Active not recruitingPHASE3INTERVENTIONAL

Tretinoin and Arsenic Trioxide in Treating Patients With Untreated Acute Promyelocytic Leukemia

This study is looking into a new way to treat acute promyelocytic leukaemia (APL) in children and young adults. The usual treatment for APL often uses strong chemotherapy medicines that can sometimes cause heart problems later in life. This trial is exploring if a combination of two specific medicines, tretinoin and arsenic trioxide, can be just as effective as traditional chemotherapy, but with fewer risks to the heart over time. Doctors want to see if these two drugs can stop cancer cells from growing and spreading, offering a kinder treatment option for newly diagnosed patients.

At a glance

Status

Active not recruiting

Phase

PHASE3

Sponsor

Children's Oncology Group

Enrolment target

158

Start

21 Jul 2015

Estimated completion

30 Jun 2029

Acute Promyelocytic Leukemia With t(15;17)(q24.1;q21.2); PML-RARA

Results

Results from this study

Posted December 2021

Results have been published for this study.

Primary outcome

Event-free Survival (EFS) in Standard Risk Acute Promyelocytic Leukemia (APL) Patients

EFS is defined as the time from on study to failure to achieve hematological complete response (CR) prior to start of consolidation, persistence of molecular positive disease after minimal residual disease (MRD) positive consolidation course, relapse (molecular, morphologic or extramedullary), or death. The Kaplan-Meier method will be used to estimate 2-year EFS along with 90% log-minus-log transformed confidence limits.

Full results on the registry

What is this study about?

This study is designed for children and young adults who have recently been diagnosed with a specific type of blood cancer called Acute Promyelocytic Leukaemia, or APL. Usually, treating APL involves powerful chemotherapy drugs. While these drugs can be very effective at fighting the cancer, one group of them, called anthracyclines, can unfortunately sometimes lead to long-term side effects, particularly affecting the heart.

In this trial, doctors are investigating an alternative approach. They want to see if using a combination of two different medicines – tretinoin and arsenic trioxide – can effectively treat APL. Tretinoin works by helping to stop cancer cells from growing, while arsenic trioxide can either kill cancer cells, stop them from dividing, or prevent them from spreading. The hope is that this combination can be just as successful at treating APL, but by reducing or even removing the need for high-dose anthracycline chemotherapy, it might also significantly lower the risk of those unwanted long-term side effects on the heart.

Ultimately, this research aims to find a treatment for APL that is not only very effective but also much gentler on the body, improving the overall health and quality of life for young people diagnosed with this condition.

Key takeaways

This study tests a new treatment for Acute Promyelocytic Leukaemia (APL).
It combines tretinoin and arsenic trioxide to reduce reliance on strong chemotherapy.
The goal is to lessen long-term side effects, particularly on the heart.
It's for newly diagnosed patients aged 12 months to 21 years.
Your quality of life and treatment response will be closely monitored.

Who may be eligible?

This study is for children and young adults, aged between 12 months and 21 years old, who have a new diagnosis of Acute Promyelocytic Leukaemia (APL). Doctors will confirm your diagnosis using samples from your bone marrow or blood. It's important that your leukaemia hasn't come back after previous treatment, or developed because of earlier chemotherapy or radiation.

Before joining, doctors will need to check your medical history to make sure this study is the right fit for you. They will also need to confirm that certain genetic markers linked to APL are present in your cells. You might be able to start taking one of the study drugs, tretinoin, for a few days before officially starting the main treatment in the study.

If you have any symptoms that make doctors suspect the cancer has spread to your brain or spinal cord, they might do extra scans. However, they will make sure any bleeding risks are under control before performing certain procedures like a spinal tap. The most important thing is that both you (if you're old enough) and your parents or legal guardians understand and agree to take part by signing a consent form.

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 between 12 months and 21 years old?
Have you recently been diagnosed with Acute Promyelocytic Leukaemia (APL)?
Has your APL NOT come back after previous treatment or developed from prior treatments?
Are you and your parents/guardians willing to provide consent?

Answer every question to see your result.

What does participation involve?

If you join this study, your treatment will involve two main medicines: tretinoin, which you'll take by mouth twice a day, and arsenic trioxide, which will be given directly into your vein over a few hours each day for about a month. Depending on your specific type of APL (whether it's considered 'standard risk' or 'high risk'), you might also receive other medicines like dexamethasone (taken by mouth or given into a vein) and idarubicin (given into a vein).

Throughout the treatment, you will have regular visits to the hospital for checks, blood tests, and to receive your medication. Doctors will closely monitor how you're responding to the treatment. You'll also be asked to fill out questionnaires to assess your quality of life and how you're feeling. The study involves different phases of treatment, starting with an 'induction therapy' phase to get the cancer under control. If this is successful, you'll likely move on to a 'consolidation therapy' phase. The total duration of the study treatment and follow-up will be explained by your medical team.

Potential risks and benefits

Taking part in this study could potentially offer a new treatment option for Acute Promyelocytic Leukaemia that might have fewer long-term side effects on the heart compared to traditional chemotherapy. However, like all medical treatments, there are potential risks, and you may experience side effects from the study medicines. Your doctors will explain these thoroughly and monitor you closely to manage any issues. You are free to withdraw from the study at any time, for any reason, without it affecting your future medical care.

Locations (174)

Children's Hospital of Alabama
Verified postcode
Birmingham, United States
USA Health Strada Patient Care Center
Verified postcode
Mobile, United States
Providence Alaska Medical Center
Verified postcode
Anchorage, United States
Banner Children's at Desert
Verified postcode
Mesa, United States
Phoenix Childrens Hospital
Verified postcode
Phoenix, United States
Arkansas Children's Hospital
Verified postcode
Little Rock, United States
Kaiser Permanente Downey Medical Center
Verified postcode
Downey, United States
City of Hope Comprehensive Cancer Center
Verified postcode
Duarte, United States
Loma Linda University Medical Center
Verified postcode
Loma Linda, United States
Children's Hospital Los Angeles
Verified postcode
Los Angeles, United States
Cedars Sinai Medical Center
Verified postcode
Los Angeles, United States
Valley Children's Hospital
Verified postcode
Madera, United States

Common questions

What is Acute Promyelocytic Leukaemia (APL)?

APL is a specific type of fast-growing blood cancer that affects certain cells in your bone marrow, which are responsible for making blood.

What are tretinoin and arsenic trioxide?

These are two medicines being tested in this study. Tretinoin helps stop cancer cells from growing, and arsenic trioxide helps kill them or stop them from spreading.

Why is this study important for patients?

This study aims to find a treatment for APL that is as effective as current methods but has fewer long-term side effects, especially on the heart, which can be affected by standard chemotherapy.

Will I still receive other treatments?

Depending on your specific condition, you might receive other medicines in addition to tretinoin and arsenic trioxide, as part of the study plan.

Can I choose not to participate?

Yes, taking part is completely voluntary. You have the right to decide whether or not to join, or to leave the study at any time, without it affecting your medical care.