Understanding primary immunodeficiency (PI)
Understanding PI
The more you understand about primary immunodeficiency (PI), the better you can live with the disease or support others in your life with PI. Learn more about PI, including the various diagnoses and treatment options.
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Living with PI
Living with PI
Living with primary immunodeficiency (PI) can be challenging, but you’re not alone—many people with PI lead full and active lives. With the right support and resources, you can, too.
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Get involved
Get involved
Be a hero for those with PI. Change lives by promoting primary immunodeficiency (PI) awareness and taking action in your community through advocacy, donating, volunteering, or fundraising.
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Advancing research and clinical care
Advancing research and clinical care
Whether you’re a clinician, researcher, or an individual with primary immunodeficiency (PI), IDF has resources to help you advance the field. Get details on surveys, grants, and clinical trials.
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Participating in clinical trials

Clinical trials help researchers develop safe, effective treatments and diagnostics for primary immunodeficiency (PI), as well as understand how these disorders develop and progress. And you can be a part of this process.

Clinical trial participation through IDF

The Immune Deficiency Foundation (IDF) routinely collaborates with industry and marketing groups to aid in the recruitment of clinical trial participants for new PI medications, devices, vaccines, blood products, or gene therapies. IDF may reach out to members of the PI community for clinical trial participation. Before we do, IDF ensures the research is legitimate by validating that it:

Protects the personal health and personal contact information of our community.
Does not allow for the sale or resale of your data or personal information.
Is supervised and monitored by an Institutional Review Board (IRB) or Data Safety Monitoring Board (DSMB).

Find a clinical trial

Healthcare providers, patients, family members, and the public can access the National Institutes of Health's free database of available clinical trials around the world that are studying a wide range of diseases and conditions.

Browse trials

Types of clinical research

Clinical research can be performed in several ways, depending on what the researchers are studying. The four main types are:

Treatment

Clinical trials or protocols for treatment studies involve a new clinical intervention such as medication, psychotherapy, medical device, or approaches to surgery or radiation therapy.

Example: new type of intravenous immunoglobulin (IVIG)

Prevention

Prevention research looks for better ways to prevent diseases or illnesses from developing or returning. Different kinds of prevention research may study medicines, vitamins, vaccines, or lifestyle changes.

Example: low salt diet to prevent high blood pressure

Diagnostic or screening

Diagnostic research looks at better ways of identifying or diagnosing a particular disorder or condition. Screening studies are also diagnostic research to learn better ways of detecting disorders.

Example: newborn screening for severe combined immune deficiency (SCID)

Natural history

Natural history studies track the development of a disorder (often present since birth) over the lifetime of an individual to see the possible complications or changes in the disorder. The data collected over time can also lead to improved care management guidelines.

Example: natural history study of common variable immune deficiency (CVID)

Clinical trial stages for treatment studies

Access to new treatments depends on clinical trial research to provide the Food and Drug Administration (FDA) with enough safety and efficacy information to decide whether to approve or reject these products. To maximize patient safety, new treatments must go through several stages, or phases, of trials.

Phase I trials

These trials test a drug or a treatment in a small group of people. This stage is usually the first time it will be tested in humans. The purpose is to determine a safe dose of the medication and if there are any side effects.

Phase II trials

Next, the drug or treatment is given to a larger group of people to test how well it works and determine what will be the most effective dose.

Phase III trials

The study drug or treatment is then given to even larger groups of patients to prove effectiveness and monitor for side effects. The study often compares the new treatment to the currently used treatment to determine safety.

Phase IV trials

These trials are for study drugs or treatments that have been approved by the FDA. This is to get more information on the usefulness and effectiveness of the drug or treatment and monitor any long-term side effects.

Pros and cons of participating in a clinical trial

Deciding to take part in clinical trials is something only you can decide with information from the study doctor (investigator) and team. Consider all possible benefits and drawbacks before making your decision.

New treatment or diagnostic options and advancing the field

Participation in a clinical trial can have immense benefits. It provides a treatment or diagnostic option that is not otherwise available and access to care from expert physicians in the field. Care and medication for the study are often free for participants.

As a research volunteer, you also have a unique chance to help others in the PI community by supporting the advancement of science.

Unknown side effects and risks

There is always a chance the treatment or diagnostic will not work. Even if some side effects are known, you may experience other side effects that are not yet known. Depending on your response to the treatment/diagnostic and possible side effects, you may need more frequent monitoring than was initially planned for in the trial.

Benefits and drawbacks of randomization and placebos

Randomization is a process used in some trials to prevent bias in who gets which treatment. One group receives the actual study treatment, while the other group receives the currently accepted treatment (standard approved treatment). Comparing the results from these two groups often shows which treatment is more effective or has fewer side effects. Your chance of being in either group is about equal. You or the study doctor do not choose which treatment you get—you are randomly assigned to a group by a study statistician or pharmacist.

Placebos are inactive “look-alike” drugs that do not include the treatment being studied. Placebos are rarely used in primary immunodeficiency drug studies. But if they are being used in your study, there is a possibility you will not receive the active drug and will not know that until later in the trial.

Your rights as a research volunteer

As a participant in any kind of research study, you have rights. You are a volunteer and can decide to participate or not based on information about the clinical trial. Here are several things to consider before you make a decision.

Informed consent

Before you decide to participate, the investigator will explain what the expectations are for you as a participant and fully explain the risks and benefits. You will have a chance to ask questions. Once you are satisfied with the information and decide you want to participate, you will be asked to sign an informed consent form. This is a legal document that says you give your permission to be involved in the research and understand the risk and benefits.

Children age 7 and older are often asked to sign an assent form, which is similar to informed consent, but written in age-appropriate language. Your child will not be forced to participate.

You may ask for the consent form to be translated into your language or ask for an interpreter to help with understanding the consent.

You will be given a copy of the signed consent form. If you have any further questions, you have options to contact your physician or nurse, the patient advocate of your institution, and the IRB for answers.

Withdrawing from a trial

Once you or your child has decided to participate in a study, you are always considered a volunteer and can withdraw at any time. You can withdraw from any research protocol without any consequences to you or your child’s care. You can continue to ask questions and be assured your privacy is maintained. Any publications that result from the study will protect your or your child’s identity and medical information.

Questions to ask before joining a clinical study

Before signing a consent form, ask questions that can help you determine if the study makes sense for you. Recommended questions include:

Why should I be in this research study?
Why is this trial being done?
How will it affect me or my child?
How long will it take?
How will we know if it’s working?
Do I have to pay for anything?
What costs will my health insurance cover?
How often will I have to come to the clinic or hospital?
What are my other choices, including standard treatment?
How can I enroll?

Organizations that connect people to trials

ResearchMatch

ResearchMatch is a nonprofit program from Vanderbilt University Medical Center funded by the National Institutes of Health (NIH). It helps to connect people interested in research studies with researchers from top medical centers across the U.S.

ResearchMatch.org

Discover more research opportunities

Patient registry

USIDNET is a national registry of PI patients who volunteered their personal information to assist and support research work.

View registry

Research grant program

IDF offers a research grant program to support patient-oriented PI research, funded by dollars raised from IDF Walk for PI teams and supporters.

Get funding

Survey research

The IDF Survey Research Center provides current information on PI through the analysis of patient and caregiver survey responses.

Browse reports

Latest resources on research

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