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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT01480076




Registration number
NCT01480076
Ethics application status
Date submitted
23/11/2011
Date registered
28/11/2011
Date last updated
21/03/2017

Titles & IDs
Public title
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
Scientific title
An Open-Label, Multicenter, Multinational Study to Assess the Effect of Long-Term Prolonged-Release Fampridine (BIIB041) 10 mg Twice Daily on Quality of Life as Reported by Subjects With Multiple Sclerosis
Secondary ID [1] 0 0
218MS403
Universal Trial Number (UTN)
Trial acronym
ENABLE
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Multiple Sclerosis 0 0
Condition category
Condition code

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Fampridine

Experimental: (BIIB041) Fampridine - All participants take 10 mg fampridine twice daily for the first 4 weeks. If deemed a treatment responder, a participant continues 10 mg fampridine twice daily for 44 weeks. Treatment non-responders can continue without treatment by completing quality of life questionnaires.


Treatment: Drugs: Fampridine
Supplied as a 10 mg twice daily tablet and taken twice daily. Doses must be spaced at least 12 hours apart.
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
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
Timepoint [1] 0 0
Baseline, Months 3, 6, 9, 12
Secondary outcome [1] 0 0
Change From Baseline in the PCS of the SF-36 at Months 3, 6, 9, and 12: Responders Versus Non-responders
Timepoint [1] 0 0
Baseline, Months 3, 6, 9, 12
Secondary outcome [2] 0 0
Change From Baseline in the MCS of the SF-36 At Months 3, 6, 9, and 12
Timepoint [2] 0 0
Baseline, Months 3, 6, 9, 12
Secondary outcome [3] 0 0
Change From Baseline in the Multiple Sclerosis Impact Scale (MSIS-29) Physical Score at Months 3, 6, 9, and 12
Timepoint [3] 0 0
Baseline, Months 3, 6, 9, 12
Secondary outcome [4] 0 0
Change From Baseline in MSIS-29 Psychological Score at Months 3, 6, 9, and 12
Timepoint [4] 0 0
Baseline, Months 3, 6, 9, 12
Secondary outcome [5] 0 0
Change From Baseline in the Activities Limitation Scale of the Patient-Reported Indices for Multiple Sclerosis (PRIMUS) at Months 3, 6, 9, and 12
Timepoint [5] 0 0
Baseline, Months 3, 6, 9, 12
Secondary outcome [6] 0 0
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
Timepoint [6] 0 0
Baseline, Months 3, 6, 9, 12
Secondary outcome [7] 0 0
Change From Baseline in the Index Scores of EQ-5D at Months 3, 6, 9, and 12
Timepoint [7] 0 0
Baseline, Months 3, 6, 9, 12
Secondary outcome [8] 0 0
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
Timepoint [8] 0 0
Baseline, Months 3, 6, 9, 12
Secondary outcome [9] 0 0
Change From Baseline in Percent Impairment While Working Due to MS, by the WPAI-SHP Questionnaire at Months 3, 6, 9, and 12
Timepoint [9] 0 0
Baseline, Months 3, 6, 9, 12
Secondary outcome [10] 0 0
Change From Baseline in Percent Overall Work Impairment Due to MS, by the WPAI-SHP Questionnaire at Months 3, 6, 9, and 12
Timepoint [10] 0 0
Baseline, Months 3, 6, 9, 12
Secondary outcome [11] 0 0
Change From Baseline in Regular Activity Productivity Loss, by the WPAI-SHP Questionnaire at Months 3, 6, 9, and 12
Timepoint [11] 0 0
Baseline, Months 3, 6, 9, 12
Secondary outcome [12] 0 0
Change From Baseline in the PCS of the SF-36 at Months 3, 6, 9, and 12 by MS Disease Type: Responders
Timepoint [12] 0 0
Baseline, Months 3, 6, 9, and 12
Secondary outcome [13] 0 0
Change From Baseline in the MCS of the SF-36 at Months 3, 6, 9, and 12 by MS Disease Type: Responders
Timepoint [13] 0 0
