A Study to Investigate the Effect of Hepatic Impairment on the Pharmacokinetics and Safety and Tolerability of a Single Oral Dose of Risdiplam Compared to Matched Healthy Participants With Normal Hepatic Function

Completed · Phase 1

Conditions studied: Muscular Atrophy, Spinal

In brief

This is a multi-center, open-label, non-randomized, parallel-group, 2-part study to evaluate the effect of hepatic impairment on the PK and safety and tolerability of a single oral dose of risdiplam compared to matched healthy participants with normal hepatic function.

Key facts

Study ID

NCT03920865

Run by

Hoffmann-La Roche

People needed

26

Starts

2019-05-16

Expected to finish

2020-01-02

Last updated by the study team

2021-02-21

Who can join

Age: 18 and older, up to 70. Sex: any. Healthy volunteers: accepted.

You may qualify if…

• All Participants:
• BMI between 18.0 and 36.0 kilograms per square metre (kg/m2), inclusive, and body weight > / = 50 kg
• Females must not be pregnant or lactating and must be of non-childbearing potential
• Male participants (whether surgically sterilized or not) with female partners of childbearing potential must use methods of contraception from Screening until 4 months after their dose of the study drug as detailed in the protocol
• Male participants must not donate sperm from Check-in (Day -1) until 4 months after their dose of the study drug
• Participants with Normal Hepatic Function Only:
• Matched to participants with mild or moderate hepatic function in sex, age, BMI, and smoking status
• In good health, as determined by no clinically significant findings from medical history, physical examination, 12-lead ECG, vital sign measurements, and clinical laboratory evaluations
• Participants with Hepatic Impairment Only:
• Documented chronic stable liver disease
• Currently on a stable medication regimen, defined as not starting new drug(s) or changing drug dose(s) within 3 months of administration of study drug
• Anemia secondary to hepatic disease will be acceptable, if hemoglobin >/= 9 gram per decilitre (g/dL). Participants must have a platelet count </= 35 000 platelets

You may not qualify if…

• All Participants
• Significant history or clinical manifestation of any metabolic, allergic, dermatological, renal, hematological, pulmonary, cardiovascular, gastrointestinal, neurological, respiratory, endocrine, or psychiatric disorder
• History of significant hypersensitivity, intolerance, or allergy to any drug compound, constituents or excipients of the study drug, food, or other substance
• History of stomach or intestinal surgery or resection that would potentially alter absorption and/or excretion of orally administered
• Ventricular dysfunction or history of risk factors for Torsades de Pointes
• Evidence of hepatorenal syndrome and estimated creatinine clearance range < 60 millilitre per minute (mL/min) or abnormal sodium and potassium levels
• Clinically significant physical examination abnormality
• History of diabetes mellitus
• Use or intend to use any medications/products known to alter drug absorption, metabolism, or elimination processes, including St. John's Wort
• Positive human immunodeficiency virus (HIV) test
• Participation in a clinical study involving administration of an investigational drug prior to dosing
• Smoke more than 10 cigarettes or use the equivalent tobacco- or nicotine-containing products per day
• Receipt of blood products within 2 months prior to study
• Donation of blood, plasma, or platelets prior to Screening
• Poor peripheral venous access
• Have previously completed or withdrawn from this study or any other study investigating risdiplam, and have previously received the investigational product
• Participants with Normal Hepatic Function Only:
• Confirmed supine blood pressure > 150 millimetre of mercury (mmHg) or < 90 mmHg
• Positive test for hepatitis B or C virus
• Clinically significant abnormal laboratory values
• Significant history or clinical manifestation of hepatic disorder
• History or presence of liver disease or liver injury
• Use or intend to use any prescription medications/products within 14 days prior to dosing
• . Use or intend to use slow-release medications/products considered to still be active within 14 days prior to dosing
• Use or intend to use any non-prescription medications/products within 7 days prior to dosing

Where it is running

• Clinical Pharmacology of Miami, Inc. — Miami, Florida, United States
• Orlando Clinical Research Center — Orlando, Florida, United States
• American Research Corporation Inc. — San Antonio, Texas, United States

Full record on ClinicalTrials.gov

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