Managed Access Program (MAP) for Patients Diagnosed With Secondary Progressive Multiple Sclerosis With Active Disease


Study First Submitted Date 2020-08-31
Study First Posted Date 2020-09-07
Last Update Posted Date 2022-09-14
Verification Month Year September 2022
Verification Date 2022-09-30
Last Update Posted Date 2022-09-14

Browse Interventions

Sequence: 96401519 Sequence: 96401520 Sequence: 96401521 Sequence: 96401522 Sequence: 96401523 Sequence: 96401524
Mesh Term Siponimod Mesh Term Sphingosine 1 Phosphate Receptor Modulators Mesh Term Molecular Mechanisms of Pharmacological Action Mesh Term Immunosuppressive Agents Mesh Term Immunologic Factors Mesh Term Physiological Effects of Drugs
Downcase Mesh Term siponimod Downcase Mesh Term sphingosine 1 phosphate receptor modulators Downcase Mesh Term molecular mechanisms of pharmacological action Downcase Mesh Term immunosuppressive agents Downcase Mesh Term immunologic factors Downcase Mesh Term physiological effects of drugs
Mesh Type mesh-list Mesh Type mesh-ancestor Mesh Type mesh-ancestor Mesh Type mesh-ancestor Mesh Type mesh-ancestor Mesh Type mesh-ancestor


Sequence: 52395755
Name Multiple Sclerosis
Downcase Name multiple sclerosis

Id Information

Sequence: 40318217
Id Source org_study_id
Id Value CBAF312A2001M


Sequence: 52705285
Intervention Type Drug
Name Siponimod
Description 1 or 2 mg film-coated tablets. The tablets have to be taken orally once daily and swallowed whole with water, with or without food.


Sequence: 80173576 Sequence: 80173577 Sequence: 80173578 Sequence: 80173579 Sequence: 80173580
Name SPMS with active disease Name SPMS Name Secondary Progressive Multiple Sclerosis Name siponimod Name Mayzent
Downcase Name spms with active disease Downcase Name spms Downcase Name secondary progressive multiple sclerosis Downcase Name siponimod Downcase Name mayzent

Browse Conditions

Sequence: 194339480 Sequence: 194339481 Sequence: 194339482 Sequence: 194339483 Sequence: 194339484 Sequence: 194339485 Sequence: 194339486 Sequence: 194339487 Sequence: 194339488 Sequence: 194339489 Sequence: 194339490 Sequence: 194339491
Mesh Term Multiple Sclerosis Mesh Term Multiple Sclerosis, Chronic Progressive Mesh Term Sclerosis Mesh Term Pathologic Processes Mesh Term Demyelinating Autoimmune Diseases, CNS Mesh Term Autoimmune Diseases of the Nervous System Mesh Term Nervous System Diseases Mesh Term Demyelinating Diseases Mesh Term Autoimmune Diseases Mesh Term Immune System Diseases Mesh Term Chronic Disease Mesh Term Disease Attributes
Downcase Mesh Term multiple sclerosis Downcase Mesh Term multiple sclerosis, chronic progressive Downcase Mesh Term sclerosis Downcase Mesh Term pathologic processes Downcase Mesh Term demyelinating autoimmune diseases, cns Downcase Mesh Term autoimmune diseases of the nervous system Downcase Mesh Term nervous system diseases Downcase Mesh Term demyelinating diseases Downcase Mesh Term autoimmune diseases Downcase Mesh Term immune system diseases Downcase Mesh Term chronic disease Downcase Mesh Term disease attributes
Mesh Type mesh-list Mesh Type mesh-list Mesh Type mesh-list Mesh Type mesh-ancestor Mesh Type mesh-ancestor Mesh Type mesh-ancestor Mesh Type mesh-ancestor Mesh Type mesh-ancestor Mesh Type mesh-ancestor Mesh Type mesh-ancestor Mesh Type mesh-ancestor Mesh Type mesh-ancestor


Sequence: 48528096
Agency Class INDUSTRY
Lead Or Collaborator lead
Name Novartis Pharmaceuticals

Overall Officials

Sequence: 29402725
Role Study Director
Name Novartis Pharmaceuticals
Affiliation Novartis Pharmaceuticals

Central Contacts

Sequence: 12066764 Sequence: 12066765
Contact Type primary Contact Type backup
Name Novartis Pharmaceuticals Name Novartis Pharmaceuticals
Phone +41613241111
Role Contact Role Contact


