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

Roche

Status and phase: Active, not recruiting; Phase 2

Conditions: Multiple Sclerosis

Treatments: Drug: Ocrelizumab

Study type: Interventional

Funder types: Industry

Identifiers

NCT04075266

WA39085

2016-002667-34 (EudraCT Number)

Details and patient eligibility

About

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.

Enrollment: 23 estimated patients

Sex: All

Ages: 10 to 18 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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23 participants in 4 patient groups

Cohort 1

Experimental group

Description:

Participants with a body weight from \>/= 25 kg to \< 40 kg (with at least 2 participants with a body weight from \>/= 25 kg to \</= 35 kg) will receive 300 milligram (mg) ocrelizumab

Treatment:

Drug: Ocrelizumab

Cohort 2

Experimental group

Description:

Participants with a body weight \>/= 40 kg (with at least 2 participants with a body weight \>/= 40 kg but \</= 50 kg) will receive 600 mg ocrelizumab

Treatment:

Drug: Ocrelizumab

Cohort 3 (optional)

Experimental group

Description:

Based on PK, PD, safety, and tolerability data analyses of Cohorts 1 and 2, additional participants with a body weight from \>/= 25 kg to \< 40 kg may be enrolled and receive another dose level of ocrelizumab

Treatment:

Drug: Ocrelizumab

Cohort 4 (optional)

Experimental group

Description:

Based on PK, PD, safety, and tolerability data analyses of Cohorts 1 and 2, additional participants with a body weight \>/= 40 kg may be enrolled and receive another dose level of ocrelizumab

Treatment:

Drug: Ocrelizumab