Study of SY-1425 (tamibarotene) alone or with azacitidine in adult patients with Acute Myeloid Leukemia (AML) or Myelodysplastic Syndrome (MDS)

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What is this study about?

This clinical trial studies two blood disorders: Acute Myeloid Leukemia (AML) and Myelodysplastic Syndrome (MDS). These conditions affect the way blood cells develop in the bone marrow. The study tests a medication called SY-1425 (also known as tamibarotene), which will be given either alone or combined with another medication called azacitidine. The purpose is to determine how well these treatments work in patients whose blood disorders have specific biological markers.

The treatment involves taking SY-1425 as tablets by mouth, while azacitidine is given through injection either under the skin or into a vein. SY-1425 works by targeting specific proteins in cancer cells that help control cell growth. Azacitidine works by helping bone marrow produce normal blood cells and by killing abnormal cells in the bone marrow.

The study will monitor how patients respond to the treatment by checking their blood cell counts and examining their bone marrow. Doctors will also track any side effects that patients may experience during treatment. The treatment period may last up to 78 weeks, depending on how well patients respond to the medication.

1 Initial evaluation

A blood test will be performed to check for specific biomarkers.

A bone marrow sample will be taken to assess your condition.

Your overall health status and organ function will be evaluated.

2 Treatment assignment

Based on your condition and test results, you will be assigned to one of the treatment groups.

The treatment may include SY-1425 tablets alone or in combination with other medications.

If combination therapy is assigned, it may include azacitidine (given through IV or under the skin) or daratumumab.

3 Treatment period

You will receive the assigned treatment according to a specific schedule.

Regular blood tests will be performed to monitor your response.

Periodic bone marrow samples will be taken to evaluate treatment effectiveness.

Your vital signs and any side effects will be monitored throughout the treatment.

4 Response monitoring

Your response to treatment will be measured by checking blood cell counts and bone marrow samples.

If you require blood transfusions, these will be recorded and monitored.

ECG tests (heart rhythm measurements) will be performed periodically.

Any side effects or changes in your condition will be documented.

5 Follow-up period

After completing treatment, you will continue to be monitored for your response.

Regular health checks and blood tests will continue.

The total study duration may extend until July 2026.

Who Can Join the Study?

Must be at least 18 years old
Must not have received any experimental treatments within 2 weeks before starting the study
Any side effects from previous therapy must be resolved or mild
Female participants who can become pregnant must have negative pregnancy tests before starting treatment
Must be willing and able to follow the study schedule, including treatments and laboratory tests
Must sign an informed consent document
Must have either:
- Leukemia that has returned after treatment or did not respond to standard therapy, with at least 5% abnormal cells in bone marrow
- High-risk bone marrow disorder (MDS) that has returned or did not respond to previous treatments
- Newly diagnosed leukemia and unable to receive standard intensive treatment due to age or health conditions
- Lower-risk MDS requiring regular blood transfusions and not responding to standard treatments
Must test positive for specific genetic markers (RARA or IRF8) through a blood test
Must be willing to undergo bone marrow samples and blood tests during the study
Must have acceptable physical functioning status (ECOG score of 0-2, with some exceptions for newly diagnosed patients)
Must have adequate liver and kidney function as shown by blood tests
Must stop any chemotherapy, radiation therapy, or growth factors at least 2 weeks before starting the study (except for hydroxyurea)

Who Cannot Join the Study?

Prior treatment with any RARA agonist (a type of drug that affects retinoic acid receptors)
Active or uncontrolled central nervous system involvement from leukemia
Acute promyelocytic leukemia (a specific type of blood cancer)
Significant heart conditions, including:
- Uncontrolled heart failure
- Unstable angina (chest pain)
- Heart attack within the past 6 months
Severe liver dysfunction (poor liver function)
Severe kidney dysfunction (poor kidney function)
Active, uncontrolled infections
Known HIV infection or active hepatitis B or C
Other active cancers requiring treatment
Pregnancy or breastfeeding
Unable to take oral medications
Any condition that would compromise the safety of the participant or affect study results

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name	City	Country	Status
Centre Hospitalier Universitaire De Bordeaux	Bordeaux	France

Other Sites

No sites found in this category

Trial status

Country	Status	Recruitment Start
France	Not recruiting	16.09.2016

Trial locations

Investigated drugs:

SY-1425 is a selective retinoic acid receptor alpha agonist. This medication works by targeting specific receptors in cancer cells to help control the growth of leukemia and myelodysplastic syndrome cells. It is being studied as a potential treatment for blood cancers.

Daratumumab is a targeted therapy that works by helping the immune system fight cancer cells. It is an antibody that attaches to specific proteins on cancer cells, helping the body’s immune system identify and destroy these cells.

Investigated diseases:

Acute myeloid leukaemia

Acute Myeloid Leukemia (AML) – A type of blood and bone marrow cancer that affects the production of blood cells. In AML, the bone marrow makes abnormal white blood cells that grow rapidly and interfere with normal blood cell production. These abnormal cells build up in the bone marrow and blood, leaving less room for healthy blood cells. The disease can progress quickly, affecting the production of red blood cells and platelets.

Myelodysplastic Syndrome (MDS) – A group of disorders where bone marrow fails to produce enough healthy blood cells. The immature blood cells (called blasts) are abnormal and either die in the bone marrow or shortly after entering the blood. This leads to low numbers of one or more types of blood cells. MDS can be divided into lower-risk and higher-risk forms, with varying degrees of blood cell production problems.

Trial ID:

2024-516281-11-00

Protocol code:

SY-1425-201

NCT ID:

NCT02807558

Trial Phase:

Therapeutic exploratory (Phase II)

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Study of SY-1425 (tamibarotene) alone or with azacitidine in adult patients with Acute Myeloid Leukemia (AML) or Myelodysplastic Syndrome (MDS)

What is this study about?

Who Can Join the Study?

Who Cannot Join the Study?