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

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ClinicalTrials.gov Identifier: NCT02038049
Recruitment Status : Terminated (The study recruitment was terminated based on strategic considerations after 8 patients were enrolled.)
First Posted : January 16, 2014
Results First Posted : October 30, 2019
Last Update Posted : October 30, 2019
Sponsor:
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Condition Relapse Remitting Multiple Sclerosis
Interventions Drug: VAY736
Drug: Placebo
Enrollment 8
Recruitment Details This study was conducted in 5 centers in 3 countries: Czech Republic (1), Ukraine (2 sites) and USA (2 sites).
Pre-assignment Details The study was planned to be conducted in approximately 96 patients. However, after enrolling 8 patients, the recruitment was terminated based on strategic considerations.
Arm/Group Title VAY736 Placebo to VAY736
Hide Arm/Group Description Intravenous infusion of VAY736 Matching placebo (infusion bag) administered intravenously. Placebo randomized patients were offered optional VAY736 administration after week 16.
Period Title: Overall Study
Started [1] 4 4
Pharmacodynamic (PD) Analysis Set [2] 4 4
Completed 3 4
Not Completed 1 0
Reason Not Completed
Lost to Follow-up             1             0
[1]
All randomized patients were included in the Full Analysis (FAS) and Safety Set
[2]
At least one dose of study drug and one evaluable PD assessment
Arm/Group Title VAY736 Placebo to VAY736 Total
Hide Arm/Group Description Intravenous infusion of VAY736 Matching placebo (infusion bag) administered intravenously. Placebo randomized patients were offered optional VAY736 administration after week 16. Total of all reporting groups
Overall Number of Baseline Participants 4 4 8
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 4 participants 4 participants 8 participants
32.0  (10.42) 42.0  (2.45) 37.0  (8.82)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 4 participants 4 participants 8 participants
Female
2
  50.0%
3
  75.0%
5
  62.5%
Male
2
  50.0%
1
  25.0%
3
  37.5%
Race/Ethnicity, Customized  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 4 participants 4 participants 8 participants
Caucasian 3 4 7
Other 1 0 1
1.Primary Outcome
Title Number of New T1-weighted Gadolinium (Gd)-Enhancing Lesions at Weeks 8, 12 and 16
Hide Description The effect of VAY736, compared to placebo on the cumulative number of new gadolinium [Gd]-enhancing lesions on T1-weighted brain MRI scans in relapsing-remitting multiple sclerosis (RRMS) patient population at weeks 8, 12 and 16. Only descriptive statistics performed.
Time Frame Week 8, Week 12, Week 16
Hide Outcome Measure Data
Hide Analysis Population Description
Pharmacodynamic (PD) analysis set, which consisted of all patients who received at least one dose of study drug during the treatment and one evaluable PD assessment, was considered
Arm/Group Title VAY736 Placebo to VAY736
Hide Arm/Group Description:
Intravenous infusion of VAY736
Matching placebo (infusion bag) administered intravenously. Placebo randomized patients were offered optional VAY736 administration after week 16.
Overall Number of Participants Analyzed 4 4
Measure Type: Number
Unit of Measure: Lesions
Week 8 5 1
Week12 5 2
Week 16 6 3
2.Secondary Outcome
Title Number of All T1-weighted Gadolinium (Gd)-Enhancing Lesions at Weeks 4, 8, 12 and 16
Hide Description Magnetic resonance imaging (MRI) scanning of the brain was performed at screening/baseline, week 4, week 8, week 12 and week 16 to assess all T1-weighted Gadolinium (Gd) enhancing lesions. Each MRI scan was reviewed by a local neuro-radiologist. Only descriptive statistics performed.
Time Frame Week 4, Week 8, Week 12, Week 16
Hide Outcome Measure Data
Hide Analysis Population Description
Pharmacodynamic (PD) analysis set, which consisted of all patients who received at least one dose of study drug during the treatment and one evaluable PD assessment, was considered
Arm/Group Title VAY736 Placebo to VAY736
Hide Arm/Group Description:
Intravenous infusion of VAY736
Matching placebo (infusion bag) administered intravenously. Placebo randomized patients were offered optional VAY736 administration after week 16.
Overall Number of Participants Analyzed 4 4
Measure Type: Number
Unit of Measure: Lesions
Week 4 19 2
Week 8 20 2
Week 12 20 3
Week 16 21 4
3.Secondary Outcome
Title Number of New T1-weighted Gadolinium (Gd)-Enhancing Lesions at Weeks 4, 8, 12 and 16
Hide Description Magnetic resonance imaging (MRI) scanning of the brain was performed at screening/baseline, week 4, week 8, week 12 and week 16 to assess all new T1-weighted Gadolinium (Gd) enhancing lesions. Each MRI scan was reviewed by a local neuro-radiologist. Only descriptive statistics performed.
