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

Study Identifier:
BP40995
ClinicalTrials.gov Identifier:
NCT03920865
EudraCT Identifier:
N/A
EU CT Number:
N/A
Sponsor:
Hoffmann-La Roche
Study Complete

Trial Results

Study Protocol
Available Languages: English
View PDF
Statistical Analysis Plan
Available Languages: English
View PDF

Study Details

Medical Condition
  • All Spinal Muscular Atrophies
Study Drug
  • Drug: Risdiplam
Date
May 2019 - Jan 2020
Phase 1
Phase 2
Phase 3
Phase 4
Patient Requirements
Sex: Female & Male
Age: 18 - 70 Years
Requirements Information
Inclusion and Exclusion Criteria
Inclusion Criteria
  • 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 \
Exclusion Criteria
  • 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
  • Participants with Hepatic Impairment Only:
  • Confirmed supine blood pressure \> 159 mmHg or \< 90 mmHg
  • Values outside the normal range for liver function tests that are not consistent with their hepatic condition
  • Use of a new medication, or a change in dose, for the treatment, or worsening of, hepatic encephalopathy
  • Use of prescription drugs within 14 days of study drug administration
  • Recent history of, or the treatment of, esophageal bleeding
  • Presence of a portosystemic shunt
  • Recent history of paracentesis
  • Current functioning organ transplant or are waiting for an organ transplant
  • Evidence of severe ascites
  • History or current symptoms of hepatic encephalopathy Grade 2 or above

Protocol Summary

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.

Trial Locations

Location
Status
Location
Clinical Pharmacology of Miami, Inc.
Miami, Florida, United States, 33014
Status
N/A
Location
Orlando Clinical Research Center
Orlando, Florida, United States, 32809
Status
N/A
Location
American Research Corporation Inc.
San Antonio, Texas, United States, 78215
Status
N/A

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