopyridine (4-AP) concentration (Cmax)
Day 1 (0 to 24 Hours After Dosing)
Time to reach Cmax following study treatment administration (Tmax)
Day 1 (0 to 24 Hours After Dosing)
Area under the time-concentration curve from time zero to infinity (AUC0-∞)
Day 1 (0 to 24 Hours After Dosing)
Apparent elimination half-life (T1/2)
Day 1 (0 to 24 Hours After Dosing)
Renal clearance of the drug from plasma
Day 1 (0 to 24 Hours After Dosing)
Renal clearance as a fraction of total clearance
Day 1 (0 to 24 Hours After Dosing)
Cumulative excreted drug amount at specified sampling intervals (calculated using the concentration data)
Day 1 (0 to 24 Hours After Dosing)

Secondary Endpoints

Number of participants with Adverse Events (AEs) and Serious Adverse Events (SAEs)
Part A (Up to 21 Weeks)
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Study Design & Arms

AllocationRANDOMIZED
MaskingTRIPLE
ModelPARALLEL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Part A PlaceboPLACEBO_COMPARATORPart A: All participants will receive placebo orally twice daily for the first 2 weeks and then be randomized to receive prolonged-release fampridine 10 mg or matching placebo tablets orally twice daily for up to 14 weeks.
Part A prolonged-release fampridineEXPERIMENTALPart A: All participants will receive placebo orally twice daily for the first 2 weeks and then be randomized to receive prolonged-release fampridine 10 mg or matching placebo tablets orally twice daily for up to 14 weeks.
Part B prolonged-release fampridineEXPERIMENTALPart B: Eligible participants will receive open label treatment with prolonged-release fampridine 10mg orally twice daily for up to 52 weeks.
Part C prolonged-release fampridineEXPERIMENTALPart C: Eligible participants will receive open label treatment with prolonged-release fampridine 10mg orally twice daily until marketed product is available.
BIIB041 (Fampridine-SR)EXPERIMENTALParticipants take 10 mg sustained-release tablets of fampridine twice daily for up to 27 months or until the product is commercially available.
Fampridine-PREXPERIMENTALProlonged-Release Fampridine (Fampridine-PR) 10 mg twice daily (every 12 hours) for up to 24 weeks.
PlaceboPLACEBO_COMPARATORMatched placebo twice daily (every 12 hours) for up to 24 weeks.
Chinese Subpopulation: Fampridine-PR 10 mgEXPERIMENTALChinese ethnic participants were administered a single dose of Fampridine-PR 10 mgs
Japanese Subpopulation: Fampridine-PR 10mgEXPERIMENTALJapanese ethnic participants were administered a single dose of Fampridine-PR 10 mgs
Caucasian Subpopulation: Fampridine-PR 10mgEXPERIMENTALCaucasian ethnic participants were administered a single dose of Fampridine-PR 10 mgs

Interventions

NameTypeDescription
PlaceboDRUGmatching placebo tablets
BIIB041 (fampridine)DRUGfampridine prolonged-release tablets
BIIB041 (Fampridine-SR)DRUG10 mg twice a day (BID) sustained-release (SR) tablets by mouth for up to 27 months (in addition to treatment in previous studies) or until the product is commercially available, whichever comes first. Doses of study treatment must be spaced at least 12 hours apart.
BIIB041 (PR Fampridine)DRUG10 mg twice daily, given orally. Doses of study treatment must be spaced at least 12 hours apart. If a dose of study treatment is delayed or missed, the participant should not dose again until their next scheduled dose. Tablets must be swallowed whole and should be taken without food.
BIIB041 (Fampridine-PR)DRUGA single 10mg dose tablet by mouth of fampridine prolonged-release (PR) for all participants
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Eligibility Criteria

Age Range18 Years to 70 Years
SexALL
Healthy VolunteersNo
Study Sites19

Key Inclusion Criteria: Part A To be eligible to participate in Part A, candidates must meet the following eligibility criteria at screening or at the timepoint specified in the individual eligibility criterion listed (potential subjects who fail screening may be rescreened 1 time): 1. Must have ...

Countries:JapanCanadaBelgiumItalyNetherlandsSwedenUnited KingdomAustraliaHong Kong
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Frequently asked questions about BIIB041

What is BIIB041 used for?

BIIB041, also known as fampridine, is an investigational small molecule being studied for the treatment of multiple sclerosis (MS), including relapsing-remitting, secondary progressive, primary progressive, and remittent progressive forms. It has also been evaluated in healthy volunteers for pharmacokinetic and safety studies.

What does BIIB041 target?

BIIB041 is a small molecule that contains fampridine, a potassium channel blocker. By blocking potassium channels, it is thought to improve nerve signal conduction in demyelinated nerves, which may help improve walking ability in people with multiple sclerosis.

Who makes BIIB041?

BIIB041 is being developed by Biogen Inc., a biotechnology company traded on the NASDAQ under the ticker symbol BIIB. Biogen is conducting clinical trials to evaluate the safety and efficacy of this oral fampridine formulation in multiple sclerosis patients.

What phase is BIIB041 in?

BIIB041 is in clinical development, with trials completed in Phase 1, Phase 2, and Phase 3. The most advanced completed trial is a Phase 3 study in Japanese participants with multiple sclerosis. However, BIIB041 is not yet approved and remains investigational.

What clinical trials is BIIB041 in?

BIIB041 has been studied in several completed trials, including NCT01215084 (Phase 1 PK study in healthy volunteers), NCT01235221 (Phase 3 open-label extension in Canadian MS patients), NCT01597297 (Phase 2 exploratory study on walking ability), and NCT01917019 (Phase 3 safety and efficacy study in Japanese MS patients).

Is BIIB041 the same as fampridine?

Yes, BIIB041 is the same as fampridine, specifically an oral prolonged-release or sustained-release formulation of fampridine. Clinical trials for BIIB041 have evaluated this formulation in healthy volunteers and in patients with multiple sclerosis to assess its safety, tolerability, and effects on walking.