Relapse Remitting Multiple SclerosisPhase 2NCT02038049
A Study to Assess the Effect of a Single Infusion of VAY736 on Disease Activity in Patients With Relapsing-remitting Multiple Sclerosis
A Phase 2 study testing VAY736 in people with Relapse Remitting Multiple Sclerosis, run by Novartis Pharmaceuticals at 5 sites in 3 countries.
Trial 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). Read more about this study
- Who is it for?
- Ages 18 to 55 years
- Conditions the study is for
- Relapse Remitting Multiple Sclerosis
- What is being tested?
- VAY736
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 8 participants
- Where
- 5 sitesin 3 countries
- Study dates
- 20 Dec 2013to 13 Sep 2018
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 55 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- 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.
Who cannot join
- 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:
Show all 10Show fewer
- 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 locations and contacts
5 sites in 3 countries.
5 sites in 3 countries
United States2 sites
Novartis Investigative Site
Long Beach, California, 90806
Novartis Investigative Site
San Diego, California, 92103
Ukraine2 sites
Novartis Investigative Site
Kharkiv, 61068
Novartis Investigative Site
Lviv, 79010
About this study
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
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).
Read the 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.
- Conditions
- Relapse Remitting Multiple Sclerosis
- Being tested
- VAY736, Placebo
- Keywords
- Multiple Sclerosis, Relapsing-Remitting Multiple Sclerosis, Magnetic Resonance Imaging, VAY736, Lanalumab, monoclonal antibody, gadolinium [Gd]-enhancing lesions, B-Cell
- Medical subject headings
- Multiple Sclerosis, Multiple Sclerosis, Relapsing-Remitting
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
VAY736Drug
Also called Lanalumab
Single intravenous infusion of VAY736 (10 mg/kg)
PlaceboDrug
Placebo to VAY736
| Study group | Role | What they receive |
|---|---|---|
| VAY736 | Experimental | Intravenous infusion of VAY736 Receives: Drug: VAY736 |
| Placebo to VAY736 | Placebo comparator | Matching placebo (infusion bag) administered intravenously. Placebo randomized patients were offered optional VAY736 administration after week 16 Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of New T1-weighted Gadolinium (Gd)-Enhancing Lesions at Weeks 8, 12 and 16
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.
Measured: Week 8, Week 12, Week 16
Secondary outcomes (7)
Number of All T1-weighted Gadolinium (Gd)-Enhancing Lesions at Weeks 4, 8, 12 and 16
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.
Measured: Week 4, Week 8, Week 12, Week 16
Number of New T1-weighted Gadolinium (Gd)-Enhancing Lesions at Weeks 4, 8, 12 and 16
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.
Measured: Week 4, Week 8, Week 12, Week 16
Number of New or Enlarging T2-weighted Gadolinium (Gd)-Enhancing Lesions at Weeks 4, 8, 12 and 16
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.
Measured: Week 4, Week 8, Week 12, Week 16
T2 Burden of Disease (Total Volume of T2-weighted Lesions) at Weeks 4, 8, 12 and 16.
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.
Measured: Week 4, Week 8, Week 12, Week 16
Number of Subjects Without Any New MRI Disease Activity at Weeks 4, 8, 12 and 16.
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.
Measured: Week 4, Week 8, Week 12, Week 16
Proportion of Relapse-free Patients Over the 16 Weeks of the Treatment Period.
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.
Measured: Week 0 (Day 1), Week 4, Week 8, Week 12, Week 16
Number of Participants With On-Treatment Adverse Events, Serious Adverse Event, and Death
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.
Measured: 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)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Jan 2014 (estimated)
- Study start20 Dec 2013
- Primary completion5 May 2015
- Study completion13 Sep 2018
Why it stopped: The study recruitment was terminated based on strategic considerations after 8 patients were enrolled.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Relapse Remitting Multiple SclerosisStudies of the same condition, open and under way.
- More trials run by Novartis PharmaceuticalsThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 8 participants (actual)
Sponsor and investigators
- Lead sponsor
- Novartis Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Novartis Pharmaceuticals, Study director, Novartis Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02038049
- Sponsor's protocol ID
- CVAY736X2202
- Other IDs
- 2013-002324-16
- Status last verified
- Oct 2019
- First submitted
- 14 Jan 2014
- Last updated
- 5 Jan 2021
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations. This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com
Study at a glance
From the registry record, last updated by the sponsor on 5 Jan 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT02038049
- Acronym / sponsor ID
- CVAY736X2202
- 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
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: The study recruitment was terminated based on strategic considerations after 8 patients were enrolled.
- Enrolment
- 8 participants (actual)
- Lead sponsor
- Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 20 Dec 2013 (actual)
- Primary completion
- 5 May 2015 (actual)
- Study completion
- 13 Sep 2018 (actual)
- First posted
- 16 Jan 2014 (estimated)
- Results
- Posted 30 Oct 2019
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| VAY736 | Experimental | Drug: VAY736Intravenous infusion of VAY736 |
| Placebo to VAY736 | Placebo comparator | Drug: PlaceboMatching placebo (infusion bag) administered intravenously. Placebo randomized patients were offered optional VAY736 administration after week 16 |
Who may qualify
- Ages
- 18 to 55 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (7 listed)
- 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:
- 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 (10 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| VAY736Also: LanalumabSingle intravenous infusion of VAY736 (10 mg/kg) | Drug | VAY736 |
| PlaceboPlacebo to VAY736 | Drug | Placebo to VAY736 |
Primary outcome
Number of New T1-weighted Gadolinium (Gd)-Enhancing Lesions at Weeks 8, 12 and 16
Time frame: Week 8, Week 12, Week 16
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.
7 secondary outcomes
Number of All T1-weighted Gadolinium (Gd)-Enhancing Lesions at Weeks 4, 8, 12 and 16
Time frame: Week 4, Week 8, Week 12, Week 16
Number of New T1-weighted Gadolinium (Gd)-Enhancing Lesions at Weeks 4, 8, 12 and 16
Time frame: Week 4, Week 8, Week 12, Week 16
Number of New or Enlarging T2-weighted Gadolinium (Gd)-Enhancing Lesions at Weeks 4, 8, 12 and 16
Time frame: Week 4, Week 8, Week 12, Week 16
T2 Burden of Disease (Total Volume of T2-weighted Lesions) at Weeks 4, 8, 12 and 16.
Time frame: Week 4, Week 8, Week 12, Week 16
Number of Subjects Without Any New MRI Disease Activity at Weeks 4, 8, 12 and 16.
Time frame: Week 4, Week 8, Week 12, Week 16
Proportion of Relapse-free Patients Over the 16 Weeks of the Treatment Period.
Time frame: Week 0 (Day 1), Week 4, Week 8, Week 12, Week 16
Number of Participants With On-Treatment Adverse Events, Serious Adverse Event, and Death
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)
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Jan 2021.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- A Plain Language Trial Summary is available on novartisclinicaltrials.comLink provided by the sponsor.
- Individual participant dataSharing planned: Yes. Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations. This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Novartis Pharmaceuticals study director
Novartis Pharmaceuticals
5 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing VAY736 in people with Relapse Remitting Multiple Sclerosis, run by Novartis Pharmaceuticals at 5 sites in 3 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 10 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 5 Jan 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 sites in United States, Ukraine, Czechia. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 30 Oct 2019). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of New T1-weighted Gadolinium (Gd)-Enhancing Lesions at Weeks 8, 12 and 16 (time frame: Week 8, Week 12, Week 16). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 5 Jan 2021.