TARPERPRUMIG for ANCA-associated vasculitis | Granulomatosis with polyangiitis (GPA) | Microscopic polyangiitis (MPA)

Inclusion criteria

• {"criterion_text":"- Age: Participants aged 18 to 80 years (inclusive) at the time of consent."}
• {"criterion_text":"- Type of Participant and Disease Characteristics: Newly diagnosed or relapsing ANCA-associated vasculitis, GPA and MPA subtypes consistent with the 2022 ACR/EULAR classification criteria for GPA and MPA for whom treatment with rituximab or cyclophosphamide is considered."}
• {"criterion_text":"- Positive test for antibodies to either PR3-ANCA or MPO-ANCA at Screening or in the past by a quantitative assay (for example, ELISA, bead assay, etc)."}
• {"criterion_text":"- At least one major item, or at least 3 minor items, or at least 2 renal items in the BVAS."}
• {"criterion_text":"- Estimated glomerular filtration rate (eGFR) ≥ 15 mL/min/1.73 m2 (calculated using the 2021 CKD EPI equation without race coefficient) at Screening (Section 8.2.6)."}
• {"criterion_text":"- Weight: There is no weight restriction in this study."}
• {"criterion_text":"- All participants must agree to follow protocol-specified contraception guidance as outlined in the study protocol. Contraceptive use must be consistent with local regulations regarding the methods of contraception for those participating in clinical studies. a.\tMale participants: Participants are eligible to participate if they agree to the following during the study intervention treatment period and for at least [CCI] after the last dose of study intervention: •\tRefrain from donating sperm •\tBe abstinent from intercourse where pregnancy can occur (abstinent on a long term and persistent basis) and agree to remain abstinent OR Must agree to use contraception/barrier as detailed below \tAgree to use an external condom and should also be advised of the benefit for a partner of CBP to use a highly effective method of contraception as a condom may break or leak when having sexual intercourse with a partner able to give birth who is not currently pregnant \tAgree to use an external condom when engaging in any activity that allows for passage of ejaculate to another person. b.\tFemale participants: A participant is eligible to participate if not pregnant or breastfeeding, and one of the following conditions applies: •\tIs a woman of NCBP as defined in Section 10.5 Contraceptive and Barrier Guidance. •\tIs of CBP and using a contraceptive method that is highly effective (with a failure rate of < 1% per year), preferably with low user dependency, as described in Section 10.5 Contraceptive and Barrier Guidance during the study intervention treatment period and for at least [CCI] after the last dose of study intervention and agrees not to donate eggs (ova, oocytes) for the purpose of reproduction during this period. The Investigator should evaluate the potential for contraceptive method failure (eg, noncompliance, recently initiated) in relationship to the first dose of study intervention. •\tA CBP participant must have a negative highly sensitive pregnancy test (urine) as required by local regulations) within 24 hours before the first dose of study intervention, see Section 8.3.6 Pregnancy Testing. \tIf a urine test cannot be confirmed as negative (eg, an ambiguous result), a serum pregnancy test is required. In such cases, the participant must be excluded from participation if the serum pregnancy result is positive. •\tAdditional requirements for pregnancy testing during and after study intervention are located in Section 8.3.6 Pregnancy Testing. •\tThe Investigator is responsible for review of medical history, menstrual history, and recent sexual activity to decrease the risk for inclusion of a participant with an early undetected pregnancy."}
• {"criterion_text":"- Informed Consent: Willing and able to give written informed consent and to comply with the requirements of the study protocol."}

