A randomized, controlled study to evaluate LNP023 (iptacopan) in patients with active ANCA-associated vasculitis.

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Novartis Pharma AG

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Immune System Diseases [C20]

Trial duration

18 Nov 2024 → ongoing

Decision date (initial)

2024-09-29

Transition trial

No

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

Funding sources

Novartis Pharma AG

External identifiers

EU CT number

2023-510525-15-00

WHO UTN

U1111-1328-7586

ClinicalTrials.gov

NCT06388941

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Therapy, Safety, Efficacy

To assess the effect of iptacopan in achieving sustained remission compared to standard of care (SOC)

Secondary objectives 3

1. To assess time to remission through Week 24
2. To assess the effect of iptacopan on disease relapse
3. To assess the effect of iptacopan on renal function

Conditions and MedDRA coding

active antineutrophilic cytoplasmic antibody (ANCA) associated vasculitis

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 5

1. Signed informed consent obtained prior to participation in the study.
2. Newly diagnosed or relapsed GPA and MPA (according to the 2022 ACR/EULAR classification criteria for GPA and MPA) requiring treatment with RTX and GC as per investigator's judgement.
3. BVAS assessment with ≥1 major item, or ≥3 minor items, or ≥2 renal items at Screening.
4. Positive antibody test for anti-proteinase 3 (PR3) or anti-myeloperoxidase (MPO) antibodies at Screening or with history of documented evidence of a positive antibody test.
5. Male or female subjects ≥18 years at Screening.

Exclusion criteria 5

1. Other systemic disease which constitutes the primary illness, including but not limited to: eosinophilic granulomatosis with polyangiitis (EGPA), moderate to severe systemic lupus erythematosus, IgA vasculitis (Purpura Schönlein-Henoch), rheumatoid vasculitis, Sjögren's syndrome, anti-glomerular basement membrane (GBM) disease, cryoglobulinemic vasculitis, autoimmune hemolytic anemia, autoimmune lymphoproliferative syndrome or mixed connective tissue disease.
2. Alveolar hemorrhage requiring invasive pulmonary ventilation support at Screening.
3. Severe kidney disease defined as estimated glomerular filtration rate <15 mL/min/1.73m2, or kidney failure defined as receiving renal replacement therapy such as hemo(dia)filtration, hemo-/peritoneal dialysis, or having received a kidney transplant.
4. Received plasma exchange/-pheresis within 12 weeks prior to Screening.
5. Received any of the following immunosuppressive, cell depleting or biological therapy (any other immunosuppressive, cell depleting, or biological therapy not listed here must be discussed with the Sponsor): • Received RTX or other B-cell depleting agent within 16 weeks prior to Screening. • Received any of the following biological or alkylating immunosuppressive medications within 12 weeks before screening, including but not limited to: • cyclophosphamide (CYC) • complement inhibitor (such as eculizumab, ravulizumab, avacopan) • anti-tumor necrosis factor • abatacept • tocilizumab • intravenous immunoglobulins • Received any of the following cell depleting agents within 24 weeks before Screening, including but not limited to: • alemtuzumab • antithymocyte globulin • Received azathioprine (AZA), methotrexate (MTX), or mycophenolate (MMF), or any other immunosuppressants with similar half-life within 1 week prior to Day 1. • Received leflunomide (LEF) within 6 weeks prior to Day 1.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Sustained remission through Week 48 defined as complete remission at Week 24 without major relapse up to Week 48.

Secondary endpoints 3

1. Time to reach BVAS = 0
2. Time to major relapse through Week 48
3. Estimated glomerular filtration rate (eGFR) using the CKD-EI formula, urinary protein excretion and hematuria over 48 weeks.

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Placebo 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Novartis Pharma AG

Sponsor organisation

Novartis Pharma AG

Address

Lichtstrasse 35

City

Basel

Postcode

4056

Country

Switzerland

Scientific contact point

Organisation

Novartis Pharma AG

Contact name

Novartis Pharma Arzneimittel GmbH

Public contact point

Organisation

Novartis Pharma AG

Contact name

Novartis Pharma Arzneimittel GmbH

Third parties 8

Locations

9 EU/EEA countries · 30 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 126 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

5 submissions — initial application plus substantial / non-substantial modifications