A Phase 1/2 Study of Enzomenib (DSP-5336) in Patients With Acute Leukemia (Horizen-1)
This study is called Horizen-1 and is investigating a new medication called Enzomenib (or DSP-5336) for patients with certain types of blood cancer, specifically acute leukaemia, myelodysplastic syndromes (MDS), or multiple myeloma. It's a Phase 1/2 study, which means it will first check the best and safest dose of Enzomenib. Then, it will see how well it works. Researchers will study Enzomenib given by itself or together with other standard treatments for these conditions. The main goals are to understand the drug's safety, how it moves through the body, and its potential to help patients whose current treatments are no longer working or who are newly diagnosed with specific genetic markers.
At a glance
What is this study about?
This research study, named Horizen-1, is looking into a new medication called Enzomenib (also known as DSP-5336). It's designed for people who have specific types of blood cancer, such as acute leukaemia (both myeloid and lymphocytic types), myelodysplastic syndromes (MDS), and multiple myeloma. When a study is called 'Phase 1/2', it means the first part (Phase 1) is focused on finding the safest and most effective dose of the new drug. Once that dose is found, the second part (Phase 2) will then expand to include more patients to see how well the drug works against the cancer.
The main purpose of this study is to carefully check the safety of Enzomenib. Researchers will also learn how the drug acts in the body – for example, how it's absorbed, where it goes, and how it's removed. Importantly, they want to see if it can help patients whose cancer hasn't responded to previous treatments, or whose cancer has returned. For some patients with newly diagnosed acute myeloid leukaemia (AML) that has specific genetic changes, the study will also test Enzomenib alongside common treatments they would already be receiving.
By taking part in this study, you would be helping doctors understand if Enzomenib could be a new treatment option for these difficult-to-treat cancers. This kind of research is vital for developing better medicines in the future, offering hope to many patients.
Key takeaways
- This study is testing a new drug called Enzomenib for certain blood cancers.
- It's for people with acute leukaemia, MDS, or multiple myeloma, especially if other treatments haven't worked.
- The study will check the drug's safety, how it works, and its potential benefits.
- Enzomenib might be given alone or with other standard chemotherapy.
- It's a Phase 1/2 study, meaning it first finds the best dose and then looks closely at its effectiveness.
Who may be eligible?
This study is looking for adults aged 12 and older who have been diagnosed with certain types of blood cancer. This includes acute myeloid leukaemia (AML), acute lymphocytic leukaemia (ALL), or acute leukaemia of uncertain type, where other standard treatments have stopped working or the disease has come back. For some patients, this might mean you've had a stem cell transplant as an option, but your cancer has returned.
In specific hospitals and regions, the study is also open to adults with myelodysplastic syndromes (MDS) if their disease has relapsed or didn't respond to at least two cycles of standard treatment, and they have at least 5% blast cells in their bone marrow. For multiple myeloma patients, you might qualify if you've tried at least three different treatments that included a proteasome inhibitor, an immunomodulatory drug, and a CD38 antibody, and your disease has progressed, and you are not able to receive other available beneficial treatments. You'll also need to meet certain other health requirements and have measurable disease.
Patients with newly diagnosed AML who have specific genetic changes (called MLLr or NPM1m) may also be eligible to receive Enzomenib along with their usual intensive or non-intensive chemotherapy treatments. Your doctor will carefully check all your medical records to see if you meet all the specific criteria for this study.
Could this study suit you?
Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.
- Are you 12 years old or older?
- Do you have acute leukaemia, MDS with specific criteria, or multiple myeloma that hasn't responded to at least three previous treatments?
- Has your cancer come back or not responded to previous standard treatments?
- Are you able to take medication by mouth?
- Are you able to attend regular hospital visits for tests and check-ups?
What does participation involve?
If you decide to join this study, you will take the study drug, Enzomenib, by mouth. This will happen over several cycles, and you might also receive other standard cancer treatments alongside it, depending on which part of the study you are in. You will have regular visits to the hospital or clinic for assessments such as blood tests, urine tests, physical examinations, and scans to check your health and how the drug is working. These visits will be more frequent at the beginning and then become less frequent over time.
The study involves carefully tracking your health, any side effects you experience, and how the cancer responds. The exact duration of your participation will depend on how your body reacts to the treatment and the stage of the study you are in, but it will involve ongoing check-ups for a certain period. The total duration will be discussed with you by the study team.
Potential risks and benefits
Locations (104)
- Hoag Family Cancer CenterVerified postcodeNewport Beach, United States· Recruiting
- Stanford UniversityVerified postcodePalo Alto, United States· Not yet recruiting
- Colorado Blood Cancer InstituteVerified postcodeDenver, United States· Recruiting
- Georgetown Lombardi Comprehensive Cancer CenterVerified postcodeWashington D.C., United States· Not yet recruiting
- University of MiamiVerified postcodeMiami, United States· Recruiting
- Miami Cancer InstituteVerified postcodeMiami, United States· Not yet recruiting
- Moffitt Cancer CenterVerified postcodeTampa, United States· Not yet recruiting
- NorthwesternVerified postcodeChicago, United States· Recruiting
- Sibley Memorial HospitalVerified postcodeBaltimore, United States· Recruiting
- University of MarylandVerified postcodeBaltimore, United States· Recruiting
- Johns Hopkins Main CenterVerified postcodeBaltimore, United States· Recruiting
- Tufts UniversityVerified postcodeBoston, United States· Withdrawn
Common questions
What is acute leukaemia?
Acute leukaemia is a fast-growing cancer of the blood and bone marrow, which are responsible for making blood cells. It interferes with the production of healthy blood cells.
What is Enzomenib (DSP-5336)?
Enzomenib is a new drug being tested in this study. It's a type of treatment that aims to target specific parts of cancer cells.
What does 'relapsed or refractory' mean?
This means your cancer has either come back after treatment (relapsed) or it hasn't responded to previous treatments (refractory).
What are the different 'phases' of a clinical trial?
Clinical trials often have phases (Phase 1, 2, 3). Phase 1 checks for safety and dosage. Phase 2 checks if the drug works and continues to monitor safety. Phase 3 compares it to existing treatments.
Will I receive Enzomenib by itself or with other drugs?
Depending on your specific condition and which part of the study you are in, you might receive Enzomenib alone or together with standard treatments you would normally receive for your cancer.
How to find out more
Matt Hitron, MD
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
Discussion
Community discussion
Powered by our forum at community.patient.info. Please be respectful — this is not medical advice.