Baseline, Months 3, 6, 9, and 12
Secondary outcome [14] 0 0
Change From Baseline in the MSIS-29 Physical Score at Months 3, 6, 9, and 12 by MS Disease Type: Responders
Timepoint [14] 0 0
Baseline, Months 3, 6, 9, and 12
Secondary outcome [15] 0 0
Change From Baseline in the MSIS-29 Psychological Score at Months 3, 6, 9, and 12 by MS Disease Type: Responders
Timepoint [15] 0 0
Baseline, Months 3, 6, 9, and 12
Secondary outcome [16] 0 0
Change From Baseline in the Activity Limitation Scale (ALS) of PRIMUS Score at Months 3, 6, 9, and 12 by MS Disease Type: Responders
Timepoint [16] 0 0
Baseline, Months 3, 6, 9, and 12
Secondary outcome [17] 0 0
Change From Baseline in Current Health State of the EQ-5D VAS at Months 3, 6, 9, and 12 by MS Disease Type: Responders
Timepoint [17] 0 0
Baseline, Months 3, 6, 9, and 12
Secondary outcome [18] 0 0
Change From Baseline in EQ-5D Index Scores at Months 3, 6, 9, and 12 by MS Disease Type: Responders
Timepoint [18] 0 0
Baseline, Months 3, 6, 9, and 12
Secondary outcome [19] 0 0
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
Timepoint [19] 0 0
Baseline, Months 3, 6, 9, and 12
Secondary outcome [20] 0 0
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
Timepoint [20] 0 0
Baseline, Months 3, 6, 9, and 12
Secondary outcome [21] 0 0
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
Timepoint [21] 0 0
Baseline, Months 3, 6, 9, and 12
Secondary outcome [22] 0 0
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
Timepoint [22] 0 0
Baseline, Months 3, 6, 9, and 12
Secondary outcome [23] 0 0
Change From Baseline in the PCS of the SF-36 at Months 3, 6, 9, and 12 by Whether Taking Additional MS Therapy
Timepoint [23] 0 0
Baseline, Months 3, 6, 9, 12
Secondary outcome [24] 0 0
Change From Baseline in the MCS of the SF-36 at Months 3, 6, 9, and 12 by Whether Taking Additional MS Therapy
Timepoint [24] 0 0
Baseline, Months 3, 6, 9, 12
Secondary outcome [25] 0 0
Change From Baseline in the MSIS-29 Physical Score at Months 3, 6, 9, and 12 by Whether Taking Additional MS Therapy
Timepoint [25] 0 0
Baseline, Months 3, 6, 9, and 12
Secondary outcome [26] 0 0
Change From Baseline in MSIS-29 Psychological Score at Months 3, 6, 9, and 12 by Whether Taking Additional MS Therapy
Timepoint [26] 0 0
Months 3, 6, 9, and 12
Secondary outcome [27] 0 0
Change From Baseline in the Activities Limitation Scale of the PRIMUS at Months 3, 6, 9, and 12 by Whether Taking Additional MS Therapy
Timepoint [27] 0 0
Baseline, Months 3, 6, 9, 12
Secondary outcome [28] 0 0
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
Timepoint [28] 0 0
Baseline, Months 3, 6, 9, 12
Secondary outcome [29] 0 0
Change From Baseline in the Index Scores of EQ-5D at Months 3, 6, 9, and 12 by Whether Taking Additional MS Therapy
Timepoint [29] 0 0
Baseline, Months 3, 6, 9, 12
Secondary outcome [30] 0 0
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
Timepoint [30] 0 0
Baseline, Months 3, 6, 9, and 12
Secondary outcome [31] 0 0
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
Timepoint [31] 0 0
Baseline, Months 3, 6, 9, and 12
Secondary outcome [32] 0 0
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
Timepoint [32] 0 0
Baseline, Months 3, 6, 9, and 12
Secondary outcome [33] 0 0
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
Timepoint [33] 0 0
Baseline, Months 3, 6, 9, and 12
Secondary outcome [34] 0 0
Number of Participants With Adverse Events (AEs) and Serious AEs (SAEs)
Timepoint [34] 0 0
From signing of Informed Consent (SAEs) or from first dose of study treatment (AEs) through Week 50 or Early Termination (14 +/- 7 days after last dose)

