What Is a Clinical Research Study?
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 companies) typically run clinical research studies. They are responsible for creating a strict study protocol (a written description of the 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 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
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 they 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.What Happens When the Study Is Completed?
Generally, at the end of a study, researchers analyze and compare the data collected from all participants. De-identified (nonpersonal) data may be shared with experts and various government agencies responsible for the approval of new drugs, biologics, and devices.What happens when the study is completed can vary among studies. After the completion of two years of assessments in this protocol, there is a long-term follow-up (LTFU) for survival, long-term toxicity, and viral vector safety that will continue under a separate protocol for up to 15 years after JCAR017 treatment. Clinical trial participants can withdraw from the study at any time without jeopardizing their standard medical care.
Following the end of the study, participants may receive the general study results directly from their study site.
A description of this clinical trial will be available on http://www.clinicaltrials.gov as required by US Law. The website will include a summary of the results from the study within approximately one year of study completion. You can search the website at any time.
More about Study Participation
Participation in the PILOT Study lasts approximately two years and includes:
• A screening period that will last up to 14 days, during which your eligibility to participate in the PILOT Study will be confirmed. If you are determined to be eligible, your T cells will be removed (through leukapheresis) and the investigational study treatment will be manufactured. This will take approximately four weeks, but timing varies from patient to patient.
• A pretreatment period that will occur up to seven days before the treatment period. During this time, the study doctor will reconfirm that you are eligible for study participation.
• A treatment period, which will last approximately 35 days.
o You will receive an infusion one time, after which you will be closely monitored for side effects for the next 30 days.
o During this time, you will receive three days of conditioning chemotherapy followed by an infusion of the investigational study treatment
• A posttreatment period, during which you will visit the study site at least eight times before your participation in the study ends. This period will last 23 months.
After completion of two years of assessments in this study, an optional long-term follow-up study for survival, long-term toxicity, and safety will continue as a separate study for up to 15 years after administration of JCAR017 investigational study treatment.
The investigational study treatment used in the PILOT Study is called JCAR017. This drug is the result of a modification to a participant’s own immune cells (T cells) with a chimeric antigen receptor (CAR). These modified T cells, (CAR T cells) may attack the cancer cells and other cells. JCAR017 is administered once as an infusion during the treatment period. As with any medicine, JCAR017 may have side effects. As an investigational study treatment, not all the side effects may be known at this time. Two of the primary side effects that you will be monitored for include cytokine release syndrome and neurotoxicity.