A Study of Efgartigimod IV in Participants From 12 Years to Less Than 18 Years of Age With Chronic Immune Thrombocytopenia (ITP)
Key facts
- Who runs it
- argenx
- Where
- Belgrade and Kragujevac, SerbiaSee the 2 sitesAlso in 10 other countries, 38 sites
- Trial code
- Status
- Recruiting
- Phase
- Phase 2/3
- People taking part
- 24 people, planned
- Started
- 20 Oct 2025
- Expected to end
- Oct 2030
- Type of study
- Interventional, 2 groupsOne group gets a placebo (a dummy treatment), for comparison.
- Treatments tested
- Efgartigimod IV
- Placebo IV
- Record updated
- 16 October 2026
Who can take part
- Age
- 12 years to 17 years
- Sex
- Women and men
- Healthy volunteers
- Cannot take part
The registry criteria, word for word
Show all 10 criteriaShow less
Who can take part
- Is aged 12 to less than 18 years when completing the informed consent process
- Has a documented duration of primary ITP of more than 12 months on the date the informed consent process is complete
- Has documented prior ITP treatment with at least 1 of the following treatments: corticosteroids, IVIg, anti-D immunoglobulin, thrombopoietin receptor agonist (TPO-RAs), or rituximab
- Has documented prior response, defined as 1 platelet count of ≥50 × 10\^9/L to at least 1 of the following ITP treatments: prednisone, other or nonspecified corticosteroids, IVIg, or anti-D immunoglobulin
- Has documented insufficient response to a prior ITP treatment with corticosteroids, IVIg, anti-D immunoglobulin, TPO-RAs, rituximab, or splenectomy
- Has documented mean platelet count of less than 30 x10\^9/L
Who cannot take part
- ITP-associated critical or severe bleeding
- History of hereditary thrombocytopenia
- Secondary ITP according to the following definition by the International Working Group (IWG): all forms of immune-mediated thrombocytopenia except primary ITP
- Nonimmune thrombocytopenia
Check if you might fit
Answer for yourself. This is not a decision: only the study team can say whether you can take part.
- Are you between 12 and 17 years old?
Where in Serbia
Also in 10 other countries, 38 sites
- Institute Of Mother And Child Healthcare Of Serbia Dr Vukan CupicBelgrade, SerbiaContact: Dragana Aleksic, MDTaking people
- University Clinical Centre of KragujevacKragujevac, SerbiaContact: Zoran Igrutinovic, MDTaking people
About the trial
What the trial wants to find out
The main purpose of this study is to confirm the correct dose of efgartigimod IV for treating patients aged 12 to younger than 18 years with chronic immune thrombocytopenia (ITP).
Read full original descriptionShow less
The study consists of a double-blinded treatment period (DBTP) in which the participants will be randomized in a 2:1 ratio to receive either efgartigimod IV or placebo IV. At the end of the treatment period (up to 24 weeks), all participants will receive efgartigimod IV during the first year open-label treatment period (OLTP1). At the end of the first OLTP1, participants may begin a second year (OLTP2). After the OLTP2, the participants will enter a follow-up period (approximately 8 weeks) while off study drug. The participants will be in the study for up to 138 weeks.
More information can be found here: https://clinicaltrials.argenx.com/advancejunior
The 2 groups in the trial
- Efgartigimod IVTreatment being tested
Participants receiving efgartigimod IV during the double-blinded treatment period and the open-label treatment period(s)
- Placebo IVPlacebo, for comparison
Participants receiving placebo IV during the double-blinded treatment period and receiving efgartigimod IV during the open-label treatment period(s)
Treatment: Efgartigimod IV and Placebo IV
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Contact the study team: see the registry record → Contacts and Locations
Print a page for my doctorData and sources
- Registry record
- Listed on ClinicalTrials.gov and on the EU's official list of trials (CTIS).
- Official title
- A Multicenter, Randomized, Double-blinded, Parallel-Arm, Placebo-Controlled, Pharmacokinetic and Pharmacodynamic Study Followed by an Open-Label Arm to Evaluate Efgartigimod IV in Pediatric Participants From 12 Years to Less Than 18 Years of Age With Chronic ITP
- Fit questions
- Built from NCT07194850, who can take part
- Record updated
- 16 October 2026