Ianalumab for Systemic Sclerosis

Recruiting at 33 trial locations

Overseen By Novartis Pharmaceuticals

Age: 18+

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 2

Recruiting

Sponsor: Novartis Pharmaceuticals

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

Disqualifiers: Rheumatic disease, Pulmonary disease, others

Prior Safety Data

Trial Summary

What is the purpose of this trial?

The purpose of this study is to evaluate efficacy, safety and tolerability of s.c. ianalumab administered in participants with diffuse cutaneous systemic sclerosis relative to placebo

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.

What makes the drug Ianalumab unique for treating systemic sclerosis?

Ianalumab is unique because it targets specific immune system components, potentially offering a new approach for systemic sclerosis, a condition with limited effective treatments. Unlike other drugs, it may work by modulating the immune response, which is a novel mechanism for this disease. ^{1 2 3 4 5}

Research Team

Novartis Pharmaceuticals

Principal Investigator

Novartis Pharmaceuticals

Eligibility Criteria

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.

I am between 18 and 70 years old.

Trial Timeline

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

Treatment Details

Interventions

• Ianalumab (Monoclonal Antibodies)

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.

Participant Groups

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

Findings from Research

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 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 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]

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]

References

1. England 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). [2019]

2. England pubmed.ncbi.nlm.nih.gov

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

3. Canada 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. [2022]

4. United States pubmed.ncbi.nlm.nih.gov

Treatment of systemic sclerosis with recombinant interferon-gamma. A phase I/II clinical trial. [2019]

5. England pubmed.ncbi.nlm.nih.gov

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