Trial of Efficacy and Safety of NS-229 Versus Placebo in Patients With Eosinophilic Granulomatosis With Polyangiitis

A Phase 2, Double-blind, Randomized, Placebo-controlled Study to Investigate the Efficacy and Safety of NS-229 in the Treatment of Eosinophilic Granulomatosis With Polyangiitis

This study will enroll male and female subjects who are 18 years of age or older with Eosinophilic Granulomatosis With Polyangiitis.

Study Overview

• Status: Active, not recruiting
• Conditions: Churg-Strauss Syndrome; Eosinophilic Granulomatosis With Polyangiitis
• Intervention / Treatment: Drug: Placebo; Drug: NS-229

Detailed Description

The purpose of this randomized, double-blind study is to investigate the efficacy and safety of NS229 compared with placebo over a 28-week study treatment period in subjects with Eosinophilic Granulomatosis with Polyangiitis (EGPA) receiving background corticosteroid therapy with or without Mepolizumab/Benralizumab therapy. During the treatment period corticosteroid dose will be tapered.

The key outcomes in the study focus on evaluation of clinical remission, defined as Birmingham Vasculitis Activity Score (BVAS)=0 with a corticosteroid dose of <=4 mg/day prednisolone/prednisone.

• Study Type: Interventional
• Enrollment (Estimated): 45
• Phase: Phase 2

Contacts and Locations

Study Locations

• Canada
  • Alberta
    • Edmonton, Alberta, Canada, T6G 2B7
      • University of Alberta
  • Ontario
    • Hamilton, Ontario, Canada, L8N4A6
      • St Joseph's Healthcare Hamilton
    • Toronto, Ontario, Canada, M5G 1X5
      • University of Toronto
• France
  • Nice, France, 06202
    • CHU Nice
  • Paris, France, 75014
    • Hôpital Cochin
  • Toulouse, France, 31059
    • CHU Rangueil
• Germany
  • Studienzentrale
    • Kirchheim unter Teck, Studienzentrale, Germany, 73230
      • medius Kliniken gGmbH
• Italy
  • Milan, Italy, 20145
    • Istituto Auxologico Italiano IRCCS
  • Roma, Italy, 00128
    • Fondazione Policlinico Universitario Campus Bio-Medico
  • Trento, Italy, 38122
    • Azienda Provinciale per i Servizi Sanitari Provincia Autonoma Trento
  • Verona, Italy, 37126
    • Azienda Ospedaliera Universitaria Integrata Verona
• Japan
  • Chiba
    • Chuo-ku, Chiba-shi, Chiba, Japan, 260-8677
      • Chiba University Hospital
  • Fukuoka
    • Kitakyushu, Fukuoka, Japan, 807-8555
      • Hospital of the University of Occupational and Environmental Health, Japan
  • Hokkaido
    • Sapporo, Hokkaido, Japan, 060-8648
      • Hokkaido University Hospital
  • Kanagawa
    • Sagamihara, Kanagawa, Japan, 252-0392
      • NHO Sagamihara National Hospital
    • Yokohama, Kanagawa, Japan, 245-8575
      • National Hospital Organization Yokohama Medical Center
  • Miyagi
    • Sendai, Miyagi, Japan, 980-8574
      • Tohoku University Hospital
  • Osaka
    • Habikino, Osaka, Japan, 583-8588
      • Osaka Habikino Medical Center
  • Saitama
    • Kawagoe, Saitama, Japan, 350-8550
      • Saitama Medical Center
  • Tokyo
    • Bunkyo-ku, Tokyo, Japan, 113-8431
      • Juntendo University Hospital
    • Mitaka, Tokyo, Japan, 181-8611
      • Kyorin University Hospital
    • Ōta-ku, Tokyo, Japan, 143-8541
      • Toho University Omori Medical Center
• Spain
  • Pamplona, Spain, 31008
    • Complejo Hospitalario de Navarra
  • A Coruna
    • Santiago de Compostela, A Coruna, Spain, 15706
      • Complejo Hospitalario Universitario de Santiago de Compostela
• United Kingdom
  • Cambridge, United Kingdom, Cb2 2qq
    • Addenbrookes Hospital
  • Reading, United Kingdom, RG1 5AN
    • Royal Berkshire NHS Foundation Trust
  • Birmingham
    • Edgbaston, Birmingham, United Kingdom, B15 2GW
      • Queen Elizabeth Hospital Birmingham
• United States
  • Colorado
    • Denver, Colorado, United States, 80206
      • National Jewish Health
  • Minnesota
    • Rochester, Minnesota, United States, 55905
      • Mayo Clinic
  • Ohio
    • Cleveland, Ohio, United States, 44195
      • Cleveland Clinic
    • Columbus, Ohio, United States, 43210
      • The Ohio State University Wexner Medical Center
  • Pennsylvania
    • Philadelphia, Pennsylvania, United States, 19104
      • University of Pennsylvania
  • South Carolina
    • Charleston, South Carolina, United States, 29425
      • Medical University of South Carolina
  • Tennessee
    • Nashville, Tennessee, United States, 37232
      • Vanderbilt University Medical Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Ability to provide written informed consent prior to participation in the study.
• Male or female subjects aged ≥18 years at the time the informed consent form is signed.
• Diagnosis of EGPA: Subjects who have been diagnosed with EGPA based on the history or presence of eosinophilia plus at least a history or presence of 2 of additional features of EGPA.
• Subjects receive background OGC dose of ≥7.5 mg/day with or without stable treatment with Mepolizumab/Benralizumab.
• Use of adequate contraception.
• Other inclusion criteria may apply.

