Overview
This is an open-label extension study to evaluate the long-term efficacy, safety and tolerability of LNP023 in subjects with C3 glomerulopathy
Description
The purpose of this extension study is to collect long-term efficacy, safety and tolerability data in eligible participants receiving open-label iptacopan after completing treatment in the C3 glomerulopathy (C3G) Phase II study CLNP023X2202 or the C3G Phase III study CLNP023B12301. Efficacy and safety assessments at the 9 month visit of this extension study in combination with data from CLNP023X2202 (baseline plus 3 months of treatment) afforded the opportunity to evaluate the effects of iptacopan on potential endpoint(s) for the Phase III trial in C3G at 12 months of treatment. In addition, the enrollment of participants from study CLNP023B12301 permits longer-term evaluation of the persistence of effects observed after 6 months (CLNP023B12301 patients randomised to placebo arm) or 12 months (CLNP023B12301 patients randomised to iptacopan arm) of iptacopan treatment. These longer term efficacy assessments may be compared to historical/concurrent control data available from relevant real world databases in C3G patients and used as supportive information for registration purposes. The extension study is expected to continue until the drug product becomes commercially available and accessible (anticipated to be up to approximately 66 months), or the benefit-risk profile is no longer positive, or the program is discontinued for business or strategic reasons.
Study CLNP023X2202 has enrolled C3G patients with native kidney disease (Cohort A) and C3G patients who have undergone kidney transplant and have recurrence of C3G (Cohort B); CLNP023B12301 will only enroll patients with native kidneys.
"Baseline" refers to the Day 1 visit (pre-dose) of CLNP023X2202 or CLNP023B12301, whereas the Day 1 visit for this C3G extension study (CLNP023B12001B) is identified as "Extension Day 1".
Eligibility
Inclusion Criteria:
- Patients must have completed the treatment period of the CLNP023X2202 or CLNP023B12301 trial on study drug
Exclusion Criteria:
- Severe concurrent co-morbidities, e.g. advanced cardiac disease (NYHA class IV), severe pulmonary arterial hypertension (WHO class IV), or any illness or medical condition that in the opinion of the investigator and sponsor is likely to prevent the patient from safely tolerating LNP023 or complying with the requirements of the study
- Participants with an active systemic bacterial, viral or fungal infection within 14 days prior to screening, or the presence of fever ≥ 38oC (100.4oF) within 7 days prior to screening.
- History or current diagnosis of ECG abnormalities indicating significant risk of safety for subjects
- History of HIV or any other immunodeficiency disease