Phase 1 Primary Biliary Cholangitis Trial, Recruiting

Written by an AI model from ClinicalTrials.gov eligibility criteria and checked on a sample basis. Please confirm against the original criteria.

Who might be able to join this trial:

Phase 1a (Healthy Volunteer Phase):

• People who are willing and able to follow all trial rules and procedures, as judged by the trial doctor
• People who have signed a consent form agreeing to take part before any trial procedures begin
• People aged 18 to 68 years old at the time of signing the consent form, regardless of sex assigned at birth
• People who are in good general health with no known heart, liver, metabolic, digestive, or kidney disease, based on tests and examinations done at the first screening visit
• People with a body mass index (BMI) below 30 kg/m² at screening (this is a measure of body weight relative to height)
• People who have not used cannabis — whether medical or recreational — on a near-daily basis in the 3 months before screening
• People assigned female at birth who are not able to become pregnant, meaning they have had a hysterectomy, had both ovaries or both fallopian tubes removed, or have been through menopause (no periods for 12 months without another medical explanation), or have no uterus due to a condition present from birth
• People assigned male at birth who are willing to use highly effective contraception from signing the consent form through to 6 months after the last dose, and who agree not to donate sperm for at least 6 months after the last dose

Phase 1b (Liver Disease Phase — Non-Cirrhotic PSC):

• People who are willing and able to follow all trial rules and procedures, as judged by the trial doctor
• People who have signed a consent form agreeing to take part before any trial procedures begin
• People aged 18 to 68 years old at the time of signing the consent form, regardless of sex assigned at birth
• People with a confirmed diagnosis of large-duct primary sclerosing cholangitis (PSC), a liver condition affecting the bile ducts, based on at least 2 of the following: a historical blood test showing an elevated liver enzyme called alkaline phosphatase; a previous liver biopsy showing features consistent with PSC; or previous imaging of the bile ducts showing changes consistent with PSC
• People whose blood tests show a fibrosis (liver scarring) marker above a pre-set level (confirm with trial site)
• People whose liver shows moderate to advanced scarring, but not full cirrhosis, based on a specific liver test score called ELF, falling between 7.7 and 11.3 at the first visit
• People with no signs of bile duct cancer or any other cancer on MRI scan at screening
• People with certain other existing conditions may be eligible if they meet pre-defined requirements (confirm with trial site)
• People whose blood test results fall within certain specified ranges at screening (confirm with trial site)
• People who have not used cannabis — whether medical or recreational — on a near-daily basis in the 3 months before screening
• People who are willing to use highly effective contraception from signing the consent form through to 6 months after the last dose, and who agree not to donate eggs or sperm for at least 6 months after the last dose
• People assigned female at birth who are able to become pregnant must have a negative pregnancy blood test at the first screening visit and a negative urine pregnancy test at the second screening visit and on the first dosing day
• People who are able to learn and correctly follow the procedure for self-administering the trial injection under the skin

Who may not be able to join:

Phase 1a (Healthy Volunteer Phase):

• People who work for the trial sponsor or trial site, or who are a close family member (such as a spouse, parent, child, or sibling) of someone directly involved in running the trial
• People with a known allergy to any ingredient in the trial drug TAK-781 or closely related compounds
• People with a history of serious or multiple allergies (for example, to food, drugs, or latex), or who have experienced a severe allergic reaction (anaphylaxis) to prescription or non-prescription medicines or foods
• People with a history or signs of abnormal bleeding or blood clotting problems
• People who have previously had their gallbladder removed (cholecystectomy)
• People who have taken part in another clinical trial using an investigational drug, device, or treatment within the past 4 weeks (or within 5 times the drug's clearance time, whichever is longer)
• People whose liver enzyme blood test results (ALT and/or AST) are above the normal range during screening
• People with abnormal cholesterol levels at screening, including total cholesterol above 240 mg/dL, LDL cholesterol above 160 mg/dL, HDL cholesterol below 35 mg/dL, or triglycerides above 250 mg/dL (these may be re-tested once under fasting conditions)
• People whose kidney filtering function (eGFR) is at or below 90 mL/min/1.73m² at screening
• People whose heart tracing (ECG) shows a certain measurement called the QTcF interval above 450 milliseconds for males or 460 milliseconds for females
• People with a history of certain serious heart rhythm problems, including a condition called torsades de pointes, significant abnormal heart rhythms, certain types of heart block, congenital long QT syndrome, or significant heart-related ECG changes
• People with a confirmed resting blood pressure at or above 140/90 mmHg at screening (measured as an average of three readings after lying down)
• People with a resting heart rate outside the range of 55 to 100 beats per minute, unless they are athletic with a heart rate above 45 beats per minute (at the trial doctor's discretion)
• People who test positive for drugs of abuse in a urine screen at screening or on Day −3
• People who have been diagnosed with or treated for any cancer in the 5 years before screening
• People who consume more than 600 mg of caffeine per day (from coffee, tea, cola, energy drinks, or similar sources)
• People who have a positive pregnancy test during screening
• People who test positive for active hepatitis B virus (HBV) DNA at screening (those with certain other hepatitis B test patterns may still be eligible — confirm with trial site)
• People who test positive for active hepatitis C virus (HCV) RNA at screening (those previously treated and cleared of hepatitis C may still be eligible — confirm with trial site)
• People who test positive for HIV at screening, or who are currently taking antiretroviral (HIV) medication
• People who have had major surgery, or donated or lost approximately 500 mL (one unit) of blood, within 4 weeks before the screening visit
• People who currently have or recently had (within 2 weeks before the first dose) a significant illness, as judged by the trial doctor
• People who are unable or unwilling to avoid certain prohibited medicines, herbal products, grapefruit juice, or other restricted substances during the trial
• People who are unable or unwilling to avoid smoking, vaping, or using nicotine products from 2 weeks before screening until the last follow-up visit
• People with clinically relevant drug or alcohol misuse within 12 months of screening, including drinking more than 4 units of alcohol on any single day or more than 14 units per week (for males), or more than 3 units on any single day or more than 7 units per week (for females) — where 1 unit equals roughly one standard drink