Sequence: 30894804
Gender All
Minimum Age 18 Years
Maximum Age N/A
Healthy Volunteers No
Criteria Inclusion Criteria: Patients eligible for inclusion in this Treatment Plan have to meet all of the following criteria: Adult patients who are able to provide written informed consent and have done so Note: proof of efficacy in the Phase III EXPAND study (Kappos et al 2018) has been obtained in patients who were aged 18 to 61 years (inclusive) at screening Diagnosis of SPMS with active disease. The SPMS diagnosis must follow an initial relapsing-remitting disease course of multiple sclerosis (MS) and align with the diagnostic criteria published (Lublin and Reingold 1996, Rovaris et al 2006, Lublin et al 2014) Patient is ambulatory, i.e., has an EDSS score of ≤6.5 Patient has no comparable or satisfactory alternative therapy available for treatment of SPMS Written patient informed consent must be obtained prior to start of treatment. If consent cannot be expressed in writing, it must be formally documented and witnessed, ideally via an independent trusted witness. Exclusion Criteria: Patients eligible for this Treatment Plan must not meet any of the following criteria: Disease exclusions Relapsing-remitting multiple sclerosis Primary progressive multiple sclerosis Secondary progressive multiple sclerosis without active disease Medical conditions precluding inclusion such as lab abnormalities or underlying diseases Patients with an active or stable but treated chronic disease of the immune system other than MS (e.g. rheumatoid arthritis, scleroderma, Sjogren’s syndrome, Crohn’s disease, ulcerative colitis, etc.) or with a known immunodeficiency syndrome (AIDS, hereditary immune deficiency, drug-induced immune deficiency) Severe active infections Patients affected by severe infections should only be included after full resolution of the condition Second-degree AV block Mobitz type II or higher, sick-sinus syndrome, sino-atrial heart block or significant QT prolongation (QTc ≥500 msec). History of cardiac arrest, recurrent syncope, symptomatic bradycardia, severe cerebrovascular disease, uncontrolled hypertension or severe untreated sleep apnea. Significant liver disease Ongoing macular edema (patients with a history of resolved macular edema are allowed to enter the program) Patients homozygous for CYP2C9*3 (CYP2C9*3*3 genotype) Patients without a health-care professional confirmed history of chickenpox or documentation of a full course of vaccination with varicella vaccine. They have to undergo antibody testing to varicella zoster virus (VZV) before initiating treatment with siponimod. A full course of vaccination for antibody-negative patients with varicella vaccine is required prior to commencing treatment with siponimod. Initiation of treatment with siponimod has be postponed for one month after completion of the vaccination course to allow full effect of vaccination to occur. History of hypersensitivity to any drugs or metabolites of similar chemical classes as siponimod. Prior therapy precluding enrollment: Natalizumab, immunosuppressive/chemotherapeutic medications (e.g. azathioprine, methotrexate) within 6 months prior to enrollment Rituximab, ofatumumab, ocrelizumab, cladribine, or cyclophosphamide within one year prior to enrollment Any mitoxantrone during previous two years prior to enrollment or evidence of cardiotoxicity following mitoxantrone or a cumulative life-time dose of more than 60 mg/m2 alemtuzumab, lymphoid irradiation, bone marrow transplantation or other immunosuppressive treatments with effects potentially lasting over 6 months, at any time Concomitant therapy precluding enrollment: Alemtuzumab, azathioprine, cyclophosphamide, cyclosporine, methotrexate, mitoxantrone, natalizumab, rituximab, ofatumumab, or ocrelizumab as immunosuppressive or immune-modulating therapies Only during treatment initiation: Class Ia (e.g. quinidine, procainamide), Class III anti-arrhythmic drugs (e.g. amiodarone, sotalol) during treatment initiation Have any of the following out-of-range laboratory values: Alanine aminotransferase (ALT) or aspartate aminotransferase (AST), or gamma-glutamyl transferase (GGT) greater than 5 times the upper limit of normal (ULN) range White blood cell (WBC) count < 3,500/mm3 (< 3.5 x 109/L) Lymphocyte count < 800/mm3 (< 0.8 x 109/L) Serum potassium > ULN Participation in a prior investigational study within 30 days prior to enrollment or within five half-lives of the investigational study drug or until the expected pharmacodynamic effect has returned to baseline, whichever is longer Pregnancy statements and contraception requirements Pregnant or nursing (lactating) women, where pregnancy is defined as the state of a female after conception and until the termination of gestation, confirmed by a positive hCG laboratory test (> 5 mIU/mL). Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant, UNLESS they are: Women whose partners have been sterilized by vasectomy or other means. Using a highly effective method of birth control for the duration of treatment with siponimod and for at least ten days after stopping treatment with siponimod. Highly effective method of birth control are such resulting in a less than 1% per year failure rate when used consistently and correctly, such as implants, injectables, combined oral contraceptives, and some intrauterine devices (IUDs); periodic abstinence (e.g. calendar, ovulation, symptothermal, post-ovulation methods) is not acceptable. Total abstinence from female-male intercourse (when this is in line with the preferred and usual lifestyle of the subject, if accepted by local regulation) Not able to understand and to comply with treatment instructions and requirements.
Adult True
Child False
Older Adult True

Calculated Values

Sequence: 254139425
Registered In Calendar Year 2020
Were Results Reported False
Has Single Facility False
Minimum Age Num 18
Minimum Age Unit Years

Intervention Other Names

Sequence: 26783345
Intervention Id 52705285
Name Mayzent

Responsible Parties

Sequence: 29007148
Responsible Party Type Sponsor