Eligibility
Key inclusion criteria
Key

* 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
Minimum age
18 Years
Maximum age
75 Years
Sex
Both males and females
Can healthy volunteers participate?
No
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.

Study design
Purpose of the study
Treatment
Allocation to intervention
Not applicable
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 4
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
1/02/2012
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
1/08/2013
Sample size
Target
Accrual to date
Final
901
Recruitment in Australia
Recruitment state(s)
NSW,QLD,VIC
Recruitment hospital [1] 0 0
Research Site - Concord
Recruitment hospital [2] 0 0
Research Site - Kogarah
Recruitment hospital [3] 0 0
Research Site - Liverpool
Recruitment hospital [4] 0 0
Research Site - New Lambton Heights
Recruitment hospital [5] 0 0
Research Site - Auchenflower
Recruitment hospital [6] 0 0
Research Site - Box Hill
Recruitment hospital [7] 0 0
Research Site - Clayton
Recruitment hospital [8] 0 0
Research Site - Fitzroy
Recruitment hospital [9] 0 0
Research Site - Heidelberg
Recruitment postcode(s) [1] 0 0
- Concord
Recruitment postcode(s) [2] 0 0
- Kogarah
Recruitment postcode(s) [3] 0 0
- Liverpool
Recruitment postcode(s) [4] 0 0
- New Lambton Heights
Recruitment postcode(s) [5] 0 0
- Auchenflower
Recruitment postcode(s) [6] 0 0
- Box Hill
Recruitment postcode(s) [7] 0 0
- Clayton
Recruitment postcode(s) [8] 0 0
- Fitzroy
Recruitment postcode(s) [9] 0 0
- Heidelberg
Recruitment outside Australia
Country [1] 0 0
Belgium
State/province [1] 0 0
Brasschaat
Country [2] 0 0
Belgium
State/province [2] 0 0
Brussels
Country [3] 0 0
Belgium
State/province [3] 0 0
Diepenbeek
Country [4] 0 0
Belgium
State/province [4] 0 0
Fraiture-en-Condroz
Country [5] 0 0
Belgium
State/province [5] 0 0
Gent
Country [6] 0 0
Belgium
State/province [6] 0 0
Liege
Country [7] 0 0
Belgium
State/province [7] 0 0
Melsbroek
Country [8] 0 0
Belgium
State/province [8] 0 0
Overpelt
Country [9] 0 0
Belgium
State/province [9] 0 0
Sijsele-Damme
Country [10] 0 0
Belgium
State/province [10] 0 0
Wilrijk
Country [11] 0 0
Denmark
State/province [11] 0 0
Copenhagen
Country [12] 0 0
France
State/province [12] 0 0
Alpes-Maritimes
Country [13] 0 0
France
State/province [13] 0 0
Bas-Rhin
Country [14] 0 0
France
State/province [14] 0 0
Calvados
Country [15] 0 0
France
State/province [15] 0 0
Gironde 5
Country [16] 0 0
France
State/province [16] 0 0
Ille-et-Vilaine
Country [17] 0 0
France
State/province [17] 0 0
Loire-Atlantique 6
Country [18] 0 0
France
State/province [18] 0 0
Marne
Country [19] 0 0
France
State/province [19] 0 0
Rhone
Country [20] 0 0
France
State/province [20] 0 0
Seine-Saint-Denis 14
Country [21] 0 0
France
State/province [21] 0 0
Somme
Country [22] 0 0
France
State/province [22] 0 0
Paris
Country [23] 0 0
Germany
State/province [23] 0 0
Bad Wuerttemberg
Country [24] 0 0
Germany
State/province [24] 0 0
Hessen
Country [25] 0 0
Germany
State/province [25] 0 0
Niedersachsen
Country [26] 0 0
Germany
State/province [26] 0 0
Nordrhein-Westfalen
Country [27] 0 0
Germany
State/province [27] 0 0
Berlin
Country [28] 0 0
Germany
State/province [28] 0 0
Erbach
Country [29] 0 0
Germany
State/province [29] 0 0
Hamburg
Country [30] 0 0
Germany
State/province [30] 0 0
Jena
Country [31] 0 0
Germany
State/province [31] 0 0
Osnabrueck
Country [32] 0 0
Germany
State/province [32] 0 0
Schwendi
Country [33] 0 0
Italy
State/province [33] 0 0
Bari
Country [34] 0 0
Italy
State/province [34] 0 0
Firenze
Country [35] 0 0
Italy
State/province [35] 0 0
Milano
Country [36] 0 0
Italy
State/province [36] 0 0
Padova
Country [37] 0 0
Italy
State/province [37] 0 0
Roma
Country [38] 0 0
Netherlands
State/province [38] 0 0
Eindhoven
Country [39] 0 0
Netherlands
State/province [39] 0 0
Hoorn
Country [40] 0 0
Netherlands
State/province [40] 0 0
Nijmegen
Country [41] 0 0
Netherlands
State/province [41] 0 0
Tilburg
Country [42] 0 0
Portugal
State/province [42] 0 0
Amadora
Country [43] 0 0
Portugal
State/province [43] 0 0
Coimbra
Country [44] 0 0
Portugal
State/province [44] 0 0
Lisboa
Country [45] 0 0
Portugal
State/province [45] 0 0
Porto
Country [46] 0 0
United Kingdom
State/province [46] 0 0
Greater Manchester
Country [47] 0 0
United Kingdom
State/province [47] 0 0
Northamptonshire
Country [48] 0 0
United Kingdom
State/province [48] 0 0
Stirlingshire
Country [49] 0 0
United Kingdom
State/province [49] 0 0
Liverpool
Country [50] 0 0
United Kingdom
State/province [50] 0 0
London

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Biogen
Address
Country

Ethics approval
Ethics application status

Summary
Brief 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.
Trial website
https://clinicaltrials.gov/study/NCT01480076
Trial related presentations / publications
Macdonell R, Nagels G, Laplaud DA, Pozzilli C, de Jong B, Martins da Silva A, Nicholas R, Lechner-Scott J, Gaebler JA, Agarwal S, Wang P, Yeh M, Hovenden M, Soelberg Sorensen P. Improved patient-reported health impact of multiple sclerosis: The ENABLE study of PR-fampridine. Mult Scler. 2016 Jun;22(7):944-54. doi: 10.1177/1352458515606809. Epub 2015 Oct 7.
Public notes

Contacts
Principal investigator
Name 0 0
Medical Director
Address 0 0
Biogen
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT01480076