Lupus and APS Clinical Research at HSS

What is a clinical research study?

A clinical research study is a highly regulated study of human volunteers, or participants, that is intended to help learn about a particular medical condition or intervention.

What are the different types of clinical research studies?

There are two main types of clinical research studies: observational studies and interventional studies (also known as clinical trials).

In observational studies, investigators do not influence the care participants receive. Investigators simply monitor participants alongside their doctors. They may collect blood samples and information about how the patient responds to treatments.

In interventional studies, investigators give participants an investigational drug or other treatment. Sometimes it is the current treatment or standard of care for the disease. The purpose of interventional studies is to determine if these treatments are safe and effective for treating a condition.

One specific type of interventional study is called a clinical trial. Clinical trials study specific treatments, such as medications, devices, or behavioral changes, to determine if they are safe and effective for treating a condition. There are four “phases” of clinical trials:

  • Phase I – studies the use of a drug in small groups of humans (20-100) for the first time, specifically looking at safety, tolerability, and factors related to dosing.
  • Phase II – studies both safety and efficacy in larger groups of people (20-300).
  • Phase III – studies both safety and efficacy in even larger groups of people (300-3000). Generally two phase III studies are required for the Food and Drug Administration (FDA) to approve a medication for use in a certain condition.
  • Phase IV – monitors effectiveness and adverse effects in the general population after a drug has received FDA approval.

Why is clinical research important?

Clinical research adds valuable information to the medical community. Trials of a new medicine, a device, or kind of treatment can help us to understand if the intervention may benefit patients with a particular condition. For example, the clinical trials of belimumab (Benlysta®) led to Food and Drug Administration (FDA) approval of this medication for the treatment of lupus in 2011. It was the first drug approved for the treatment of lupus by the FDA in over 50 years.

Sometimes drugs that are used in one condition are studied for potential use in a different condition. For example, hydroxychloroquine (Plaquenil®), is a medication that has long been used for the treatment of lupus. It is currently being studied in people with antiphospholipid antibodies to determine if it may play a role in preventing blood clots in these patients.

The goal of all clinical research studies is to learn more about the treatments being studied to benefit the larger population in the future.

Am I eligible for a clinical research study?

Protocols, which are rules for the study, define which patients are eligible for participation in a clinical research study. Some factors that determine whether a patient is a candidate for a particular study include the status of the disease being studied (active versus inactive), age, and current medications. Your doctor can help determine if you are eligible for a study.

What do I have to do as a participant in a clinical research study?

Each clinical research study has a rule book or protocol for investigators and participants to follow. Protocols for interventional studies determine the intervention a participant will receive (such as the investigational medication, behavioral change, standard of care, or placebo (inactive medication)), the schedule of study visits, phone encounters, and blood draws or other tests that might be required in the study. The duration of the study varies and may range from a few weeks to several years. Investigators will carefully discuss what is required to participate in a study when they invite a patient to enroll.

What are the risks and benefits of participating in a study?

The risks and benefits of participating in a clinical research study vary with the type of study being conducted. For observational studies, participants may benefit from the additional care and attention they receive from the research team. For interventional studies, participants may gain access to experimental and novel therapies. Interventional studies involving experimental/investigational procedures may not give you immediate benefit or any benefit. The knowledge gained may benefit others in the future.

For all studies, risks include side effects of investigational treatments, extra blood draws or additional radiation exposure, and time commitment to participate in the study. It is important to remember that all clinical research studies are completely voluntary, and once enrolled, participants have the right to withdraw from a study at any time without any change in clinical care at Hospital for Special Surgery.

How am I protected if I participate in a clinical research study?

The protocols for all clinical research studies are approved by an institution’s Institutional Review Board (IRB) or Ethics Committee (EC). The IRB consists of a panel of physicians, researchers, and members of the general public that review clinical research study proposals and ensure that the rights and welfare of all participants are protected. Additionally, the IRB ensures that participants’ privacy is protected under the Health Insurance Portability and Accountability Act (HIPAA) which protects the privacy of health information that one would expect to remain confidential.

As part of the review, the IRB ensures that patients are given accurate and detailed information about study details before agreeing to participate. This is documented in a form and via dialogue between a study team member and an eligible participant, and is referred to as informed consent.

Finally, certain types of studies, such as interventional clinical trials, have an additional independent group called a Data Safety Monitoring Board (DSMB). The DSMB is composed of experts who meet regularly to review patient safety data during the course of a clinical trial. Additionally, a DSMB may monitor treatment efficacy to determine is a clinical trial should continue or be halted. These boards help to protect the safety of participants in clinical research studies.

How can I learn more about clinical research at Hospital for Special Surgery?

Hospital for Special Surgery has many active clinical research studies for patients with lupus and/or antiphospholipid antibodies. Additional information about ongoing clinical research studies across the country and around the world is available at


Alana B. Levine, MD
Assistant Attending Physician, Hospital for Special Surgery
Clinical Associate, Barbara Volcker Center for Women and Rheumatic Diseases

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