CLL clinical trials evaluate the safety and effectiveness of potential new treatments for CLL.
Health authorities, such as the Food and Drug Administration (FDA), use the data from clinical trials to determine whether these new CLL treatments should be available outside of clinical trials.
Phase 1 focuses on how safe a new treatment is in the human body, including how to dose the treatment.
Phase 2 focuses on how safe a new treatment is against a certain type of cancer, such as CLL.
Phase 1 and 2 may be combined (Phase 1 / 2).
Phase 3 focuses on how effective and safe a new treatment for a certain type of cancer is, like CLL, compared to an existing treatment.
Phase 4 continues to study how effective and safe a treatment is over the long term, after it has been approved by FDA.
Clinical trials follow a research plan, called a protocol, that determines who is eligible to participate in the trial, which treatments are being studied, how participants will be cared for during the trial, and what research questions the trial seeks to answer.
Normally, clinical trials for cancer treatments, like those for CLL, are planned in phases:
Participation in a clinical trial should start with a conversation with your doctor about the potential risks and benefits. Your doctor will be able to help you determine if you may be a good candidate for a certain clinical trial. Some of the considerations may include your age, health status, and treatment history.
Often, the doctors and care team who run a clinical trial will be different from your everyday CLL doctor, but they will stay in communication about your care.
If you are eligible for a clinical trial and decide to participate, you will need to sign a document called “informed consent” that acknowledges that you understand what to expect in the clinical trial, including the risks and benefits.
It is very rare for cancer trials to use a placebo (an inactive version of a treatment). If you have questions about whether a clinical trial involves a placebo, it is best to ask your doctor.
Instead, cancer trials will often test a new treatment option against a “comparator,” which is the treatment that is used as a standard.
If you are considering participation in a clinical trial, be sure to discuss with your doctor what the comparator is to ensure that you are comfortable with receiving that treatment as an option.
You do not have to pay to enroll in a clinical trial or to receive clinical trial treatments. Discuss with your clinical trial team whether there is financial assistance for non-treatment-related costs, such as transportation or lodging.
Clinical trials use specific terminology to explain how the trial will work. Some key terms to know include:
This means that patients are placed into different treatment groups by chance, like flipping a coin. This is done to make sure the groups are similar and that the results are fair.
This means that researchers observe and measure outcomes in participants who are receiving standard treatment without intervention. Researchers or doctors could be providing the standard treatment, which is being observed.
Refers to a clinical trial in which researchers actively provide a treatment, or intervention, to the participants.
This refers to the most used or accepted treatment(s) for a specific disease. The standard of care is usually what potential new treatments are studied against in a clinical trial.
This is a group of patients in a clinical trial that does not receive the new treatment. The control group may receive either a placebo, the standard of care, or no treatment depending on the study design. The control group serves as the baseline against which the effects of the new treatment are compared.
In a blinded study, either the patients, the doctors, or both do not know who is receiving the new treatment and who is receiving the control. This helps prevent bias, as knowing what kind of treatment someone is getting could influence their experience or reporting.
In an open-label study, everyone knows what treatment the patients are receiving (both the patients and the doctors).
This is commonly reflected in:
Progression-Free Survival (PFS), which measures the time during and after treatment that a patient lives without the disease getting worse (progressing)
and
Overall Survival (OS), which measures the time from the start of treatment until a patient dies from any cause
Both PFS and OS data can be reported at many time points, so it is critical to understand which treatments have and have not reported favorable long-term results (typically measured at 5+ years after starting treatment).
In some cases, short-term survival data does not correlate with long term survival data.
A clinical trial endpoint is a specific outcome used to evaluate the effectiveness of a treatment.
In CLL clinical trials, endpoints usually measure how well the CLL patient is responding to treatment.
Because CLL is a chronic (slow-growing) disease, it is important to understand the long-term effects of treatments on patient survival and safety.
Additional Endpoints that Measure How Well the Treatment Fights Cancer (Efficacy Endpoints)
Reflects the percentage of patients whose cancer shrinks or disappears after treatment. It combines different responses such as complete response (CR) and partial response (PR).
Measures how long a patient maintains a response (CR or PR) after the treatment starts before the cancer starts to grow again. It indicates how long the benefits of the treatment last.
Measures the time from the start of treatment until the cancer starts to worsen or progress.
Endpoints that Measure How Well Patients Tolerate the Treatment (Safety Endpoints)
Any undesirable experiences or medical conditions that occur during the trial. These can be mild to severe and can provide insight into the safety of the treatment.
A subset of adverse events that are severe enough to result in significant health issues, hospitalization, or death.
The percentage of patients who must stop the treatment due to safety issues or side effects.
The percentage of patients who must reduce treatment due to safety issues or side effects.
