A Randomized, Double-blind, Placebo Controlled, 2-arm Multicenter Phase 3 Study to Assess the Efficacy and Safety of Ianalumab in Patients With Active Sjogren's Syndrome (NEPTUNUS-1)

Plain English Summary

Two-arm Study to Assess Efficacy and Safety of Ianalumab (VAY736) in Patients With Active Sjogren's Syndrome is a Phase 3 clinical trial sponsored by Novartis Pharmaceuticals studying Sjogren Syndrome. Tests Ianalumab, a new drug, to see if it helps people with active Sjogren's Syndrome. For adults 18 years and older with Sjogren's Syndrome, who have had it for up to 7.5 years. Participation involves taking Ianalumab or a placebo for 48 weeks and having regular check-ups. Alternative treatments include other medications or no treatment. The trial aims to enroll 276 participants.

Official Summary

A randomized, double-blind, placebo controlled, 2-arm multicenter phase 3 study to assess the efficacy and safety of ianalumab in patients with active Sjogren's syndrome (NEPTUNUS-1)

Who Can Participate

Here is what you need to know about eligibility for this trial. Adults 18 years and older can join. People with Sjogren's Syndrome and certain lab test results can join. Women and men can join, but must be able to communicate well. People with other autoimmune diseases or infections cannot join. This trial is studying Sjogren Syndrome, so participants generally need a confirmed diagnosis.

What They're Measuring

The primary outcome measures how Ianalumab affects the ESSDAI score, which helps track disease activity. The specific primary outcome measures are: Change from baseline in EULAR Sjogren Syndrome Disease Activity Index (ESSDAI) score at Week 48 as compared to placebo (48 weeks). These endpoints are how researchers determine whether the treatment is effective and will form the basis of any future regulatory submissions.

About This Phase

This trial is in Phase 3, the final and most rigorous stage before seeking FDA approval. Phase 3 trials involve 300-3,000+ patients across multiple sites and compare the new treatment directly against the current standard of care. These pivotal trials generate the evidence needed for regulatory review. About 58% of Phase 3 drugs receive FDA approval. Successful Phase 3 results typically lead to a New Drug Application submission.

Why This Trial Matters

This trial is important as it aims to fill a treatment gap for those with active Sjogren's Syndrome. As a Phase 3 trial, positive results could directly lead to FDA approval, making this treatment available to the broader patient population. This research targets Sjogren Syndrome, where improved treatment options are needed.

Investor Insight

The market for Sjogren's Syndrome treatments is growing, with limited approved options, making this trial potentially significant. Phase 3 trials have approximately a 50-60% chance of gaining FDA approval if they reach this stage.

Is This Trial Right for Me?

Ask your doctor if you meet the age and health requirements. Participation involves regular check-ups and taking Ianalumab or a placebo for 48 weeks. The trial is being conducted at 20 sites. Always discuss clinical trial participation with your healthcare provider before making any decisions. This information is for educational purposes only and is not medical advice.

AI-generated analysis for educational purposes only. This is not medical advice. Discuss clinical trial participation with your doctor. Data sourced from ClinicalTrials.gov.

Study Design

• Study Type: INTERVENTIONAL
• Allocation: RANDOMIZED
• Model: PARALLEL
• Masking: QUADRUPLE
• Enrollment: 276 participants

Interventions

• BIOLOGICAL: VAY736 — ianalumab s.c.
• OTHER: Placebo — placebo s.c.

Primary Outcomes

• Change from baseline in EULAR Sjogren Syndrome Disease Activity Index (ESSDAI) score at Week 48 as compared to placebo (48 weeks)

Secondary Outcomes

• Achieving ESSDAI response at Week 48 (48 weeks)
• Achieving ESSDAI score <5 at Week 48 (48 weeks)
• Achieving ESSDAI response at Week 24 (24 weeks)
• Achieving SSSD response at Week 48 (48 weeks)
• Change from baseline in stimulated whole salivary flow rate at Week 48 (48 weeks)

Full Eligibility Criteria

Inclusion Criteria:

* Signed informed consent must be obtained prior to participation in the study
* Women and men ≥ 18 years of age
* Classification of Sjögren's syndrome according to the ACR/EULAR 2016 criteria
* Time since diagnosis of Sjögren's of ≤ 7.5 years at screening
* Positive anti-Ro/SSA antibody at screening