Exclusion Criteria:

• Current diagnosis of either granulomatosis with polyangiitis or microscopic polyangiitis
• Imminently life-threatening EGPA at the time of screening.
• History or presence of any form of cancer within 5 years prior to screening.
• Serious liver, renal, blood, or psychiatric disease
• Severe or clinically significant cardiovascular disease uncontrolled with standard treatment
• Active systemic infections (including TB, pneumonia, Pneumocystis pneumonia, sepsis, and opportunistic infections)
• Parasitic infection: Subjects with a known parasitic infestation within 6 months prior to screening.
• HIV positive status
• Active hepatitis due to hepatitis B virus or hepatitis C virus
• Known history or presence of venous thromboembolism/venous thrombotic events (deep vein thrombosis and/or pulmonary embolus)

• laboratory parameter exclusions:

  1. Estimated glomerular filtration rate of <30 mL/min/1.73 m2 by Chronic Kidney Disease Epidemiology Collaboration equations
  2. WBC count <4 × 109/L
  3. Absolute lymphocyte count <500 cells/mm3
  4. Absolute neutrophil count <1000 cells/mm3
  5. Platelet count <120,000/mm3
  6. Hemoglobin <8 g/dL (<80 g/L)
• Subjects who are pregnant, breastfeeding, or planning to become pregnant during the time of study participation
• History of clinically significant drug or alcohol abuse within the last 6 months
• Other exclusion criteria may apply.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Triple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: NS-229; Self-administer NS-229 in consecutive 28 weeks.	Drug: NS-229; Experimental
Placebo Comparator: Placebo; Self-administer matching placebo in consecutive 28 weeks.	Drug: Placebo; Placebo comparator

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
The proportion of subjects in remission [OGC 4.0]	The proportion of subjects in remission (oral glucocorticoid [OGC] 4.0) at Week 28 of the study treatment period. Definition of remission (OGC 4.0): BVAS of 0 AND OGC dose of prednisolone/prednisone ≤4 mg/day	From Baseline to week 28

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
The proportion of subjects in remission [OGC 7.5]	The proportion of subjects in remission (OGC 7.5) at Week 28 of the study treatment period Definition of remission (OGC 7.5): BVAS of 0 AND OGC dose of prednisolone/prednisone ≤7.5 mg/day	From Baseline to week 28
Time to first relapse of EGPA	Relapse of EGPA will be defined as active disease since the last visit after remission (OGC 4.0) was achieved, characterized by: Active vasculitis (BVAS of >0); OR; Signs and/or symptoms of active asthma with a corresponding worsening in answers on the 6-item Asthma Control Questionnaire (compared with the most recent previous results); OR; Active nasal and/or sinus disease (attributable to EGPA) with a corresponding worsening in at least 1 of the answers on the sinonasal symptom questionnaire (compared with the most recent previous assessment).	Up to Week 28
Time to first worsening of EGPA	Worsening of EGPA will be defined as worsening of active disease since the last visit, characterized by: Active vasculitis (BVAS >0) and the score greater than the previous visit; OR; Signs and/or symptoms of active asthma with a corresponding worsening in answers on the 6-item Asthma Control Questionnaire (compared to the most recent previous score); OR; Active nasal and/or sinus disease (attributable to EGPA) with a corresponding worsening in at least 1 of the answers on the sinonasal symptom questionnaire (compared to the most recent previous assessment).	Up to Week 28

Collaborators and Investigators

• Sponsor: NS Pharma, Inc.
• Collaborators: Nippon Shinyaku Co., Ltd.

Study record dates

Study Major Dates

• Study Start (Actual): December 20, 2023
• Primary Completion (Estimated): December 1, 2026
• Study Completion (Estimated): December 1, 2026

Study Registration Dates

• First Submitted: September 14, 2023
• First Submitted That Met QC Criteria: September 14, 2023
• First Posted (Actual): September 21, 2023

Study Record Updates

• Last Update Posted (Actual): May 26, 2026
• Last Update Submitted That Met QC Criteria: May 22, 2026
• Last Verified: April 1, 2026

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Vascular Diseases; Cardiovascular Diseases; Autoimmune Diseases; Immune System Diseases; Skin Diseases; Lymphatic Diseases; Lymphoproliferative Disorders; Skin Diseases, Vascular; Vasculitis; Anti-Neutrophil Cytoplasmic Antibody-Associated Vasculitis; Granuloma; Systemic Vasculitis; Skin and Connective Tissue Diseases; Hemic and Lymphatic Diseases; Churg-Strauss Syndrome
• Other Study ID Numbers: NS229-P2-01

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO
• IPD Plan Description: Submission to the FDA

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No