Active not recruitingPHASE1, PHASE2INTERVENTIONAL

Total Therapy for Infants With Acute Lymphoblastic Leukemia (ALL) I

This research study, called 'Total Therapy for Infants With Acute Lymphoblastic Leukemia (ALL) I', is for babies diagnosed with a specific blood cancer called acute lymphoblastic leukaemia (ALL). Doctors are testing two new medications, bortezomib and vorinostat, alongside the usual chemotherapy. They want to see if adding these new drugs can help reduce the number of cancer cells more effectively, but also understand any new side effects they might cause. These new drugs are approved for other cancers in adults but haven't been widely used for children with leukaemia before. The main goals are to find out if these combinations are safe and tolerable, how well they help children survive without the cancer coming back, and to closely monitor the cancer cells during and after treatment.

At a glance

Status

Active not recruiting

Phase

PHASE1, PHASE2

Sponsor

St. Jude Children's Research Hospital

Enrolment target

Start

29 Jan 2016

Estimated completion

01 Oct 2031

Acute Lymphoblastic Leukemia

Results

Results from this study

Posted June 2023

Results have been published for this study.

Primary outcome

Percentage of Treatment-related Mortality (TRM)

Number of treatment related deaths divided by total number of patients during induction or reinduction therapy. Presented as percentage

Full results on the registry

What is this study about?

This study is designed for babies who have been recently diagnosed with a type of blood cancer called acute lymphoblastic leukaemia (ALL). ALL is a serious condition where the body makes too many abnormal white blood cells. Researchers are looking into whether adding two newer drugs, bortezomib and vorinostat, to the standard chemotherapy treatments can make a difference. These drugs are already used to treat other cancers in adults, but doctors need to carefully check if they are safe and effective for very young children with ALL.

The main aim of this study is to understand how well these new drug combinations are tolerated by infants. This means looking closely at any side effects and how the babies cope with the treatment. The researchers also want to find out if adding these drugs can improve the long-term health of these children, specifically how long they stay free from the cancer and their overall survival. They will also be meticulously checking for any remaining cancer cells during different stages of treatment to see how well the drugs are working.

The treatment journey in this study is divided into several stages. It starts with an 'Induction' phase to get the cancer into remission, followed by 'Consolidation' to kill any remaining cancer cells, and then 'Reinduction' to try to achieve remission again if needed. Some children might also have a 'Maintenance' phase to keep the cancer from coming back. This structured approach helps doctors target the cancer effectively at different points, with the goal of giving these very young patients the best possible chance.

Key takeaways

This study is for babies 1 year old or younger with newly diagnosed ALL.
It tests new drug combinations (bortezomib and vorinostat) with standard chemotherapy.
The main goals are to check for safety, tolerability, and better treatment outcomes.
Treatment involves multiple phases with careful monitoring and follow-up.
Parents must give informed consent for their baby to participate.
Participation is voluntary, and you can withdraw at any time.

Who may be eligible?

This study is specifically for babies who are 365 days old or younger at the time they are diagnosed with acute lymphoblastic leukaemia (ALL) or a very similar diagnosis called acute undifferentiated leukaemia. We are looking for infants who have just been diagnosed and haven't had much treatment yet. This includes babies with different types of ALL, including T-cell ALL.

There are some reasons why a baby might not be able to join the study. For example, if they have already had extensive treatment for their leukaemia, or if they have other specific types of leukaemia like mature B-cell ALL or acute myelogenous leukaemia (AML), they wouldn't be suitable. Babies born with Down syndrome are also not able to take part in this particular study.

Before a baby can join, their parent or legal guardian must fully understand the study and agree to their participation in writing. This is a very important step to make sure everyone is comfortable and well-informed.

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.

Is your baby 1 year old or younger?
Has your baby been newly diagnosed with acute lymphoblastic leukaemia (ALL) or acute undifferentiated leukaemia?
Has your baby had very little or no prior leukaemia treatment?
Does your baby NOT have Down syndrome?
Are you able and willing to sign a consent form for your baby to join?

Answer every question to see your result.

What does participation involve?

If your baby takes part in this study, the treatment will be divided into specific phases over several months. You will need to visit the hospital regularly for your baby to receive their medications, which are given in different ways, including infusions (into a vein) and directly into the fluid around the spinal cord. Doctors and nurses will check your baby very closely throughout the treatment to see how they are responding and to manage any side effects.

Regular tests, such as blood tests and bone marrow checks, will be done to monitor the leukaemia cells and your baby's general health. The total duration of the full treatment can vary, with the maintenance phase lasting up to 20 cycles, which can be a long-term commitment. You will be given detailed information about the schedule and what to expect at each stage. After the main treatment, there will also be follow-up appointments to ensure your baby continues to do well.

Potential risks and benefits

Participating in this study might offer potential benefits to your baby, as they would be receiving new drug combinations that could potentially improve their chances of getting better. However, there are also potential risks involved, as with any medical treatment. The new drugs being tested may cause side effects, some of which could be serious or currently unknown in infants. The research team will carefully monitor your baby for any side effects and provide the best possible care. You have the right to withdraw your baby from the study at any time, for any reason, without it affecting their ongoing medical care.

Locations (15)

Children's Hospital Los Angeles
Verified postcode
Los Angeles, United States
Children's Hospital of Orange County
Verified postcode
Orange, United States
Lucile Packard Children's Hospital Stanford University
Verified postcode
Palo Alto, United States
Children's Hospital and Clinics of Minnesota
Verified postcode
Minneapolis, United States
St. Jude Affiliate-Charlotte
Verified postcode
Charlotte, United States
Cincinnati Children's Hospital
Verified postcode
Cincinnati, United States
Oregon Health and Science University
Verified postcode
Portland, United States
St. Jude Children's Research Hospital
Verified postcode
Memphis, United States
Children's Hospital of the King's Daughters (CHKD)
Verified postcode
Norfolk, United States
Alberta Children's Hospital
Verified postcode
Calgary, Canada
Stollery Children's Hospital
Verified postcode
Edmonton, Canada
Children's & Women's Health Centre of British Columbia
Verified postcode
Vancouver, Canada

Common questions

What is ALL?

ALL (acute lymphoblastic leukaemia) is a type of cancer that affects the white blood cells, which are part of the body's immune system. In ALL, the body makes too many abnormal white blood cells.

Why are these new drugs being tested?

Bortezomib and vorinostat are approved for other types of cancer in adults, and doctors believe they might also help treat leukaemia in babies more effectively when combined with standard chemotherapy. This study aims to find out if they are safe and work well.

Will my baby receive extra care in this study?

Yes, your baby will be closely monitored by a dedicated research team throughout the study. This means they will receive very careful attention to track their progress and manage any side effects.

What does 'remission' mean?

'Remission' means that tests can no longer find signs of the cancer in the body. The aim of treatment is to achieve and maintain remission.

What if my baby needs a stem cell transplant?

This study focuses on drug treatments, but if a stem cell transplant becomes necessary, the study team will discuss this with you, and it would be arranged separately from this specific trial.