Multiple Sclerosis
Conditions
Keywords
Multiple sclerosis, Mavenclad®, Cladribine, Health-related Quality of Life
Brief summary
The main purpose of the study was to assess the health-related quality of life (HRQoL) through the Multiple Sclerosis Quality of Life-54 Questionnaire (MSQoL-54) scale in highly-active relapsing multiple sclerosis (RMS) particpants treated with Mavenclad® for 2 years (24 months).
Interventions
DRUGMavenclad®
Participants with Relapsing Multiple Sclerosis (RMS) received Mavenclad® 3.5 milligram per kilogram (mg/kg) of body weight over 2 years, administered as 1 treatment course of 1.75 mg/kg per year. Each treatment course consisted of 2 treatment weeks, one at the beginning of the first month and one at the beginning of the second month of the respective year.
Sponsors
Merck KGaA, Darmstadt, Germany
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Highly active RMS as defined by: * One relapse in the previous year and at least 1 T1 Gadolinium (Gd)+ lesion or 9 or more T2 lesions, while on therapy with other disease modifying drugs (DMDs). * Two or more relapses in the previous year, whether on DMD treatment or not. * Expanded Disability Status Scale (EDSS) score less than equals to (\<=) 5.0. * Other protocol defined inclusion criteria could apply.
Exclusion criteria
* Positive hepatitis C or hepatitis B surface antigen test and/or core antibody test for immunoglobulin G (IgG) and/or immunoglobulin M (IgM). * Current or previous history of immune deficiency disorders including a positive human immunodeficiency virus (HIV) result. * Currently receiving immunosuppressive or myelosuppressive therapy with, for example, monoclonal antibodies, methotrexate, cyclophosphamide, cyclosporine or azathioprine, or chronic use of corticosteroids. * History of tuberculosis, presence of active tuberculosis, or latent tuberculosis * Presence of Progressive Multifocal Leukoencephalopathy (PML) in Magnetic Resonance Imaging (MRI). * Active malignancy. * Other protocol defined
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Change From Baseline in Multiple Sclerosis Quality of Life-54 Questionnaire (MSQoL-54) Physical Health Composite Summary and Mental Health Composite Summary Scores at Month 24 | Baseline, Month 24 | The MSQOL-54 was a multidimensional health-related QOL measure that combines both generic and MS-specific items into a single instrument. This 54-item instrument generates 12 sub-scales along with two summary scores, and two additional single-item measures. Sub-scales are: physical function, role limitations-physical, role limitations-emotional, pain, emotional well-being, energy, health perceptions, social function, cognitive function, health distress, overall quality of life, and sexual function. The two summary scores physical health and mental health are derived from a weighted combination of scale scores. Each composite summary score has a range from 0-100 where higher scores indicate better QOL. A positive change from baseline indicates improvement. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Treatment Global Satisfaction Determined by Treatment Satisfaction Questionnaire Medication Version 1.4 (TSQM v1.4) Scale at Month 6 | At Month 6 | TSQM version 1.4 was a global satisfaction scale used to assess the overall level of participant's satisfaction or dissatisfaction with their medications. It comprises of 14 items assessing the following 4 domains: effectiveness (questions: 1-3), side effects (questions: 4-8), convenience (questions: 9-11), global satisfaction (questions:12-14). Global Satisfaction- Question 12 scored as 1 (not at all confident) to 5 (extremely confident); question 13 scored as 1 (not at all certain) to 5 (extremely certain); and question 14 scored as 1 (extremely dissatisfied) to 7 (extremely satisfied). The scores of the domain were added together and an algorithm was used to create a score of 0 to 100. Higher scores indicated greater satisfaction. |
Countries
Austria, Belgium, Czechia, Denmark, Finland, France, Greece, Hungary, Italy, Lithuania, Netherlands, Norway, Poland, Portugal, Slovakia, Spain, Sweden, United Kingdom
Participant flow
Pre-assignment details
A total of 485 participants were enrolled in the study from different trial sites across Poland, Czechia, Slovakia, Hungary, Lithuania, Austria, Denmark, Finland, Sweden, Norway, Italy, Spain, Portugal, Greece, France, Netherlands, United Kingdom of Great Britain and Northern Ireland, and Belgium. Out of 485 participants, three participants were enrolled, but did not receive study medication.
