Actively Recruiting
A Phase I Study to Investigate the Safety of the Ubiquitin Activating Enzyme Inhibitor TAK-243 in Adult Solid Tumor and Lymphoma Patients
Led by National Cancer Institute (NCI) ยท Updated on 2026-05-13
95
Participants Needed
1
Research Sites
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating TAK-243, a drug that inhibits the ubiquitin-activating enzyme, in patients with advanced or metastatic solid tumors and lymphomas. This phase I trial aims to determine the safety, tolerability, and best dose of TAK-243 given intravenously. The study also explores pharmacokinetics, effects on tumor markers, and preliminary anti-tumor activity and survival outcomes. TAK-243 targets an enzyme that is more active in cancer cells, potentially affecting tumor growth and survival. Participants are assigned to one of two treatment arms: Arm A receives TAK-243 intravenously on days 1, 4, 8, and 11 of a 21-day cycle, while Arm B receives the drug once weekly on days 1, 8, and 15 of a 28-day cycle. Patients undergo biopsies during the study and have imaging tests including CT and MRI scans as well as blood collection. Echocardiograms may be performed if clinically needed. Treatment continues until disease progression or unacceptable side effects occur. During the study, patients have regular assessments including tumor biopsies, imaging scans, and blood tests to monitor treatment effects and safety. Researchers measure the maximum tolerated dose, recommended phase II dose, and incidence of dose-limiting toxicities within the first treatment cycle. Follow-up occurs for 30 days after treatment ends to observe pharmacodynamics and tumor response. The total duration of participation depends on treatment cycles and response.
CONDITIONS
Brief Title
Studying TAK-243 in Patients With Advanced Cancer
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Patients with histologically confirmed advanced or metastatic solid tumors or aggressive lymphomas who have received standard or approved therapies with clinical benefit, or indolent lymphomas meeting treatment criteria
- Measurable or evaluable disease
- Age 18 years or older
- ECOG performance status of 0, 1, or 2
- Hemoglobin 9 g/dL or higher (transfusion allowed)
- Absolute neutrophil count 1,000/mcL or higher
- Platelets 75,000/mcL or higher
- Total bilirubin less than or equal to 1.5 times institutional upper limit of normal
- AST/ALT less than or equal to 3 times institutional upper limit of normal or up to 5 times if liver metastases present
- Creatinine less than or equal to 1.5 times institutional upper limit or creatinine clearance 60 mL/min/1.73 m2 or higher
- Prior therapies completed at least 4 weeks or 5 half-lives before enrollment
- Female patients must be postmenopausal, surgically sterile, or agree to use effective contraception
- Male patients must agree to use effective barrier contraception or abstinence during and 4 months after treatment
- Ability and willingness to sign informed consent
- Willingness to provide blood and tumor biopsies for research
- Eligible on anticoagulation if no drug interactions with TAK-243 are expected
You will not qualify if you...
- Receiving other investigational agents
- Uncontrolled illnesses including active infection or psychiatric/social issues limiting study compliance
- Life-threatening illness unrelated to cancer
- Uncontrolled bleeding disorders
- Known severe liver disease or cirrhosis
- Cardiopulmonary disease including unstable angina, severe heart failure, recent heart attack within 6 months, or cardiomyopathy
- Significant arrhythmias including certain types of atrial fibrillation and ventricular fibrillation history
- Prolonged QTc interval 470 msec or higher
- Uncontrolled high blood pressure (systolic >180 mm Hg, diastolic >95 mm Hg)
- Moderate to severe lung disease including COPD, interstitial lung disease, or pulmonary fibrosis
- Major surgery within 14 days prior to first dose
- Females intending to donate eggs or males intending to donate sperm during and 4 months after treatment
- Active or unstable brain metastases except stable treated disease for at least 1 month
- Use of strong CYP3A4/5 inhibitors or inducers, or strong inhibitors of BCRP or OATP transporters from 14 days before enrollment through study end
- Use of live vaccines within 30 days before and 100 days after study drug
- History of allergy to TAK-243 or similar compounds
- Pregnant or breastfeeding women due to potential risks of TAK-243
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 - Multiple 21-day or 28-day cycles until disease progression or unacceptable toxicity
Participants receive UAE inhibitor TAK-243 intravenously on a schedule depending on their assigned arm. Arm A receives the drug on days 1, 4, 8, and 11 of each 21-day cycle. Arm B receives the drug on days 1, 8, and 15 of each 28-day cycle. Treatment cycles repeat in the absence of disease progression or unacceptable toxicity. Participants undergo biopsies, CT and MRI scans, blood collection throughout the study, and may have echocardiograms as clinically indicated.
1 to 4 visits per cycle depending on arm and assessments throughout treatment
Duration - 30 days
After completing treatment, participants are followed for 30 days to monitor safety and collect additional data.
1 follow-up visit
Trial Site Locations
Total: 1 location
1
National Cancer Institute Developmental Therapeutics Clinic
Bethesda, Maryland, United States, 20892
Actively Recruiting
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
2
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