Exclusion criteria

• {"criterion_text":"- Medical Conditions: Other systemic diseases that, in the judgment of the Investigator, constitute the primary illness, including but not limited to: eosinophilic granulomatosis with polyangiitis (EGPA), systemic lupus erythematosus, IgA nephropathy and/or IgA-associated vasculitis with or without Henoch-Schönlein purpura, rheumatoid vasculitis, Sjögren's syndrome, anti-GBM disease, cryoglobulinemic vasculitis, autoimmune hemolytic anemia, or mixed connective tissue disease."}
• {"criterion_text":"- Evidence of latent or active TB (Section 8.1.9)."}
• {"criterion_text":"- History of complement deficiency."}
• {"criterion_text":"- History of splenectomy."}
• {"criterion_text":"- History of malignancy, lymphoproliferative, or myeloproliferative disorder within 5 years prior to Screening with the exception of nonmelanoma skin cancer or carcinoma in situ of the cervix that has been treated with no evidence of recurrence."}
• {"criterion_text":"- Prior/Concomitant Therapy Has received any of the following: a.\tA kidney transplant b.\tDialysis or plasma exchange within [CCI] before Screening c.\tCyclophosphamide within [CCI] before Screening d.\t[CCI] or other B cell or plasma cell-depleting agent within 12 months to > 14 days before Screening e.\tA cumulative dose of intravenous [CCI] greater than 3 g methylprednisolone equivalent within 4 weeks of Screening f.\tOral daily dose of a glucocorticoid of > 10 mg prednisone equivalent for more than [CCI] continuously prior to the Screening Visit g.\t[CCI] or other complement inhibitors (eculizumab, ravulizumab, etc) within 30 days or 5 half-lives, whichever is longer, before Screening h.\tAnti-tumor necrosis factor treatment, abatacept, alemtuzumab, IVIG, antithymocyte globulin, or any other experimental or biological therapy within 12 weeks or 5 half-lives, whichever is longer, before Screening."}
• {"criterion_text":"- Prior/Concurrent Clinical Study Experience Participation in a clinical study involving an investigational medical product or device within 30 days or 5 half-lives of the investigational drug, whichever is longer, before screening"}
• {"criterion_text":"- Diagnostic Assessments: The following abnormal laboratory findings at Screening: •\tIgG < 5 g/L (if [CCI] treatment is initiated before Screening, then test results before initiation of [CCI] and within 6 months of the Screening can be used) •\tWBC count (3500/µL), or neutrophil count less than 1500/μL"}
• {"criterion_text":"- Women who are pregnant (positive pregnancy test) or breastfeeding at study entry"}
• {"criterion_text":"- Participants with an active systemic bacterial, viral, fungal, or parasitic infection, with the diagnostic assessment at the discretion of the investigator, that required use of intravenous antibacterials, antivirals, antifungals, or anti-parasitic agents within 14 days prior to Day 1."}
• {"criterion_text":"- Participants with any contraindication to rituximab per the locally approved product information, or any label warning/precaution that in the Investigator’s judgment would prevent safe rituximab administration in this study."}
• {"criterion_text":"- Alveolar hemorrhage requiring invasive pulmonary ventilation support at Screening."}
• {"criterion_text":"- Any diseases or conditions that, in the judgment of the Investigator, present a substantial clinical risk to participate in this study."}
• {"criterion_text":"- For patients with a previous diagnosis of CKD, patients known to have a stable eGFR for greater than 3 months prior to Screening and a decline less than 25% of previous eGFR at Screening will be excluded."}
• {"criterion_text":"- Known hypersensitivity to any ingredient contained in the study intervention."}
• {"criterion_text":"- History of serious N meningitidis infection."}
• {"criterion_text":"- Current and/or history (within the previous 5 years) of drug and/or alcohol abuse and/or dependence."}
• {"criterion_text":"- Evidence of active hepatitis B, hepatitis C, and/or HIV infection. Only participants with documented negative historical results (within [CCI] before Screening) for HBV, HCV, and HIV or a negative test by screening can be included into the study. Evidence of hepatitis B or hepatitis C infections will be according to the following criteria at Screening: •\tTesting positive for HBsAg, OR •\tTesting positive for HBcAb while having a negative HBsAb. •\tPositive hepatitis C antibody test result at Screening or within 3 months unless HCV RNA negative test is documented. Note: HBV DNA testing may be performed instead of HBsAb as per local standard practice."}
• {"criterion_text":"- Evidence of hepatic disease: AST, ALT, ALP, or bilirubin > 3 times the ULN at Screening."}

Primary endpoints

• {"endpoint_text":"- Incidence of TEAEs and TESAEs.","definition_or_measurement_approach":"Incidence of treatment-emergent adverse events (TEAEs) and treatment-emergent serious adverse events (TESAEs) reported during the study (safety reporting per protocol)."}
• {"endpoint_text":"- Changes from Baseline in safety assessments (vital sign measurements, physical examination, clinical laboratory tests, and ECG results).","definition_or_measurement_approach":"Change from baseline in vital signs, physical exam findings, clinical laboratory test values, and ECG results collected at scheduled safety assessments."}

