Purpose of this Study
If you join this study, you will be randomly placed into one of two groups. One group will get the study medicine called efgartigimod, and the other group will get a placebo, which is a fake medicine. Both are given through a tube in your vein, called an IV.
You will get the medicine or placebo for the first 4 weeks. After that, depending on your platelet count, you will keep getting it every week or every other week for 20 more weeks. After 24 weeks, you may be able to join the next part of the study, where everyone gets the real medicine for up to 52 weeks. If the medicine does not work well enough after 12 weeks, you might be able to switch to the real medicine earlier.
Who Can Participate?
Eligibility
To join this study, people must:
- Have been diagnosed with a blood condition called primary ITP more than a year ago.
- Have tried medicine for ITP before, but it did not work well.
- Have a platelet count higher than 30 billion per liter of blood.
- Not have had cancer in the last 3 years.
- Not have had a blood clot in the last 12 months.
Age Range
18-110
Sex/Genders
Male (cisgender)
Female (cisgender)
Non-binary or gender fluid
Transgender male
Transgender female
Looking for Healthy Participants
No
What is Involved?
Description
This study is testing a new medicine called efgartigimod to see if it is safe and works well for people with a blood condition called primary immune thrombocytopenia, or ITP. These are people who tried other treatments but did not get better.
In the study, people will be randomly placed into one of two groups. One group will get the real medicine, and the other group will get a placebo, which is a fake medicine. Both are given through a tube in the arm called an IV.
For the first 4 weeks, everyone gets either the medicine or the placebo. After that, depending on their blood test results, they will keep getting it every week or every other week for 20 more weeks. After 24 weeks, people can join the next part of the study where everyone gets the real medicine for up to 52 weeks. If someone does not get better after 12 weeks, they may be able to switch to the real medicine sooner.
Locations
Duke University Hospital
Visit Timing
Weekdays
Compensation
Yes
Spanish Materials Available
No
Study Details
Full Title
A Phase 3, Multicenter, Randomized, Double-Blinded, Placebo-
Controlled, Parallel-Arm Study Followed by an Open-Label Arm to
Evaluate the Efficacy and Safety of Efgartigimod IV in Adult
Participants With Primary Immune Thrombocytopenia
Controlled, Parallel-Arm Study Followed by an Open-Label Arm to
Evaluate the Efficacy and Safety of Efgartigimod IV in Adult
Participants With Primary Immune Thrombocytopenia
Study Website
Principal Investigator
Ara
Metjian
Protocol Number
PRO00117299
NCT ID
NCT06544499
Phase
III
Enrollment Status
Open to Enrollment