Active not recruitingPHASE1, PHASE2INTERVENTIONAL

A Study to Assess the Safety and Efficacy of Two Combinations of Isocitrate Dehydrogenase (IDH) Mutant Targeted Therapies Plus Azacitidine in Participants With Newly Diagnosed Acute Myeloid Leukemia (AML) Harboring IDH Mutations Who Are Not Candidates to Receive Intensive Induction Chemotherapy

This research study is for adults newly diagnosed with a specific type of blood cancer called Acute Myeloid Leukaemia (AML). This AML has a particular gene change (called an IDH mutation). This study is designed for individuals who aren't able to receive the usual strong chemotherapy. We're looking at two new combinations of medicines: one new drug called AG-120 with azacitidine, and another new drug called AG-221 with azacitidine. The main goals are to find the best and safest dose of these combinations, and to understand how well they work compared to azacitidine alone. This is to see if these new combinations can be a better option for treating AML.

At a glance

Status

Active not recruiting

Phase

PHASE1, PHASE2

Sponsor

Celgene

Enrolment target

130

Start

03 Jun 2016

Estimated completion

30 Sep 2026

Leukemia, Myeloid, Acute

What is this study about?

This study is about finding better treatments for a type of blood cancer called Acute Myeloid Leukaemia (AML). AML affects the blood and bone marrow, which is the spongy part inside your bones that makes blood cells. In AML, the body makes too many abnormal white blood cells.

Specifically, this study is for people whose AML has a particular change in their genes, called an IDH mutation. This mutation can cause the cancer cells to grow. The study is particularly for those individuals who, for various health reasons, might not be suitable for the standard, very strong chemotherapy treatments that are often used for AML.

The researchers want to test two new combinations of medicines. One combination involves a new drug called AG-120 with an existing drug called azacitidine. The other combination uses a different new drug called AG-221, also with azacitidine. The main aims are to work out the safest and most effective dose for these combinations, and to see how well they treat the AML compared to using azacitidine on its own.

Key takeaways

This study explores new treatments for a specific type of AML (with an IDH gene change).
It's for adults who can't have usual strong chemotherapy.
Two new drug combinations are being tested against an existing treatment.
The main goals are to find safe doses and see how well they work.
Participation involves treatments, regular check-ups, and bone marrow samples.
You can withdraw from the study at any time.

Who may be eligible?

This study is looking for adults who have recently been diagnosed with a type of blood cancer called Acute Myeloid Leukaemia (AML). Your AML must have a specific gene change (called an IDH mutation).

You might be able to take part if you are generally well enough to do most routine daily activities. You must also be willing to have samples taken from your bone marrow (the spongy part inside your bones) more than once during the study.

You cannot join if you have certain other specific types of leukaemia, or if you've already had a different targeted treatment for your IDH mutation. If there's a concern your cancer has spread to your brain or spinal cord, you also wouldn't be able to join.

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 an adult (18 years or older)?
Have you been newly diagnosed with Acute Myeloid Leukaemia (AML)?
Does your AML have an IDH gene mutation?
Are you unable to receive strong, intensive chemotherapy?
Are you generally well enough to do most daily activities?

Answer every question to see your result.

What does participation involve?

If you join this study, you would receive either one of the new drug combinations (AG-120 plus azacitidine, or AG-221 plus azacitidine) or azacitidine on its own. You would have regular appointments at the hospital for treatments, blood tests, and check-ups to see how you are responding to the medication and to monitor for any side effects. You would also need to have bone marrow samples taken at certain times. The total length of your participation would depend on how your AML responds to the treatment and what your doctors decide is best for you.

Potential risks and benefits

Participating in this study might offer potential benefits, such as access to new treatments for AML that are not yet widely available. It could also help researchers learn more about treating AML with an IDH mutation, which could help others in the future. However, like all medications, the study drugs may have side effects, which could be mild or more serious. The research team will carefully monitor you for any side effects throughout the study. You will be given more detailed information about the potential risks and benefits before deciding to take part, and you are always free to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (49)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

Local Institution - 105
Verified postcode
Duarte, United States
Local Institution - 107
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New Haven, United States
Local Institution - 108
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Chicago, United States
Local Institution - 103
Verified postcode
Chicago, United States
Local Institution - 102
Verified postcode
Boston, United States
Beth Israel Deaconess Medical Center
Verified postcode
Boston, United States
Local Institution - 902
Verified postcode
Boston, United States
Local Institution - 106
Verified postcode
New York, United States
Local Institution - 110
Verified postcode
Dallas, United States
Local Institution - 178
Verified postcode
Adelaide, Australia
Local Institution - 175
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Melbourne, Australia
Local Institution - 177
Verified postcode
Perth, Australia

Common questions

What is AML?

AML stands for Acute Myeloid Leukaemia. It's a fast-growing type of cancer that affects the blood and bone marrow, where new blood cells are made.

What is an IDH mutation?

An IDH mutation is a specific change in a gene that can be found in some people with AML. This change can contribute to the cancer growing.

Why is this study for people who can't have strong chemotherapy?

Some people might not be well enough for standard strong chemotherapy due to other health conditions or their general fitness. This study is looking for gentler, targeted treatment options for them.

What are AZ-120, AZ-221, and azacitidine?

AG-120 and AG-221 are new medications being tested to target the specific IDH mutation in AML. Azacitidine is an existing medication used to treat certain blood cancers.

Will I definitely get the new drugs if I join?

This study compares the new drug combinations to azacitidine alone. You might receive one of the new combinations or azacitidine by itself, depending on what the study design decides for you.