A clinical trial to compare how risdiplam is processed by the body in people with mild or moderate liver disease compared to people with healthy livers

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

  • Spinal Muscular Atrophy (SMA)
Trial Status:

Completed

This trial runs in
Cities
  • Hialeah
  • Orlando
  • San Antonio
Trial Identifier:

NCT03920865 BP40995

      Show trial locations

      The source of the below information is the publicly available website ClinicalTrials.gov. It has been summarised and edited into simpler language.

      The below information is taken directly from the publicly available website ClinicalTrials.gov within a week of any updates, and has not been edited.

      Results Disclaimer

      Trial 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.

      Hoffmann-La Roche Sponsor
      Phase 1 Phase
      NCT03920865 , BP40995 Trial Identifier
      All Gender
      ≥18 Years & ≤ 70 Years Age
      Accepts Healthy Volunteers Healthy Volunteers

       

      How does the BP40995 clinical trial work?
      This clinical trial is recruiting people who have mild or moderate liver disease. It is also recruiting healthy volunteers with normal livers.

      Risdiplam is currently being tested as a possible treatment for people with spinal muscular atrophy (or SMA). People with SMA can also be affected by liver disease, so it is important for doctors to know if risdiplam works differently in these patients. By taking part in this trial, you will help us to find out how liver disease affects the way the body processes risdiplam, so that doctors know how best to use risdiplam in people who have SMA and liver disease.

      How do I take part in this clinical trial?
      To be able to take part in this clinical trial, you must have been diagnosed with mild or moderate liver disease, or be in good health with a normal liver. Volunteers with a healthy liver will be matched to a person with liver disease based on sex, smoking status, age and BMI (body mass index).

      You must not have previously been given risdiplam and you must not have diabetes. Women who are pregnant, breastfeeding or able to have children will not be able to join the trial.

      If you think this clinical trial may be suitable for you and would like to take part, please talk to your doctor. If your doctor thinks that you might be able to take part in this clinical trial, he/she may refer you to the closest clinical trial doctor. They will give you all the information you need to make your decision about taking part in the clinical trial. You can also find the clinical trial locations on this page.

      You will have some further tests to make sure you will be able to take the treatments given in this clinical trial. Some of these tests or procedures may be part of your regular medical care. They may be done even if you do not take part in the clinical trial. If you have had some of the tests recently, they may not need to be done again.

      Before starting the clinical trial, you will be told about any risks and benefits of taking part in the trial. You will also be told what other treatments are available so that you may decide if you still want to take part.

      While taking part in the clinical trial, men with female partners who are able become pregnant will need to either not have heterosexual intercourse or take 2 methods of contraception for safety reasons.

      Everyone who joins this clinical trial will be given risdiplam, given as a liquid to drink once. You will have to stay in the Clinical Research Unit (or CRU) for 15 days in total, starting the day before you are given risdiplam, so that doctors can monitor how your body processes risdiplam.

      How often will I be seen in follow-up appointments, and for how long?
      You will be given the clinical trial treatment risdiplam once, the day after you arrive at the CRU. Once you have finished your 2 week stay at the CRU, you will still have to visit the CRU every 1–2 days for 2 weeks. These visits will include checks to see how your body is processing risdiplam and any side effects that you may be having. You are free to leave the clinical trial at any time.

       

      What happens if I am unable to take part in this clinical trial?
      If this clinical trial is not suitable for you, you will not be able to take part. You will not lose access to any of your regular care.

      For more information about this clinical trial see the For Expert tab on the specific ForPatient page or follow this link to ClinicalTrials.gov https://clinicaltrials.gov/ct2/show/NCT03920865

      Trial-identifier: NCT03920865

      Trial 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.

      Hoffmann-La Roche Sponsor
      Phase 1 Phase
      NCT03920865 , BP40995 Trial Identifier
      Risdiplam Treatments
      Muscular Atrophy, Spinal Condition
      Official Title

      An Open-Label, Single-Dose, Parallel-Group, Two-Part Study to Evaluate the Pharmacokinetics and Safety of Risdiplam in Subjects With Mild or Moderate Hepatic Impairment Compared to Subjects With Normal Hepatic Function

      Eligibility Criteria

      All Gender
      ≥18 Years & ≤ 70 Years Age
      Accepts Healthy Volunteers Healthy Volunteers
      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 </= 35 000 platelets
      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

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