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Methyl-(1-{[6-{[(1S)-1-Cyclopropylethyl]Amino}-2-(Pyrazolo[5,1-B][1,3]Thiazol-7-Yl)-Pyrimidin-4-Yl]Carbonyl}Piperidin-4-Yl)Carbamate-Mono(4-Methylbenzenesulfonate) Monohydrate

A groundbreaking clinical trial is underway to investigate the effectiveness and safety of NS-229, a new drug for treating Eosinophilic Granulomatosis with Polyangiitis (EGPA). This Phase 2 study aims to compare NS-229 with a placebo in patients diagnosed with EGPA, a rare autoimmune condition. The trial will focus on achieving remission and monitoring potential side effects, offering hope for improved treatment options for those affected by this challenging disease.

Table of Contents

What is NS-229?

NS-229 is a new medication being developed to treat a rare autoimmune condition called Eosinophilic Granulomatosis with Polyangiitis (EGPA). It is classified as a Janus kinase (JAK) 1 inhibitor, which means it works by blocking certain signals in the immune system that contribute to inflammation[1]. The active substance in NS-229 has a long scientific name: methyl-(1-{[6-{[(1S)-1-cyclopropylethyl]amino}-2-(pyrazolo[5,1-b][1,3]thiazol-7-yl)-pyrimidin-4-yl]carbonyl}piperidin-4-yl)carbamate-mono(4-methylbenzenesulfonate) monohydrate. It comes in the form of tablets that are taken orally (by mouth).

What is Eosinophilic Granulomatosis with Polyangiitis (EGPA)?

Eosinophilic Granulomatosis with Polyangiitis (EGPA) is a rare autoimmune disorder that causes inflammation in small and medium-sized blood vessels[1]. This inflammation can affect various organs in the body, including the lungs, sinuses, skin, heart, and nerves. EGPA is characterized by an increased number of eosinophils (a type of white blood cell) in the blood and tissues, which contribute to the inflammation.

Some common symptoms and features of EGPA include:

  • Asthma
  • Sinus problems
  • Skin rashes or purpura (purple spots on the skin)
  • Nerve pain or numbness
  • Lung infiltrates (abnormal substances in the lungs)
  • Heart problems
  • Kidney issues

How NS-229 Works

NS-229 is a JAK1 inhibitor, which means it targets a specific enzyme called Janus kinase 1. This enzyme plays a crucial role in the signaling pathways that lead to inflammation in the body. By inhibiting JAK1, NS-229 aims to reduce the inflammatory processes that cause damage in EGPA[1].

The goal of this treatment is to help patients achieve remission, which means a significant reduction in disease activity and symptoms, while also allowing them to reduce their use of other medications, particularly oral glucocorticoids (a type of steroid).

Clinical Trial of NS-229 for EGPA

A clinical trial is currently underway to investigate the effectiveness and safety of NS-229 in treating EGPA. This trial is known as a Phase 2, double-blind, randomized, placebo-controlled study[1]. Here’s what these terms mean:

  • Phase 2: This stage of clinical research focuses on determining the effectiveness of the drug and further evaluating its safety.
  • Double-blind: Neither the patients nor the researchers know who is receiving the actual drug and who is receiving a placebo.
  • Randomized: Participants are randomly assigned to either the NS-229 group or the placebo group.
  • Placebo-controlled: Some participants receive an inactive substance (placebo) instead of the actual drug, which helps researchers compare the effects of the drug to no treatment.

In this study, participants are divided into two groups: one group receives NS-229, while the other receives a placebo. The ratio of assignment is 2:1, meaning for every two people who receive NS-229, one person receives the placebo[1].

Potential Benefits of NS-229

The main goals of the NS-229 treatment in this clinical trial are:

  1. To achieve remission in patients with EGPA. This is defined as a significant reduction in disease activity while using a low dose of oral glucocorticoids (4.0 mg or less) by week 28 of the treatment[1].
  2. To maintain remission for a longer period and delay the time to the first relapse or worsening of EGPA[1].
  3. To potentially reduce the need for high doses of steroids, which can have significant side effects when used long-term.

Who Can Participate in the NS-229 Trial?

The clinical trial has specific criteria for who can participate. Some key inclusion criteria are:

  • Adults aged 18 years or older
  • Diagnosed with EGPA based on specific criteria, including a history of eosinophilia (high levels of eosinophils) and at least two other features of the disease[1]
  • Active disease as measured by a score of 3 or higher on the Birmingham Vasculitis Activity Score (BVAS), which is a tool used to assess disease activity in vasculitis[1]

There are also several exclusion criteria, which include certain other medical conditions, recent use of specific medications, and pregnancy or breastfeeding[1].

