A clinical trial to find a safe and suitable dose of idasanutlin, and to see how well idasanutlin works when given with chemotherapy or venetoclax, in children and young adults with leukemia or solid tumours

How does the iMATRIX idasa clinical trial work?

This clinical trial is recruiting children and young adults who have a type of blood cancer called leukemia, or cancer of any tissue or organs that form solid tumours. In order to take part, patients with leukemia must have either acute myeloid leukemia (or AML) or acute lymphocytic leukemia (or ALL). All patients, including those with solid tumours, must have disease that has not gone into remission after treatment or that has come back after going into remission.

The purpose of the first part of this clinical trial is to find a safe and suitable dose of idasanutlin for children.

The purpose of the second part of this clinical trial is to compare the effects, good or bad, of idasanutlin plus other medicines in patients with leukemia or a particular type of solid tumour called neuroblastoma. If you join the second part of the trial, you will get either idasanutlin plus chemotherapy or idasanutlin plus venetoclax.

How do I take part in this clinical trial?
To be able to take part in this clinical trial, you must have been diagnosed with AML, ALL, or any type of solid tumour that has not gone into remission after treatment or that has come back after going into remission.

You must not have previously been given idasanutlin (or venetoclax if you are in a group that can be given this drug in the clinical trial) and you cannot join the trial if you are pregnant or breastfeeding.

If you think this clinical trial may be suitable for you and would like to take part, please talk to your doctor. If your doctor thinks that you might be able to take part in this clinical trial, he/she may refer you to the closest clinical trial doctor. They will give you all the information you need to make your decision about taking part in the clinical trial. You can also find the clinical trial locations on this page.

You will have some further tests to make sure you will be able to take the treatments given in this clinical trial. Some of these tests or procedures may be part of your regular medical care. They may be done even if you do not take part in the clinical trial. If you have had some of the tests recently, they may not need to be done again.

Before starting the clinical trial, you will be told about any risks and benefits of taking part in the trial. You will also be told what other treatments are available so that you may decide if you still want to take part.

While taking part in the clinical trial, both men and women (if you are not currently pregnant but can become pregnant) will need to either not have heterosexual intercourse or take contraceptive medication for safety reasons.

What treatment will I be given if I join this clinical trial?
This clinical trial will be split into 3 parts. You will only be able to join Part 1 or Part 2/3.

Part 1

Only people with solid tumours under the age of 18 will be able to join Part 1.

Part 1 will find a safe and suitable dose of idasanutlin for children. If you are enrolled in Part 1, you will receive idasanutlin given as a tablet or a drink to swallow every day for the first 5 days of a 4 week treatment cycle.

If idasanutlin does not make you too sick after 4 weeks, you will have the following options:

• Continue to receive idasanutlin given as a tablet or a drink to swallow every day for the first 5 days of each 4 week treatment cycle
• OR receive idasanutlin given as a tablet or a drink to swallow and chemotherapy (cyclophosphamide and topotecan) given as an infusion into your vein (all treatments given every day for the first 5 days of each 4 week treatment cycle)

If idasanutlin makes you too sick during the first cycle of treatment, you will receive idasanutlin every day for the first 5 days of each 4 week treatment cycle, but at a lower dose.

Once a suitable dose of idasanutlin has been found, the trial will try to find the best dose of idasanutlin when given with chemotherapy or venetoclax in patients with solid tumours, AML and ALL. You may be given idasanutlin plus chemotherapy or idasanutlin plus venetoclax depending on your type of cancer. If you have AML or ALL, you will also be given chemotherapy directly into your spine with a lumbar puncture.

The trial will then move on to Part 2.

Part 2

Everyone who joins Part 2 of this clinical trial will be split into groups based on whether they have acute myeloid leukemia (or AML), acute lymphocytic leukemia (or ALL), or a specific type of solid tumour called neuroblastoma.

AML

Everyone with AML will be split into 2 groups and given either:

• idasanutlin given as a tablet or a drink to swallow and chemotherapy (fludarabine and cytarabine) given as an infusion into your vein (treatments given every day for the first 5 days of each 4 week treatment cycle)
• OR idasanutlin given as a tablet or a drink to swallow every day for the first 5 days of each 4 week treatment cycle, and venetoclax given as a tablet or a drink to swallow every day.

Your doctor will tell you which treatment you will receive. You might be placed in a treatment group randomly (like flipping a coin). If this is the case, you will have an equal chance of being placed in either group. You will also be given chemotherapy directly into your spine with a lumbar puncture, regardless of which treatment you are given.

ALL

Everyone with ALL will be given idasanutlin given as a tablet or a drink to swallow every day for the first 5 days of each 4 week treatment cycle, and venetoclax given as a tablet or a drink to swallow every day. You will also be given chemotherapy directly into your spine with a lumbar puncture.

Neuroblastoma

Everyone with neuroblastoma will be split into 2 groups and given either:

• idasanutlin given as a tablet or a drink to swallow and chemotherapy (cyclophosphamide and topotecan) given as an infusion into your vein (treatments given every day for the first 5 days of each 4 week treatment cycle)
• OR idasanutlin given as a tablet or a drink to swallow every day for the first 5 days of each 4 week treatment cycle, and venetoclax given as a tablet or a drink to swallow every day.

Your doctor will tell you which treatment you will receive. You might be placed in a treatment group randomly (like flipping a coin). If this is the case, you will have an equal chance of being placed in either group.

Part 3

If Part 2 shows that the drug works well, more patients may be added to the clinical trial and given the same treatments as in Part 2.

How often will I be seen in follow-up appointments, and for how long?

You will be given the treatment for as long as it can help you. You are free to stop this treatment at any time. After being given your last treatment, the clinical trial doctor will see you after 1 month and then you will be contacted every 3 months. These checks will monitor how you are responding to the treatment and any side effects that you may be having. 

What happens if I am unable to take part in this clinical trial?
If this clinical trial is not suitable for you, you will not be able to take part. Your doctor will suggest other clinical trials that you may be able to take part in or other treatments that you can be given. You will not lose access to any of your regular care.

For more information about this clinical trial see the For Expert tab on the specific ForPatient page or follow this link to ClinicalTrials.gov https://clinicaltrials.gov/ct2/show/NCT04029688?term=GO40871&rank=1 

Trial-identifier: NCT04029688