A Phase 2 interventional study of VAY736 and Placebo in Relapse Remitting Multiple Sclerosis, sponsored by Novartis Pharmaceuticals. Terminated at 5 sites in 3 countries. Open to participants aged 18 Years to 55 Years. Per ClinicalTrials.gov, last updated 2021-01-05.
Sponsored by Novartis Pharmaceuticals · Phase 2, Interventional, and Treatment
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).
The study was planned to be conducted in approximately 96 patients. However, after enrolling 8 patients, the recruitment was terminated based on strategic considerations.
3,460 studies on the registry are indexed under Multiple Sclerosis; 661 are open to participants now.
This study's enrollment of 8 is below the median of 50 across 2,342 interventional studies indexed under Multiple Sclerosis.
Browse Multiple Sclerosis studies →Novartis Pharmaceuticals is the lead sponsor of 2,673 studies on the registry; 228 are open to participants now.
Of its 576 completed or terminated interventional studies of FDA-regulated products, 431 (75%) have results posted.
Counted across the registry records on this site, refreshed daily.
Key inclusion criteria:
A relapsing-remitting course of disease with:
Key exclusion criteria:
Intravenous infusion of VAY736
Drug: VAY736
Matching placebo (infusion bag) administered intravenously. Placebo randomized patients were offered optional VAY736 administration after week 16
Drug: Placebo
Single intravenous infusion of VAY736 (10 mg/kg)
Also known as: Lanalumab
Placebo to VAY736
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.
Time frame: Week 8, Week 12, Week 16
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.
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
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
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.
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.
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
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.
Time frame: 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.
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
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)
This study was conducted in 5 centers in 3 countries: Czech Republic (1), Ukraine (2 sites) and USA (2 sites).
| Milestone | VAY736 | Placebo to VAY736 |
|---|---|---|
| Started | 4 | 4 |
| Pharmacodynamic (pd) analysis set | 4 | 4 |
| Completed | 3 | 4 |
| Not completed | 1 | 0 |
| Withdrew: Lost to follow-up | 1 | 0 |
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.
| Lesions | VAY736 | Placebo to VAY736 |
|---|---|---|
| Week 8 | 5 | 1 |
| Week12 | 5 | 2 |
| Week 16 | 6 | 3 |
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.
| Lesions | VAY736 | Placebo to VAY736 |
|---|---|---|
| Week 4 | 19 | 2 |
| Week 8 | 20 | 2 |
| Week 12 | 20 | 3 |
| Week 16 | 21 | 4 |
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.
| Lesions | VAY736 | Placebo to VAY736 |
|---|---|---|
| Week 4 | 4 | 1 |
| Week 8 | 1 | 0 |
| Week 12 | 0 | 1 |
| Week 16 | 1 | 1 |
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.
| Lesions | VAY736 | Placebo to VAY736 |
|---|---|---|
| Week 4 | 279 | 94 |
| Week 8 | 277 | 93 |
| Week 12 | 276 | 91 |
| Week 16 | 264 | 91 |
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.
| mm3 of T2-weighted lesions | VAY736 | Placebo to VAY736 |
|---|---|---|
| Week 4 | 20108.9 | 17706 |
| Week 8 | 18998.9 | 16785 |
| Week 12 | 18484.1 | 15996 |
| Week 16 | 18102.7 | 17919 |
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.
| Participants | VAY736 | Placebo to VAY736 |
|---|---|---|
| Week 4 | 4 | 2 |
| Week 8 | 1 | 0 |
| Week 12 | 0 | 1 |
| Week 16 | 3 | 3 |
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.
| Participants | VAY736 | Placebo to VAY736 |
|---|---|---|
| Week 0 (Day 1) — Relapse-free | 4 | 4 |
| Week 0 (Day 1) — Relapse | 0 | 0 |
| Week 4 — Relapse-free | 4 | 4 |
| Week 4 — Relapse | 0 | 0 |
| Week 8 — Relapse-free | 4 | 4 |
| Week 8 — Relapse | 0 | 0 |
| Week 12 — Relapse-free | 4 | 4 |
| Week 12 — Relapse | 0 | 0 |
| Week 16 — Relapse-free | 3 | 3 |
| Week 16 — Relapse | 1 | 1 |
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.
| Participants | VAY736 Administered at Visit 2 (Day 1) | VAY736 Administered at Visit 7 (Week 16 - Week 17) | Placebo Administered at Visit 2 |
|---|---|---|---|
| On-treatment Adverse Events (AEs) | 4 | 3 | 1 |
| On-treatment Serious Adverse Events (SAEs) | 0 | 0 | 0 |
| On-treatment Deaths | 0 | 0 | 0 |
Collected over 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).. Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| VAY736 Administered at Visit 2 (Day 1) | 0/4 (0%) | 0/4 (0%) | 4/4 (100%) |
| VAY736 Administered at Visit 7 (Week 16 - Week 17) | 0/4 (0%) | 0/4 (0%) | 3/4 (75%) |
| Placebo Administered at Visit 2 | 0/4 (0%) | 0/4 (0%) | 1/4 (25%) |
| Event | VAY736 Administered at Visit 2 (Day 1) | VAY736 Administered at Visit 7 (Week 16 - Week 17) | Placebo Administered at Visit 2 |
|---|---|---|---|
| TachycardiaCardiac disorders | 3/4 | 0/4 | 0/4 |
| NauseaGastrointestinal disorders | 3/4 | 1/4 | 0/4 |
| HeadacheNervous system disorders | 3/4 | 1/4 | 1/4 |
| AstheniaGeneral disorders | 2/4 | 0/4 | 0/4 |
| ChillsGeneral disorders | 2/4 | 0/4 | 0/4 |
| LymphopeniaBlood and lymphatic system disorders | 0/4 | 1/4 | 0/4 |
| PalpitationsCardiac disorders | 1/4 | 0/4 | 0/4 |
| VomitingGastrointestinal disorders | 1/4 | 0/4 | 0/4 |
| HyperthermiaGeneral disorders | 1/4 | 0/4 | 0/4 |
| PyrexiaGeneral disorders | 1/4 | 1/4 | 0/4 |
| Age, Continuous(Years) | VAY736 | Placebo to VAY736 | Total |
|---|---|---|---|
| Mean | 32.0 ± 10.42 | 42.0 ± 2.45 | 37.0 ± 8.82 |
| Sex: Female, Male(Participants) | VAY736 | Placebo to VAY736 | Total |
|---|---|---|---|
| Female | 2 | 3 | 5 |
| Male | 2 | 1 | 3 |
| Race/Ethnicity, Customized(Participants) | VAY736 | Placebo to VAY736 | Total |
|---|---|---|---|
| Caucasian | 3 | 4 | 7 |
| Other | 1 | 0 | 1 |
Plan to share: 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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