Safety Considerations

As with any new medication, safety is a primary concern. The clinical trial is designed to carefully monitor for any side effects or adverse events that may occur during treatment with NS-229. Some potential areas of concern that are being monitored include:

  • Infections, including tuberculosis and viral hepatitis
  • Changes in blood cell counts
  • Liver and kidney function
  • Cardiovascular health
  • Possible interactions with other medications

It’s important to note that all participants in the trial are closely monitored by healthcare professionals throughout the study period[1].

Aspect Details
Study Type Phase 2, double-blind, randomized, placebo-controlled
Condition Eosinophilic Granulomatosis with Polyangiitis (EGPA)
Intervention NS-229 (JAK1 inhibitor) vs Placebo
Primary Endpoints Proportion of subjects in remission at Week 28, Number of adverse events
Secondary Endpoints Time to first relapse, Time to first worsening of EGPA
Key Inclusion Criteria Adults ≥18 years, Diagnosed EGPA, Active disease (BVAS ≥3)
Key Exclusion Criteria Life-threatening EGPA, Recent cancer, Severe liver/kidney disease, Active infections
Treatment Duration 28 weeks
Dosing NS-229: 18.2 mg daily (1 tablet 14mg + 3 tablets 1.4mg) or Placebo: 4 tablets

FAQ

  • What is Eosinophilic Granulomatosis with Polyangiitis (EGPA)? EGPA is a rare autoimmune condition characterized by inflammation of blood vessels, typically involving the lungs, sinuses, and other organs. It is associated with high levels of eosinophils (a type of white blood cell) in the blood and tissues.
  • What is NS-229 and how does it work? NS-229 is a new drug being tested for EGPA treatment. It is a Janus kinase (JAK) 1 inhibitor, which means it works by blocking certain enzymes involved in inflammation and immune responses in the body.
  • Who is eligible to participate in this clinical trial? The trial is open to adults (18 years and older) diagnosed with EGPA who meet specific criteria, including having active disease and being on certain doses of prednisone or prednisolone. However, there are several exclusion criteria, such as having other severe medical conditions or recent use of certain medications.
  • What are the main goals of this clinical trial? The primary objectives are to determine if NS-229 is more effective than a placebo in achieving remission in EGPA patients and to assess its safety by monitoring adverse events. Secondary goals include evaluating the time to relapse or worsening of EGPA symptoms.
  • How long does the trial last and what does participation involve? The study treatment period lasts 28 weeks. Participants will be randomly assigned to receive either NS-229 or a placebo in a 2:1 ratio. They will need to take the medication orally and attend regular check-ups to monitor their condition and any potential side effects.

Ongoing Clinical Trials on Methyl-(1-{[6-{[(1S)-1-Cyclopropylethyl]Amino}-2-(Pyrazolo[5,1-B][1,3]Thiazol-7-Yl)-Pyrimidin-4-Yl]Carbonyl}Piperidin-4-Yl)Carbamate-Mono(4-Methylbenzenesulfonate) Monohydrate

  • Study on the Effectiveness and Safety of NS-229 for Patients with Eosinophilic Granulomatosis with Polyangiitis

    Recruiting

    2 1
    Investigated diseases:
    Investigated drugs:
    France Germany Italy Spain

Glossary

  • Eosinophilic Granulomatosis with Polyangiitis (EGPA): A rare autoimmune disorder characterized by inflammation of blood vessels, typically affecting the lungs, sinuses, and other organs. It is associated with high levels of eosinophils in the blood and tissues.
  • Eosinophil: A type of white blood cell that plays a role in fighting parasitic infections and is involved in allergic reactions and inflammation.
  • Remission: A state in which the signs and symptoms of a disease are reduced or disappear, often achieved through treatment.
  • Janus kinase (JAK) inhibitor: A type of medication that works by blocking specific enzymes involved in inflammation and immune responses in the body.
  • Placebo: An inactive substance or treatment used in clinical trials to compare the effects of a new drug against a control group.
  • Adverse event (AE): Any unfavorable or unintended sign, symptom, or disease that occurs during a clinical trial, which may or may not be related to the treatment being studied.
  • Oral glucocorticoid (OGC): A type of steroid medication taken by mouth to reduce inflammation and suppress the immune system, such as prednisone or prednisolone.
  • Biopsy: A medical procedure in which a small sample of tissue is removed from the body for examination under a microscope.
  • ANCA (Anti-Neutrophil Cytoplasmic Antibody): A type of antibody found in some autoimmune disorders, including certain forms of vasculitis like EGPA.
  • Vasculitis: Inflammation of blood vessels, which can affect various organs and tissues in the body.

References

  1. http://clinicaltrials.eu/trial/study-on-the-effectiveness-and-safety-of-ns-229-for-patients-with-eosinophilic-granulomatosis-with-polyangiitis/