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Taking people in PortugalPhase 3

Efgartigimod given by infusion for adults with primary immune thrombocytopenia

Tests efgartigimod against placebo in adults with primary immune thrombocytopenia.

Key facts

Who runs it
argenx
Where
Braga and Vila Nova de Gaia, PortugalSee the 2 sitesAlso in 17 other countries, 96 sites
Registry title
A Study to Assess the Efficacy and Safety of Efgartigimod IV in Adult Participants With Primary Immune Thrombocytopenia
Status
Recruiting
Phase
Phase 3
People taking part
69 people, planned
Started
18 Oct 2024
Expected to end
Jun 2028
Type of study
Interventional, 2 groups
Treatments tested
  • Efgartigimod IV
  • Placebo IV
Record updated
1 May 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 adults who have had primary immune thrombocytopenia (ITP) for more than 12 months and have low platelet counts. ITP is a condition where the immune system destroys platelets.

Age
18 years and older, and at least the local legal age of consent.
Sex
All sexes can join.
Disease stage
Primary ITP lasting more than 12 months when the consent form is signed.
Earlier treatment
People must have had at least 1 earlier ITP treatment: corticosteroids, intravenous immunoglobulin (IVIg), anti-D immunoglobulin, thrombopoietin receptor agonist (TPO-RAs), or rituximab. A prior ITP treatment must not have worked well enough. In the 3 years before signing consent, platelets must have reached ≥50 × 109/L at least once after prednisone, dexamethasone, other corticosteroids, IVIg, or anti-D immunoglobulin.
Biomarker or test result
Average platelet count at the start below 30 x 10^9/L before randomization.

Main reasons for not taking part

  • Secondary ITP, meaning ITP caused by another disease
  • Low platelets not caused by the immune system
  • Autoimmune hemolytic anemia, or another autoimmune disease
  • Critical or severe bleeding linked to ITP
  • Other medical conditions that could confuse the results or put the person at undue risk

The registry criteria, word for word

Show all 11 criteriaShow less

Who can take part

  • Is at least 18 years of age and the local legal age of consent for clinical studies when signing the informed consent form (ICF)
  • Has documented baseline mean platelet count of <30 x 10\^9/L before randomization
  • Has a documented duration of primary immune thrombocytopenia (ITP) of more than 12 months on the date of informed consent form (ICF) signature
  • Has documented prior ITP treatment with at least 1 of the following treatments: corticosteroids, intravenous immunoglobulin (IVIg), anti-D immunoglobulin, thrombopoietin receptor agonist (TPO-RAs), or rituximab
  • Has documented insufficient response to a prior ITP treatment (the specific criteria can be found in the protocol)
  • Has documented prior response defined as 1 platelet count of ≥50 × 109/L to at least 1 of the following ITP treatments in the 3 years before the date of ICF signature: prednisone, dexamethasone, other or nonspecified corticosteroids, IVIg, or anti-D immunoglobulin

Who cannot take part

  • Other than the indication under study, known autoimmune disease or any medical condition that would interfere with an accurate assessment of clinical symptoms of ITP, confound the results of the study or put the participant at undue risk
  • Autoimmune hemolytic anemia
  • ITP-associated critical or severe bleeding The complete list of criteria can be found in the protocol
  • Secondary ITP
  • Nonimmune thrombocytopenia

Where in Portugal

Also in 17 other countries, 96 sites

  • CCAB Centro Clinico Academico Braga AssociacaoBraga, PortugalTaking people
  • Unidade Local de Saúde de Gaia/Espinho, E. P. EVila Nova de Gaia, PortugalTaking people

About the trial

What the trial wants to find out

The main purpose of this study is to look at the effect (efficacy) and safety of efgartigimod IV in participants with primary immune thrombocytopenia (ITP). After an up to 2 weeks screening period, eligible participants will be randomized in a 2:1 ratio to receive either efgartigimod IV or placebo IV, respectively during the double-blinded treatment period (DBTP). At the end of the treatment period (up to 24 weeks), all participants will receive efgartigimod IV during the first 52-week open-label treatment period (OLTP1). At the end of the first OLTP1, participants may begin a second 52-week 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.

Read full original descriptionShow less

More information can be found here: https://clinicaltrials.argenx.com/advancenext

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 IVTreatment being tested

    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 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
Plain-language title
Efgartigimod given by infusion for adults with primary 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.
Record updated
1 May 2026