Last Update: Oct 04, 2024
A 2-year Randomized, 3-arm, Double-blind, Non-inferiority Study Comparing the Efficacy and Safety of Ofatumumab and Siponimod Versus Fingolimod in Pediatric Patients With Multiple Sclerosis Followed by an Open-label Extension
ClinicalTrials.gov Identifier:
Novartis Reference Number:CBAF312D2301
All compounds are either investigational or being studied for (a) new use(s). Efficacy and safety have not been established. There is no guarantee that they will become commercially available for the use(s) under investigation.

Study Description

Efficacy and safety of ofatumumab and siponimod compared to fingolimod in pediatric patients with multiple sclerosis The study is divided into a Core Part and Extension Part. The Core Part is a 24-month, double-blind, triple dummy, randomized, 3-arm active-controlled in children/adolescent patients aged 10-17 years old with Multiple Sclerosis (MS). The Extension Part is 60-month (5 year) open label (except for first 12 weeks transition which will remain double-blind) treatment for patients who complete the Core Part of the study and meet all inclusion/exclusion criteria. The targeted enrollment is 120 participants with multiple sclerosis which will include at least 5 participants with body weight (BW) ≤40 kg and at least 5 participants with age 10 to 12 years in each of the ofatumumab and siponimod arms. There is a minimum 6 month follow up period for all participants (core and extension). Total duration of the study could be up to 7 years.

Multiple Sclerosis (MS)
Phase3
Recruiting
120
Oct 05, 2021
Dec 23, 2031
All
10 Years - 17 Years (Child)

Interventions

Drug

Fingolimod

Fingolimod capsule administered orally once daily at a dose of either 0.5 mg or 0.25 mg (depending on patient's body weight).
Other

Fingolimod placebo

Fingolimod matching placebo capsule
Drug

Ofatumumab

Ofatumumab as a solution for injection in an autoinjector containing 20 mg ofatumumab (50 mg/mL, 0.4 mL content) for subcutaneous administration. A loading dose at Day1, Day 7 and Day 14 and then injections every 4 weeks/ 6 weeks (depending on patient's body weight).
Other

Ofatumumab placebo

Ofatumumab matching placebo autoinjector
Drug

Siponimod

Siponimod tablet administered orally once daily. Titration period, Day 1 to Day 6, first dose is either 0.1 mg or 0.25 mg up to daily dose of either 0.5 mg, 1 mg or 2 mg (depending on CYP2C9 genotype and body weight).
Other

Siponimod placebo

Siponimod matching placebo tablet

Eligibility Criteria

Inclusion Criteria:

1. Between 10 to \<18 years of age (i.e., have not yet had their 18th birthday) at randomization
2. Diagnosis of multiple sclerosis
3. EDSS score of 0 to 5.5, inclusive
4. At least one MS relapse/attack during the previous year or two MS relapses in the previous two years prior or evidence of one or more new T2 lesions within 12 months

Exclusion Criteria:

1. Participants with progressive MS
2. Participants with an active, chronic disease of the immune system other than MS
3. Participants meeting the definition of ADEM
4. Participants with severe cardiac disease or significant findings on the screening ECG.
5. Participants with severe renal insufficiency

Novartis Investigative Site

Recruiting

Caba,Buenos Aires,C1181ach,Argentina

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Parkville,Victoria,3052,Australia

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Wien,1090,Austria

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Esneux,4130,Belgium

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Gent,9000,Belgium

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Curitiba,PR,81210-310,Brazil

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Porto Alegre,RS,90430-001,Brazil

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Sao Paulo,SP,05403-000,Brazil

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Montreal,Quebec,H3a 2b4,Canada

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Montreal,Quebec,H3t 1c5,Canada

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Lo Barnechea,Santiago,7691236,Chile

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Zagreb,10000,Croatia

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Tallinn,11315,Estonia

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Montpellier,34295,France

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Strasbourg,67000,France

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Le Kremlin Bicetre,94275,France

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Gottingen,37075,Germany

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Berlin,13353,Germany

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Bochum,44791,Germany

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Erlangen,91054,Germany

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Freiburg,79106,Germany

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Guatemala,01015,Guatemala

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New Delhi,Delhi,110 017,India

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Kochi,Kerala,682041,India

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Lucknow,Uttar Pradesh,226014,India

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Kolkata,West Bengal,700017,India

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Petach Tikva,49202,Israel

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Milano,MI,20132,Italy

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Roma,RM,00165,Italy

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Napoli,80131,Italy

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Riga,Lv-1004,Latvia

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Ciudad de Mexico,Distrito Federal,06700,Mexico

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Mexico,Distrito Federal,06720,Mexico

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Gdansk,80 952,Poland

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Poznan,60-355,Poland

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Lodz,93-338,Poland

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Warsaw,04 730,Poland

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Coimbra,3000-602,Portugal

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Lisboa,1169 050,Portugal

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Bucuresti,041914,Romania

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Belgrade,11000,Serbia

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Bratislava,833 40,Slovakia

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Sevilla,Andalucia,41009,Spain

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Oviedo,Asturias,33011,Spain

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Baracaldo,Vizcaya,48903,Spain

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Madrid,28034,Spain

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Tainan,70403,Taiwan

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Taipei,10002,Taiwan

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Istanbul,TUR,34098,Turkey

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Izmir,35575,Turkey

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Kocaeli,41380,Turkey

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Samsun,55139,Turkey

Childrens Healthcare Of Atlanta .

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Atlanta,Georgia,30322,United States

Grace Gombolay
Arpit Dosanjh

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Recruiting

San Diego,California,92103,United States

Childrens Hospital of Philadelphia .

Recruiting

Philadelphia,Pennsylvania,19104 4399,United States

Amy Waldman
Kat Gallison

Uni of Louisville Clncl Trials Unit Novak Center

Recruiting

Louisville,Kentucky,40202,United States

Michael Sweeney

Childrens National Medical Center .

Recruiting

Washington,District of Columbia,20010,United States

Alexandra Kornbluh
Hameenat Adekoya

Mayo Clinic - Rochester

Recruiting

Rochester,Minnesota,55905,United States

Jan-Mendelt Tillema
Lexi DeLay

Novartis Investigative Site

Recruiting

Dallas,Texas,75390,United States

Arkansas Childrens Hosp Rsch Inst .

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Little Rock,Arkansas,72202,United States

Kat Turbeville
Virginia Erin Willis

Axiom Clinical Research of Florida

Recruiting

Tampa,Florida,33609,United States

Chloe Rzeppa
Mark Cascione

Medical College of Wisconsin .

Recruiting

Milwaukee,Wisconsin,53226,United States

Ahmed Obeidat
Andrew Crow

WA Uni School Of Med Main Center

Recruiting

Saint Louis,Missouri,63110,United States

Olga Novak
Soe Mar

Childrens Hospital Los Angeles .

Recruiting

Los Angeles,California,90027,United States

Fatima Menendez
Jonathan Santoro

Novartis Investigative Site

Recruiting

Portland,Oregon,97225,United States

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Novartis Pharmaceuticals

Novartis Pharmaceuticals