A Study to Assess the Effect of a Single Infusion of VAY736 on Disease Activity in Patients With Relapsing-remitting Multiple Sclerosis

Novartis

Status and phase: Terminated; Phase 2

Conditions: Relapse Remitting Multiple Sclerosis

Treatments: Drug: VAY736; Drug: Placebo

Study type: Interventional

Funder types: Industry

Identifiers

NCT02038049

2013-002324-16 (EudraCT Number)

CVAY736X2202

Details and patient eligibility

About

This was a randomized, partially blinded, placebo-controlled, non-confirmatory study to assess the effects of a single infusion of VAY736 on disease activity as measured by brain MRI scans in patients with relapsing-remitting multiple sclerosis (RRMS).

Full description

The study was planned to be conducted in approximately 96 patients. However, after enrolling 8 patients, the recruitment was terminated based on strategic considerations.

Enrollment: 8 patients

Sex: All

Ages: 18 to 55 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

Key inclusion criteria:

• Male and female patients aged 18 to 55 years.

• Diagnosis of MS as defined by the 2010 revised McDonald criteria (Polman et al 2011).

• A relapsing-remitting course of disease with:

  • at least 1 documented relapse during the previous 12 months (but not within 30 days prior to randomization ), or
  • a positive Gd-enhancing lesion on brain MRI scan at screening.

• An Expanded Disability Status Scale (EDSS) score of 0-5.0 inclusive at screening.

• No evidence of a relapse within 30 days prior to randomization.

Key exclusion criteria:

• A manifestation of another type of MS other than RRMS.
• Findings on screening or baseline brain MRI inconsistent with the diagnosis of MS.
• History of chronic disease of the immune system other than MS, or a known immunodeficiency syndrome.
• Score "yes" on item 4 or item 5 of the Suicidal Ideation section of the C-SSRS, if this ideation occurred in the past 6 months, or "yes" on any item of the Suicidal Behavior section, except for the "Non-Suicidal Self-Injurious Behavior" (item also included in the Suicidal Behavior section), if this behavior occurred in the past 2 years.
• Women of child-bearing potential and Pregnant or nursing (lactating) women.
• Screening CBC (complete blood count) laboratory values as follows:
• Hemoglobin levels below 10.0 g/dL
• Total leukocyte count less than 3,000 cells/µL
• Neutropenia, defined as absolute neutrophil counts less than 1500 cells/mm3
• Platelets less than 100,000/µL

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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8 participants in 2 patient groups, including a placebo group

VAY736

Experimental group

Description:

Intravenous infusion of VAY736

Treatment:

Drug: VAY736

Placebo to VAY736

Placebo Comparator group

Description:

Matching placebo (infusion bag) administered intravenously. Placebo randomized patients were offered optional VAY736 administration after week 16

Treatment:

Drug: Placebo