Phase 4
N=901
Open-Label Study to Assess the Effect of Long-Term Prolonged-Release Fampridine (BIIB041) on Quality of Life as Reported by Participants With Multiple Sclerosis
Multiple Sclerosis
Bottom Line
View on ClinicalTrials.gov: NCT01480076 ↗Enrolled (actual)
901
Serious AEs
9.5%
Results posted
Nov 2016
Primary outcome: Primary: Change From Baseline in the Physical Component Scale (PCS) of the Short Form 36 Health Status Questionnaire (SF-36) At Months 3, 6, 9, and 12: Responders — 4.2; 3.4; 3.2; 2.9 units on a scale — p=<0.0001
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 4
- Interventions
- Fampridine (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Biogen
- Primary completion
- Jul 2013
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Change From Baseline in the Physical Component Scale (PCS) of the Short Form 36 Health Status Questionnaire (SF-36) At Months 3, 6, 9, and 12: Responders |
4.2; 3.4; 3.2; 2.9 | <0.0001 sig |
| SECONDARY Change From Baseline in the PCS of the SF-36 at Months 3, 6, 9, and 12: Responders Versus Non-responders |
3.3; -0.4; 4.0; -0.2; 3.3; 0.2 | <0.0001 sig |
| SECONDARY Change From Baseline in the MCS of the SF-36 At Months 3, 6, 9, and 12 |
3.1; 0.1; 4.2; -0.6; 3.3; 0.6 | <0.0001 sig |
| SECONDARY Change From Baseline in the Multiple Sclerosis Impact Scale (MSIS-29) Physical Score at Months 3, 6, 9, and 12 |
-10.3; -2.1; -13.0; -2.1; -10.6; -3.0 | <0.0001 sig |
| SECONDARY Change From Baseline in MSIS-29 Psychological Score at Months 3, 6, 9, and 12 |
-7.8; -1.0; -9.6; -0.2; -7.7; -2.6 | <0.0001 sig |
| SECONDARY Change From Baseline in the Activities Limitation Scale of the Patient-Reported Indices for Multiple Sclerosis (PRIMUS) at Months 3, 6, 9, and 12 |
-1.4; 0.8; -2.3; -0.4; -1.4; 0.8 | <0.0001 sig |
| SECONDARY Change From Baseline in the Current Health State of EuroQoL Descriptive System of Health-related Quality of Life States Consisting of 5 Dimensions (EQ-5D) Visual Analog Scale (VAS) at Months 3, 6, 9, And 12 |
6.7; -4.4; 8.0; -3.2; 6.1; -3.6 | <0.0001 sig |
| SECONDARY Change From Baseline in the Index Scores of EQ-5D at Months 3, 6, 9, and 12 |
0.05; 0.00; 0.07; 0.01; 0.05; 0.01 | <0.0001 sig |
| SECONDARY Change From Baseline in Percent Work Time Missed Due to MS, by the Work Productivity and Activity Impairment-Specific Health Problem (WPAI-SHP) Questionnaire at Months 3, 6, 9, and 12 |
-2.5; -8.3; -3.0; -4.0; -5.3; -7.6 | 0.1795 |
| SECONDARY Change From Baseline in Percent Impairment While Working Due to MS, by the WPAI-SHP Questionnaire at Months 3, 6, 9, and 12 |
-4.2; -8.4; -6.9; -1.8; -6.5; -5.6 | 0.0281 sig |
| SECONDARY Change From Baseline in Percent Overall Work Impairment Due to MS, by the WPAI-SHP Questionnaire at Months 3, 6, 9, and 12 |
-5.2; -8.7; -7.7; -4.5; -7.8; -2.9 | 0.0219 sig |
| SECONDARY Change From Baseline in Regular Activity Productivity Loss, by the WPAI-SHP Questionnaire at Months 3, 6, 9, and 12 |
-11.4; -4.1; -13.5; -3.1; -12.3; -4.7 | <0.0001 sig |
| SECONDARY Change From Baseline in the PCS of the SF-36 at Months 3, 6, 9, and 12 by MS Disease Type: Responders |
4.5; 3.8; 2.5; 3.2; 5.5; 4.5 | <0.0001 sig |
| SECONDARY Change From Baseline in the MCS of the SF-36 at Months 3, 6, 9, and 12 by MS Disease Type: Responders |
