Long-term efficacy, safety and tolerability of LNP023 in C3G

JRCT ID: jRCT2041220097

Registered date:25/11/2022

Basic Information

• Recruitment status: Recruiting
• Health condition(s) or Problem(s) studied: Complement component 3 glomerulopathy
• Date of first enrollment: 01/12/2022
• 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): Iptacopan(LNP023) 200 mg b.i.d.

Outcome(s)

• Primary Outcome: Occurrence of clinically significant vital signs (msDBP, msSBP, heart rate), ECGs, and safety laboratory measurements, as well as adverse events (AEs), AEs of special interest, and study drug discontinuation due to an AE (or any safety issue).

Key inclusion & exclusion criteria

• Age minimum: >= 18age old
• Age maximum: Not applicable
• Gender: Both

Include criteria

1. Written informed consent must be obtained before any assessment is performed.
2. Male and female patients 18 years of age or greater at screening.
3. Patients must have completed the treatment period of the CLNP023X2202 or CLNP023B12301 trial on study drug.
4. Patients must be able to communicate well with the investigator, understand and comply with the requirements of the study [applicable in Germany only]
4a. Patients or their legal representatives must be able to communicate well with the investigator, understand and comply with the requirements of the study [applicable in all countries except Germany].

Exclude criteria

1. 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 iptacopan or complying with the requirements of the study.
2. Participants with an active systemic bacterial, viral or fungal infection within 14 days prior
to screening, or The presence of fever >= 38 degree Celsius (100.4 degree Fahrenheit) within 7 days prior to screening.
3. History or current diagnosis of ECG abnormalities indicating significant risk of safety for participants participating in the study such as:
-Concomitant clinically significant cardiac arrhythmias, e.g., sustained ventricular tachycardia, and clinically significant second- or third-degree AV block without a pacemaker
-History of familial long QT syndrome or known family history of Torsades de Pointe.
4. Use of agents known to prolong the QT interval unless they can be permanently discontinued for the duration of the study.
5. History of malignancy of any organ system (other than localized basal cell carcinoma of the skin or in-situ cervical cancer), treated or untreated, within the past 5 years, regardless of whether there is evidence of local recurrence or metastases.

Related Information

• Primary Sponsor: Yamada Hiroyuki