Ongoing, recruitingPhase I and Phase II (Integrated)- OtherInterventional

Phase I/II multicenter study to assess efficacy and safety of ribociclib (LEE011) in combination with topotecan and temozolomide (TOTEM) in pediatric patients with relapsed or refractory neuroblastoma and other solid tumors

This study is for children and young people with specific cancers, like neuroblastoma or some solid tumours, that haven't responded well to previous treatments or have returned. Researchers are testing a combination of existing cancer drugs (topotecan and temozolomide) with a newer medicine called ribociclib (LEE011). The study has two main parts: Phase I focuses on finding the safest and most effective dose of the drug combination and checking for any serious side effects. Phase II then checks how well this new combination shrinks tumours or stops them from growing. The goal is to see if this treatment could offer a new option for these challenging cancers.

At a glance

Status

Ongoing, recruiting

Phase

Phase I and Phase II (Integrated)- Other

Sponsor

Novartis Pharma AG

Enrolment target

Start

19 Apr 2024

Relapsed or refractory neuroblastoma and other solid tumors (including medulloblastoma high grade glioma malignant rhabdoid tumors and rhabdomyosarcoma)

What is this study about?

This research study is looking into a new way to treat certain cancers in children and young people. These include neuroblastoma and other solid tumours like medulloblastoma, high-grade glioma, malignant rhabdoid tumours, and rhabdomyosarcoma. The study is for patients whose cancer has either come back after treatment or hasn't responded to earlier treatments.

The study is testing a combination of medicines. Two of these, topotecan and temozolomide, are chemotherapy drugs already used to treat cancer. The third medicine, ribociclib (also known as LEE011), is a newer drug that works in a different way to fight cancer cells. The main aim is to see if combining these medicines can be more effective than current treatments and to make sure this combination is safe for young patients.

The study is divided into two phases. In Phase I, the doctors will carefully increase the dose of the new medicine to find the highest dose that can be given without causing too many serious side effects. This helps them find the recommended dose for further study. In Phase II, once the best dose is found, more patients will be given this combination. The doctors will then look closely at how well the tumours respond to the treatment, checking if they shrink or stop growing, and continue to monitor for any side effects.

Key takeaways

This study is for children and young people with certain cancers that have returned or not responded to previous treatments.
It tests a new combination of medicines: ribociclib with two existing chemotherapy drugs.
The first part of the study aims to find the safest and most effective dose.
The second part will assess how well the combination shrinks tumours or stops them growing.
You will have regular checks and tests to monitor your health during the study.

Who may be eligible?

This study is looking for children and young adults who are 18 years old or younger. Both boys and girls can take part.

To be considered, participants must have certain types of cancer, specifically neuroblastoma or other solid tumours such as medulloblastoma, high-grade glioma, malignant rhabdoid tumours, or rhabdomyosarcoma. Importantly, their cancer must have either returned after previous treatment or not responded well to earlier treatments.

There will be other detailed medical criteria that the study doctors will check to make sure the study is a safe and suitable option for each individual patient. If you're interested, your doctor will be able to tell you if you meet all the necessary requirements.

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 18 years old or younger?
Do you have neuroblastoma or a similar solid tumour (like medulloblastoma or high-grade glioma)?
Has your cancer come back after treatment, or not responded to previous treatments?
Are you able to attend regular hospital visits for appointments and tests?
Are you or your parent/guardian willing to provide informed consent?

Answer every question to see your result.

What does participation involve?

If you join this study, you will receive a combination of medicines: topotecan, temozolomide, and ribociclib (LEE011). These medicines will be given in cycles, meaning you'll have periods of treatment followed by breaks. Each treatment cycle usually lasts for 28 days.

During your time in the study, you will have regular hospital visits. These visits will involve various tests and checks to monitor your health, how the treatment is affecting your cancer, and any possible side effects. This will include blood tests, checks of your organs like your heart and liver, and imaging scans (like MRI or CT scans) to see if the tumours are shrinking or stable. The specific number of visits and tests will depend on which phase of the study you are in and how you are responding to treatment. You will be closely monitored throughout the study period.

Potential risks and benefits

Participating in this study might offer a chance to try a new combination therapy that could potentially help treat your cancer, especially if standard treatments haven't worked. However, like all medicines, these drugs can have side effects, which might include feeling unwell, changes in blood counts, or effects on other organs. The study team will monitor you very closely for any side effects and manage them as best they can. It's important to remember that you have the right to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (7)

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

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Unverified
Spain
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Unverified
Germany
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Unverified
France
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Unverified
Denmark
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Unverified
Hungary
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Unverified
Czechia
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Unverified
Italy

Common questions

What is neuroblastoma?

Neuroblastoma is a rare type of cancer that usually starts in certain nerve cells, mainly found in babies and young children.

What does 'relapsed or refractory' mean?

It means the cancer has either returned after being treated (relapsed) or it hasn't responded to previous treatments (refractory).

Are the medicines in this study new?

Two of the medicines, topotecan and temozolomide, are existing cancer drugs. Ribociclib is a newer medicine, and this study is testing it in combination with the others for your type of cancer.

How long will the study last?

The overall study duration for each patient varies, depending on how they respond to the treatment and what the doctors decide is best for their health. Your study team can give you a more personal estimate.

Will I still receive my regular care if I join?

Yes, you will continue to receive all necessary medical care and support from your healthcare team, whether you join the study or not.