What is a clinical research study?
Clinical research studies, also called clinical trials, are research studies in which participants are asked to take a study drug under the supervision of a physician and other research professionals. A study drug is one that is not approved by a health authority such as the US Food and Drug Administration (FDA) or the European Medicines Agency (EMA).
Clinical research studies must be reviewed by an institutional review board (IRB) or ethics committee (EC). An IRB or EC is a group that is responsible for helping to protect the rights and welfare of study participants. In addition, every study participant is monitored with study-related medical tests and exams before, during, and sometimes even after the study.
Who conducts clinical research studies?
Governments and sponsor companies (such as pharmaceutical or biotechnology) typically run clinical research studies. They are responsible for creating a strict study protocol (a written description of a research study) and ensuring that all people involved follow it throughout the course of the study.
What should I expect?
Before you participate in a clinical research study, a detailed description of the study – as well as possible risks and benefits – will be provided in writing in an “informed consent form” and will be discussed with you. You will be asked to review and sign the informed consent form prior to participating to show that you understand the study and its possible risks and benefits.
If you qualify, you may be enrolled in the clinical research study. Once enrolled, feel free to discuss your research care with the study doctor or study team members at any time during the course of your study participation.
It is important for you to:- Attend all scheduled visits
- Adhere to the treatment and follow-up schedules
- Describe your physical state, feelings, and well-being accurately and honestly to the study doctor
- Discuss any questions regarding the study with the study doctor
As a study participant, you will have the right to contact the study’s IRB or EC representative regarding your rights as a research participant. You will also have the right to leave the study at any time for any reason.
Can I leave the study if I change my mind?
Participation in any clinical research study is completely voluntary, and participants may withdraw from a clinical research study at any time for any reason without penalty or loss of medical care or benefits to which you are entitled. If you would like to leave the study, you should discuss this with your study doctor, who will give you information about how to do this safely and make arrangements for further medical care and follow-up.
Frequently asked questions
How can I participate in this study?
You will first need to take part in a screening visit for a series of medical tests to determine whether you qualify for the study. The visit will include disease assessment, blood and urine sample testing, a physical exam, questions about your health and treatments, and a full review of your medical history. If you qualify and agree to participate, you will be entered into the study.
What will I be asked to do?
If you qualify and agree to participate, you will be asked to follow instructions of the study doctor. Your participation is expected to last up to three years. Below is a breakout of the different stages of the TRANSFORM Study.
- Pre-Treatment:
- You will undergo a series of tests to check if you qualify for the study.
- You will be randomly assigned (by chance) to receive either standard of care or JCAR017 at the start of the study.
- Treatment:
- You will be randomly (by chance) assigned to one of two treatment groups below:
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- Arm A: Standard of Care (whatever your doctor feels is the best treatment for you that has already been approved and defined in the study protocol)
- Arm B: Investigational study drug JCAR017 infusion, following intravenous (IV) lymphodepleting chemotherapy
- Post-Treatment:
- Your study doctor will continue to follow up with you. The visit schedule for follow-up will depend on which group you were assigned and if/when you may have crossed over from Arm A (standard of care) to Arm B (investigational study drug).
If you join the TRANSFORM Study, it will be important for you to keep in communication with the study team and to ask questions. Treatments for cancer and for side effects can be complicated, so be sure to talk openly about your disease, your overall health, and what treatment options may be best for you at any time. The study doctor and team want you to be informed and are available to support you throughout your participation and study treatment.
Remember, you can choose to leave the study at any time for any reason, without penalty to you.
What is the study drug?
JCAR017 is known as a Chimeric Antigen Receptor T cell, or CAR-T cell therapy. This involves taking T-cells from your body, modifying them in a laboratory, then injecting them back into your body. These modified cells may have the potential to recognize, target, and destroy the mutated B-cells.
You will be asked to undergo lymphodepleting chemotherapy before the modified cells are injected back into your body.
Is there any cost to participate?
You will receive study treatment at no cost.
What are the risks and benefits of taking part?
There are risks and benefits of taking part in any clinical research study. For example, you may benefit from the frequent health checks. You may also benefit from taking the investigational study drug, although this cannot be guaranteed. Your health may get better, it may stay the same, or it may get worse. You should discuss the risks and benefits of participation with your health care providers. These risk and benefits are also outlined in detail in the informed consent form (ICF) that you must read and sign before you can take part in the study.
Study treatment will be provided at no cost. You may be reimbursed for reasonable travel expenses as long as relevant receipts are provided.