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onrelapsing secondary progressive multiple sclerosis (NRSPMS) study of Bruton tyrosine kinase (BTK) inhibitor tolebrutinib SAR442168 (HERCULES)

Phase 1
Conditions
on-relapsing Secondary Progressive Multiple Sclerosis
MedDRA version: 21.1Level: PTClassification code 10063400Term: Secondary progressive multiple sclerosisSystem Organ Class: 10029205 - Nervous system disorders
Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Registration Number
EUCTR2020-000647-30-NL
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
1700
Inclusion Criteria

- 18 to 60 years of age inclusive
- Diagnosis of nonrelapsing secondary progressive multiple sclerosis according to the 2017 McDonald criteria
- Expanded disability status scale (EDSS) between 3.0 to 6.5 points inclusive, at screening
- The participant must have documented evidence of disability progression observed during the 12 months before screening
- Absence of clinical relapses for at least 24 months
- Contraceptive use by men or women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies
- Participant is eligible to participate if she is not pregnant or breastfeeding, and at least one of the following conditions applies:
- Is not a woman of child bearing potential (WOCBP)
OR
- Is a WOCBP and agrees to use an acceptable contraceptive method
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 1700
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

- The participant has conditions that would adversely affect study participation such as short life expectancy.
- History of organ transplant.
- Evidence of infection with human immuodeficiency virus (HIV), transplantation, progressive multifocal leukoencephalopathy (PML), active hepatitis B or C, active or latent tuberculosis or other active infections that would adversely affect study participation.
- Persistent chronic or active or recurring system infection, that may adversely affect participation or IMP administration in this study, as judged by the Investigator.
- 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
- A bleeding disorder or known platelet dysfunction at any time prior to the screening visit.
- A platelet count <150 000/µL at the screening visit.
-A history of significant bleeding event within 6 months prior to screening, according to the Investigator’s judgment such as, but not limited to cerebral or gastrointestinal bleeding.
- 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 potent and moderate inducers of cytochrome P450 3A (CYP3A) or potent inhibitors of CYP2C8 hepatic enzymes
- Receiving anticoagulant or antiplatelet therapy (such as aspirin >81mg/day, 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
NameTimeMethod
Main Objective: To determine the efficacy of SAR442168 compared to placebo in delaying disability progression in<br>NRSPMS;Secondary Objective: - To evaluate efficacy of SAR442168 compared to placebo on clinical endpoints, magnetic resonsnce<br>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 NRSPMS and its relationship to efficacy and safety<br>- To evaluate pharmacodynamics (PD) of SAR442168;Primary end point(s): 6-month confirmed disability progression (CDP) ; Time to onset of 6 months CDP defined as follows:<br>-Increase of =1.0 point from the baseline expanded disability status scale (EDSS) score when the baseline score is =5.0, or<br>-Increase of =0.5 point when the baseline EDSS score is >5.0;Timepoint(s) of evaluation of this end point: Up to approximately 48 months
Secondary Outcome Measures
NameTimeMethod
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