Treatment Guided by Comprehensive Genome and Transcriptome Analysis Versus Standard of Care in Advanced Rare Cancers
This study, called RATIONALE, is for people with advanced rare cancers. Rare cancers are less common and often harder to treat. Researchers want to see if a very detailed genetic analysis of a patient's cancer, looking at all its genes (like a blueprint), can help doctors choose more effective treatments. They will compare this 'tailored' approach to the usual standard care. Patients will be split into two groups: one gets the tailored treatment right away, and the other gets standard treatment first, with the option for tailored treatment later if needed. The main goal is to find out if the tailored treatment can stop the cancer from getting worse for longer, and if it improves patients' quality of life.
At a glance
What is this study about?
Imagine your cancer cells have a unique instruction manual. This study, called RATIONALE, wants to read that manual very carefully. For people with rare cancers, which are less common and often don't have many treatment options, understanding these instructions could be key. We know that rare cancers can be tough to treat, partly because there hasn't been enough research specifically for them. This study aims to change that by using cutting-edge genetic tests to get a much clearer picture of what makes each person's cancer tick.
Researchers will use advanced techniques, like looking at all the genes in a cancer cell (called 'genome and transcriptome analysis'), to find specific changes or weaknesses. A team of experts will then review these findings to suggest the most suitable treatments. This might include medicines already available, or even matching patients to other specialized trials. The idea is that a treatment specifically chosen for your cancer's unique genetic make-up might be more effective than a 'one-size-fits-all' approach.
The study will compare two groups of patients: one group will get this 'molecularly guided' treatment straight away. The other group will receive the standard treatment first, as decided by their doctor. If the standard treatment isn't working or causes too many side effects, patients in this second group would then have the option to switch to a molecularly guided approach. The main thing the researchers want to find out is if the molecularly guided treatment can help patients live longer without their cancer progressing, but they will also look at how patients feel and how well they respond to treatment.
Key takeaways
- This study is for people with advanced rare cancers.
- It compares standard care with treatment tailored to your cancer's genetics.
- The goal is to see if tailored treatment can stop cancer progression for longer.
- Patients are assigned to groups randomly, either getting tailored treatment immediately or later.
- Being part of the study involves genetic testing, regular check-ups, and questionnaires.
- You can withdraw from the study at any time.
Who may be eligible?
To be considered for this study, you need to be at least 18 years old and have an advanced rare cancer (either epithelial or mesenchymal type) that has spread or is locally advanced. Your cancer should be getting worse or expected to get worse, and you should have already tried at least one standard treatment, or there isn't a standard treatment available for your specific type of cancer.
It's important that we can get a small sample of your tumor tissue (a biopsy) or use a recent sample taken within the last three months. You also need to be generally well enough to take part in the study, meaning you are able to carry out most daily activities without too much help. You must be able to understand what the study involves and be willing to consider the treatment recommendations that come from the genetic testing.
There are also some reasons why you might not be able to join. For example, if you have severe memory problems, epilepsy that needs medication, or have had a bone marrow or organ transplant in the past. People with blood cancers (like leukaemia) or primary brain tumours are also not included. If your cancer has spread to your brain or spinal cord and is causing severe symptoms, you might not be eligible unless it's been successfully treated and isn't causing problems anymore.
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 or older?
- Do you have an advanced rare cancer?
- Has your cancer started to progress, or is it expected to?
- Have you tried at least one standard treatment, or is there no standard option?
- Are you generally well enough to manage daily activities?
- Can you provide a fresh or recent tumour sample?
What does participation involve?
If you join this study, you'll first have detailed genetic tests done on your cancer tissue. You'll then be randomly assigned to one of two groups. One group will start treatment based on these genetic results right away. The other group will receive standard care first, with the option to switch to genetically guided treatment later if needed. The specific treatments you receive will depend on your cancer's genetic make-up and what your doctors recommend.
Throughout the study, you'll have regular appointments for check-ups, scans (like CT or MRI), and blood tests to monitor your cancer and your overall health. You'll also be asked to complete questionnaires about how you're feeling and your quality of life. The exact duration of your participation will depend on how your treatment progresses, but the study will regularly assess how well the treatments are working and how you are coping. The total duration for individual patients is not universally fixed but will be guided by treatment response and patient wellbeing.
Potential risks and benefits
Locations (16)
- Universitätsklinikum AugsburgVerified postcodeAugsburg, Germany· Recruiting
- Charité BerlinVerified postcodeBerlin, Germany· Recruiting
- Universitäts-Klinikum KölnVerified postcodeCologne, Germany· Recruiting
- Medizinische Fakultät der TU DresdenVerified postcodeDresden, Germany· Recruiting
- Uniklinikum ErlangenVerified postcodeErlangen, Germany· Recruiting
- Universitätsmedizin EssenVerified postcodeEssen, Germany· Not yet recruiting
- Universitätsklinikum Freiburg, Tumorzentrum Freiburg - CCCFVerified postcodeFreiburg im Breisgau, Germany· Not yet recruiting
- Universitätsklinikum Hamburg-Eppendorf (UKE)Verified postcodeHamburg, Germany· Not yet recruiting
- Universitätsklinikum HeidelbergVerified postcodeHeidelberg, Germany· Recruiting
- Universitätsmedizin der Johannes Gutenberg- Universität MainzVerified postcodeMainz, Germany· Not yet recruiting
- Comprehensive Cancer Center , LMU MünchenVerified postcodeMünchen, Germany· Not yet recruiting
- Comprehensive Cancer Center Ostbayern (CCCO) Universitätsklinikum Regensburg,Verified postcodeRegensburg, Germany· Recruiting
Common questions
What is a 'rare cancer'?
A rare cancer is one diagnosed in fewer than 6 out of every 100,000 people each year. There are many different types.
What does 'molecularly guided' treatment mean?
It means doctors choose treatments based on a detailed genetic fingerprint of your specific cancer cells, rather than a general approach.
Will I definitely get the new type of treatment?
You will be randomly assigned to one of two groups. One group gets the molecularly guided treatment first, the other gets standard treatment first with a chance for the guided treatment later.
What are the main tests involved?
The main tests involve detailed genetic analysis of your tumour tissue (like whole-genome sequencing) to understand its unique characteristics.
How long will I be in the study?
Your time in the study will depend on how your treatment progresses and how your cancer responds, but you'll have regular check-ups and assessments.
How to find out more
CTC-NCT Heidelberg Trial Management and Services
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
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