Study of efficacy and safety of iptacopan in patients with C3 glomerulopathy

JRCT ID: jRCT2041210082

Registered date:11/10/2021

Basic Information

• Recruitment status: Recruiting
• Health condition(s) or Problem(s) studied: C3G
• Date of first enrollment: 11/10/2021
• Target sample size: 68
• Countries of recruitment: Argentina,Japan,Belgium,Japan,Brazil,Japan,Canada,Japan,China,Japan,Czech Republic,Japan,France,Japan,Germany,Japan,Greece,Japan,India,Japan,Israel,Japan,Italy,Japan,Netherlands,Japan,Spain,Japan,Switzerland,Japan,Turkey,Japan,UK,Japan,US,Japan
• Study type: Interventional

Intervention(s)

Arm A: LNP023
Arm B: Placebo to LNP023

Outcome(s)

Primary Outcome

Change from baseline in eGFR. [ Time Frame: 6 months (double-blind) ]
- To demonstrate the superiority of iptacopan vs. placebo in improving eGFR

Key inclusion & exclusion criteria

• Age minimum: >= 18age old
• Age maximum: <= 60age old
• Gender: Both

Include criteria

1. Male and female participants age >= 18 and =< 60 years at screening.
2. Diagnosis of C3G as confirmed by renal biopsy within 12 months prior to enrollment.
3. Prior to randomization, all participants must have been on a maximally recommended or tolerated dose of an angiotensin converting enzyme inhibitor (ACEI) or angiotensin receptor blocker (ARB) for at least 90 days. The doses of other antiproteinuric medications including mycophenolic acid, corticosteroids and mineralocorticoid receptor antagonists should be stable for at least 90 days prior to randomization.
4. Reduced serum C3 (defined as less than 0.85 x lower limit of the central laboratory normal range) at Screening.
5. UPCR >= 1.0 g/g sampled from the first morning void urine sample at Day -75 and Day -15.
6. Estimated GFR (using the CKD-EPI formula) or measured GFR >= 30 ml/min/1.73m2 at screening and Day -15.
7. Vaccination against Neisseria meningitidis infection prior to the start of study treatment. Vaccination against Streptococcus pneumoniae and Haemophilus influenzae infections should be given, if available and according to local regulations.

Exclude criteria

1. Participants who have received any cell or organ transplantation, including a kidney transplantation.
2. Rapidly progressive crescentic glomerulonephritis defined as a 50% decline in the eGFR within 3 months with renal biopsy findings of glomerular crescent formation seen in at least 50% of glomeruli.
3. Renal biopsy showing interstitial fibrosis/tubular atrophy (IF/TA) of more than 50%.
4. Monoclonal gammopathy of undetermined significance (MGUS) confirmed by the measurement of serum free light chains or other investigation as per local standard of care.
5. Participants with an active systemic bacterial, viral or fungal infection within 14 days prior to study treatment administration
6. The presence of fever >= 38 degree (100.4 degree Fahrenheit) within 7 days prior to study treatment administration.
7. A history of recurrent invasive infections caused by encapsulated organisms, e.g., N. meningitidis and S. pneumoniae.
8. The use of inhibitors of complement factors (e.g., Factor B, Factor D, C3 inhibitors, anti C5 antibodies, C5a receptor antagonists) within 6 months prior to the Screening visit.
9. The use of immunosuppressants (except mycophenolic acids), cyclophosphamide or systemic corticosteroids at a dose >7.5 mg/day (or equivalent for a similar medication) within 90 days of study drug administration.

Related Information

• Primary Sponsor: Yamada Hiroyuki
• Secondary ID(s): NCT04578834