A Study to Evaluate Safety and Efficacy of Ocrelizumab in Comparison With Fingolimod in Children and Adolescents With Relapsing-remitting Multiple Sclerosis (RRMS)

NCT05123703 | Active Not Recruiting Phase 3 Results DMC FDA Drug

• 3 other identifiers: WN420862020-004128-412023-506516-40-00
• Study Type: interventional
• Target: 188
• Locations: 23 countries
• Sites: 65

Brief Summary

This double-blind, double-dummy study will evaluate the safety and efficacy of ocrelizumab compared with fingolimod in children and adolescents with RRMS aged between 10 and \< 18 years over a flexible duration. The double-blind period will last until after the last participant randomized has completed 24 weeks.

Conditions

Relapsing-remitting Multiple Sclerosis

Keywords

pediatric Multiple Sclerosis; pediatric MS; children MS; children Multiple Sclerosis; pediatric ocrelizumab

Outcome Measures

Primary Outcomes (1)

• Protocol-defined Annualized Relapse Rate (ARR)

  The population-level summary is rate ratio of ARR. The ARR is calculated as the total number of protocol-defined relapses divided by the total patient-years. Protocol-defined relapse is the occurrence of new/worsening neurological symptoms attributable to multiple sclerosis (MS) and immediately preceded by relatively stable or improving neurological state for ≥ 30 days. Symptoms must persist for \>24 hours \& should not be attributable to confounding clinical factors. The new/worsening neurological symptoms must be accompanied by objective neurological worsening consistent with an increase of at least half a step on the expanded disability status scale (EDSS) score, or 2 points on one of the appropriate functional system (FS) scores, or 1 point on two or more of the appropriate FS scores. The change must affect the selected FS (i.e., pyramidal, ambulation, cerebellar, brainstem, sensory, or visual). Adjusted values were reported. OM assessed non inferiority of ocrelizumab vs fingolimod.

  Up to after the last participant randomized completed 24 weeks since first study treatment administration (up to approximately 160 weeks)

Secondary Outcomes (8)

• Protocol-defined ARR

  Up to after the last participant randomized completed 24 weeks since first study treatment administration (up to approximately 160 weeks)

• Number of New or Enlarging T2-hyperintense Lesions (T2 Lesions), as Detected by Brain Magnetic Resonance Imaging (MRI)

  Up to after the last participant randomized completed 24 weeks since first study treatment administration (up to approximately 160 weeks)

• Number of T1 Gd Lesions at Week 12

  At Week 12

• Number of Participants With Adverse Events (AEs)

  Up to approximately 7 years

• Maximum Serum Concentration (Cmax) of Ocrelizumab

  Cycle (1 Cycle=24 weeks)

Study Arms (2)

Ocrelizumab

EXPERIMENTAL

Participants will receive ocrelizumab, as IV infusion Q24W. The first dose is given as dual infusions of half the dose of ocrelizumab on Days 1 and 15, and subsequent doses are given as single infusions of ocrelizumab Q24W. Participants will also receive a placebo of fingolimod administered as QD capsule.

Drug: Ocrelizumab; Other: Fingolimod Placebo

Fingolimod

ACTIVE COMPARATOR

Participants will receive fingolimod PO, QD as per the prescribing information provided with fingolimod. Participants will also receive a placebo of ocrelizumab administered as IV infusion on Days 1 and 15, and Q24W thereafter.

Other: Ocrelizumab Placebo; Drug: Fingolimod

Interventions

Ocrelizumab DRUG

Ocrelizumab, 300 milligrams (mg) will be administered as IV infusion to participants who weigh \< 35 kilograms (kg), and ocrelizumab 600 mg, IV will be administered to participants who weigh ≥ 35 kg on Days 1 and 15 (half the dose, 2 weeks apart), and Q24W thereafter.

Also known as: RO4964913; Ocrevus

Ocrelizumab

Ocrelizumab Placebo OTHER

Ocrelizumab matching placebo will be administered as IV infusion on Day 1 and Day 15, and Q24W thereafter.

Fingolimod

Fingolimod DRUG

Fingolimod will be administered QD as a capsule per the prescribing information (0.25 mg to participants who weigh ≤ 40 kg and 0.5 mg to participants who weigh \> 40 kg).

Also known as: Gilenya®

Fingolimod

Fingolimod Placebo OTHER

Fingolimod matching placebo will be administered QD as a capsule.

