Study to Evaluate Efficacy, Safety, and Tolerability of IMU-838 in Patients With Progressive Multiple Sclerosis

Study Overview
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Brief Summary
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Multicenter, Randomized, Double-blind, Placebo-controlled Study to Evaluate Efficacy, Safety, and Tolerability of IMU-838 in Patients with Progressive Multiple Sclerosis - CALLIPER

Detailed Description
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This study will be a multicenter, randomized, double-blind, placebo-controlled study with a blinded Main Treatment Period (MT) and an Open Label Period (OLE) to evaluate the efficacy, safety, and tolerability of IMU838 in adult patients with PMS. The study will consist of the following periods: Screening Period: Approximately 28 days Main Treatment Period: Up to 120 weeks (approximately 2 years) Open Label Extension Period: Up to approximately 8 years

Official Title
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Multicenter, Randomized, Double-blind, Placebo-controlled Study to Evaluate Efficacy, Safety, and Tolerability of IMU-838 in Patients With Progressive Multiple Sclerosis

Conditions
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Multiple Sclerosis

Intervention / Treatment
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* Drug: IMU-838
* Drug: Placebo matching IMU-838


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Adult patients, age 18 to 65 years (inclusive). EDSS score at screening between 3.0 to 6.5 (both inclusive) No evidence of relapse in the last 24 months before randomization, AND Patients diagnosed according to 2017 revised McDonald Criteria 1 and the 2013 revised classification of disease courses 2 as either SPMS inpatients showing evidence of Gd+MRI lesions (active SPMS) or without Gd+MRI lesions (non-active SPMS) in the last 12 months, OR PPMS Willingness and ability to comply with the protocol. Written informed consent given by the patient before the beginning of any study-related procedure. Documented evidence of disability progression not temporarily related to a relapse in the last 24 months before randomization, adjudicated by a central independent reviewer Exclusion Criteria: Any disease other than MS that may better explain the signs and symptoms, including a history of complete transverse myelitis. Clinical signs or presence of laboratory findings suggestive for neuromyelitis optica (NMO) spectrum disorders or myelin oligodendrocyte glycoprotein (MOG)-associated encephalomyelitis (i.e.,presence of anti-NMO [aquaporin-4] antibodies or anti-MOG antibodies). Previous or current use of MS treatments lifelong, or within a pre-specified time period. Use of any investigational product within 8 weeks or 5 the respective PK half- life before the date of informed consent, whichever is longer, and throughout the study.For some investigational products, prolonged biological effects beyond 8 weeks should be considered. Positive test for severe acute respiratory syndrome coronavirus 2 (SARS- CoV-2) within14 days before randomization. In case of known SARS-CoV-2 infection, patients should be randomized no earlier than 14 days after 2 consecutive negative tests confirming virus negative status.The screening period can be extended for these patients to accommodate the required virus negativity. Positive IFN-gamma release assay (IGRA) for Mycobacterium tuberculosis at SV1. Positive hepatitis B virus (HBV) surface antigen, hepatitis B core antibody, positive hepatitis C virus (HCV) antibody, and/or HIV-antigen-antibody test at SV1.

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 65 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: IMU-838<br>IMU-838 as tablet; Administration: Oral - daily | Drug: IMU-838<br>* IMU-838 tablets<br>* Other names: Vidofludimus calcium;|
| Placebo Comparator: Placebo<br>Matching placebo as tablet; Administration: Oral - daily | Drug: Placebo matching IMU-838<br>* Placebo matching IMU-838 tablets<br>* Other names: Placebo Arm;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Efficacy of IMU-838 versus placebo | Annualized rate of percent brain volume change (PBVC) during MT period | 24 weeks | 
| Efficacy IMU-838 versus placebo | Time to 24-week confirmed disability progression based on expanded disability status scale (EDSS) during the MT period | 24 weeks | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Safety IMU-838 versus placebo | Adverse events (AEs) and serious AEs (SAEs) | 24 weeks | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Progressive Multiple Sclerosis