Active not recruitingPHASE2INTERVENTIONAL

Study Of Palbociclib Combined With Chemotherapy In Pediatric Patients With Recurrent/Refractory Solid Tumors

This research study is investigating a new medication called palbociclib when given alongside traditional chemotherapy treatments. It's for children, teenagers, and young adults who have specific types of cancer, like Ewing sarcoma or neuroblastoma, that have either returned after treatment or haven't responded well to previous therapies. The study has a few stages: first, it aims to find the safest dose of palbociclib when combined with different chemotherapy drugs. Later, it will check how effective this combination is, particularly for neuroblastoma and Ewing sarcoma. The main goal is to understand if this new treatment approach can improve outcomes for these young patients.

At a glance

Status

Active not recruiting

Phase

PHASE2

Sponsor

Pfizer

Enrolment target

128

Start

24 May 2019

Estimated completion

18 Oct 2025

Ewing Sarcoma Solid Tumors Rhabdoid Tumor Rhabdomyosarcoma Neuroblastoma Medulloblastoma Diffuse Intrinsic Pontine Glioma

What is this study about?

This study is looking into a new way to treat certain cancers in children, teenagers, and young adults. These are cancers that have either returned after treatment or haven't responded well to standard therapies, making them difficult to treat. The study focuses on different types of solid tumours, including cancers like Ewing sarcoma, neuroblastoma, and medulloblastoma. The main idea is to combine a new drug called palbociclib with commonly used chemotherapy drugs.

Palbociclib works differently from traditional chemotherapy by targeting specific processes within cancer cells. Researchers want to see if adding palbociclib to chemotherapy can make the treatment more effective. The study is divided into different parts. In the first part, the main goal is to find the safest dose of palbociclib when given with different combinations of chemotherapy. This is really important to make sure the treatment is as safe as possible for young patients.

Once a safe dose is found, the study will move on to see how well this new combination treatment works. For example, it will specially look at how effective palbociclib plus chemotherapy is for a type of cancer called neuroblastoma. Another part of the study will compare palbociclib combined with chemotherapy against chemotherapy alone for Ewing sarcoma, to see if adding palbociclib makes a difference. This research is vital for finding better treatment options for young people facing challenging cancers.

Key takeaways

Targets children, teenagers, and young adults with challenging cancers.
Investigates a new drug (palbociclib) combined with chemotherapy.
Aims to find safe doses and assess how well the treatment works.
Focuses on solid tumours like Ewing sarcoma and neuroblastoma.
Offers a potential new treatment option for relapsed or refractory cancers.

Who may be eligible?

To be part of this study, participants generally need to be children, teenagers, or young adults aged between 2 and under 21 years old. They must have a solid tumour that has either returned after previous treatment or hasn't responded well to other treatments. For some parts of the study, this includes specific cancers like Ewing sarcoma, rhabdomyosarcoma, and neuroblastoma. Even if the cancer is in the brain (like a DIPG), participants might be able to join.

Participants need to be well enough to take part, as assessed by doctors. This means their general health, including blood counts (like white blood cells that fight infection, and platelets that help stop bleeding), kidney function, and liver function, must be within healthy limits. Some of these can be okay even if they've had transfusions, but generally, their body systems need to be working quite well.

Specifically for Ewing sarcoma, if you're considering the comparison part of the study, your cancer needs to have certain genetic changes (called EWSR1-ETS or FUS-ETS) that are typical for this type of cancer. Also, your cancer needs to be measurable, meaning doctors can see it and track its progress, often with scans. The study team will check all these details carefully to make sure it's the right fit for each young person.

Quick self-check

Are you between 2 and 20 years old?
Do you have a solid tumour that has returned or is hard to treat?
Are you generally well enough to participate, as determined by your doctor?
Are your blood tests and organ functions (like kidneys and liver) within acceptable limits?
For Ewing sarcoma, does your cancer have specific genetic markers and is it measurable?

This is a guide only — the research team will confirm whether you can take part.

What does participation involve?

If you decide to take part in this study, you will receive the study medication (palbociclib) along with specific chemotherapy drugs. The exact medications and schedule will depend on which part of the study you are in and your specific cancer. You will have regular hospital visits for check-ups, blood tests, and scans to monitor your health and how the treatment is working. These assessments are important to track any side effects and see if the cancer is responding. The duration of your participation will vary depending on the study phase and how you respond to treatment, but it will involve multiple cycles of medication and follow-up appointments. The study team will explain the full schedule and what to expect during your visits.

Potential risks and benefits

Taking part in a clinical trial might offer the chance to receive a new treatment that isn't yet widely available, which could potentially work better for your cancer. However, there are also potential risks, including side effects from the new drug and chemotherapy which could be mild or severe. The research team will closely monitor you for any side effects and provide care. You can also choose to leave the study at any time, for any reason, without it affecting your future medical care.

Locations (101)

University of Alabama at Birmingham/Children's of Alabama
Birmingham, United States
Phoenix Children's Hospital
Phoenix, United States
MemorialCare Health System - Long Beach Medical Center
Long Beach, United States
Children's Hospital Los Angeles
Los Angeles, United States
Children's Hospital and Research Center at Oakland
Oakland, United States
Children's Hospital of Orange County
Orange, United States
Lucile Packard Children's Hospital
Palo Alto, United States
UCSF Medical Center
San Francisco, United States
University of California San Francisco,
San Francisco, United States
Children's Hospital Colorado
Aurora, United States
Children's National Hospital
Washington D.C., United States
UF Health Shands Hospital
Gainesville, United States

+89 more sites — see the official record for the full list.

Common questions

What is palbociclib?

Palbociclib is a medicine that targets specific processes in cancer cells to stop them from growing and dividing. It's not a traditional chemotherapy drug.

Why is this study being done?

This study aims to find safer and more effective ways to treat difficult cancers in young people, especially those that have come back or haven't responded to other treatments.

Will I still receive standard cancer treatment?

Yes, palbociclib will be given in combination with standard chemotherapy drugs that are already used to treat these types of cancers.

What are the common side effects of treatment?

While the study aims to understand side effects, common side effects from similar drugs can include feeling tired, nausea, hair loss, or changes to blood counts. The study team will discuss these with you.

How long will I be in the study?

The length of time you participate can vary, depending on how your body reacts to the treatment and the specific part of the study you are in. The doctors will let you know what to expect.