Clinical trial NCT02038049: A Study to Assess the Effect of a Single Infusion of VAY736 on Disease Activity in Patients With Relapsing-remitting Multiple Sclerosis
Official title: A Randomized, Partially Blind, Placebo-controlled, Proof-of-concept Study to Assess the Effect of a Single Infusion of VAY736 on Disease Activity as Measured by Brain MRI Scans in Patients With Relapsing-remitting Multiple Sclerosis
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE2
Conditions: Relapse Remitting Multiple Sclerosis
Keywords: Multiple Sclerosis
Relapsing-Remitting Multiple Sclerosis
Magnetic Resonance Imaging
VAY736
Lanalumab
monoclonal antibody
gadolinium [Gd]-enhancing lesions
B-Cell
Lead sponsor: Novartis
Sponsor class: INDUSTRY
First posted: 2014-01-16
Enrollment: 8
Sex: ALL
Minimum age (years): 18
Maximum age (years): 55
Standard ages: ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: 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).

Detailed 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.

Eligibility 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

Primary outcomes:
measure: Number of New T1-weighted Gadolinium (Gd)-Enhancing Lesions at Weeks 8, 12 and 16
timeFrame: Week 8, Week 12, Week 16
Secondary outcomes:
measure: Number of All T1-weighted Gadolinium (Gd)-Enhancing Lesions at Weeks 4, 8, 12 and 16
timeFrame: Week 4, Week 8, Week 12, Week 16
measure: Number of New T1-weighted Gadolinium (Gd)-Enhancing Lesions at Weeks 4, 8, 12 and 16
timeFrame: Week 4, Week 8, Week 12, Week 16
measure: Number of New or Enlarging T2-weighted Gadolinium (Gd)-Enhancing Lesions at Weeks 4, 8, 12 and 16
timeFrame: Week 4, Week 8, Week 12, Week 16
measure: T2 Burden of Disease (Total Volume of T2-weighted Lesions) at Weeks 4, 8, 12 and 16.
timeFrame: Week 4, Week 8, Week 12, Week 16
measure: Number of Subjects Without Any New MRI Disease Activity at Weeks 4, 8, 12 and 16.
timeFrame: Week 4, Week 8, Week 12, Week 16
measure: Proportion of Relapse-free Patients Over the 16 Weeks of the Treatment Period.
timeFrame: Week 0 (Day 1), Week 4, Week 8, Week 12, Week 16
measure: Number of Participants With On-Treatment Adverse Events, Serious Adverse Event, and Death
timeFrame: From first dosing (single administration, Day 1) up to End of Study Visit (EOS) depending on B cell recovery (ranging from week 48 to 216)
Other outcomes:
Locations:
city: Long Beach
country: United States
facility: Novartis Investigative Site
state: California
city: San Diego
country: United States
facility: Novartis Investigative Site
state: California
city: Hradec Kralove
country: Czechia
facility: Novartis Investigative Site
city: Kharkiv
country: Ukraine
facility: Novartis Investigative Site
city: Lviv
country: Ukraine
facility: Novartis Investigative Site
Study officials:
affiliation: Novartis Pharmaceuticals
name: Novartis Pharmaceuticals
role: STUDY_DIRECTOR
References: