This study focuses on patients with Higher-risk Myelodysplastic Syndrome, a condition where bone marrow does not produce enough healthy blood cells. The study will test a combination of two medications: tamibarotene (also known as SY-1425) and azacitidine, comparing them to treatment with azacitidine and placebo. The purpose is to determine how well the combination therapy works in newly diagnosed adult patients whose disease tests positive for a specific marker called RARA.
The treatment involves taking tamibarotene tablets by mouth and receiving azacitidine through injections under the skin or into a vein. Azacitidine is given at a dose of 75 mg per square meter of body surface for 7 days, while tamibarotene is given as a daily tablet. Some patients will receive a placebo tablet instead of tamibarotene, but all patients will receive azacitidine.
The study will track how many patients achieve complete remission (when signs of cancer disappear) and will also monitor how long patients survive, whether they need fewer blood transfusions, and how the treatment affects their quality of life. Doctors will regularly check patients’ blood counts and bone marrow to assess how well the treatment is working and watch for any side effects.
1Initial assessment
Your eligibility for the study will be confirmed through medical tests and examinations
A bone marrow test will be performed to verify that bone marrow blasts are greater than 5%
Laboratory tests will check your liver and kidney function
If you are a woman who can become pregnant, a pregnancy test will be conducted
2Treatment assignment
You will be randomly assigned to one of two treatment groups
Group 1: Tamibarotene tablets plus Azacitidine injections
Group 2: Placebo tablets plus Azacitidine injections
Neither you nor your doctor will know which tablet treatment group you are in
3Treatment administration
Azacitidine will be given as an injection under the skin or into a vein
You will take either Tamibarotene or placebo tablets by mouth
Regular blood tests and medical examinations will monitor your response to treatment
Your quality of life will be assessed through questionnaires
4Monitoring period
Your condition will be regularly monitored for any changes in disease status
Blood cell counts will be checked regularly
Side effects will be tracked through laboratory tests and physical examinations
The study will continue until February 2031
Who Can Join the Study?
Must be at least 18 years old when signing the consent form
Must test positive for RARA (a specific protein marker) using a special test
Must be newly diagnosed with higher-risk myelodysplastic syndrome (HR-MDS) with specific risk scores:
Very High (score greater than 6)
High (score 4.5 to 6)
Intermediate (score 3 to 4.5)
Must have more than 5% blast cells (immature blood cells) in bone marrow
Must have an ECOG Performance Status of 2 or less (ability to carry out daily activities)
Must have adequate organ function, including:
Acceptable liver function tests
Kidney function showing filtration rate of at least 30 mL/min
Women who can become pregnant must have a negative pregnancy test
Must be willing to:
Attend all scheduled study visits
Follow treatment plans
Complete required laboratory tests
Use two methods of birth control, including a barrier method
Must be able to understand and sign an informed consent document
Who Cannot Join the Study?
Prior treatment with hypomethylating agents (drugs that affect DNA)
Active or prior acute myeloid leukemia (a type of blood cancer)
History of other cancers within the past 3 years, except for successfully treated skin cancer or early-stage cancer
Severe heart conditions, including:
Uncontrolled high blood pressure
Recent heart attack
Unstable heart disease
Severe liver problems or active liver disease
Severe kidney problems requiring dialysis
Active, uncontrolled infections
Known HIV infection or active hepatitis B or C
Pregnancy or breastfeeding
Mental conditions that could interfere with following study procedures
Participation in other clinical trials within 30 days before this study
Tamibarotene
A medication that belongs to a class of drugs called retinoids. It works by targeting specific genes and proteins in cancer cells. In this trial, it is being studied for treating patients with higher-risk myelodysplastic syndrome, a condition where the bone marrow doesn’t produce enough healthy blood cells.
Azacitidine
A chemotherapy medication that helps bone marrow produce normal blood cells and kills abnormal cells in the bone marrow. It is commonly used to treat myelodysplastic syndrome. This medication works by interfering with the DNA of cancer cells, which helps prevent them from growing and dividing.
Placebo
An inactive substance that contains no medication. It is used as a control to help determine if the experimental treatment is effective. Some patients in this study receive a placebo along with azacitidine to compare results with those who receive tamibarotene plus azacitidine.
Myelodysplastic Syndrome – A group of blood disorders where the bone marrow does not produce enough healthy blood cells. The condition occurs when blood-forming cells in the bone marrow become abnormal and create defective cells. This leads to low levels of one or more types of blood cells: red blood cells, white blood cells, or platelets. The condition primarily affects older adults and develops gradually over time. People with this condition may experience fatigue, frequent infections, and easy bruising or bleeding due to the lack of proper blood cell production.
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