FAQ

You can always start by asking your doctor if he or she knows of any studies that might be right for you. If you are part of a patient support or advocacy group, they may have resources or know of studies that are ongoing or coming soon. ClinicalTrials.gov is an excellent resource and registry of all clinical studies ongoing in the US, and often contains a study contact for more information. If you are interested in any clinical trials at Ionis, you may reach out to [email protected] at any time. 

No. Typically, all study-related procedures and investigational drug costs are covered by the study sponsor and provided at no cost to the study participant. Additional cost incurred as a result of participation, such as travel, are often reimbursed.   

In some clinical studies, neither the participant nor the researchers know to which group the participant has been assigned. This is called ‘double-blinding’ and it is designed to eliminate potential bias on the part of the researchers. In other clinical studies, only the participant does not know to which group they have been assigned. These are called ‘blinded’ trials.  

The process that determines what type of intervention the participant will receive. For example, if the trial is designed with a placebo control arm, randomization may be used to determine whether a participant will be assigned to the group receiving the investigational drug or the group receiving a placebo. This process is done by chance, like flipping a coin. 

A placebo is an inactive version of whatever drug is being tested, be it a pill, a liquid, an injectable, or a powder. Placebos are incorporated into the design of some clinical studies so that the patients receiving the placebo can be compared to those receiving the active drug. This can speed up a study by allowing the effectiveness of the drug to become evident and better understood in a shorter period of time than without incorporating a placebo. 

Phase I: This is a clinical study done in a small number of people. Participants can be people who have a certain disease or illness, or they can be healthy volunteers. The number one goal of a phase I study is to find out how safe a new drug is. These studies establish a safe dosage and sometime even a dosing schedule or route of administration. Phase 1 studies may be the first time a certain drug has been given to humans. 

Phase II: These clinical studies are done in a larger number of people than phase I studies. The number one goal of these studies is to see if a drug is effective and to closely monitor the safety of the participants and the side effects they experience. 

Phase III: These are the largest of the clinical studies, and often health care sites all over the world will participate. The number one goal of these trials is to compare the new treatment to the existing treatment(s), which is sometimes no treatment at all. 

Phase IV: These studies take place after a treatment has been approved by the U.S. Food and Drug Administration and are used to study long-term safety and effectiveness of the new treatment. The number of participants varies depending on the disease and how many people are typically affected. 

Participating in a clinical study is an important decision, and you should take as much time as you need to make it. Talk to your doctor and/or your family members about it. Bring a family member or close friend to help you ask questions or remember key information. Take notes. While you should learn as much as you can ahead of time, here are some examples of the types of questions you should ask before you volunteer:

• Why is this study being done? 
• How long will I be in this study? 
• What tests or procedures will I need to have done if I participate?  
• How many visits will be required and how long are the visits? 
• Will I need someone to come with me to my visits?
• What are the possible risks and side effects, and what are the potential benefits? How do these risks and benefits compare to those I would experience if I didn’t participate in this study?
• Is there a chance of getting a placebo? If so, what are my chances?  

There are always risks involved in participating in clinical studies. The side effects of new drugs or devices are not fully understood, which is part of the reason the research is being done. However, clinical study protocols are carefully controlled and designed to monitor the safety of all the participants. In addition, investigational drugs and devices go through extensive pre-clinical testing before they even make it into a clinical study. All clinical study protocols must be reviewed by an Institutional Review Board, or IRB, which may be comprised of doctors, nurses, and/or medical ethics experts, whose job it is to review the study and ensure the potential benefits outweigh the risks.

Participating in a clinical study allows researchers to gather valuable information about potential new therapies for medical conditions. In some cases, the medical conditions are extremely rare and have limited or no available treatments. Participating in a clinical study may enable a patient to take a more active role in their own healthcare or a chance to gain access to new investigational drugs or devices before they are available, and the information gathered from these studies may help others with the same disease or condition in the future. 

All clinical studies have different eligibility criteria that will determine whether or not a person can participate. These criteria typically include things such as age, gender, the type and/or severity of a disease, previous treatment history, and other medical conditions or history. Eligibility criteria are designed to ensure the researchers are testing a new investigational drug or device in a population that will give them the best information about how safe and effective the study treatment is. These criteria are also designed to reduce unnecessary risk. 

Yes, clinical studies can fall into one of several categories. Here are some examples. 

• Treatment studies: These test investigational new drugs, new combinations of drugs, new medical devices, or new approaches to interventions, like surgery. 
• Prevention studies: These look for better ways to prevent disease in people who have never had the disease or to prevent a disease from returning. These studies may test a vaccine, a medication, or even a lifestyle change.
• Natural History Studies: These studies collect information about a group of people who have, or are at risk of developing, a certain medical condition or disease.
• Quality of Life Studies: These explore ways to improve comfort and the quality of life for people with chronic illnesses. 

Clinical studies are research studies conducted to learn about the effects of investigational new drugs. The studies follow a pre-defined protocol, and they are the most important way for researchers to find out if a new treatment, like a drug or a medical device, is safe and effective for people.   

For more information on the Ionis expanded access policy, see here.