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Taking people in United KingdomPhase 2/3

Efgartigimod infusions for teenagers with long-lasting immune thrombocytopenia

Tests efgartigimod given by infusion in teenagers with chronic immune thrombocytopenia.

Key facts

Who runs it
argenx
Where
Cardiff, Glasgow, and Manchester, United KingdomSee the 3 sitesAlso in 10 other countries, 37 sites
Registry title
A Study of Efgartigimod IV in Participants From 12 Years to Less Than 18 Years of Age With Chronic Immune Thrombocytopenia (ITP)
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

AI summary

From the trial's eligibility criteria. Machine-read from the registry text on this page, so it can be wrong. The registry text below and the trial team decide who can take part.

The trial is looking for young people aged 12 to under 18 with chronic primary immune thrombocytopenia (ITP), a condition with low platelets. It tests efgartigimod IV against a placebo.

Age
12 to less than 18 years (12 Years to 17 Years)
Sex
All sexes
Disease stage
Primary ITP that has lasted more than 12 months.
Earlier treatment
Must have had at least 1 ITP treatment: corticosteroids, IVIg, anti-D immunoglobulin, TPO-RAs or rituximab. Must have once reached a platelet count of ≥50 × 10^9/L with prednisone, other corticosteroids, IVIg or anti-D immunoglobulin. Must have had too little response to a past treatment (corticosteroids, IVIg, anti-D immunoglobulin, TPO-RAs, rituximab or splenectomy, which is removal of the spleen).
Biomarker or test result
Average platelet count less than 30 x10^9/L.

Main reasons for not taking part

  • Secondary ITP, meaning low platelets caused by another immune condition
  • Low platelets not caused by the immune system
  • Critical or severe bleeding linked to ITP
  • History of inherited low platelets

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 United Kingdom

Also in 10 other countries, 37 sites

  • Cardiff and Vale Nhs Trust - University Hospital of Wales (Uhw)Cardiff, United KingdomContact: Philip Connor, MDTaking people
  • The Royal Hospital For Children - Glasgow Health BoardGlasgow, United KingdomContact: Sarah Clarke, MDTaking people
  • Royal Manchester Children'S Hospital - Manchester University Nhs Foundation TrustManchester, United KingdomContact: John Grainger, 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

  1. How often do I need to come in, and for how long each time?
  2. Are travel costs paid?
  3. Can I keep my own doctor?
  4. What happens if I want to stop?

Contact the study team: see the registry record → Contacts and Locations

Print a page for my doctor

Data 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
Plain-language title
Efgartigimod infusions for teenagers with long-lasting immune thrombocytopeniaGenerated from the registry record. The registry title is the official one.
AI summary
From the trial's eligibility criteria. Read by machine on 14 November 2026.
Fit questions
Built from NCT07194850, who can take part
Record updated
16 October 2026