Multiple Sclerosis
Conditions
Brief summary
This study aims to assess the real-world treatment patterns, data from patient reported outcomes, the effectiveness profile of tolebrutinib, and health care utilization data in routine practice. The decision to initiate tolebrutinib treatment is made by the treating neurologist as part of standard of care. Consideration and option of participation in the study will only be done once the treatment decision has been made, independent of study participation. Only upon signing the informed consent form (ICF) will the patient be enrolled in the study. The patient's participation in the study will not influence the MS treatment. Total study duration of study is 5 years per country comprising an approximately 24-months of enrollment period followed by a 36-month individual observational period.
Interventions
DRUGTolebrutinib
This study will not administer any treatment, only observe the treatment as prescribed in real-world clinical practice.
Sponsors
Sanofi
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Willing to participate with the ability to consent and sign an Informed Consent Form (ICF). * Treatment decision according to approved local label. Retrospective inclusion of patients who had initiated tolebrutinib up to a maximum of 4 weeks prior to signing the ICF is allowed.
Exclusion criteria
* Participation in tolebrutinib long-term safety (LTS) study or any prior tolebrutinib study; experience with any other Bruton's Tyrosine Kinase (BTK) inhibitors. * Current or planned/pending participation in a clinical study. * Inability to comply with the study protocol The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Socio-demographic Characteristics | Baseline | Number and percentage of participants with socio-demographic characteristics including age, gender, occupational status will be collected. |
| Disease History: Most Recent Disability Status Scale (EDSS) score | Baseline | Disease history including EDSS score will be collected. The EDSS is a clinician-rated scale, ranging from 0 (normal neurological status) and 10 (death due to MS). Patients with EDSS score up to 4.5 are fully ambulant. Up to this point, the main determinant of EDSS are functional systems (FS), the ambulation status is the main determinant in the degree of disability after 4.5. |
| Disease History: EDSS Progression in Past 12 Months | Baseline | Disease history including EDSS progression in past 12 Months will be collected. The EDSS is a clinician-rated scale, ranging from 0 (normal neurological status) and 10 (death due to MS). Patients with EDSS score up to 4.5 are fully ambulant. Up to this point, the main determinant of EDSS are functional systems (FS), the ambulation status is the main determinant in the degree of disability after 4.5. |
| Disease History: Time Since Relapsing-Remitting Multiple Sclerosis (RRMS) Symptom Onset | Baseline | Disease history including time (in years) since RRMS symptom onset will be collected. |
| Disease History: Time Since Secondary Progressive Multiple Sclerosis (SPMS) Diagnosis | Baseline | Disease history including time (in years) since SPMS diagnosis will be collected. |
| Disease History: Time Since Most Recent Clinical Relapse | Baseline | Disease history including time (in years) since most recent clinical relapse will be collected. |
| Disease History: Number and Type of Previous MS Treatments | Baseline | Disease history including number and type of previous MS treatments will be collected. |
| Disease History: Number and Type of Prior and Current Disease Modifying Therapies (DMTs) | Baseline | Disease history including number and type prior and current DMTs will be collected. |
| Disease History: Number of Participants with Comorbidities | Baseline | Disease history including comorbidities will be collected. |
| Disease History: Number of Participants with Medical History | Baseline | Disease history including medical history will be collected. |
| Disease History: Number of Participants with Concomitant Medication | Baseline | Disease history including concomitant medication will be collected. |
| Magnetic Resonance Imaging Data | Baseline | MRI data will be collected. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change from Baseline in Individual Expanded Disability Status Scale (EDSS) Score | Baseline up to 36 months | The EDSS is a clinician-rated scale, ranging from 0 (normal neurological status) and 10 (death due to MS). Patients with EDSS score up to 4.5 are fully ambulant. Up to this point, the main determinant of EDSS are functional systems (FS), the ambulation status is the main determinant in the degree of disability after 4.5. |
| Change from Baseline in Timed 25-Foot Walk Test (T25FW) | Baseline up to 36 months | The mean walk time will be used for assessment of the patient's walking ability. An increase of more than 20 percent (%) from the baseline in the T25-FW test is considered meaningful worsening. |
| Change from Baseline in 9-Hole Peg Test (9-HPT) | Baseline up to 36 months | Time to complete the 9-HPT will be used to assess a participant's manual dexterity and fine motor skills. The mean time to test completion will serve for assessment of the patient's hand dexterity. An increase of more than 20 percent (%) from the baseline in the 9-HPT is considered meaningful worsening. |
| Change from Baseline in Symbol Digit Modalities Test (SDMT) | Baseline up to 36 months | The SDMT is used to assess visual scanning, tracking and motor speed, and has been designed to detect cognitive impairment. The SDMT involves a simple substitution task that normal children and adults can easily perform. Using a reference key, the examinee has 90 seconds to pair specific numbers with given geometric figures. |
| Change from Baseline in Multiple Sclerosis Impact Scale-29 (MSIS-29) | Baseline up to 36 months | The MSIS-29 is a scale that measures the physical (20 items) and psychological (9 items) impact of MS. Two summary scores (eg, the physical scale and the psychological scale) are calculated by summing up individual items in each scale and transforming the scores into a 0-100 scale. High scores indicate a higher impact of MS on the patient's physical and/or psychological health. |
