Pharmacokinetic (PK) and Safety Study of Iptacopan (LNP023) in Participants With Mild, Moderate, and Severe Hepatic Impairment Compared to Matched Control Healthy Participants With Normal Hepatic Function.

Novartis

Status and phase: Completed; Phase 1

Conditions: Hepatic Impairment

Treatments: Drug: Iptacopan

Study type: Interventional

Funder types: Industry

Identifiers

NCT05078580

CLNP023A2105

Details and patient eligibility

About

This was an open-label, single dose parallel group study to evaluate the PK of iptacopan in participants with mild, moderate, and severe hepatic impairment compared to matched healthy control participants.

Full description

The study comprised of a screening period up to 28 days, one Baseline evaluation on Day -1, a single dose administration of 200 mg of iptacopan followed by PK sampling to 240 hr, and an End-of-Study (EOS) visit (Day 11). All participants had a post study safety follow-up call conducted approximately 30 days after last administration of study drug.

Enrollment: 38 patients

Sex: All

Ages: 18 to 75 years old

Volunteers: Accepts Healthy Volunteers

Inclusion criteria

Participants eligible for inclusion in this study met the following key criteria:

• Written informed consent was obtained before performing any assessment.
• Male participants and female participants of non-childbearing potential 18 to 75 years of age (inclusive).
• Participants were to weigh at least 55 kg to participate in the study and were to have a body mass index (BMI) within the range of 18 to 35 kg/m2 for healthy participants.

For hepatic impairment participants without overt ascites, the BMI was to be within the range of 18 to 40 kg/m2. For hepatic impairment participants with overt ascites, the BMI was to be within the range of 18 to 45 kg/m2.

• Ability to communicate well with the Investigator, to understand and comply with the requirements of the study.
• Participant was willing to remain in the clinical research unit as required by the protocol

Exclusion criteria

Participants meeting any of the following criteria were excluded from this study:

• Use of other investigational drugs within the last 30 days or 5 half-lives prior to dosing, whichever was longer.
• History of hypersensitivity to the investigational compound/compound class or its excipients being used in this study.
• Pregnant or nursing (lactating) women. Pregnancy was defined as the state of a female after conception and until termination of gestation, confirmed by a positive human chorionic gonadotropin (hCG) laboratory test.
• Known history of, or current clinically significant arrhythmias, history of prolonged QT correction formula (QTcF) interval or QTcF > 450 msec (males) or QTcF > 460 msec (females) at screening in healthy participants and history of prolonged QTcF interval or QTcF > 470 msec (males) or QTcF > 480 msec (females) at screening in participants with hepatic impairment.

Trial design

Primary purpose: Other

Allocation: Non-Randomized

Interventional model: Single Group Assignment

Masking: Single Blind

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38 participants in 4 patient groups

Healthy participants

Experimental group

Description:

Iptacopan 200 mg single dose

Treatment:

Drug: Iptacopan

Drug: Iptacopan

Drug: Iptacopan

Drug: Iptacopan

Mild hepatic impairment patients

Experimental group

Description:

Iptacopan 200 mg single dose

Treatment:

Drug: Iptacopan

Drug: Iptacopan

Drug: Iptacopan

Drug: Iptacopan

Moderate hepatic impairment patients

Experimental group

Description:

Iptacopan 200 mg single dose

Treatment:

Drug: Iptacopan

Drug: Iptacopan

Drug: Iptacopan

Drug: Iptacopan

Severe hepatic impairment patients

Experimental group

Description:

Iptacopan 200 mg single dose

Treatment:

Drug: Iptacopan

Drug: Iptacopan

Drug: Iptacopan

Drug: Iptacopan