Time Frame Week 4, Week 8, Week 12, Week 16
Hide Outcome Measure Data
Hide Analysis Population Description
Pharmacodynamic (PD) analysis set, which consisted of all patients who received at least one dose of study drug during the treatment and one evaluable PD assessment, was considered
Arm/Group Title VAY736 Placebo to VAY736
Hide Arm/Group Description:
Intravenous infusion of VAY736
Matching placebo (infusion bag) administered intravenously. Placebo randomized patients were offered optional VAY736 administration after week 16.
Overall Number of Participants Analyzed 4 4
Measure Type: Number
Unit of Measure: Lesions
Week 4 4 1
Week 8 1 0
Week 12 0 1
Week 16 1 1
4.Secondary Outcome
Title Number of New or Enlarging T2-weighted Gadolinium (Gd)-Enhancing Lesions at Weeks 4, 8, 12 and 16
Hide Description Magnetic resonance imaging (MRI) scanning of the brain was performed at screening/baseline, week 4, week 8, week 12 and week 16 to assess T2 hyperintense lesions (new or enlarging T2-weighted lesions). Each MRI scan was reviewed by a local neuro-radiologist. Only descriptive statistics performed.
Time Frame Week 4, Week 8, Week 12, Week 16
Hide Outcome Measure Data
Hide Analysis Population Description
Pharmacodynamic (PD) analysis set, which consisted of all patients who received at least one dose of study drug during the treatment and one evaluable PD assessment, was considered
Arm/Group Title VAY736 Placebo to VAY736
Hide Arm/Group Description:
Intravenous infusion of VAY736
Matching placebo (infusion bag) administered intravenously. Placebo randomized patients were offered optional VAY736 administration after week 16.
Overall Number of Participants Analyzed 4 4
Measure Type: Number
Unit of Measure: Lesions
Week 4 279 94
Week 8 277 93
Week 12 276 91
Week 16 264 91
5.Secondary Outcome
Title T2 Burden of Disease (Total Volume of T2-weighted Lesions) at Weeks 4, 8, 12 and 16.
Hide Description Magnetic resonance imaging (MRI) scanning of the brain was performed at screening/baseline, week 4, week 8, week 12 and week 16 to assess T2 burden of disease. Each MRI scan was reviewed by a local neuro-radiologist. Only descriptive statistics performed.
Time Frame Week 4, Week 8, Week 12, Week 16
Hide Outcome Measure Data
Hide Analysis Population Description
Pharmacodynamic (PD) analysis set, which consisted of all patients who received at least one dose of study drug during the treatment and one evaluable PD assessment, was considered
Arm/Group Title VAY736 Placebo to VAY736
Hide Arm/Group Description:
Intravenous infusion of VAY736
Matching placebo (infusion bag) administered intravenously. Placebo randomized patients were offered optional VAY736 administration after week 16.
Overall Number of Participants Analyzed 4 4
Measure Type: Number
Unit of Measure: mm3 of T2-weighted lesions
Week 4 20108.9 17706
Week 8 18998.9 16785
Week 12 18484.1 15996
Week 16 18102.7 17919
6.Secondary Outcome
Title Number of Subjects Without Any New MRI Disease Activity at Weeks 4, 8, 12 and 16.
Hide Description Magnetic resonance imaging (MRI) scanning of the brain was performed at screening/baseline, week 4, week 8, week 12 and week 16 to assess patients without any new MRI disease activity (no new Gd-enhancing lesions nor new or enlarging T2 lesions). Each MRI scan was reviewed by a local neuro-radiologist. Only descriptive statistics performed.
Time Frame Week 4, Week 8, Week 12, Week 16
Hide Outcome Measure Data
Hide Analysis Population Description
Pharmacodynamic (PD) analysis set, which consisted of all patients who received at least one dose of study drug during the treatment and one evaluable PD assessment, was considered
Arm/Group Title VAY736 Placebo to VAY736
Hide Arm/Group Description:
Intravenous infusion of VAY736
Matching placebo (infusion bag) administered intravenously. Placebo randomized patients were offered optional VAY736 administration after week 16.
Overall Number of Participants Analyzed 4 4
Measure Type: Number
Unit of Measure: Participants
Week 4 4 2
Week 8 1 0
Week 12 0 1
Week 16 3 3
7.Secondary Outcome
Title Proportion of Relapse-free Patients Over the 16 Weeks of the Treatment Period.