Participants by arm
| Arm | Count |
|---|---|
| Mavenclad® Participants with Relapsing Multiple Sclerosis (RMS) received Mavenclad® 3.5 milligram per kilogram (mg/kg) of body weight over 2 years, administered as 1 treatment course of 1.75 mg/kg per year. Each treatment course consisted of 2 treatment weeks, one at the beginning of the first month and one at the beginning of the second month of the respective year. | 482 |
| Total | 482 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Overall Study | Adverse Event | 3 |
| Overall Study | Lost to Follow-up | 7 |
| Overall Study | Not classified | 3 |
| Overall Study | Progression Disease | 2 |
| Overall Study | Withdrawal by Subject | 15 |
Baseline characteristics
| Characteristic | Mavenclad® |
|---|---|
| Age, Continuous | 37.4 Years STANDARD_DEVIATION 10.39 |
| Ethnicity (NIH/OMB) Hispanic or Latino | 21 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 379 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 82 Participants |
| Race/Ethnicity, Customized Race Asian | 2 Participants |
| Race/Ethnicity, Customized Race Not collected at this site | 120 Participants |
| Race/Ethnicity, Customized Race White | 360 Participants |
| Sex: Female, Male Female | 338 Participants |
| Sex: Female, Male Male | 144 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 0 / 482 |
| other Total, other adverse events | 375 / 482 |
| serious Total, serious adverse events | 26 / 482 |
Outcome results
Primary
Change From Baseline in Multiple Sclerosis Quality of Life-54 Questionnaire (MSQoL-54) Physical Health Composite Summary and Mental Health Composite Summary Scores at Month 24
The MSQOL-54 was a multidimensional health-related QOL measure that combines both generic and MS-specific items into a single instrument. This 54-item instrument generates 12 sub-scales along with two summary scores, and two additional single-item measures. Sub-scales are: physical function, role limitations-physical, role limitations-emotional, pain, emotional well-being, energy, health perceptions, social function, cognitive function, health distress, overall quality of life, and sexual function. The two summary scores physical health and mental health are derived from a weighted combination of scale scores. Each composite summary score has a range from 0-100 where higher scores indicate better QOL. A positive change from baseline indicates improvement.
Time frame: Baseline, Month 24
Population: FAS consisted of all participants from the ITT set treated with at least one dose of study medication. Here, overall number of participants analyzed signifies number of participants who were evaluable for this outcome measure.
| Arm | Measure | Group | Value (LEAST_SQUARES_MEAN) | Dispersion |
|---|---|---|---|---|
| Mavenclad® | Change From Baseline in Multiple Sclerosis Quality of Life-54 Questionnaire (MSQoL-54) Physical Health Composite Summary and Mental Health Composite Summary Scores at Month 24 | Physical health composite summary | 4.86 Score on a Scale | Standard Error 0.769 |
| Mavenclad® | Change From Baseline in Multiple Sclerosis Quality of Life-54 Questionnaire (MSQoL-54) Physical Health Composite Summary and Mental Health Composite Summary Scores at Month 24 | Mental health composite summary | 4.80 Score on a Scale | Standard Error 0.845 |
Secondary
Treatment Global Satisfaction Determined by Treatment Satisfaction Questionnaire Medication Version 1.4 (TSQM v1.4) Scale at Month 6
TSQM version 1.4 was a global satisfaction scale used to assess the overall level of participant's satisfaction or dissatisfaction with their medications. It comprises of 14 items assessing the following 4 domains: effectiveness (questions: 1-3), side effects (questions: 4-8), convenience (questions: 9-11), global satisfaction (questions:12-14). Global Satisfaction- Question 12 scored as 1 (not at all confident) to 5 (extremely confident); question 13 scored as 1 (not at all certain) to 5 (extremely certain); and question 14 scored as 1 (extremely dissatisfied) to 7 (extremely satisfied). The scores of the domain were added together and an algorithm was used to create a score of 0 to 100. Higher scores indicated greater satisfaction.
Time frame: At Month 6
Population: FAS consisted of all participants from the ITT set treated with at least one dose of study medication. Here, overall number of participants analyzed signifies number of participants who were evaluable for this outcome measure.
| Arm | Measure | Value (LEAST_SQUARES_MEAN) | Dispersion |
|---|---|---|---|
| Mavenclad® | Treatment Global Satisfaction Determined by Treatment Satisfaction Questionnaire Medication Version 1.4 (TSQM v1.4) Scale at Month 6 | 72.02 Score on a Scale | Standard Error 1.528 |