A Randomized, Double-blind 2-arm, Followed by an Open-label 1-arm, NEPTUNUS Extension Study to Assess the Long-term Safety and Efficacy of Ianalumab in Patients With Sjögren's Syndrome

Plain English Summary

A Randomized, Double-blind 2-arm, Followed by an Open-label 1-arm, NEPTUNUS Extension Study to Assess the Long-term Safety and Efficacy of Ianalumab in Patients With Sjogrens Syndrome. is a Phase 3 clinical trial sponsored by Novartis Pharmaceuticals studying Sjogrens Syndrome. Tests Ianalumab's long-term safety and effectiveness in patients with Sjögren's Syndrome who have completed previous NEPTUNUS studies. For patients who have completed NEPTUNUS core studies and are expected to benefit from Ianalumab. Participation involves monthly injections of Ianalumab or placebo for up to 4 years, with regular check-ups. Alternative treatments include other medications or no treatment, depending on individual health. The trial aims to enroll 612 participants.

Official Summary

The purpose of this study is to measure the long-term safety and tolerability of ianalumab in participants with Sjögren's syndrome who have previously completed treatment from one of two NEPTUNUS 1 year core studies (CVAY736A2301 \[NCT05350072\] or CVAY736A2302 \[NCT05349214\]).

Who Can Participate

Here is what you need to know about eligibility for this trial. Eligible if you have completed NEPTUNUS core studies and are expected to benefit from Ianalumab. Not eligible if you are pregnant, nursing, or planning to become pregnant, or if you are sexually active without contraception. Age: 18 years and older. Health: Must have Sjögren's Syndrome. This trial is studying Sjogrens Syndrome, so participants generally need a confirmed diagnosis.

What They're Measuring

The primary outcome measures Ianalumab's safety and effectiveness in reducing symptoms and improving quality of life for patients. The specific primary outcome measures are: Number of Treatment-emergent AEs (TEAEs)/SAEs (Week 52 to Week 464). 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 addresses the long-term safety and efficacy gap for Ianalumab in Sjögren's Syndrome patients, providing crucial data for future treatments. 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 Sjogrens Syndrome, where improved treatment options are needed.

Investor Insight

Market size is significant, with a large unmet need for effective treatments in Sjögren's Syndrome, making this trial potentially impactful. Phase 3 trials have approximately a 50-60% chance of gaining FDA approval if they reach this stage. The large enrollment target of 612 participants suggests significant investment in this program.

Is This Trial Right for Me?

Ask your doctor if you have completed NEPTUNUS core studies and are expected to benefit from Ianalumab. Participation involves monthly injections and regular check-ups. 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: 612 participants

Interventions

• DRUG: Ianalumab (VAY736) — Ianalumab 300 mg / 2 mL, PFS or AI, monthly or every 3 months, solution for injection for subcutaneous use
• OTHER: Placebo — Placebo 0 mg/2 mL PFS or AI, once monthly between doses of Ianalumab 3 monthly , solution for injection for subcutaneous Use

Primary Outcomes

• Number of Treatment-emergent AEs (TEAEs)/SAEs (Week 52 to Week 464)

Secondary Outcomes

• Percentage of participants achieving ≥ 5 points reduction from baseline in ESSDAI (Over time up to Week 360)
• Percentage of participants achieving ≥2.3 points reduction from baseline in ESSPRI among participants with baseline ESSPRI ≥3 score over time (Over time up to Week 360)
• Percentage of participants achieving ≥2.0 points reduction from baseline in SSSD among participants with baseline SSSD ≥3 (Over time up to Week 360)
• Pre-dose Ianalumab serum concentrations (pre-dose at Week 48 (from core study) and Week 64)
• On-treatment Ianalumab serum concentrations (During treatment (from Week 52 to Week 204) and follow-up (From Week 208 to Week 304))

Full Eligibility Criteria

Inclusion Criteria:

1. Signed informed consent prior to participation in the extension study.
2. Participants must have participated in either one of the two NEPTUNUS core studies, CVAY736A2301 or CVAY736A2302, and must have completed the entire treatment up to Week 48 without treatment discontinuation in core NEPTUNUS studies.
3. In the judgement of the Investigator, participants must be expected to clinically benefit from continued ianalumab therapy.