Hide Description A relapse is defined as the appearance of a new neurological abnormality, or worsening of previously stable, or improving pre-existing neurological abnormality, separated by at least 30 days from the onset of a preceding clinical demyelinating event. The abnormality must be present for at least 24 hours and occur in the absence of fever (<37.5 C) or infection. A relapse was considered confirmed when confirmed by an Extended disability status scale (EDSS)-certified physician who was not involved in the treatment of the patient, was blinded to treatment allocation, and had no access to patient medical records. It was recommended that this occurs within 5 days of the onset of symptoms. A relapse was confirmed when it was accompanied by an increase of at least half a point (0.5) on the EDSS or an increase of 1 point on two different Functional Systems (FS) of the EDSS or 2 points on one of the FS (excluding Bowel/Bladder or Cerebral FS). Only descriptive statistics performed.
Time Frame Week 0 (Day 1), Week 4, Week 8, Week 12, Week 16
Hide Outcome Measure Data
Hide Analysis Population Description
Pharmacodynamic (PD) analysis set, which consisted of all patients who received at least one dose of study drug during the treatment and one evaluable PD assessment, was considered
Arm/Group Title VAY736 Placebo to VAY736
Hide Arm/Group Description:
Intravenous infusion of VAY736
Matching placebo (infusion bag) administered intravenously. Placebo randomized patients were offered optional VAY736 administration after week 16.
Overall Number of Participants Analyzed 4 4
Measure Type: Count of Participants
Unit of Measure: Participants
Week 0 (Day 1) Relapse-free
4
 100.0%
4
 100.0%
Relapse
0
   0.0%
0
   0.0%
Week 4 Relapse-free
4
 100.0%
4
 100.0%
Relapse
0
   0.0%
0
   0.0%
Week 8 Relapse-free
4
 100.0%
4
 100.0%
Relapse
0
   0.0%
0
   0.0%
Week 12 Relapse-free
4
 100.0%
4
 100.0%
Relapse
0
   0.0%
0
   0.0%
Week 16 Relapse-free
3
  75.0%
3
  75.0%
Relapse
1
  25.0%
1
  25.0%
8.Secondary Outcome
Title Number of Participants With On-Treatment Adverse Events, Serious Adverse Event, and Death
Hide Description Analysis of absolute and relative frequencies for treatment emergent Adverse Event (AE), Serious Adverse Event (SAE) and Deaths by primary System Organ Class (SOC) to demonstrate that VAY736 is safe for the treatment of patients with relapsing-remitting multiple sclerosis through the monitoring of relevant clinical and laboratory safety parameters. Only descriptive statistics performed.
Time Frame 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)
Hide Outcome Measure Data
Hide Analysis Population Description
The Safety Set, which consisted of all patients who received at least one dose of study drug during the treatment period, was considered
Arm/Group Title VAY736 Administered at Visit 2 (Day 1) VAY736 Administered at Visit 7 (Week 16 - Week 17) Placebo Administered at Visit 2
Hide Arm/Group Description:
Intravenous infusion of VAY736 at Visit 2 (Day 1)
Intravenous infusion of VAY736 at Visit 7 (Week 16 - Week 17)
Matching placebo (infusion bag) administered intravenously at Visit 2. Placebo randomized patients were offered optional VAY736 administration after week 16.
Overall Number of Participants Analyzed 4 4 4
Measure Type: Count of Participants
Unit of Measure: Participants
On-treatment Adverse Events (AEs)
4
 100.0%
3
  75.0%
1
  25.0%
On-treatment Serious Adverse Events (SAEs)
0
   0.0%
0
   0.0%
0
   0.0%
On-treatment Deaths
0
   0.0%
0
   0.0%
0
   0.0%
Time Frame Adverse events and serious adverse events were collected 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).
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title VAY736 Administered at Visit 2 (Day 1) VAY736 Administered at Visit 7 (Week 16 - Week 17) Placebo Administered at Visit 2
Hide Arm/Group Description Intravenous infusion of VAY736 at Visit 2 (Day 1) Intravenous infusion of VAY736 at Visit 7 (Week 16 - Week 17) Matching placebo (infusion bag) administered intravenously at Visit 2. Placebo randomized patients were offered optional VAY736 administration after week 16.
