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OpenTrials
Active, Not Recruiting

NCT Number: NCT04075266

A Study of Ocrelizumab in Children and Adolescents With Relapsing-Remitting Multiple Sclerosis

This 2-year study will evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamic (PD) effects of ocrelizumab in children and adolescents ages ≥ 10 to ≤ 18 years with relapsing-remitting multiple sclerosis (RRMS). The data from this study will serve to determine the dosing regimen of ocrelizumab to be further investigated in the subsequent Phase III study in children and adolescents.

Active, Not Recruiting

This study is active but is not currently recruiting participants.

Key information

Conditions

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

Age range

10 year–18 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Azienda Ospedaliera Sant'Andrea, Rome, Lazio, Italy

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Body weight >/= 25 kg
  • Children and adolescents must have received all childhood required vaccinations
  • Female participants of childbearing potential must agree to either remain completely abstinent or to use reliable means of contraception
  • Diagnosis of relapsing-remitting multiple sclerosis (RRMS)
  • Expanded Disability Status Scale (EDSS) at screening: 0-5.5, inclusive
  • Neurologic stability for >/= 30 days prior to screening, and between screening and baseline
  • Participants naive to prior disease-modifying therapy (DMT)
  • Participants who have had at least 6 contiguous months of DMT within the past 1 year must have evidence of disease activity occurring after the full 6-month course of treatment, that is, at least one relapse or >/= 1 Gd-enhancing lesion(s) on a T1-weighted brain MRI

Exclusion criteria

  • Known presence or suspicion of other neurologic disorders that may mimic MS, including, but not limited to, acute disseminated encephalomyelitis, neuromyelitis optica or neuromyelitis optica spectrum disorders and any neurologic, somatic, or metabolic condition that could interfere with brain function or normal cognitive or neurological development
  • Patients that are aquaporin 4 positive and myelin oligodendrocyte glycoprotein (MOG) antibody positive are not eligible to participate in the study.
  • In case of an ADEM-like appearance of the first MS attack, a second attack with clear MS-like features is required.
  • Infection requiring hospitalization or treatment with IV anti-infective agents
  • History or known presence of recurrent or chronic infection (e.g., HIV, syphilis, tuberculosis)
  • Receipt of a live or live-attenuated vaccine within 6 weeks prior to treatment allocation
  • History or laboratory evidence of coagulation disorders
  • Peripheral venous access that precludes IV administration and venous blood sampling
  • Inability to complete a magnetic resonance imaging (MRI) scan
  • History of cancer, including solid tumors, hematologic malignancies, and carcinoma in situ
  • History of a severe allergic or anaphylactic reaction to humanized or murine monoclonal antibody (mAbs) or known hypersensitivity to any component of ocrelizumab solution
  • Previous treatment with B-cell-targeted therapies
  • Percentage of CD4 < 30%
  • Absolute Neutrophil Count < 1.5x1000/microliter
  • Lymphocyte count below the lower limit of normal (LLN) for age- and sex-specific reference range

Treatment and study plan

Ocrelizumab

Drug

Ocrelizumab is administered as two infusions of half the dose given 14 days apart for the first dose, then subsequent doses are administered as a single infusion every 24 weeks.

Cohort 1: total dose of 300 mg Cohort 2: total dose of 600 mg Cohort 3 and 4: additional dose level(s) may be lower than 300 mg, between 300 mg and 600 mg, or higher than 600 mg, but will be no higher than 1200 mg

Primary outcomes

  1. Dose Exploration Period: Area Under the Concentration Versus Time Curve of Ocrelizumab

    Time frame: Pre-dose 5- 30 minutes and post-dose 30 mins on Days 1, 15 and 169; At any time on Days 29, 57, 85 and 113

    The population PK model was used to simulate concentration-time course and predict individual area under the concentration versus time curve. The data for the PK parameter: area under the concentration versus time curve was collected and analyzed as per body weight range (<40kg to ≥40 kg).

  2. Dose Exploration Period: Maximum Concentration (Cmax) of Ocrelizumab

    Time frame: Pre-dose 5- 30 minutes and post-dose 30 mins on Days 1, 15 and 169; At any time on Days 29, 57, 85 and 113

    The population PK model was used to simulate concentration-time course and predict individual Cmax. The data for the PK parameter: Cmax was collected and analyzed as per body weight range (<40kg to ≥40 kg).

  3. Dose Exploration Period: Levels of CD 19+ B-cell Count in Blood

    Time frame: At Week 24

Secondary outcomes

  1. Number of Participants With Adverse Events (AEs)

    Time frame: Up to 7 years

    An AE is untoward medical occurrence in participant administered a pharmaceutical product & regardless of causal relationship with this treatment. An AE can therefore be any unfavorable & unintended sign (including an abnormal laboratory finding), symptom/disease temporally associated with use of investigational product, whether or not considered related to investigational product.

  2. Dose Exploration Period: Level of Circulating T Cells and Natural Killer (NK) Cells

    Time frame: At Week 24

  3. OOE Period: Level of Circulating T Cells and NK Cells

    Time frame: Up to 5 years

  4. Dose Exploration Period: Level of Circulating Lymphocyte, Neutrophil, Monocyte and Leukocyte

    Time frame: At Week 24

  5. OOE Period: Level of Circulating Lymphocyte, Neutrophil, Monocyte and Leukocyte

    Time frame: Up to 5 years

  6. Developmental Milestones - Growth Velocity: Change in Height

    Time frame: Up to 7 years

  7. Developmental Milestones: Bone Age Assessment by Wrist/Hand Radiographs

    Time frame: Up to 7 years

  8. Developmental Milestones: Male and Female Puberty Assessed by Tanner Staging

    Time frame: Up to 7 years

  9. Developmental Milestones: Age at Menarche, Related With the Female Reproductive Status

    Time frame: Up to 7 years

  10. Dose Exploration Period: Number of Participants With Shift From Baseline in Non-MS Central Nervous System (CNS) Pathology as Measured by Brain Magnetic Resonance Imaging (MRI)

    Time frame: Up to Week 24

    The change in the non-MS CNS pathology was assessed using MRI scans.

  11. OOE Period: Number of Participants With Shift From Baseline in Non-MS CNS Pathology as Measured by Brain MRI

    Time frame: Up to 5 years

  12. Dose Exploration Period: Levels of Blood Immunoglobulins

    Time frame: At Week 24

  13. OOE Period: Levels of Blood Immunoglobulins

    Time frame: Up to 5 years

  14. Dose Exploration Period: Number of Participants With Antibody Titers Against Standard Vaccines

    Time frame: At Week 24

    Measurement of antibody titers to common antigens (mumps, rubella, varicella, and Streptococcus pneumoniae [S. pneumoniae]) were performed.

  15. OOE Period: Number of Participants With Antibody Titers Against Standard Vaccines

    Time frame: Up to 5 years

  16. Dose Exploration Period: Number of Participants With Anti-Drug Antibodies (ADAs) to Ocrelizumab

    Time frame: At Week 24

  17. OOE Period: Number of Participants With ADAs to Ocrelizumab

    Time frame: Up to 5 years

Sponsors and collaborators

Lead sponsor

Hoffmann-La Roche

Industry

Registry information

Official study title

An Open-Label, Parallel-Group Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamic Effects of Ocrelizumab in Children and Adolescents With Relapsing-Remitting Multiple Sclerosis

Important dates

Study start
2020
Primary completion
2023
Study completion
2029
First posted
Aug 30, 2019
Registry last updated
Dec 23, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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