A study to evaluate specific unmet needs in the current clinical practice of multiple sclerosis
| ISRCTN | ISRCTN40939838 |
|---|---|
| DOI | https://doi.org/10.1186/ISRCTN40939838 |
| Secondary identifying numbers | ML39348 |
- Submission date
- 09/12/2021
- Registration date
- 17/12/2021
- Last edited
- 16/12/2021
- Recruitment status
- No longer recruiting
- Overall study status
- Completed
- Condition category
- Nervous System Diseases
Plain English summary of protocol
Background and study aims
Multiple sclerosis (MS) is an incurable condition where the immune system attacks myelin, the protective layer around nerve fibers. This makes it difficult for the brain to send signals to the rest of the body. Common symptoms include tiredness, vision problems, and problems with walking or balance. A majority of people experience a form of MS called relapsing-remitting MS (RRMS) which is characterised by a pattern of clearly defined relapses that are divided by symptom-free periods. If left untreated RRMS will transition through an intermediate state of relapsing secondary progressive MS (rSPMS) into secondary progressive MS (SPMS), which is characterised by increased disability in addition to relapses. Several disease-modifying treatments (DMTs) have been shown to reduce the number of relapses, slow the progression of the disease and thus delay disability. However, in spite of this there are still several critical questions remaining: What constitutes a truly effective medication in MS management? What are the actual unmet needs of MS medications? The aims of this study are:
1. To describe the different patient sub-groups (profiles) seen in clinical practice and to identify the unmet medical needs in the current care of relapsing multiple sclerosis (RMS) (RRMS and rSPMS). For this purpose, five specific subgroups of RMS (RRMS and rSPMS) patients with significant unmet needs will be prospectively monitored under real-life conditions to assess disease- and patient-related outcomes
2. To study MS activity using various assessment parameters such as magnetic resonance imaging (MRI), clinical assessments, patient-reported treatment satisfaction, disability, MS symptoms, adapted clinical global impression (CGI) scale as reported by the patient and the physician
3. To find out the nature, frequency, and severity of adverse events (AEs) and serious adverse events (SAEs)
4. To study the nature, frequency, and severity of adverse drug reactions (ADRs) and serious adverse drug reactions (SADRs)
Who can participate?
People over 18 years of age with RMS (RRMS and rSPMS)
What does the study involve?
Participants may be asked to be in the study for up to 12 months. This includes:
A screening period where participants will be assessed for eligibility to participate in the study and further into the following subgroups:
Subgroup 1 will include participants with MS who have been on DMT for the past 12 months.
Subgroup 2 will include participants with MS who have experienced significant side effects with DMTs.
Subgroup 3 will include participants with MS who have low treatment satisfaction.
Subgroup 4 will include participants with MS who have never received any treatment for the disease.
Subgroup 5 will include participants with MS who are without treatment currently but had been previously treated with a DMT.
During the observation period three visits are recorded by the study doctor in a standardised form either on paper or electronically. The first visit will be the baseline and the second and the third observation visits should take place around 6 and 12 months (±2 weeks) after the baseline visit.
During clinic visits, participants will be assessed for various parameters such as progression of the disease, treatment satisfaction, extent of disability, frequency of relapses, overall disease status, and so forth. Assessments will be made using patient questionnaires, lab tests, MRI scans, etc.
What are the possible benefits and risks of participating?
Participants will not receive any benefit from participating in this study, but the information that is learned from this study may help researchers and doctors to learn more about MS in general and other people who have a similar medical condition may benefit from the results of such research in the future. There are no risks from participating in the study.
Where is the study run from?
Roche Pharma AG (Germany)
When is the study starting and how long is it expected to run for?
November 2016 to September 2023
Who is funding the study?
Roche Pharma AG (Germany)
Who is the main contact?