Ocrelizumab

Eligibility Criteria

• Age: 10 Years - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Body weight ≥ 25 kg
• Diagnosis of RRMS in accordance with the International Pediatric Multiple Sclerosis Study Group (IPMSSG) criteria for pediatric multiple sclerosis (MS), Version 2012, or McDonald criteria 2017
• Expanded Disability Status Scale (EDSS) at screening: 0-5.5, inclusive
• For all countries except Germany, at least one MS relapse during the previous year or two MS relapses in the previous 2 years or evidence of at least one Gd-enhancing lesion on MRI within 6 months prior to randomization
• Participants in Group A (ocrelizumab in the DBP) and Group B (fingolimod in the DBP) who, in the opinion of the investigator, may benefit from switching to ocrelizumab and who have completed the DBP with study treatment (ocrelizumab/fingolimod), may participate in the OLE period

You may not qualify if:

• Known presence or suspicion of other neurologic disorders that may mimic MS
• Significant uncontrolled somatic diseases, known active infection or any other significant condition that may preclude participant from participating in the study
• Participants with severe cardiac disease or significant findings on the screening electrocardiograph (ECG)
• Participants who have discontinued the study during the DBP

Sponsors & Collaborators

• Hoffmann-La Roche: lead
• PPD Development, LP: collaborator

Study Sites (71)

UC San Diego

La Jolla, California, 92037-1337, United States

Location

Children's Hospital Colorado

Aurora, Colorado, 80045, United States

Location

Children's National Hospital

Washington D.C., District of Columbia, 20010, United States

Location

Johns Hopkins Medicine

Baltimore, Maryland, 21287, United States

Location

Boston Children's Hospital Central Pharmacy

Boston, Massachusetts, 02115-5724, United States

Location

Washington University

St Louis, Missouri, 63101, United States

Location

Cleveland Clinic, Mellen Center for Multiple Sclerosis

Cleveland, Ohio, 44195-0001, United States

Location

The Boster Center for Multiple Sclerosis a Singlepoint Healthcare Company

Columbus, Ohio, 43235, United States

Location

The Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104-4319, United States

Location

Baylor College of Medicine/Texas Children's Hospital

Houston, Texas, 77030-2608, United States

Location

Centro de Investigaciones Médicas Tucuman

San Miguel de Tucumán, T4000AXL, Argentina

Location

Royal Children's Hospital Melbourne - PIN

Parkville, Victoria, 3052, Australia

Location

Medizinische Universität Wien

Vienna, 1090, Austria

Location

UZ Gent

Ghent, 9000, Belgium

Location

L2 Ip - Instituto de Pesquisas Clinicas Ltda - ME

Brasília, Federal District, 70200-730, Brazil

Location

Instituto de Neurologia de Curitiba

Curitiba, Paraná, 81210-310, Brazil

Location

Nucleo de Pesquisa Clinica do Rio Grande do Sul NPCR

Porto Alegre, Rio Grande do Sul, 90430-001, Brazil

Location

Hospital Sao Lucas - PUCRS

Porto Alegre, Rio Grande do Sul, 90610-000, Brazil

Location

CPQuali Pesquisa Clínica Sao Paulo

São Paulo, São Paulo, 01228-000, Brazil

Location

Inst. Da Criança- Faculdade de Medicina Usp

São Paulo, 05403-900, Brazil

Location

Children's Hospital of Eastern Ontario

Ottawa, Ontario, K1H 8L1, Canada

Location

The Hospital for Sick Children

Toronto, Ontario, M5G 1X8, Canada

Location

Astra Kliinik

Tallinn, 11315, Estonia

Location

Tartu University Hospital

Tartu, 50406, Estonia

Location

Hospices Civils de Lyon - Hôpital Pierre Wertheimer

Bron, Rhône, 69003, France

Location

Centre Hospitalier Universitaire de Bicêtre

Le Kremlin-Bicêtre, 94275, France

Location

CHRU de Montpellier, Hopital Gui de Chauliac

Montpellier, 34295, France

Location

Hopital de Hautepierre

Strasbourg, 67091, France

Location

Vestische Kinder- und Jugendklinik Datteln

Datteln, 45711, Germany

Location

Universitaetsklinikum Carl Gustav Carus an der TU Dresden

Dresden, 01307, Germany

Location

Eginitio University General Hospital of Athens

Athens, Attica, 115 28, Greece

Location

University General Hospital ''ATTIKON'' - General Hospital of West Attica H AGIA VARVARA

Chaïdári, Attica, 124 62, Greece

Location

St. Luke's Hospital

Thessaloniki, 552 36, Greece

Location

Semmelweis Egyetem

Budapest, 1094, Hungary

Location

Debreceni Egyetem Klinikai Kozpont

Debrecen, H-4032, Hungary

Location

Sparsh Super Speciality Hospital

Bangalore North, Karnataka, 560022, India

Location

Universita? G. D'Annunzio

Chieti, Abruzzo, 66100, Italy

Location

Azienda Ospedaliero-Universitaria Consorziale Pol. di Bari

Bari, Apulia, 70124, Italy

Location

Ospedale Pediatrico Bambino Gesù

Rome, Lazio, 00165, Italy

Location

Azienda Ospedaliera Sant'Andrea

Rome, Lazio, 00189, Italy

Location

Fondazione IRCCS Istituto Neurologico Carlo Besta

Milan, Lombardy, 20133, Italy

Location

Azienda Ospedaliero Universitaria Policlinico Vittorio Emanuele

Catania, Sicily, 95123, Italy

Location

Children's Clinical University Hospital

Riga, LV-1004, Latvia

Location

Hospital Civil Fray Antonio Alcalde

Guadalajara, Jalisco, 44280, Mexico

Location

Clinstile S.A de C.V.

