About the ADVANCESC
Clinical Research Study

This clinical research study is testing the effectiveness and safety of an investigational study drug 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

Find a participating site location near you and take a questionnaire to determine your eligibility.

Find out if you qualify for this study.

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About the ADVANCESC Study

The ADVANCESC study is designed to assess how effective and safe the investigational study drug (subcutaneous efgartigimod*) is compared to a placebo (drug 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 drug that is currently being studied in multiple disease states. Efgartigimod is an investigational study drug that is not currently approved for use by any regulatory agency as 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 therapies 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 therapy 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 variation in patients’ needs and response to treatment.

The opportunity to continue to receive the investigational study drug following the trial.

How the study tries 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 both 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 the 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.

Subcutaneous efgartigimod treatment frequency will be adjusted based on platelet count.

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

*Efgartigimod is an investigational study drug that is currently being studied in multiple disease states. Efgartigimod is an investigational study drug 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

INFORMED CONSENT

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
• Potential risks and benefits
• Confidentiality
• Information on how to withdraw from the study

RANDOMIZATION

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 (as by chance, like the flip 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 your current treatments for ITP.

VISIT PROCEDURES

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

SCREENING

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

STUDY LENGTH

Enrolled patients will be asked to go to weekly study site visits, for approximately 28 times. If all criteria are met, after the 24-week treatment period, 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 drug that is currently being studied in multiple disease states. Efgartigimod is an investigational study drug 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 drug or treatment can be approved for use, there must be proof that the investigational study drug 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 drugs to understand if these potential treatments can be used in the future.

Will compensation be provided?

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

What happens to 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 unauthorized 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 can withdraw at any time for any reason. Care 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 learn more about the investigational study drug and this study?

More information on this clinical study can be found at ClinicalTrials.gov (NCT04687072) and information on the investigation study drug 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 (IRB), Ethics Committees (EC) or Ministry of Health (MOH) 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 country 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 drug?

An investigational study drug is a product that has not been approved by a country’s regulatory authorities (e.g., the U.S. Food and Drug Administration) for medical use. The product must go through a clinical research study in order to assess the safety and effectiveness.

What is a placebo?

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

WHERE WE ARE

Study Locations

The ADVANCESC study will be taking place in countries all over the world. 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 “Find out if you qualify” and complete the questionnaire to find out if you may 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 upon request.

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