Ianalumab for Systemic Sclerosis

Recruiting at 123 trial locations

Overseen By Novartis Pharmaceuticals

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Novartis Pharmaceuticals

Must not be taking: Cyclophosphamide, Biologics, Anti-fibrotics, others

Disqualifiers: Rheumatic disease, Pulmonary disease, others

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

200 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment called ianalumab for individuals with diffuse cutaneous systemic sclerosis, a condition that causes hardening and tightening of the skin. The goal is to determine if ianalumab is safe and effective compared to a placebo, which contains no active treatment. Participants will receive the treatment through a subcutaneous injection. The study seeks individuals diagnosed with systemic sclerosis who have experienced symptoms like skin tightening or lung issues for less than five years. As a Phase 2 trial, this research focuses on assessing the treatment's effectiveness in an initial, smaller group of participants.

Will I have to stop taking my current medications?

The trial requires stopping certain medications before participating. You must not have taken cyclophosphamide within 12 weeks, B-cell depleting therapies within 36 weeks, or biologic agents within 12 weeks before starting the trial. Additionally, anti-fibrotic agents should not be used in the 4 weeks prior to the trial.

Is there any evidence suggesting that Ianalumab is likely to be safe for humans?

Research has shown that ianalumab has been tested in patients with conditions like Sjögren's disease. Studies found it well-tolerated during trials. For example, one study found that ianalumab was generally safe at doses up to 300 mg over a year.

No major safety issues emerged in these trials. This suggests that ianalumab could be safe for people, but it's important to remember that the current trial is still in an early stage. Researchers are still closely monitoring safety and gathering more information. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for systemic sclerosis?

Ianalumab is unique because it targets B cells, a type of immune cell, by blocking the BAFF receptor. Unlike standard treatments for systemic sclerosis, which often focus on managing symptoms with immunosuppressants like methotrexate or mycophenolate mofetil, Ianalumab aims to directly modulate the immune system's activity. Researchers are excited about Ianalumab because it has the potential to more precisely target the underlying immune dysfunctions in systemic sclerosis, offering a more tailored approach. Additionally, its subcutaneous delivery method can provide a more convenient and potentially less invasive option for patients.

What evidence suggests that Ianalumab might be an effective treatment for systemic sclerosis?

Research shows that ianalumab, which participants in this trial may receive, can help reduce symptoms in people with autoimmune diseases. In studies with patients who have Sjögren's syndrome, both doctors and patients observed improvements. Ianalumab effectively eased symptoms and met key safety and efficacy goals. Although systemic sclerosis is a different condition, ianalumab's method of targeting specific parts of the immune system suggests it might help manage this disease as well. Early signs from similar conditions offer hope for its use in treating systemic sclerosis. ^{3 4 6 7 8}

Who Is on the Research Team?

Novartis Pharmaceuticals

Principal Investigator

Novartis Pharmaceuticals

Are You a Good Fit for This Trial?

This trial is for adults aged 18-70 with diffuse cutaneous systemic sclerosis, a type of scleroderma. Participants must have interstitial lung disease, ATA autoantibody positivity, and a specific disease duration and severity as measured by skin thickness score (mRSS). They should not have had the condition for more than 60 months.

Inclusion Criteria

My skin thickness score is between 15 and 45.

My condition started less than 18 months ago, not counting Raynaud's phenomenon.

My symptoms started less than 5 years ago, not counting Raynaud's.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

6 weeks

Treatment Period 1

Participants receive Ianalumab or placebo subcutaneous injections

52 weeks

Regular visits as defined in the protocol

Treatment Period 2 (Open-label)

Participants receive open-label Ianalumab subcutaneous injections

52 weeks

Regular visits as defined in the protocol

Post-treatment Follow-up

Participants are monitored for safety and effectiveness after treatment

20 weeks to 2 years

What Are the Treatments Tested in This Trial?

Interventions

• Ianalumab

Trial Overview The study tests Ianalumab's effectiveness compared to a placebo in treating systemic sclerosis. It measures how well it works, its safety, and if there are any negative effects when given subcutaneously (under the skin) to participants.

How Is the Trial Designed?

Group I: VAY736 (Ianalumab) Experimental Treatment 1 Intervention

Treatment Period 1: Ianalumab subcutaneous (s.c.) injection as defined in the protocol Treatment Period 2: Open-label (OL) Ianalumab subcutaneous (s.c.) injection as defined in the protocol

Group II: Placebo Placebo Group 2 Interventions

Treatment Period 1: Placebo to Ianalumab subcutaneous (s.c.) injection as defined in the protocol Treatment Period 2: Open-label (OL) Ianalumab subcutaneous (s.c.) injection as defined in the protocol

Who Is Running the Clinical Trial?

Novartis Pharmaceuticals

Lead Sponsor

Trials

2,963

Recruited

4,275,000+

Founded

1996

Headquarters

Basel, Switzerland

Known For

Precision medicine

Top Products

Gleevec, Cosentyx, Entresto, Kisqali

Dr. Vas Narasimhan

Novartis Pharmaceuticals

Chief Executive Officer since 2018

MD from Harvard Medical School

Dr. Shreeram Aradhye

Novartis Pharmaceuticals

Chief Medical Officer since 2021

MD

Published Research Related to This Trial

The review highlights the importance of novel biomarkers in systemic sclerosis (SSc) for improving early diagnosis, classification, and management of the disease, which currently lacks effective therapies.

