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

• ClinicalTrials.gov Identifier: NCT06046222
• Recruitment Status: Recruiting
• First Posted: September 21, 2023
• Last Update Posted: April 23, 2024
• Sponsor: NS Pharma, Inc.
• Collaborator: Nippon Shinyaku Co., Ltd.
• Information provided by (Responsible Party): NS Pharma, Inc.

Tracking Information

• First Submitted Date: September 14, 2023
• First Posted Date: September 21, 2023
• Last Update Posted Date: April 23, 2024
• Actual Study Start Date: December 20, 2023
• Estimated Primary Completion Date: April 2025 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: September 14, 2023)

The proportion of subjects in remission [OGC 4.0] [ Time Frame: From Baseline to week 28 ]

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

• Original Primary Outcome Measures: Same as current

Current Secondary Outcome Measures (submitted: September 14, 2023)

• The proportion of subjects in remission [OGC 7.5] [ Time Frame: From Baseline to week 28 ]
  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
• Time to first relapse of EGPA [ Time Frame: Up to Week 28 ]
  Relapse of EGPA will be defined as active disease since the last visit after remission (OGC 4.0) was achieved, characterized by:

  1. Active vasculitis (BVAS of >0); OR
  2. 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
  3. 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).
• Time to first worsening of EGPA [ Time Frame: Up to Week 28 ]
  Worsening of EGPA will be defined as worsening of active disease since the last visit, characterized by:

  1. Active vasculitis (BVAS >0) and the score greater than the previous visit; OR
  2. 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
  3. 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).

• Original Secondary Outcome Measures: Same as current
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: Trial of Efficacy and Safety of NS-229 Versus Placebo in Patients With Eosinophilic Granulomatosis With Polyangiitis
• Official Title: 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
• Brief Summary: This study will enroll male and female subjects who are 18 years of age or older with Eosinophilic Granulomatosis With Polyangiitis.

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 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
• Study Phase: Phase 2
• Study Design: Allocation: Randomized; Intervention Model: Parallel Assignment; Masking: Triple (Participant, Care Provider, Investigator); Primary Purpose: Treatment

Condition

• Eosinophilic Granulomatosis With Polyangiitis
• Churg-Strauss Syndrome

Intervention

• Drug: NS-229
  Experimental
• Drug: Placebo
  Placebo comparator

Study Arms

• Experimental: NS-229
  Self-administer NS-229 in consecutive 28 weeks.
  Intervention: Drug: NS-229
• Placebo Comparator: Placebo
  Self-administer matching placebo in consecutive 28 weeks.
  Intervention: Drug: Placebo

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Recruiting
• Estimated Enrollment (submitted: September 14, 2023): 45
• Original Estimated Enrollment: Same as current
• Estimated Study Completion Date: May 2025
• Estimated Primary Completion Date: April 2025 (Final data collection date for primary outcome measure)

Eligibility Criteria

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.
• 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 <500 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.

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 18 Years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Contacts

Contact: NS Pharma, Inc.; 1-866-677-4276; trialinfo@nspharma.com

• Listed Location Countries: Japan, United States

Administrative Information

• NCT Number: NCT06046222
• Other Study ID Numbers: NS229-P2-01
• Has Data Monitoring Committee: Yes

U.S. FDA-regulated Product

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No

IPD Sharing Statement

• Plan to Share IPD: No
• Plan Description: Submission to the FDA

• Current Responsible Party: NS Pharma, Inc.
• Original Responsible Party: Same as current
• Current Study Sponsor: NS Pharma, Inc.
• Original Study Sponsor: Same as current
• Collaborators: Nippon Shinyaku Co., Ltd.
• Investigators: Not Provided
• PRS Account: NS Pharma, Inc.
• Verification Date: April 2024