An open-label long-term study of LNP023 in C3G patients
Research type
Research Study
Full title
An open-label, non-randomized extension study to evaluate the long-term efficacy, safety and tolerability of LNP023 in subjects with C3 glomerulopathy
IRAS ID
265062
Contact name
Edwin Wong
Contact email
Sponsor organisation
Novartis Pharma AG
Eudract number
2018-004253-24
Duration of Study in the UK
7 years, 4 months, 0 days
Research summary
C3 glomerulopathy (C3G) is a rare inflammatory kidney disease caused by abnormalities in the “alternative pathway of complement” - part of our immune system which provides protection against infections.
Most patients with C3G have significant disease, with approximately 50% developing end stage renal disease within 10 years. Even after kidney transplantation, the disease often recurs. Currently, there are no approved drugs available to patients to treat C3G.
The purpose of this extension study is to collect long-term efficacy, safety and tolerability data in eligible subjects receiving open-label LNP023 after completing the C3G proof of concept (PoC) study CLNP023X2202. Efficacy assessments at the 9 month visit of the extension study in combination with data from CLNP023X2202 (baseline plus 3 months of treatment) will afford the opportunity to evaluate the effects of LNP023 on potential endpoint(s) for the Phase III trial in C3G at 12 months of treatment. Longer-term efficacy assessments may be used as supportive information for registration purposes. Patients will be on study for a total of 5.5 years followed by a 90-day follow-up period. During that time they will receive open-label medication, visit the study site around 17 times and have all study-related assessments including blood and urine sampling, ECGs and optional renal biopsies.REC name
North East - Tyne & Wear South Research Ethics Committee
REC reference
19/NE/0260
Date of REC Opinion
28 Aug 2019
REC opinion
Further Information Favourable Opinion