Secondary endpoints

• {"endpoint_text":"- Achieving disease remission at Week 26.","definition_or_measurement_approach":"Assessment of disease remission at Week 26 per protocol-defined remission criteria."}
• {"endpoint_text":"- Achieving sustained remission at Week 52.","definition_or_measurement_approach":"Assessment of sustained remission at Week 52 per protocol-defined criteria."}
• {"endpoint_text":"- Achieving BVAS = 0 at [CCI], 26, and 52.","definition_or_measurement_approach":"BVAS (Birmingham Vasculitis Activity Score) assessed at specified visits ([CCI], Week 26, Week 52); endpoint is BVAS equal to 0."}
• {"endpoint_text":"- Relapse after previously achieving disease remission at Week 26.","definition_or_measurement_approach":"Occurrence of disease relapse following initial remission at Week 26 as defined by protocol."}
• {"endpoint_text":"- Time to first relapse after having achieved disease remission at Week 26.","definition_or_measurement_approach":"Time from Week 26 remission to first documented relapse."}
• {"endpoint_text":"- Change from Baseline in Vasculitis Damage Index (VDI) at [CCI] 26, and 52.","definition_or_measurement_approach":"Change from baseline in VDI score measured at specified timepoints."}
• {"endpoint_text":"- Time to first occurrence of BVAS = 0.","definition_or_measurement_approach":"Time from baseline to first visit with BVAS equal to 0."}
• {"endpoint_text":"- Change from Baseline in BVAS at all timepoints.","definition_or_measurement_approach":"Change in BVAS score from baseline measured across all scheduled visits."}
• {"endpoint_text":"- Change from Baseline in eGFR (mL/min per 1.73 m2) at [CCI] 26, and 52.","definition_or_measurement_approach":"Change from baseline in estimated glomerular filtration rate (eGFR) at specified timepoints."}
• {"endpoint_text":"- Change from Baseline in proteinuria based on spot UPCR (mg/g) and % change from Baseline at [CCI] 26, and 52.","definition_or_measurement_approach":"Change from baseline in urine protein-to-creatinine ratio (UPCR) from spot urine and percent change at specified visits."}
• {"endpoint_text":"- Change from Baseline in proteinuria based on spot UACR (mg/g) and % change from Baseline at [CCI] 26, and 52.","definition_or_measurement_approach":"Change from baseline in urine albumin-to-creatinine ratio (UACR) from spot urine and percent change at specified visits."}
• {"endpoint_text":"- Hematuria change from Baseline (RBCs/HPF) and % change from Baseline at [CCI] 26, and 52.","definition_or_measurement_approach":"Change from baseline in hematuria quantified as RBCs per high power field (HPF) and percent change at specified visits."}

Methods

• Patient brochures (country-specific versions)
• Invitation letters to potential participants
• Posters (patient posters) at clinical sites
• Patient Advocacy Group (PAG) materials: website/newsletter posts, PAG slides, PAG study factsheets
• PAG adverts (digital) and country-specific recruitment materials (ES, IT, FR, DE, PL)

France

Earliest CTIS Part Ii Submission Date

28-10-2025

Latest Decision Or Authorization Date

21-11-2025

Processing Time Days

24

Number Of Sites

5

Number Of Participants

4

Poland

Earliest CTIS Part Ii Submission Date

24-10-2025

Latest Decision Or Authorization Date

21-11-2025

Processing Time Days

28

Number Of Sites

4

Number Of Participants

4

Spain

Earliest CTIS Part Ii Submission Date

27-10-2025

Latest Decision Or Authorization Date

20-11-2025

Processing Time Days

24

Number Of Sites

5

Number Of Participants

4

Italy

Earliest CTIS Part Ii Submission Date

22-08-2025

Latest Decision Or Authorization Date

17-11-2025

Processing Time Days

87

Number Of Sites

4

Number Of Participants

4

Germany

Earliest CTIS Part Ii Submission Date

03-10-2025

Latest Decision Or Authorization Date

18-11-2025

Processing Time Days

46

Number Of Sites

5

Number Of Participants

5

Primary sponsor

Full Name

Alexion Pharmaceuticals Inc.

Organisation Type

Pharmaceutical company

Country Of Registered Address

United States