4.0; 3.0; 3.2; 2.0; 4.9; 4.5 | <0.0001 sig |
| SECONDARY Change From Baseline in the MSIS-29 Physical Score at Months 3, 6, 9, and 12 by MS Disease Type: Responders |
-12.1; -10.3; -8.3; -12.5; -15.0; -13.3 | <0.0001 sig |
| SECONDARY Change From Baseline in the MSIS-29 Psychological Score at Months 3, 6, 9, and 12 by MS Disease Type: Responders |
-9.4; -6.5; -8.3; -7.5; -11.2; -8.9 | <0.0001 sig |
| SECONDARY Change From Baseline in the Activity Limitation Scale (ALS) of PRIMUS Score at Months 3, 6, 9, and 12 by MS Disease Type: Responders |
-2.5; -1.1; -0.5; -1.7; -3.2; -2.1 | <0.0001 sig |
| SECONDARY Change From Baseline in Current Health State of the EQ-5D VAS at Months 3, 6, 9, and 12 by MS Disease Type: Responders |
8.8; 6.5; 6.7; 4.7; 10.1; 8.2 | <0.0001 sig |
| SECONDARY Change From Baseline in EQ-5D Index Scores at Months 3, 6, 9, and 12 by MS Disease Type: Responders |
0.05; 0.04; 0.05; 0.07; 0.07; 0.06 | <0.0001 sig |
| SECONDARY Change From Baseline in Percent Work Time Missed Due to MS on the WPAI-SHP Questionnaire at Months 3, 6, 9, and 12 by MS Disease Type: Responders |
-2.9; -1.5; -2.5; -4.0; -3.1; -2.6 | 0.1471 |
| SECONDARY Change From Baseline in Percent Impairment While Working Due to MS on the WPAI-SHP Questionnaire at Months 3, 6, 9, and 12 by MS Disease Type: Responders |
-5.8; -4.2; 0.8; -7.3; -8.6; -8.0 | 0.0032 sig |
| SECONDARY Change From Baseline in Percent Overall Work Impairment Due to MS on the WPAI-SHP Questionnaire at Months 3, 6, 9, and 12 by MS Disease Type: Responders |
-7.5; -6.8; -0.8; -6.5; -10.7; -10.4 | 0.0022 sig |
| SECONDARY Change From Baseline in Regular Activity Productivity Loss on the WPAI-SHP Questionnaire at Months 3, 6, 9, and 12 by MS Disease Type: Responders |
-14.9; -9.8; -7.1; -12.2; -16.2; -13.4 | <0.0001 sig |
| SECONDARY Change From Baseline in the PCS of the SF-36 at Months 3, 6, 9, and 12 by Whether Taking Additional MS Therapy |
3.1; -0.3; 3.9; -0.6; 4.0; 0.1 | <0.0001 sig |
| SECONDARY Change From Baseline in the MCS of the SF-36 at Months 3, 6, 9, and 12 by Whether Taking Additional MS Therapy |
2.8; -0.1; 3.9; 0.6; 4.0; -1.3 | <0.0001 sig |
| SECONDARY Change From Baseline in the MSIS-29 Physical Score at Months 3, 6, 9, and 12 by Whether Taking Additional MS Therapy |
-9.6; -1.5; -12.2; -3.2; -12.6; -1.4 | <0.0001 sig |
| SECONDARY Change From Baseline in MSIS-29 Psychological Score at Months 3, 6, 9, and 12 by Whether Taking Additional MS Therapy |
-7.1; -0.3; -9.7; -2.8; -9.0; 0.2 | <0.0001 sig |
| SECONDARY Change From Baseline in the Activities Limitation Scale of the PRIMUS at Months 3, 6, 9, and 12 by Whether Taking Additional MS Therapy |
-1.3; 0.8; -1.9; 0.8; -2.1; -0.5 | <0.0001 sig |
| SECONDARY Change From Baseline in the Current Health State of EQ-5D VAS at Months 3, 6, 9, and 12 by Whether Taking Additional MS Therapy |
6.0; -5.8; 8.6; -0.8; 7.6; -3.9 | <0.0001 sig |
| SECONDARY Change From Baseline in the Index Scores of EQ-5D at Months 3, 6, 9, and 12 by Whether Taking Additional MS Therapy |
0.04; -0.00; 0.06; 0.02; 0.07; 0.02 | 0.0001 sig |
| SECONDARY Change From Baseline in Percent Work Time Missed Due to MS on the WPAI-SHP Questionnaire at Months 3, 6, 9, and 12 by Whether Taking Additional MS Therapy |
-3.0; -11.9; -2.4; 14.9; -3.7; -8.3 | 0.1544 |
