Clinical Research Study

This clinical research study is testing the effectiveness and safety of an investigational study medication for qualified adults living with primary ITP; primary being the absence of other causes or disorders associated with thrombocytopenia.

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Study Locations

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About the ADVANCESC Study

The ADVANCESC study is designed to assess how effective and safe the investigational study medication (subcutaneous efgartigimod*) is compared to a placebo (medication with no active ingredient) as a possible treatment for adults living with primary ITP, primary being the absence of other causes or disorders associated with thrombocytopenia.

*Efgartigimod is an investigational study medication that is currently being studied in multiple disease states. Efgartigimod is an investigational study medication that is not currently approved for use by any regulatory agency as its efficacy and safety have not been established.

About Primary Immune Thrombocytopenia (ITP)

ITP is a rare autoimmune bleeding disorder where the body’s immune system attacks and destroys blood platelets causing many complications for people living with this disease. There are different forms of treatments used in the management of ITP but it is still a chronic disease. Many people develop a resistance to current treatments and some experience difficult side effects, such as severe fatigue. A new form of treatment is required to address this unmet need, which is why we are conducting the ADVANCE SC study.

Seeking to address the needs of ITP patients

What patients said about the unmet need in ITP

More treatment options for patients with difficult-to-treat ITP.

A treatment designed to target the underlying cause of ITP.

A treatment that provides a durable response with a strong safety profile and limited side effects.

A treatment that accounts for the variation in patients’ needs and response to treatment.

The opportunity to continue receiving the investigational study medication following the trial.

How the study is trying to address these unmet needs

Patients included in the trials will have been exposed to at least 2 treatments for ITP.

Efgartigimod* is thought to reduce the presence of antibodies that contribute to the low platelet count in ITP and has the potential to impact on both the destruction and production of the platelets.

The study is designed to evaluate the durability of a sustained platelet count response over a 24-week treatment period, in a 35-week trial. Additionally, the safety of subcutaneous efgartigimod and the incidence and severity of adverse events will be monitored and measured.

Patients who complete the treatment period will have the opportunity to receive subcutaneous efgartigimod in an open-label (no placebo) study if the appropriate criteria are met.

The subcutaneous efgartigimod treatment frequency will be adjusted based on the platelet count.

The study will also seek to measure other factors of ITP, such as fatigue and general health.

*Efgartigimod is an investigational study medication that is currently being studied in multiple disease states. Efgartigimod is an investigational study medication that is not currently approved for use by any regulatory agency as efficacy and safety have not been established.

All data in the above chart references the Platelet Disorder Support Association report1


  1. Published report “Voice of the Patient” from the EL PFDD meeting https://pdsa.org/voice-of-the-patient

What to expect in the ADVANCESC Study


To participate in this study, patients will be asked to read and sign an informed consent form (ICF). The form explains:
• the purpose of the study
• study visits and procedures
• the potential risks and benefits
• confidentiality
• information on how to withdraw from the study


Approximately 156 qualifying adults will take part in the ADVANCE SC study in countries around the world. Patients enrolled in the study will be randomly assigned by a computer (by chance, like the toss of a coin) to either the subcutaneous efgartigimod* group or the placebo group. Patients will have 2 chances out of 3 to receive subcutaneous efgartigimod and 1 chance out of 3 to receive placebo. The study is blinded, meaning that neither you nor the study team will know which treatment you are receiving. In both groups, you will be able to continue on your current treatments for ITP.


As safety is our top priority, all enrolled patients will be monitored and supported by the study team. Procedures to make sure patients are doing well will be required in this study, some of them are:
• health questions
• physical examinations
• blood and urine tests


During screening, patients will be evaluated to see if they meet the study criteria. The screening period can last up to 2 weeks.


Enrolled patients will be asked to come to weekly study site visits, for approximately 28 times. If all criteria are met, at the conclusion of the study you may be given the opportunity to join an open-label extension study where everyone participating will receive subcutaneous efgartigimod.

*Efgartigimod is an investigational study medication that is currently being studied in multiple disease states. Efgartigimod is an investigational study medication that is not currently approved for use by any regulatory agency as efficacy and safety have not been established.

Frequently Asked Questions

What is a clinical research study?

Before a medication or treatment can be approved for use, there must be proof that the investigational study medication works and is considered safe enough for the tested population. Clinical research studies, also called clinical trials, look for new ways to prevent, detect, and treat disease. They are designed to test the safety and effectiveness of investigational study medications and to understand if these potential treatments can be used in the future.

Will compensation be provided?

The study-related treatments and assessments will be provided at no cost. Participants will not be paid to take part in this study; however, reasonable study-related expenses, such as meals and parking, will be reimbursed. Travel arrangements may be provided with no out-of-pocket expenses for the patient.

What happens to your personal information?

There are security measures to protect personal information. This is to avoid personal information from being lost, used, altered or disclosed or accessed in any unauthorised way. To protect the identity of the participant, personal study information will be coded (e.g. unique study numbers are assigned to each patient). More information about privacy maintenance will be shared to those who choose to participate in the study.

Can participants withdraw from the clinical trial after they’ve started?

Taking part in a clinical research study is entirely voluntary. Participants may withdraw at any time for any reason. Care for you by doctors and nurses will not change.

Who is sponsoring this clinical research study?

argenx, a global biopharmaceutical company, is sponsoring this clinical research study. For more information about argenx, please visit www.argenx.com.

How can I find out more about the investigational study medication and this study?

More information about this clinical study can be found at ClinicalTrials.gov (NCT04687072) and information on the investigational study medication can be found on the argenx website.

Who oversees clinical research studies?

To help ensure that a research study is ethical and that patients’ rights are protected, Institutional Review Boards (IRBs), Ethics Committees (ECs) or Departments of Health (DoH) review and approve study protocols that detail all aspects of how the study is conducted. There are also teams that monitor the study called Clinical Research Associates; they visit the study sites to make sure the study protocol procedures, research requirements and state and federal regulations are being followed throughout the study. Health authorities may also inspect the study sites to ensure the safety and rights of the subjects participating in a trial and the integrity of the research study objectives.

What is an investigational study medication?

An investigational study medication is a product that has not been approved by a country’s regulatory authorities (e.g. the Therapeutic Goods Administration) for medical use. The product must undergo a clinical research study in order to assess its safety and effectiveness.

What is a placebo?

A placebo has no active ingredient and is used to compare the effects of the investigational study medication. The placebo will look like the investigational study medication and be administered the same way to make sure that no one, not even the study doctor, will know which medication has been assigned to whom.


Study Locations

The ADVANCESC study will be taking place in countries all over the world. The current study locations are listed below. Those highlighted in green are currently open and those highlighted in red will be open soon. If you are interested in participating, click “See if you qualify” and complete the questionnaire to find out if you might be eligible for the study.

      There will be no study-associated costs. You will not be paid to participate in this study, but you will be reimbursed for study-related travel costs upon request.

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