Phase 2 Study Evaluating Rapcabtagene Autoleucel in Participants With Severe Active GPA or MPA

Phase 2

Recruiting

• Conditions: ANCA Associated Vasculitis (AAV)
• Interventions: Biological: Rapcabtagene autoleucel; Other: Active Comparator; Drug: Glucocorticoids

• Registration Number: NCT06868290
• Lead Sponsor: Novartis Pharmaceuticals

Brief Summary

The purpose of this study is to evaluate the efficacy and safety of rapcabtagene autoleucel versus comparator in participants with severe active Granulomatosis with Polyangiitis (GPA) or Microscopic Polyangiitis (MPA)

Detailed Description

This is a Phase 2, randomized, assessor-blinded active controlled study. This study comprises two cohorts:

* A lead-in cohort enrolling participants to receive rapcabtagene autoleucel

* A randomized cohort with participants receiving either rapcabtagene autoleucel or comparator.

After end of study (EOS), participants who received rapcabtagene autoleucel infusion will enter a long-term follow-up (LTFU) period lasting up to 15 years after rapcabtagene autoleucel infusion. This LTFU will be described in a separate study protocol.

Study Timeline

• Study First Submitted: 2025-03-07
• Study First Submitted QC: 2025-03-07
• Study First Posted: 2025-03-10
• Study Start: 2025-03-13
• Status Verified: 2025-05
• Last Update Submitted: 2025-05-05
• Last Update Posted: 2025-05-08
• Primary Completion: 2029-06-07
• Study Completion: 2030-05-24

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 126

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Rapcabtagene autoleucel	Rapcabtagene autoleucel	Single infusion of rapcabtagene autoleucel (YTB323) and concomitant glucocorticoids as per protocol
Rapcabtagene autoleucel	Glucocorticoids	Single infusion of rapcabtagene autoleucel (YTB323) and concomitant glucocorticoids as per protocol
Active comparator	Active Comparator	Comparator and concomitant glucocorticoids as per protocol
Active comparator	Glucocorticoids	Comparator and concomitant glucocorticoids as per protocol

Primary Outcome Measures

Name	Time	Method
Event-free survival (EFS)	From randomization until the occurrence of an EFS event, up to approx. 4 years after randomization	Event-free survival (EFS) defined as the time from Randomization to the first occurrence of as per protocol defined events.

Secondary Outcome Measures

Name	Time	Method
Percentage of patients achieving complete remission	Up to Week 13	Complete remission is defined by Birmingham Vasculitis Activity Score version 3 (BVASv3)
ANCA seronegativity and sustaining ANCA seronegativity until the analysis cutoff date	From randomization until the occurrence of an EFS event, up to approx. 4 years after randomization	ANCA seronegativity means that the patient tests negative for ANCA antibodies.
Change from baseline in estimated glomerular filtration rate (eGFR) at Week 39	Up to Week 39	Change from baseline in estimated glomerular filtration rate (eGFR) at Week 39.
Adjusted annual cumulative GC dose between Randomization and analysis cutoff date	From randomization until the occurrence of an EFS event, up to approx. 4 years after randomization	The adjusted annual cumulative glucocorticoid dose refers to the total amount of glucocorticoids administered to a patient over the course of a year, adjusted for any changes in dosage, and measured up to the defined week of treatment as per protocol.
Change from baseline in symptoms of GPA/ MPA using the AAV-PRO at Week 39	Up to Week 39	Change from baseline in symptoms of GPA/ MPA using the AAV-PRO at Week 39.
Change from baseline in Patient- Reported Outcome Measurement Information System (PROMIS)- Fatigue 7a at Week 91.	Up to Week 91	Change from baseline in PROMIS- Fatigue 7a at Week 91.

Trial Locations

Locations (2)

Oregon Health Sciences University; 🇺🇸 Portland, Oregon, United States

Novartis Investigative Site; 🇸🇬 Singapore, Singapore