grenzach.medical_information@roche.com
Contact information
Public
Emil-Barell-Str. 1
Grenzach-Wyhlen
79639
Germany
| Phone | +49 (0)7624142015 |
|---|---|
| grenzach.medical_information@roche.com |
Study information
| Study design | Multicenter prospective observational primary data collection, non-interventional study (NIS) |
|---|---|
| Primary study design | Observational |
| Secondary study design | Cohort study |
| Study setting(s) | Other |
| Study type | Other |
| Scientific title | Evaluation of specific unmet needs in the current clinical practice of multiple sclerosis: characterization of different profiles of relapsing multiple sclerosis patients defined by disease activity and patient-reported outcomes (Profile RMS) |
| Study objectives | The aim of this study is to characterize different participant profiles in clinical practice regarding the unmet medical needs in current care of relapsing multiple sclerosis (RMS) (relapsing-remitting multiple sclerosis (RRMS) and relapsing secondary progressive multiple sclerosis (rSPMS). |
| Ethics approval(s) | Approved 10/04/2017, Ethics committee at the TU Dresden (Fetscherstrasse 74, 01307 Dresden, Germany; +49 (0)351 458 2992; ethics committee@mailbox.tu-dresden.de), ref: EK 170042017 |
| Health condition(s) or problem(s) studied | Relapsing multiple sclerosis |
| Intervention | Cohort 1: Participants with multiple sclerosis (MS) disease activity on any disease-modifying therapy (DMT) in the past 12 months. Cohort 2: Participants with significant adverse drug reactions of DMTs or findings of theoretical safety concerns as assessed by the treating physician and showing no disease activity. Cohort 3: Participants with subjective low treatment satisfaction. Cohort 4: Treatment-naïve MS participants. Cohort 5: Participants without current treatment who had been previously treated with a DMT. During the observation period three visits are recorded by the study doctor in a standardised form either on paper or electronically. The first visit will be the baseline and the second and the third observation visits should take place around 6 and 12 months (±2 weeks) after the baseline visit. During clinic visits, participants will be assessed for various parameters such as progression of the disease, treatment satisfaction, extent of disability, frequency of relapses, overall disease status, and so forth. Assessments will be made using patient questionnaires, lab tests, MRI scans, etc. |
| Intervention type | Other |
| Primary outcome measure | Percentage of participants with treatment failure as determined by the occurrence of first confirmed relapse or Expanded Disability Status Scale (EDSS) progression (increase by ≥1.5 EDSS Points if the initial EDSS is 0, increase by ≥1.0 EDSS Point if the initial EDSS is ≥0.5 ≤5.5, increase by ≥0.5 EDSS points if the initial EDSS is > 5.5) or magnetic resonance imaging (MRI) activity (new T2 or Gadolinium (Gd) enhancing lesion in spinal or cerebral MRI) or treatment change, whichever occurs first by 48 weeks |
| Secondary outcome measures | 1. Time to first confirmed relapse during the study (up to 12 months). A relapse is defined as an episode of neurological symptoms that happens at least 30 days after any previous episode began, lasts at least 24 hours and is not attributable to another cause and occurs in the absence of an infection or fever. Changes in neurological examination or an increase in the EDSS Score after confirmation by the treating physician is defined as a confirmed relapse. 2. Percentage of relapse-free participants at the end of study (month 12) where relapse is defined as an episode of neurological symptoms that happens at least 30 days after any previous episode began, lasts at least 24 hours and is not attributable to another cause and occurs in the absence of an infection or fever., and is confirmed by the treating physician through e.g. changes in neurological examination or an increase in the EDSS score the relapse is called a confirmed relapse 3. Change from Baseline in measure of disability and disease progression using the EDSS score around months 6 and 12 4. Time to EDSS progression during the study (up to 12 months). EDSS progression is defined as an increase by ≥1.5 EDSS points if the initial EDSS is 0, increase by ≥1.0 EDSS points if the initial EDSS is ≥ 0.5 ≤ 5.5, increase by ≥0.5 EDSS points if the initial EDSS is >5.5 5. Percentage of participants with treatment change and the type of treatments from baseline up to month 12. Treatment change is defined as any change of the actual treatment status at baseline i.e., due to ongoing disease activity, actual or theoretical safety concerns, low treatment satisfaction, or low quality of life (Qol) 6. Change from Baseline in global impression on the disease course measured using the adapted Clinical Global Impression (CGI) as reported by participant and physician around months 6 and 12 7. Change from baseline in quality of life and participant-reported treatment satisfaction as measured by Health-Related Quality of Life (HR-Qol) at months 6 and 12 8. Change from baseline in quality of life and participant-reported treatment satisfaction as measured by Multiple Sclerosis Impact Scale-29 Version 2 (MSIS-29 v2) at months 6 and 12. The MSIS-29 is a 29-item participant-reported measure of the physical and psychological impacts of MS 9. Change from baseline in quality of life and participant-reported treatment satisfaction as measured by TSQM at months 6 and 12. The TSQM questionnaire covers three dimensions (effectiveness, side effects, and convenience) obtaining four different scores: effectiveness score, side effects score, convenience score, and global satisfaction score 10. Change from baseline in MS signs and symptoms (including mobility, cognition, fatigue) as measured by 2 minutes (min) walking test (2 MWT) at months 6 and 12. 