Actively Recruiting
A Phase 1, Randomized, Placebo-Controlled, Double-Blind Study Evaluating Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of TAK-781 in Healthy Volunteers and Participants With Non-Cirrhotic Primary Sclerosing Cholangitis
Led by Takeda · Updated on 2026-03-05
134
Participants Needed
1
Research Sites
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating the drug TAK-781 to determine its safety, tolerability, and how the body processes it in both healthy volunteers and participants with non-cirrhotic Primary Sclerosing Cholangitis (PSC). This Phase 1 study aims to understand how TAK-781 affects the immune system and its pharmacokinetics and pharmacodynamics. The study is sponsored by Takeda and involves detailed monitoring of participants' responses to the drug. The study includes two main phases: Phase 1a and Phase 1b. In Phase 1a, healthy participants receive either a single dose or multiple doses of TAK-781 or a placebo through injections. Different dose levels and regimens are tested across various cohorts, with some cohorts potentially repeated to assess safety and target engagement. In Phase 1b, participants with large duct, non-cirrhotic PSC receive a single dose of TAK-781. The Phase 1b part only starts after completion of the single ascending dose portion of Phase 1a. Participants will be involved for about 36 weeks, during which they will undergo clinical evaluations including laboratory tests, ECGs, and vital sign monitoring to check for treatment-emergent adverse events and any clinically significant changes. Researchers will also measure drug levels in the blood, immune responses, and fibrosis biomarkers. Safety and tolerability will be carefully monitored throughout the study periods, with follow-up visits to assess ongoing effects and adverse events.
CONDITIONS
Brief Title
A Study of TAK-781 in Healthy Volunteers and in Participants With Non-Cirrhotic Primary Sclerosing Cholangitis (PSC)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Participants aged 18 to 68 years at the time of consent
- Male and female at birth participants
- Willing and able to comply with all trial procedures and requirements
- Provided informed consent prior to any trial procedures
- For healthy participants: judged to be in good health by investigator based on clinical evaluations
- Body mass index less than 30.0 kg/m² at screening
- No frequent or heavy use of medical or recreational cannabis for at least 3 months before screening
- Not a person of childbearing potential (POCBP) or meeting specified criteria for surgical sterility, postmenopausal status, or congenital absence of uterus
- Male participants must use highly effective contraception during and for 6 months after the trial
- For PSC participants: confirmed diagnosis of large-duct PSC by at least two diagnostic criteria
- Fibrogenesis biomarker above predefined level
- Evidence of moderate to advanced fibrosis but no cirrhosis based on specific tests
- No evidence of cholangiocarcinoma or other malignancy on MRI at screening
- Able to learn and comply with subcutaneous injection procedures throughout the trial
You will not qualify if you...
- Employees of sponsor, trial site, or their immediate family members involved in the trial
- Known hypersensitivity to TAK-781 or related compounds
- History of severe allergies or anaphylactic reactions to drugs or food
- History or evidence of bleeding or coagulation disorders
- Prior cholecystectomy
- Participation in another investigational trial within 4 weeks or 5 half-lives before screening
- Elevated liver enzymes (ALT or AST) above normal limits
- Abnormal cholesterol or triglyceride levels at screening
- Reduced kidney function (eGFR ≤90 mL/min/1.73m²)
- ECG abnormalities including prolonged QT interval or significant arrhythmias
- High blood pressure or abnormal resting heart rate outside specified ranges
- Positive drug or alcohol abuse screen within 12 months
- Positive pregnancy test during screening
- Positive HIV, active hepatitis B or C infection at screening
- Major surgery or blood donation within 4 weeks before screening
- Significant illness within 2 weeks before first dose
- Use of prohibited medications or substances during trial
- Smoking or nicotine use exceeding specified limits
- History of malignancy within 5 years
- Evidence or suspicion of cirrhosis or decompensated liver disease
- Overlap syndromes with autoimmune hepatitis or primary biliary cholangitis
- Presence of dominant bile duct strictures of clinical concern
- Prior liver transplantation
- Severe allergic reactions to related RNA therapeutics
- Use of investigational drugs or devices within restricted periods
- Any condition that limits ability to participate or comply with the trial
- Social or geographic factors limiting participation
- Use of ursodeoxycholic acid without completing washout period
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Up to 20 weeks for SAD and Phase 1b; up to 32 weeks for MAD
Participants receive TAK-781 or placebo injections as part of the study drug administration. Healthy participants in single ascending dose (SAD) cohorts receive one dose on Day 1, while others receive multiple doses in multiple ascending dose (MAD) cohorts or a single dose if they have non-cirrhotic PSC. Treatment includes monitoring for safety, tolerability, pharmacokinetics, and pharmacodynamics.
Multiple visits depending on dosing regimen, including dosing day and follow-up visits
Trial Site Locations
Total: 1 location
1
ICON
Salt Lake City, Utah, United States, 84124
Actively Recruiting
Research Team
T
Takeda Contact
How is the study designed?
Study Type
INTERVENTIONAL
Masking
DOUBLE
Allocation
RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
5
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