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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT02776072




Registration number
NCT02776072
Ethics application status
Date submitted
16/05/2016
Date registered
18/05/2016
Date last updated
23/03/2017

Titles & IDs
Public title
Observational Study to Characterize Real-world Clinical Outcomes With Relapsing-remitting Multiple Sclerosis (RRMS)
Scientific title
A Multicenter, Global, Retrospective, Observational Study to Characterize Real-world Clinical Outcomes in Patients With Relapsing-remitting Multiple Sclerosis Treated With Disease-modifying Therapies (Tecfidera®, Copaxone®, Aubagio®, or Gilenya®)
Secondary ID [1] 0 0
109MS421
Universal Trial Number (UTN)
Trial acronym
EFFECT
Linked study record

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

Intervention/exposure
Study type
Observational
Patient registry
Target follow-up duration
Target follow-up type
Description of intervention(s) / exposure
dimethyl fumarate (DMF) - Participants who initiated DMF during the specified time period

glatiramer acetate (GA) - Participants who initiated GA during the specified time period

teriflunomide - Participants who initiated teriflunomide during the specified time period

fingolimod - Participants who initiated fingolimod during the specified time period
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Proportion of participants relapsed at 12 months in participants treated with DMF
Timepoint [1] 0 0
One day
Secondary outcome [1] 0 0
Proportion of participants relapsed at 12 months treated with index therapy in the overall population as well as the subgroup of MS participants who were naïve to DMTs and were diagnosed with MS within 3 years of starting the index therapy
Timepoint [1] 0 0
One day
Secondary outcome [2] 0 0
ARR at 12 months in participants treated with index therapy in the overall population as well as in the matched cohorts
Timepoint [2] 0 0
One day
Secondary outcome [3] 0 0
Proportion of relapsed participants with one or more MS-related hospitalizations during the 12 months following treatment initiation in participants treated with index therapy in the overall population and the matched cohorts
Timepoint [3] 0 0
One day
Secondary outcome [4] 0 0
Proportion of relapsed participants requiring treatment with intravenous corticosteroids during the 12 months following treatment initiation in participants treated with index therapy in the overall participant population and the matched cohorts
Timepoint [4] 0 0
One day

Eligibility
Key inclusion criteria
Key

* Diagnosis of RRMS per McDonald criteria
* Initiated treatment with DMF, GA, teriflunomide, or fingolimod (defined as index therapy) no earlier than January 2011 and no later than 12 months prior to the date of medical record abstraction (i.e., patient has at least 12 months of data available in the medical record following initiation of index treatment). Note: Patients who have initiated the index therapy but subsequently discontinued or switched to other therapies are allowed in this study as long as there is at least 12 months of follow-up following the initiation of the index therapy.
* Have sufficient available medical records for data abstraction to meet the objectives of the study, i.e., the patient was either under the medical care of the investigating site during the entire period of the index treatment or the patient's complete MS disease and treatment history is otherwise available at the investigating site

Key
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
* Diagnosis of a progressive form of MS (progressive relapsing, primary progressive, secondary progressive) at any time before or during the period for which data will be collected
* Have received disease-modifying therapies other than one platform therapy (IFN or GA) prior to initiation of index therapy. In patients for whom GA is index therapy, only one prior IFN therapy is allowed.
* Have received any formulations of DMF, Fumaderm®, or compounded fumarates (e.g., Psorinovo) at any time prior to initiation of treatment with Tecfidera®
* Concurrent enrollment in any interventional clinical trial of an investigational product during time evaluated for medical record abstraction

NOTE: Other protocol defined Inclusion/Exclusion criteria may apply

Study design
Purpose
Duration
Selection
Timing
Retrospective
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
1/05/2016
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
1/12/2016
Sample size
Target
Accrual to date
Final
2978
Recruitment in Australia
Recruitment state(s)
NSW,VIC
Recruitment hospital [1] 0 0
Research Site - New Lambton Heights
Recruitment hospital [2] 0 0
Research Site - Box Hill
Recruitment hospital [3] 0 0
Research Site - Heidelberg
Recruitment hospital [4] 0 0
Research Site - Melbourne
Recruitment postcode(s) [1] 0 0
2305 - New Lambton Heights
Recruitment postcode(s) [2] 0 0
3128 - Box Hill
Recruitment postcode(s) [3] 0 0
3084 - Heidelberg
Recruitment postcode(s) [4] 0 0
3050 - Melbourne
Recruitment outside Australia
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United States of America
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Alabama
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Catania
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Lausanne
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Lugano
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Cornwall
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Devon
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Essex
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Greater London
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Strathclyde
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Surrey

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 evaluate the real-world clinical effectiveness, as measured by the proportion of participants relapsed at 12 months, in participants treated with dimethyl fumarate (DMF).

Secondary objectives of the study are: To evaluate the real-world clinical effectiveness, as measured by the proportion of participants relapsed at 12 months, in participants treated with DMF, glatiramer acetate (GA), teriflunomide, or fingolimod both in the overall participant cohort and in a subset of participants who were naïve to disease-modifying therapy (DMT) and were diagnosed with multiple sclerosis (MS) within 3 years of starting the index therapy; To compare relapse activity, defined as annualized relapse rate (ARR), among participants treated with DMF, GA, teriflunomide, or fingolimod; To compare MS-related hospitalizations among participants treated with DMF, GA, teriflunomide, or fingolimod; To compare intravenous corticosteroid use among participants treated with DMF, GA, teriflunomide, or fingolimod.
Trial website
https://clinicaltrials.gov/study/NCT02776072
Trial related presentations / publications
Min J, Cohan S, Alvarez E, Sloane J, Phillips JT, van der Walt A, Koulinska I, Fang F, Miller C, Chan A. Real-World Characterization of Dimethyl Fumarate-Related Gastrointestinal Events in Multiple Sclerosis: Management Strategies to Improve Persistence on Treatment and Patient Outcomes. Neurol Ther. 2019 Jun;8(1):109-119. doi: 10.1007/s40120-019-0127-2. Epub 2019 Jan 31.
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/NCT02776072