Exclusion Criteria:

1. Use of therapies excluded by the NEPTUNUS-1 and NEPTUNUS-2 study protocols (see NEPTUNUS studies protocols exclusion criteria in Section 5.2 for details).
2. Plans for administration of live vaccines during the study period.
3. Pregnant or nursing (breastfeeding) women, where pregnancy is defined as the state of a female after conception and until the termination of gestation, confirmed by a positive human Chorionic Gonadotropin (hCG) laboratory test.
4. Women of child-bearing potential (WOCBP), defined as all women physiologically capable of becoming pregnant, from menarche until becoming post-menopausal, unless they have had surgical bilateral oophorectomy (with or without hysterectomy), total hysterectomy or bilateral salpingectomy at least six weeks before taking study treatment. In the case of oophorectomy alone, the reproductive status of the woman needs to have been confirmed by follow-up hormone level assessment. Women are considered post-menopausal if they have had 12 months of natural (spontaneous) amenorrhea with an appropriate clinical profile (e.g., hormonal profile confirming menopause and/or age-appropriate history of vasomotor symptoms).

   WOCBP are excluded unless they are using highly effective methods of contraception (failure rate \< 1% per year) while taking study treatment during dosing and for 6 months after stopping of investigational drug. Highly effective contraception methods include:
   * Total abstinence (when this is in line with the preferred and usual lifestyle of the participant. Periodic abstinence (e.g., calendar, ovulation, symptothermal, post- ovulation methods) and withdrawal are not acceptable methods of contraception.
   * Bilateral tubal occlusion or bilateral tubal ligation (at least six weeks before taking study treatment).
   * Sterilization (vasectomy) of male partner (s) of the female participant at least 6 months prior to screening provided partner(s) has(have) received medical confirmation of surgical process.
   * Use of hormonal contraception methods:
   * Combined (estrogen and progestogen-containing) hormonal contraception associated with inhibition of ovulation; oral, intravaginal or transdermal.
   * Progestogen-only hormonal contraception associated with inhibition of ovulation: oral, injectable or implantable.
   * Intrauterine device (IUD) or intrauterine hormone-releasing system (IUS) In case of use of oral contraception women should have been stable on the same pill for a minimum of 3 months before taking study treatment.

   Contraception should be used in accordance with locally approved prescribing information of concomitant medications administered.

   If local regulations are more stringent than the contraception methods listed above, local regulations apply and will be described in the informed consent form (ICF).
5. United States (and other countries, if male contraception is locally required): Sexually active males, unless they agree to use barrier protection during intercourse with a woman of child-bearing potential, while taking study treatment. As condom use alone has a reported failure rate exceeding 1% per year, it is recommended that female partners of male study participants use a second method of birth control. Although ianalumab is not teratogenic and/or genotoxic, and not transferred to semen, male contraception is required, as requested by FDA.

Globally, for all sexually active males, contraception should be used in accordance with the locally approved prescribing information of concomitant medications administered.

Trial Locations

• Providence Medical Foundation, Fullerton, California, United States
• Advanced Medical Research, La Palma, California, United States
• Medvin Clinical Research, Van Nuys, California, United States
• Bay Area Arthritis And Osteoporosis, Brandon, Florida, United States
• GNP Research, Cooper City, Florida, United States
• Sarasota Arthritis Res Ctr, Sarasota, Florida, United States
• West Broward Rheumatology Associates Inc, Tamarac, Florida, United States
• Augusta University Georgia, Augusta, Georgia, United States
• North GA Rheumatology Group PC, Suwanee, Georgia, United States
• Clin Invest Specialists Inc, Orland Park, Illinois, United States
• ...and 10 more locations

Frequently Asked Questions

Related Conditions

• Sjögren's Syndrome
• Autoimmune diseases
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