Participating in Clinical Trials

Clinical trials are research that determine the safety and effectiveness of new ROS1+ cancer treatments in humans.

 

Cancer clinical trials determine the safety and effectiveness of cancer treatments in humans under the supervision of trained researchers and medical professionals. Clinical trials are an alternative to standard of care treatment for patients who have ROS1+ cancer. For patients in countries where ROS1+ cancer does not have an approved targeted therapy, clinical trials may be the best option for accessing a ROS1 TKI. 

Cancer clinical trials might study:

  • A new treatment on its own

  • A new treatment added to a standard treatment

  • A new treatment on its own (or added to standard treatment) compared with standard treatment alone -- a randomized study

  • Some studies are observational rather than treatment trials – for instance, assessing accuracy of a new cancer detection method or biomarker test.

Some key clinical trial terms that are helpful to know:

  • Open-Label: participants know which intervention they are receiving

  • Randomization: participants are assigned by chance to separate groups that are given different interventions

  • Blinded: you (and probably your doctor) don’t know what treatment you’re receiving

  • Basket trial: the same treatment options are given to people with different cancers

What Are Clinical Trial Phases?

Clinical trials are conducted in phases. A Phase 1 trial tests a new treatment in humans for the first time; it enrolls a small group of people and focuses on evaluating safety and appropriate dosage.  If Phase 1 shows the treatment is safe and has some effectiveness, a Phase 2 trial is conducted in a somewhat larger group of people and focuses on establishing effectiveness and side effects associated with the treatment.  A Phase 3 trial compares the effectiveness of different treatments in a much larger group of people. 

 
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If the Phase 2 or Phase 3 results look good, the trial sponsor can submit the results to a country's regulatory agency for approval (in the USA, this is the US Food and Drug Administration, or FDA).  Once approval of the treatment is granted, then the treatment can be marketed to doctors, prescribed for patients, and (depending on the country) paid for by the healthcare system or insurance.  A treatment must obtain a specific country's regulatory approval separately before the treatment can be made available in that country.  In the USA, the FDA has approved TKIs for ROS1+ cancer based on Phase 2 trial data (rather than Phase 3 trials) because the number of patients with ROS1+ cancer is likely too small to support a full Phase 3 trial.

Sometimes patients may be able to access an experimental drug that has strong evidence of effectiveness from their own physician through Compassionate Use offered by the drug's manufacturer.  This may be an Expanded Access Program (EAP) or some other means (e.g., Right to Try Laws in the USA).

Should I Join a Clinical Trial?

Participating in a clinical trial has potential advantages:

  • Your data may help determine the best available ROS1 treatment.

  • The treatment you receive might be more effective and/or less toxic than other options.

  • You’ll form a close relationship with an expert team that provides a higher level of care than a typical clinic.

 

Some people who joined early phase trials for ROS1 targeted therapies gained many months or years with good quality of life before their cancer progressed. However, clinical trials do not hold any guarantees of being able to participate or obtain successful treatment. Be aware:

  • You will be taking a risk because the treatment might not work for you.

  • You might not receive the experimental treatment if you are in a randomized phase 3 trial (but you WILL receive the best standard of care).

When assessing your clinical options, differences between trial phases become important.  Phase 1 trials involve more risk than Phase 2 trials because the safety of the drug has not been established.  Phase 2 trials involve more risk than Phase 3 trials because the side effects and efficacy of the drug have not been established in a large group of people.  However, some patients have found a Phase 1 trial for a targeted therapy may still be more tolerable and/or effective treatment than the standard of care (such as long-term chemotherapy), especially if the study drug has already completed Phase 1 or Phase 2 trials in another country.

Before joining a trial, ask questions to be sure you understand what the trial involves and how much risk you are willing to tolerate.

  • Is my general health good enough for a trial?

  • Do I meet the eligibility criteria--for instance, is my type of ROS1+ cancer eligible?  

  • Is the trial available near me? Can I handle the required clinic visit schedule and potential travel? 

  • What procedures are required?  Will these expenses be covered by the trial sponsor?

  • What expenses must I pay for?  Will my health insurance cover any of those expenses?

  • Does the trial require a fresh tissue biopsy?  If so, am I able to provide it?

  • What do we know about this treatment?  Is there any preclinical data available?

  • Might participating in this trial disqualify me from participating in a different trial later on?  (Some trials limit the number or type of treatments you can receive before enrolling).

  • Is a trial the best treatment option for my particular cancer?

Choosing to participate in a study is an important personal decision. Talk with your doctor, family members, or friends who might help support your participation and cancer patients who have participated in trials before deciding to join.  If your doctor is not familiar with ROS1+ cancer treatments and clinical trials, it might be wise for your doctor (or you) to consult a ROS1 Clinician-Researcher. You can also talk to other ROS1ders in our private Facebook group--many of us have participated in clinical trials. The ROS1ders community is a good place to learn about the most promising ROS1+ cancer clinical trials because the pharmaceutical firms keep us informed about new TKI trials. Some clinical trials might assist with travel planning and expense—if this interests you, inquire at the trial site where you would like to participate or contact The ROS1ders.

 

You can find clinical trials for ROS1+ cancer anywhere in the world by searching the clinicaltrials.gov website (or clinicaltrialsregister.eu for trials in Europe).  All clinical trials conducted in the USA are required to be listed on this site, and most trials conducted by major drug companies in other countries are also listed.  However, not every trial in every country is listed.  Various clinical trial finder apps exist to make searching this database easier, but they are all searching the same database.

You can learn more about clinical trials on websites of the US National Institutes of Health, US National Cancer Institute, regulatory agencies, academic cancer centers, research organizations, and cancer nonprofits.

 

Medically reviewed by ROS1 Clinician-Researchers
Last updated 19-Feb-2022