2 MWT allows evaluation of the endurance of the participant assessing the walking distance over 2 minutes 11. Change from baseline in MS signs and symptoms (including mobility, cognition, fatigue) as measured by Symbol Digit Modalities Test (SDMT) at months 6 and 12. SDMT is a brief screening tool for cognitive dysfunction and processing speed in MS 12. Change from baseline in MS signs and symptoms (including mobility, cognition, fatigue) as measured by Fatigue Scale for Motor and Cognitive Functions (FSMC) at months 6 and 12. Fatigue is rated using this self-administered FSMC questionnaire that evaluates both motor fatigue and cognitive fatigue in MS 13. Change from baseline in MS signs and symptoms (including mobility, cognition, fatigue) as measured by the Hospital Anxiety and Depression Scale (HADS) at months 6 and 12. HADS is a common depression and anxiety measurement instrument used in MS. The minimum score is 0 and the maximum score is 21 14. Percentage of participants with adverse events (AEs) and serious adverse events (SAEs) collected prospectively from study start-up to month 12 for participants of cohorts 1 to 3 and in participants of cohorts 4 and 5 in case DMT is started during the study 15. Percentage of participants with adverse events of special interests (AESIs) and pregnancies collected prospectively from study start-up to month 12 for participants of cohorts 1 to 3 and in participants of cohorts 4 and 5 in case DMT is started during the study 16. Percentage of participants with severity of AEs, SAEs and AESIs as per physician’s assessment using National Cancer Institute-Common Terminology Criteria for Adverse Events Version 4.0 (NCI CTCAE v4.0) collected prospectively from study start up to month 12 for participants of cohorts 1 to 3 and in participants of cohorts 4 and 5 in case DMT is started during the study 17. Percentage of participants with adverse drug reactions (ADRs) and serious adverse drug reaction (SADRs) of DMTs extracted from participant charts (secondary data use) retrospectively for 12 months before start of the study for participants in cohort 2 only (recorded at baseline) 18. Percentage of participants with and severity of ADRs and SADRs of DMTs as per physician’s assessment using NCI CTCAE v4.0 extracted from participant charts (secondary data use) retrospectively for 12 months before start of the study for participants in cohort 2 only (recorded at baseline) |
| Overall study start date | 07/11/2016 |
| Completion date | 30/09/2023 |
Eligibility
| Participant type(s) | Patient |
|---|---|
| Age group | Adult |
| Sex | Both |
| Target number of participants | 1250 |
| Total final enrolment | 1250 |
| Key inclusion criteria | 1. RRMS or rSPMS, diagnosed by revised McDonald 2010 criteria 2. Treatment naïve participants or participants without current treatment or participants being treated with an approved DMT according to the respective summary of product characteristics (SPC) |
| Key exclusion criteria | 1. Participation in an interventional study or in NIS Confidence (ML39632) 2. Participants not receiving treatment for MS with the studied DMTs according to the standard of care and in line with the current SPC 3. Off-label use of any medication |
| Date of first enrolment | 05/07/2018 |
| Date of final enrolment | 31/07/2021 |
Locations
Countries of recruitment
- Germany
Study participating centre
Dresden
74-01307
Germany
Sponsor information
Industry
Emil-Barell-Str. 1
Grenzach-Wyhlen
79639
Germany
| Phone | +49 (0)7624142015 |
|---|---|
| grenzach.medical_information@roche.com | |
| Website | https://www.roche.com/about_roche/roche_worldwide.htm |
Funders
Funder type
Industry
Government organisation / For-profit companies (industry)
- Alternative name(s)
- F. Hoffmann-La Roche Ltd, F. Hoffmann-La Roche & Co, F. Hoffmann-La Roche AG, Roche Holding AG, Roche Holding Ltd, Roche Holding, Roche Holding A.G., Roche Holding, Limited, F. Hoffmann-La Roche & Co.
- Location
- Switzerland
Results and Publications
| Intention to publish date | 30/09/2023 |
|---|---|
| Individual participant data (IPD) Intention to share | No |
| IPD sharing plan summary | Not expected to be made available |
| Publication and dissemination plan | Planned publication in a high-impact peer-reviewed journal |
| IPD sharing plan | The datasets generated during and/or analysed during the current study are not expected to be made available due to participant-level data not being a regulatory requirement. |
Editorial Notes
16/12/2021: Trial's existence confirmed by TU Dresden.
