PPMS Study of Bruton's tyrosine kinase (BTK) inhibitor SAR442168 (PERSEUS)
- Conditions
- Primary Progressive Multiple SclerosisMedDRA version: 21.1Level: PTClassification code 10063401Term: Primary progressive multiple sclerosisSystem Organ Class: 10029205 - Nervous system disordersTherapeutic area: Diseases [C] - Nervous System Diseases [C10]
- Registration Number
- EUCTR2020-000645-14-HR
- Lead Sponsor
- Genzyme Corporation
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Authorised-recruitment may be ongoing or finished
- Sex
- All
- Target Recruitment
- 1320
- Diagnosis of PPMS according to the 2017 McDonald criteria
- Expanded disability status scale (EDSS) score between 2.0 to 6.5 points, at screening inclusive
- Disease duration from the onset of MS symptoms of <15 years if screening EDSS score of >5.0 OR <10 years if screening EDSS score of =5.0.
- Positive cerebrospinal fluid oligoclonal bands and/or elevated Immunoglobulin G (IgG) index either during screening or documented previous history.
- Contraceptive use consistent with local regulations for individuals participating in clinical studies
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 1320
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range
- Participant has conditions that would adversely affect study participation such as short life expectancy.
- History of organ transplant.
- Evidence of infection with human immunodeficiency virus (HIV), progressive multifocal leukoencephalopathy (PML), active hepatitis B or C, active or latent tuberculosis or other active infection that would adversely affect study participation.
- History of malignancy within 5 years prior to screening.
- History of alchohol or drug abuse within 1 year prior to Screening.
- Hospitalized for psychiatric disease within 2 years prior to Screening.
- Clinically significant laboratory abnormalities (including evidence of liver injury) or electrocardiogram abnormalities at Screening.
- Bleeding disorder, known platelet dysfunction or platelet count <150 000/µL at Screening or history of significant bleeding event within 6 months prior to Screening.
- Lymphocyte count below the lower limit of normal at Screening.
- Recent live (attenuated) vaccine within 2 months before the first treatment visit.
- Recent major surgery (within 4 weeks of Screening) or planned major surgery during the study.
- The participant has received medications/treatments for MS within a specified time frame.
- Receiving strong inducers or inhibitors of cytochrome P450 3A (CYP3A) or CYP2C8 hepatic enzymes.
- Receiving anticoagulant or antiplatelet therapy (such as aspirin, clopidogrel, warfarin).
- Contraindications to magnetic resonance imaging (MRI).
NOTE: Other Inclusion/Exclusion criteria may apply
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: To determine the efficacy of SAR442168 compared to placebo in delaying disability progression in Primary Progressive Multiple Sclerosis (PPMS);Secondary Objective: To evaluate efficacy of SAR442168 compared to placebo on clinical endpoints, magnetic resonance imaging (MRI) lesions, cognitive performance, physical function, and quality of life<br>To evaluate safety and tolerability of SAR442168<br>To evaluate population pharmacokinetics (PK) of SAR442168 in PPMS and its relationship to efficacy and safety<br>To evaluate pharmacodynamics of SAR442168 <br>;Primary end point(s): 6 month Confirmed Disability Progression (CDP) ; Time to onset of 6 month CDP defined as follows:<br>Increase of =1.0 point from the baseline expanded disability status scale (EDSS) score when the baseline score is =5.5, or Increase of =0.5 points when the baseline EDSS score is >5.5<br>;Timepoint(s) of evaluation of this end point: Up to approximately 48 months
- Secondary Outcome Measures
Name Time Method