Phase 1b (Liver Disease Phase — Non-Cirrhotic PSC):

• People with a diagnosis of secondary sclerosing cholangitis (bile duct scarring caused by another condition, such as chronic pancreatitis, stones, injury, or infection, rather than PSC itself)
• People with a diagnosis of IgG4-related sclerosing cholangitis (a different condition that can look similar to PSC)
• People with evidence of cirrhosis (advanced liver scarring), assessed using a detailed checklist involving blood tests, imaging, and clinical review; people with 2 or more cirrhosis-related signs on imaging or blood tests will be excluded (confirm with trial site for full details)
• People who have previously had their gallbladder removed (cholecystectomy)
• People with decompensated liver disease (meaning the liver is no longer able to function adequately), or signs of significant worsening of liver function during screening
• People with a history of fluid build-up in the abdomen (ascites), confusion caused by liver failure (hepatic encephalopathy), bleeding from enlarged veins in the digestive system (variceal bleeding), a liver disease severity score (Child-Pugh) above 6, or a liver disease score (MELD) above 11
• People with an overlap condition involving autoimmune hepatitis, based on certain blood test and biopsy results at screening
• People with an overlap condition involving primary biliary cholangitis (PBC), based on certain antibody tests or biopsy results
• People with significant liver disease caused by something other than PSC
• People with a concerning narrowing (dominant stricture) of the bile duct seen on imaging at screening, unless the trial doctor determines it poses no concern for cancer and is unlikely to cause significant fluctuations in test results during the trial
• People who have had a bile duct stent placed or a drain inserted within 3 months of screening (those who had a balloon dilation procedure may be eligible after at least 4 weeks have passed since the procedure)
• People with a history, confirmed diagnosis, or strong suspicion of bile duct cancer or other liver or bile duct cancer
• People who have had a bile duct infection (ascending cholangitis) within 60 days before screening or before the first dose
• People who have previously had a liver transplant
• People whose ECG shows clinically significant abnormalities, as determined by the trial doctor
• People who test positive for active hepatitis B virus (HBV) DNA at screening (those with certain other hepatitis B test patterns may still be eligible — confirm with trial site)
• People who test positive for active hepatitis C virus (HCV) RNA at screening (those previously treated and cleared of hepatitis C may still be eligible — confirm with trial site)
• People who test positive for HIV at screening, or who are currently taking antiretroviral (HIV) medication
• People with a history of cancer diagnosed or treated within the 5 years before screening
• People who are unable or unwilling to avoid certain prohibited medicines, herbal products, grapefruit juice, or other restricted substances during the trial
• People who smoke, vape, or use nicotine products at a level of 20 or more cigarettes per day, or who have smoking-related health complications
• People with clinically relevant drug or alcohol misuse within 12 months of screening, including drinking more than 4 units on any single day or more than 14 units per week (for males), or more than 3 units on any single day or more than 7 units per week (for females)
• People currently taking prohibited medications who have not completed the required washout (clearance) period before the first dose
• People currently taking ursodeoxycholic acid (a bile acid medication) who have not completed the required washout period before the first dose
• People with a history of severe allergic reactions to a type of drug called N-acetylgalactosamine small-interfering RNA therapeutics
• People who have taken part in another clinical trial or used an investigational drug, device, or biological treatment within 4 weeks before screening (or within 5 times the drug's clearance time, whichever is longer)
• People with a significant unstable or untreated medical condition, or another serious medical disorder, that could interfere with their participation or safety in the trial
• People with any condition — whether short-term or ongoing — that, in the trial doctor's opinion, would prevent full participation in the trial
• People whose personal circumstances (for example, location or social situation) would make it difficult or impossible to participate for the full duration of the trial
• People who work for the trial sponsor, a contract research organisation involved in the trial, or the trial site — including permanent, temporary, or contract staff directly involved in running the trial — or who are a close family member of such an employee

Important: Always verify eligibility with the trial site directly before applying.