  * Patients negative for anti-Ro/SSA antibody are eligible, if they have a positive salivary gland biopsy confirmed by central expert review
  * Enrollment of anti-Ro/SSA-negative patients will be limited up to ≤10% of the study population
* Screening ESSDAI score of ≥ 5 within the following 8 domains: constitutional, lymphadenopathy, glandular, articular, cutaneous, renal, hematological and biologic.
* Stimulated whole salivary flow (sSF) rate of ≥ 0.05 mL/min at screening
* Ability to communicate well with the Investigator, understand and agree to comply with the requirements of the study
* Patients taking hydroxychloroquine (≤ 400 mg/day), methotrexate (≤ 25 mg/week) or azathioprine (≤ 150 mg/day) alone or in combination, are allowed to continue their medication, and must have been on a stable dose for at least 30 days prior to randomization.
* Patients taking systemic corticosteroids have to be on a stable dose of ≤ 10 mg/day predniso(lo)ne or equivalent for at least 30 days before randomization.
* Patients taking

  * disease-modifying antirheumatic drugs (DMARDs) other than specifically allowed by protocol
  * the following Traditional Chinese Medicines: Total glucoside of peony (TGP) or Tripterium glycosides (TG) must discontinue these medications at least 30 days prior to randomization, except for leflunomide, which has to be discontinued for 8 weeks prior to randomization unless a cholestyramine wash-out has been performed.

Exclusion Criteria:

* Presence of another autoimmune rheumatic disease that is active and constitutes the principal illness
* Use of other investigational drugs within 5 half-lives of enrollment, or within 30 days or until the expected pharmacodynamic effect has returned to baseline, whichever is longer3. Prior treatment with ianalumab
* Prior use of a B-cell depleting therapy other than ianalumab within 36 weeks prior to randomization or as long as B-cell count is less than the lower limit of normal or baseline value prior to receipt of previous B cell-depleting therapy (whichever is lower)
* Prior treatment with any of the following:

  1. Within 24 weeks prior to randomization: iscalimab (anti CD-40 mAb), belimumab , abatacept, anti-tumor necrosis factor alpha biologic agents, immunoglobulins plasmapheresis;
  2. Within 12 weeks prior to randomization: i.v. or oral cyclophosphamide and mycophenolate mofetil, i.v. or oral cyclosporine A or any other immunosuppressants (e.g., JAK inhibitors or other kinase inhibitors) unless explicitly allowed by protocol
* Use of corticosteroids (predniso(lo)ne or equivalent corticosteroid) at dose \>10 mg/day
* Any one of the following laboratory values at screening:

  * Hemoglobin levels \< 8.0 g/dL
  * White blood cells (WBC) count \< 2.0 x 10E3/µL
  * Platelet count \< 80 x 10E3/µL
  * Absolute neutrophil count (ANC) \< 0.8 x 10E3/µL
* Active viral, bacterial or other infections requiring systemic treatment at the time of screening or randomization, or history of recurrent clinically significant infection or of recurrent bacterial infections with encapsulated organisms
* History of hypersensitivity to any of the study drugs or its excipients or to drugs of similar chemical classes (e.g., mAb of IgG1 class) or to any of the constituents of the study drug formulation (sucrose, L-histidine hydrochloride/ L-histidine, polysorbate 20)
* History of major organ, hematopoietic stem cell or bone marrow transplant
* Required regular use of medications known to cause dry mouth/eyes as a regular and major side effect, and which have not been on a stable dose for at least 30 days prior to Screening, or any anticipated change in the treatment regimen during the course of the study
* Use of topical ocular prescription medications (excluding artificial tears, gels, lubricants) that have not been on a stable dose for at least 90 days prior to randomization, or any anticipated change in the treatment regimen during the course of the study
* Receipt of live/attenuated vaccine within a 4-week period prior to randomization
* History of primary or secondary immunodeficiency, including a positive human immunodeficiency virus (HIV) test result
* History of malignancy of any organ system (other than localized basal cell carcinoma of the skin or in situ cervical cancer or Sjögren's related lymphoma), treated or untreated, within the past 5 years, regardless of whether there is evidence of local recurrence or metastases.
* History of sarcoidosis
* Any surgical, medical (e.g., uncontrolled hypertension, heart failure or diabetes mellitus), psychiatric or additional physical condition that the Investigator feels may jeopardize the patient in case

Trial Locations

• Medvin Clinical Research, Van Nuys, California, United States
• West Broward Rheumatology Associates Inc, Tamarac, Florida, United States
• Indiana Univ School of Dentistry, Indianapolis, Indiana, United States
• Ochsner Health System, Baton Rouge, Louisiana, United States
• The John Hopkins Jerome L Greene Sjogren, Baltimore, Maryland, United States
• Winthrop University Hospital, Mineola, New York, United States
• Carolina Arthritis Associates, Wilmington, North Carolina, United States
• STAT Research Inc, Dayton, Ohio, United States
• Altoona Center for Clin Res, Duncansville, Pennsylvania, United States
• Prisma Health, Columbia, South Carolina, United States
• ...and 10 more locations

Frequently Asked Questions

Related Conditions

• Related conditions include autoimmune diseases like lupus and rheumatoid arthritis.
• Therapeutic areas include rheumatology and immunology.

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