All-Cause Mortality
VAY736 Administered at Visit 2 (Day 1) VAY736 Administered at Visit 7 (Week 16 - Week 17) Placebo Administered at Visit 2
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   0/4 (0.00%)   0/4 (0.00%)   0/4 (0.00%) 
Hide Serious Adverse Events
VAY736 Administered at Visit 2 (Day 1) VAY736 Administered at Visit 7 (Week 16 - Week 17) Placebo Administered at Visit 2
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   0/4 (0.00%)   0/4 (0.00%)   0/4 (0.00%) 
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
VAY736 Administered at Visit 2 (Day 1) VAY736 Administered at Visit 7 (Week 16 - Week 17) Placebo Administered at Visit 2
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   4/4 (100.00%)   3/4 (75.00%)   1/4 (25.00%) 
Blood and lymphatic system disorders       
Lymphopenia  1  0/4 (0.00%)  1/4 (25.00%)  0/4 (0.00%) 
Cardiac disorders       
Palpitations  1  1/4 (25.00%)  0/4 (0.00%)  0/4 (0.00%) 
Tachycardia  1  3/4 (75.00%)  0/4 (0.00%)  0/4 (0.00%) 
Gastrointestinal disorders       
Nausea  1  3/4 (75.00%)  1/4 (25.00%)  0/4 (0.00%) 
Vomiting  1  1/4 (25.00%)  0/4 (0.00%)  0/4 (0.00%) 
General disorders       
Asthenia  1  2/4 (50.00%)  0/4 (0.00%)  0/4 (0.00%) 
Chills  1  2/4 (50.00%)  0/4 (0.00%)  0/4 (0.00%) 
Hyperthermia  1  1/4 (25.00%)  0/4 (0.00%)  0/4 (0.00%) 
Pyrexia  1  1/4 (25.00%)  1/4 (25.00%)  0/4 (0.00%) 
Infections and infestations       
Respiratory tract infection viral  1  1/4 (25.00%)  0/4 (0.00%)  0/4 (0.00%) 
Rhinitis  1  0/4 (0.00%)  1/4 (25.00%)  0/4 (0.00%) 
Upper respiratory tract infection  1  0/4 (0.00%)  1/4 (25.00%)  0/4 (0.00%) 
Injury, poisoning and procedural complications       
Infusion related reaction  1  0/4 (0.00%)  1/4 (25.00%)  0/4 (0.00%) 
Road traffic accident  1  1/4 (25.00%)  0/4 (0.00%)  0/4 (0.00%) 
Investigations       
Glucose urine present  1  0/4 (0.00%)  1/4 (25.00%)  0/4 (0.00%) 
Metabolism and nutrition disorders       
Decreased appetite  1  1/4 (25.00%)  0/4 (0.00%)  0/4 (0.00%) 
Musculoskeletal and connective tissue disorders       
Arthralgia  1  1/4 (25.00%)  0/4 (0.00%)  0/4 (0.00%) 
Back pain  1  1/4 (25.00%)  0/4 (0.00%)  0/4 (0.00%) 
Musculoskeletal pain  1  1/4 (25.00%)  0/4 (0.00%)  0/4 (0.00%) 
Nervous system disorders       
Headache  1  3/4 (75.00%)  1/4 (25.00%)  1/4 (25.00%) 
Hypotonia  1  1/4 (25.00%)  0/4 (0.00%)  0/4 (0.00%) 
Somnolence  1  1/4 (25.00%)  0/4 (0.00%)  0/4 (0.00%) 
Psychiatric disorders       
Anxiety  1  1/4 (25.00%)  0/4 (0.00%)  0/4 (0.00%) 
Respiratory, thoracic and mediastinal disorders       
Dyspnoea  1  1/4 (25.00%)  0/4 (0.00%)  0/4 (0.00%) 
Nasal congestion  1  1/4 (25.00%)  0/4 (0.00%)  0/4 (0.00%) 
Rhinorrhoea  1  1/4 (25.00%)  0/4 (0.00%)  0/4 (0.00%) 
1
Term from vocabulary, MedDRA (21.0)
Indicates events were collected by systematic assessment
After enrolling 8 patients, the recruitment was terminated based on strategic considerations.
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
The terms and conditions of Novartis' agreements with its investigators may vary. However, Novartis does not prohibit any investigator from publishing. Any publications from a single-site are postponed until the publication of the pooled data (ie, data from all sites) in the clinical trial.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Study Director
Organization: Novartis Pharmaceuticals
Phone: 862-778-8300
EMail: Novartis.email@novartis.com
Layout table for additonal information
Responsible Party: Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier: NCT02038049    
Other Study ID Numbers: CVAY736X2202
2013-002324-16 ( EudraCT Number )
First Submitted: January 14, 2014
First Posted: January 16, 2014
Results First Submitted: August 13, 2019
Results First Posted: October 30, 2019
Last Update Posted: October 30, 2019