| SECONDARY Change From Baseline in Percent Impairment While Working Due to MS on the WPAI-SHP Questionnaire at Months 3, 6, 9, and 12 by Whether Taking Additional MS Therapy |
-3.1; -7.3; -6.4; -11.5; -6.1; -1.0 | 0.1392 |
| SECONDARY Change From Baseline in Percent Overall Work Impairment Due to MS on the WPAI-SHP Questionnaire at Months 3, 6, 9, and 12 by Whether Taking Additional MS Therapy |
-4.8; -9.9; -6.7; 1.2; -7.9; -6.6 | 0.0628 |
| SECONDARY Change From Baseline in Regular Activity Productivity Loss on the WPAI-SHP Questionnaire at Months 3, 6, 9, and 12 by Whether Taking Additional MS Therapy |
-10.6; -4.2; -13.3; -3.1; -12.7; -3.4 | <0.0001 sig |
| SECONDARY Number of Participants With Adverse Events (AEs) and Serious AEs (SAEs) |
522; 64; 586; 76; 3; 79 | — |
Summary
The primary objective of the study is to assess the effect of long-term treatment with prolonged-release fampridine (BIIB041) 10 mg twice daily on the physical component scale (PCS) of the Short Form 36 Health Status Questionnaire (SF-36) as reported by treatment responders. The secondary objectives of this study are to compare the change in the PCS of the SF-36 between treatment responders and non-responders, to evaluate change from baseline in additional quality of life measures among treatment responders as well as changes from baseline in treatment responders versus non-responders and to assess the safety and tolerability of prolonged-release fampridine 10 mg twice daily.
Eligibility Criteria
Key Inclusion Criteria
- Ability to understand the purpose and risks of the study and provide signed and dated informed consent and authorization to use protected health information in accordance with national and local subject privacy regulations.
- Must have a diagnosis of primary-progressive, secondary-progressive, progressive-remitting, or relapsing-remitting multiple sclerosis (MS) per revised McDonald Committee criteria ([Polman et al, 2011]) as defined by Lublin and Reingold [Lublin and Reingold 1996] of at least 3 months duration.
- Have a walking impairment as determined by the Investigator.
- Able to perform the Timed 25-foot Walk Test with or without a walking aid.
- Female subjects of childbearing potential must practice effective contraception during the study and be willing and able to continue contraception for 30 days after their last dose of study treatment.
- Able to understand and comply with the requirements of the protocol.
Key Exclusion Criteria
- Known allergy to pyridine-containing substances or to any of the inactive ingredients in the prolonged-release fampridine tablet.
- Any history of seizure, epilepsy, or other convulsive disorder, with the exception of febrile seizures in childhood.
- An estimated creatinine clearance of <80 mL/minute.
- Subject needs to take medicinal products that are inhibitors of organic cation transporter 2 (OCT2 [e.g., cimetidine]).
- Female subjects who are currently pregnant or who are considering becoming pregnant while participating in the study.
- Female subjects who are currently breastfeeding.
- Previous exposure to fampridine.
NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.
Data sourced from ClinicalTrials.gov (NCT01480076). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.