| Change from Baseline in Short Form Health Survey (SF-36) The | Baseline up to 36 months | The SF-36 is a 36-item questionnaire designed to measure health-related quality of life. It assesses eight domains of health, including physical functioning, bodily pain, general health perceptions, vitality, social functioning, role limitations due to physical health problems, role limitations due to emotional problems, and mental health. |
| Change from Baseline in Multiple Sclerosis International Quality of Life (MuSIQoL) | Baseline up to 36 months | The MusiQoL questionnaire is a 31-item, MS-specific tool that measures quality of life from the patient's perspective. It covers nine domains-daily activities, psychological well-being, symptoms, relationships with friends and family, sentimental/sexual life, coping, and experiences of rejection-plus an overall index score. Items use a five-point scale, with higher scores reflecting better quality of life. |
| Change from Baseline in Multiple Sclerosis Walking Scale (MSWS-12) | Baseline up to 36 months | The 12-item MSWS is a validated patient-reported measure of the impact of MS on the individual's walking ability. A low MSWS-12 score indicates minimal impact of MS on walking ability, reflecting better mobility and fewer limitations in daily activities. Conversely, a high MSWS-12 score indicates a greater impact of MS on walking reflecting more significant mobility impairment and increased difficulty performing walking-related tasks. |
| Change from Baseline in Modified Fatigue Impact Scale (MFIS) | Baseline up to 36 months | The MFIS, 21-item questionnaire, is designed to evaluate how fatigue affects physical, cognitive, and psychosocial functioning. A lower MFIS score indicates that fatigue has minimal impact on a person's physical, cognitive, and psychosocial functioning. In contrast, a higher MFIS score reflects a greater impact of fatigue, suggesting more substantial interference with daily activities and overall quality of life. |
| Change from Baseline in Fatigue Scale for Motor and Cognitive Functions (FSMC) | Baseline up to 36 months | The FSMC is a 20-item questionnaire designed to assess both cognitive and motor fatigue, particularly in individuals with MS. Each item is rated on a 5-point Likert scale, ranging from "does not apply at all" to "applies completely," resulting in a total score between 20 (no fatigue) and 100 (severe fatigue). |
| Change from Baseline in Treatment Satisfaction Questionnaire for Medication (TSQM) | Baseline up to 36 months | The 14-item TSQM is a tool to assess patient satisfaction with medication. The tool includes four domains: Effectiveness (3 items), Side Effects (5 items), Convenience (3 items), and Global Satisfaction (3 items). It uses 5- or 7-point Likert-type scales and Yes/No responses. |
| Change from Baseline in Work Productivity and Activity Impairment - Multiple Sclerosis (WPAI-MS) | Baseline up to 36 months | The WPAI-MS questionnaire is a 6-item instrument to measure impairments over the past 7 days in both paid work and unpaid work due to one's health. Respondents are asked questions about work and activity impairment due to health problems. |
| Change from Baseline in Chronic Treatment Acceptance Questionnaire (ACCEPT) | Baseline up to 36 months | The ACCEPT is a patient questionnaire with 25 items which evaluates the patient's acceptance of long-term treatment in real-life. Low scores indicate poor acceptance, moderate scores reflect a neutral stance, and high scores indicate strong acceptance. |
| Change from Baseline in Patient Global Impression of Change (PGIC) | Baseline up to 36 months | The PGIC scale is administered at the end of the test-retest period and asks respondents to rate their symptom severity now/today compared to the beginning of the test-retest period. PGIC response options typically use a bi-directional response scale, eg, 5 to 7 options ranging from "Much better" to "Much worse" with "No change" at the midpoint. Lower scores indicate improvement, 4 indicates no change, and higher scores indicate worsening of the patient's condition. |
| Change from Baseline in Patient Global Impression of Severity (PGIS) | Baseline up to 36 months | The PGIS Scale asks respondents to rate their symptom or disease severity at a given time, and it is administered at multiple timepoints, including the beginning and the end of the test-retest period. Response options, in the form of a unidirectional verbal rating scale (VRS), often range from "None" to "Very severe". Lower scores indicate mild or no symptoms, moderate scores indicate noticeable impact, and higher scores indicate severe or very severe symptoms. |
| Change from Baseline in Health resource utilization questionnaire (HRUQ) | Baseline up to 36 months | The HRUQ is a PRO designed to capture patient-reported data on healthcare resource use, medication adherence, and work productivity over the past month, specifically in the context of MS management. Data collected from the HRUQ can be used to estimate healthcare utilization patterns, economic burden, and cost outcomes. |
| Number of Participants with at Least one AE, SAE, AEs by Maximum Severity, AEs Leading to treatment Discontinuation and AESI | Baseline up to 36 months | An adverse event (AE) was defined as any untoward medical occurrence in a subject who received study drug and did not necessarily had a causal relationship with the study treatment. Serious AEs (SAEs): Any untoward medical occurrence that resulted in any of the following outcomes: death, life-threatening, required initial or prolonged in-patient hospitalisation, persistent or significant disability/incapacity, congenital anomaly/birth defect, or considered as medically important event. An adverse event of special interest (AESI) is an adverse event (serious or non-serious) of scientific and medical concern specific to the product, for which ongoing monitoring and rapid communication to the provider is required. |
Countries
United Arab Emirates
Contacts
CONTACTTrial Transparency email recommended (Toll free for US & Canada)
Outcome results
None listed