Clinical Trial for the treatment of ITP

The ADVANCE study is designed to assess how effective and safe the investigational study drug (efgartigimod) is compared to a placebo* as a possible treatment for adults living with primary ITP, primary being the absence of other causes or disorders associated with thrombocytopenia.  


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

Who is Eligible to Participate?

To join, you or someone you know must:

  • Be at least 18 years old.
  • Not be pregnant, not be lactating, and not be actively trying to get pregnant. 
  • Have been diagnosed with primary ITP for more than three (3) months.
  • Meet other criteria which the study staff will discuss.

What to expect with the ADVANCE study

INFORMED CONSENT
To participate in this study, patients will be asked to read and sign an Informed Consent form. 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

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

RANDOMIZATION
There will be approximately 156 adults taking part in the ADVANCE 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 efgartigimod group or the placebo group. Patients will have 2 chances out of 3 to receive 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.

STUDY LENGTH
Enrolled patients will be asked to come weekly to approximately 29 visits for about 31 weeks. If all criteria are met, you may be given the opportunity to join an open label extension study where everyone participating will receive efgartigimod for an additional year.

VISIT PROCEDURES
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

STUDY-RELATED EXPENSES
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 to expect with the ADVANCE study

INFORMED CONSENT
To participate in this study, patients will be asked to read and sign an Informed Consent form. The form explains:

  • The purpose of the study
  • Study visits and procedures
  • Potential risks and benefits
  • Confidentiality
  • Information on withdrawal from the study

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

RANDOMIZATION
There will be approximately 156 adults taking part in the ADVANCE 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 efgartigimod group or the placebo group. Patients will have 2 chances out of 3 to receive 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.

STUDY LENGTH
Enrolled patients will be asked to come weekly to approximately 29 visits for about 31 weeks. If all criteria are met, you may be given the opportunity to join an open label extension study where everyone participating will receive efgartigimod for an additional year.

VISIT PROCEDURES
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

STUDY-RELATED EXPENSES
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.

Seeking to address the needs of ITP patients

The ADVANCE clinical study was designed with input from patients and expert clinicians.

What we heard
There is still an unmet need for…		   How the study addresses these unmet needs
Treatment options for patients with difficult to treat ITP Patients included in the trials will have been exposed to at least 2 treatments for ITP
A therapy designed to target the underlying cause of 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
A treatment that provides a durable response with a strong safety profile and limited side effects. The study is designed to show the durability of a sustained platelet count response over the 24-week trial period. Additionally, the safety of efgartigimod and the incidence and severity of adverse events will be monitored and measured.
A treatment that accounts for variation in patients’ needs and response to treatment. Efgartigimod treatment frequency will be adjusted based on platelet count.
The opportunity to continue to receive active drug following the trial. Patients who complete the 24-week trial period will have the opportunity to receive efgartigimod in a 1-year open label extension study if the appropriate criteria are met.
Assistance with travel to clinical trial visits Travel and logistical assistance, including transportation, lodging and meals associated with travel to and from the study site, may be provided with approval

*Efgartigimod is an investigational product candidate, without approval by any regulatory body, and it is being studied in clinical trials as a therapeutic for ITP, among other indications.

 

Seeking to address the needs of ITP patients

The ADVANCE clinical study was designed with input from patients and expert clinicians.

What we heard
There is still an unmet need for…		   How the study addresses these unmet needs
Treatment options for patients with difficult to treat ITP Patients included in the trials will have been exposed to at least 2 treatments for ITP
A therapy designed to target the underlying cause of 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
A treatment that provides a durable response with a strong safety profile and limited side effects. The study is designed to show the durability of a sustained platelet count response over the 24-week trial period. Additionally, the safety of efgartigimod and the incidence and severity of adverse events will be monitored and measured.
A treatment that accounts for variation in patients’ needs and response to treatment. Efgartigimod treatment frequency will be adjusted based on platelet count.
The opportunity to continue to receive active drug following the trial. Patients who complete the 24-week trial period will have the opportunity to receive efgartigimod in a 1-year open label extension study if the appropriate criteria are met.
Assistance with travel to clinical trial visits Travel and logistical assistance, including transportation, lodging and meals associated with travel to and from the study site, may be provided with approval

*Efgartigimod is an investigational product candidate, without approval by any regulatory body, and it is being studied in clinical trials as a therapeutic for ITP, among other indications.