Emerging biomarkers, including SSc-specific autoantibodies and genome-wide expression analysis, could significantly enhance the prediction of disease progression and therapeutic responses, potentially transforming clinical trial designs for new treatments.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Current status of systemic sclerosis biomarkers: applications for diagnosis, management and drug development. Castelino, FV., Varga, J.[2021]

Recent advancements have led to the approval of the first drug for treating interstitial lung disease related to systemic sclerosis (SSc), highlighting a significant step forward in targeted therapies for this condition.

A systematic review of 21 studies, including 15 phase 1/2 trials and 2 phase 3 trials, indicates that drugs like nintedanib and tocilizumab show promise in treating SSc, particularly in patients with early diffuse SSc and interstitial lung disease.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

An update on targeted therapies in systemic sclerosis based on a systematic review from the last 3 years. Campochiaro, C., Allanore, Y.[2023]

In a study of 21 patients with difficult-to-treat systemic sclerosis, tocilizumab (TCZ) significantly improved skin and joint symptoms after one year, with a notable reduction in the modified Rodnan skin score and Disease Activity Score in 28 joints.

TCZ was generally well tolerated, with most patients experiencing sustained benefits over a follow-up period of up to 2.2 years, indicating its potential as an effective treatment option for systemic sclerosis despite some patients experiencing complications.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Anti-interleukin 6 Therapy Effect for Refractory Joint and Skin Involvement in Systemic Sclerosis: A Real-world, Single-center Experience. Panopoulos, ST., Tektonidou, MG., Bournia, VK., et al.[2022]

In a phase II study involving patients with systemic sclerosis (SSc), tocilizumab treatment for 48 weeks led to a significant improvement in skin scores and stabilization of lung function, as measured by the modified Rodnan Skin Score (mRSS) and forced vital capacity (%pFVC).

While tocilizumab was associated with an increased rate of serious infections compared to placebo, no new safety concerns were identified, suggesting that while the drug is effective, monitoring for infections is important during treatment.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Safety and efficacy of subcutaneous tocilizumab in systemic sclerosis: results from the open-label period of a phase II randomised controlled trial (faSScinate). Khanna, D., Denton, CP., Lin, CJF., et al.[2019]

In an 18-week trial involving 18 patients with rapidly progressive systemic sclerosis (SSc), interferon-gamma (IFN gamma) therapy significantly reduced skin thickness and affected body surface area, indicating its potential efficacy in treating skin involvement in SSc.

While most patients showed improvement, three patients experienced renal crises, suggesting that further investigation is needed to assess the safety of IFN gamma in this population, and a placebo-controlled study is recommended to confirm these findings.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Treatment of systemic sclerosis with recombinant interferon-gamma. A phase I/II clinical trial. Freundlich, B., Jimenez, SA., Steen, VD., et al.[2019]

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT06470048

NCT06470048 | A Clinical Study to Evaluate Ianalumab in ... Study Overview. Brief Summary. The purpose of this study is to evaluate efficacy, safety and tolerability of s.c. ianalumab administered in participants with ...

2. novartis.com novartis.com/us-en/news/media-releases/novartis-ianalumab-first-drug-reduce-disease-activity-and-patient-burden-sjogrens-disease-phase-iii-trials

Novartis ianalumab first drug to reduce disease activity and ... Ianalumab 300 mg monthly numerically improved physician- and patient-reported outcomes1. Nominal significance was observed in NEPTUNUS-1 and the ...

3. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/34861168/

Safety and efficacy of subcutaneous ianalumab (VAY736) ... This dose-finding trial aimed to assess the safety and efficacy of different subcutaneous doses of ianalumab in patients with moderate to severe primary Sjö ...

4. clinicaltrials.eu clinicaltrials.eu/inn/ianalumab/

Ianalumab: A Promising Treatment for Autoimmune Diseases This article summarizes key information about ongoing clinical trials evaluating ianalumab for conditions like systemic lupus erythematosus, Sjögren's syndrome, ...

5. neurologylive.com neurologylive.com/view/ianalumab-meets-primary-end-points-phase-3-neptunus-trials-sjogren-disease

Ianalumab Meets Primary End Points in Phase 3 ... Ianalumab showed significant efficacy and safety in reducing Sjögren's disease activity in phase 3 trials, meeting primary endpoints. The trials ...

6. clinicaltrials.gov clinicaltrials.gov/study/NCT06470048

NCT06470048 | A Clinical Study to Evaluate Ianalumab in ... Also called a data safety and monitoring board, or DSMB. ... Safety and Tolerability of Ianalumab in Participants With Diffuse Cutaneous Systemic Sclerosis.

7. novartis.com novartis.com/clinicaltrials/study/nct06470048

A Clinical Study to Evaluate Ianalumab in Participants With ... The purpose of this study is to evaluate efficacy, safety and tolerability of sc ianalumab administered in participants with diffuse cutaneous systemic ...

8. ard.bmj.com ard.bmj.com/content/80/Suppl_1/593

POS0692 IANALUMAB (VAY736) SAFETY AND ... Ianalumab 300 mg was well tolerated up to 52 Wks. Exploratory efficacy showed that continuous dosing of 300 mg sc q4w provided sustained clinical benefit.