At a glance
- What the study gets you
- Access to the study treatment being tested
- Type of study
- Interventional (receives a drug or procedure)
- Time in hospital
- In-person visits at study sites — visit count not specified by the sponsor
- Drug or intervention
- Efgartigimod IV (biological), Placebo IV (other)
- How long the study runs
- Study runs about 60 months (dates as stated)
- About the drug or intervention
- Efgartigimod IV — biological: Intravenous infusion of efgartigimod · Placebo IV — other: Intravenous infusion of placebo
- Patient visit burden
- Not specified by the sponsor
- Type of study
- Testing a treatment
- Ages
- 12 Years to 17 Years
- Who
- All
- Number of participants
- 24
- Started
- 2025-10-20
- Last checked
- 2026-09
Plain English Summary
What is this study?
- • Testing a new treatment for immune thrombocytopenia (itp)
- • Phase2/Phase3 - 24 participants
- • 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)
Who can take part?
- • Ages 12 Years to 17 Years
- • Diagnosed with immune thrombocytopenia (itp)
Where?
- • Cardiff - Cardiff and Vale NHS Trust - University Hospital of Wales (UHW)
- • Glasgow - The Royal Hospital for Children - Glasgow Health Board
- • Manchester - Royal Manchester Children's Hospital - Manchester University NHS Foundation Trust
This is a simplified summary. Always discuss with your doctor before making any decisions.
About This Trial
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). 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
How this trial compares with your answers
Answer 2 more questions to improve match
What we know so far
Still need:
- • Tell us your age for better matching
- • Tell us your sex for better matching
Preliminary match based on your answers. Full eligibility requires on-site assessment including medical history, physical exam, and lab tests. This does not guarantee enrolment.
Eligibility at a Glance
Key info
- Age: 12 Years - 17 Years
- Who can join: All genders
What the study is looking for
- ✓Is aged 12 to less than 18 years when completing the agreement to take part process
- ✓Has a documented duration of primary ITP of more than 12 months on the date the agreement to take part process is complete
- ✓Has documented prior ITP treatment with at least 1 of the following treatments: corticosteroids, IVIg, anti-D...
- ✓Has documented insufficient response to a prior ITP treatment with corticosteroids, IVIg, anti-D immunoglobulin,...
- ✓Has documented mean blood clotting cell count of less than 30 x10\^9/L
Who cannot take part
- ✗Secondary ITP according to the following definition by the International Working Group (IWG): all forms of...
- ✗Nonimmune thrombocytopenia
- ✗ITP-associated critical or severe bleeding
- ✗History of hereditary thrombocytopenia
See the full criteria
Where Is This Study? (3 UK sites)
Cardiff and Vale NHS Trust - University Hospital of Wales (UHW)
Cardiff CF14 4XW, United Kingdom
The Royal Hospital for Children - Glasgow Health Board
Glasgow G51 4TF, United Kingdom
Royal Manchester Children's Hospital - Manchester University NHS Foundation Trust
Manchester M139WL, United Kingdom
How to Get in Touch
Sabine Coppieters, MD
Sponsor contactCONTACT
