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