Study on Efficacy and Safety of LNP023 in C3 Glomerulopathy Patients Transplanted and Not Transplanted

Novartis

Status and phase: Completed; Phase 2

Conditions: Glomerulonephritis

Treatments: Drug: LNP023

Study type: Interventional

Funder types: Industry

Identifiers

NCT03832114

CLNP023X2202

Details and patient eligibility

About

The study is an open-label, two cohort non-randomized study evaluating the efficacy, safety, and pharmacokinetics of LNP023 in patients with C3G (Cohort A) and patients who have undergone kidney transplant and have C3G recurrence (Cohort B).

Enrollment: 27 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

Inclusion Criteria for Cohort A and B:

• Written informed consent must be obtained before any assessment is performed
• Male and female patients between the ages of 18 to 65 (inclusive) at screening
• C3G patients wit proteinuria
• Able to communicate well with the investigator, to understand and comply with the requirements of the study
• At screening and baseline visits, patients must weigh at least 35 kg
• Supine vital signs should be within the following ranges :

oral body temperature between 35.0-37.5 °C systolic blood pressure, 80-170 mm Hg diastolic blood pressure, 50-105 mm Hg pulse rate, 45 - 100 bpm

.

Inclusion Criteria for Cohort A:

• Estimated GFR (using the CKD-EPI formula) or measured GFR ≥30 mL/min per 1.73 m2 for patients on a maximum recommended or maximum tolerated dose of an angiotensin converting enzyme inhibitor (ACEI) or angiotensin receptor blocker (ARB)
• UPCR ≥ 100 mg/mmol (equivalent to ≥ 1 g/24h total urinary protein excretion)
• Prior to entry, all patients must have been on supportive care including a maximally tolerated dose of ACEi or ARB for at least 30 days.

Inclusion Criteria for Cohort B:

• No histological/laboratory/clinical signs of allorejection
• If applicable, induction treatment after allotransplantation needs to be completed >30 days before inclusion.
• Transplantation of a kidney allograft >90 days before inclusion
• Patients need to be on a stable dose of immunosuppressive regimen prior to inclusion. Any approved treatments are allowed for this purpose.

Exclusion Criteria for Cohort A and B:

• Use of other investigational drugs at the time of enrollment, or within 5 half-lives of randomization, or within 30 days, whichever is longer; or longer if required by local regulations
• A history of clinically significant ECG abnormalities,
• Known family history or known presence of long QT syndrome or Torsades de Pointes
• Use of agents known to prolong the QT interval unless they can be permanently discontinued for the duration of the study
• Pregnant or nursing (lactating) women, where pregnancy is defined as the state of a female after conception and until the termination of gestation, confirmed by a positive hCG laboratory test.
• Women of child bearing potential, defined as all women physiologically capable of becoming pregnant, unless they are using highly effective methods of contraception during dosing and for 1 week after stopping of investigational drug.
• History of immunodeficiency diseases, or a positive HIV test result.
• Chronic infection with Hepatitis B (HBV) or Hepatitis C (HCV).
• Patients who cannot receive vaccinations against N. meningitidis, S. pneumoniae, or H. influenzae

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Single Group Assignment

Masking: None (Open label)

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27 participants in 2 patient groups

Cohort A - no kidney transplant

Experimental group

Description:

C3G patients who have not received a kidney transplant and have reduced C3 blood levels.

Treatment:

Drug: LNP023

Cohort B - kidney transplant

Experimental group

Description:

C3G patients who have received a kidney transplant and have C3G recurrence.

Treatment:

Drug: LNP023