Mexico City, Mexico CITY (federal District), 06700, Mexico

Location

Grupo Médico Camino S.C.

Mexico City, Mexico CITY (federal District), 3310, Mexico

Location

Centro de Investigacion Clinica Chapultepec S. A. de C. V.

Morelia, Michoacán, 58260, Mexico

Location

Neurociencias Estudios Clinicos S.C.

Culiacán, Sinaloa, 80020, Mexico

Location

FAICIC S de R.L. de C.V

Veracruz, 91900, Mexico

Location

CHU Mohammed VI

Marrakesh, 40000, Morocco

Location

Uniwersyteckie Centrum Kliniczne

Gda?sk, 80-952, Poland

Location

Uniwersytecki Szpital Kliniczny w Poznaniu

Późna, 60-355, Poland

Location

Dzieci?cy Szpital Kliniczny im. Józefa Polikarpa Brudzi?skiego

Warsaw, 02-091, Poland

Location

Instytut Pomnik Centrum Zdrowia Dziecka

Warsaw, 04-730, Poland

Location

Hospital de Braga

Braga, 4710-243, Portugal

Location

ULS de Coimbra, EPE - Hospitais da Universidade de Coimbra

Coimbra, 3000-602, Portugal

Location

Hospital Santo Antonio dos Capuchos

Lisbon, 1169-050, Portugal

Location

Victor Gomoiu Clinical Hospital for Children

Bucharest, 022102, Romania

Location

Prof Dr Alexandru Obregia Clinical Psychiatric Hospital

Bucharest, 041914, Romania

Location

Childrens University Hospital

Belgrade, 11000, Serbia

Location

Clinic for Neurology and Psychiatry for Children and Youth

Belgrade, 11000, Serbia

Location

Mother and Child Health Care Institute of Serbia Dr Vukan Cupic

Belgrade, 11000, Serbia

Location

University Clinical Centre of Nis

Niš, 18000, Serbia

Location

Hospital Sant Joan De Deu

Esplugues de Llobregas, Barcelona, 08950, Spain

Location

Hospital Universitari Vall d'Hebron

Barcelona, 08035, Spain

Location

Hospital Universitario de la Princesa

Madrid, 28006, Spain

Location

Hospital Universitario Ramon y Cajal

Madrid, 28034, Spain

Location

Hospital Universitario Virgen Macarena

Seville, 41009, Spain

Location

Universitäts-Kinderspital Zürich - Eleonorenstiftung

Zurich, 8008, Switzerland

Location

Communal noncommercial enterprise of Lviv Regional Council Lviv Regional Clinical Hospital

Lviv, Kharkiv Governorate, 79010, Ukraine

Location

Royal Hospital for Children and Young People

Edinburgh, EH51, United Kingdom

Location

Related Publications (1)

• Mar S, Valeriani M, Steinborn B, Schreiner T, Waubant E, Filippi M, Kotulska K, Mazurkiewicz-Beldzinska M, El Azzouzi B, Lin CJ, Shen YA, Kletzl H, Evershed J, Hogea A, Manlius C, Bonati U, Banwell B. Ocrelizumab dose selection for treatment of pediatric relapsing-remitting multiple sclerosis: results of the OPERETTA I study. J Neurol. 2025 Jan 15;272(2):137. doi: 10.1007/s00415-024-12879-z.

  PMID: 39812825 DERIVED

MeSH Terms

Conditions

Multiple Sclerosis, Relapsing-Remitting

Interventions

ocrelizumab; Fingolimod Hydrochloride

Condition Hierarchy (Ancestors)

Multiple Sclerosis; Demyelinating Autoimmune Diseases, CNS; Autoimmune Diseases of the Nervous System; Nervous System Diseases; Demyelinating Diseases; Autoimmune Diseases; Immune System Diseases

Intervention Hierarchy (Ancestors)

Sphingosine; Amino Alcohols; Alcohols; Organic Chemicals; Propylene Glycols; Glycols; Amines

Results Point of Contact

• Title: Medical Communications
• Organization: Hoffmann-La Roche

genentech@druginfo.com

800 821-8590

Study Officials

• Clinical Trials

  Hoffmann-La Roche

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 16, 2021
• First Posted: November 17, 2021
• Study Start: May 19, 2022
• Primary Completion: June 9, 2025
• Study Completion (Estimated): September 17, 2029
• Last Updated: July 15, 2026
• Results First Posted: July 15, 2026

Record last verified: 2026-07

Data Sharing

• IPD Sharing: Will share

Locations

IN (1); MO (1); AU (1); ME (6); US (10); FR (4); PL (4); BE (1); LA (1); RO (2); AR (1); CH (1); SE (4); GB (1); DE (2); ES (2); BR (6); UA (1); HU (2); PT (3); GR (3); CA (2); IT (6)