A Study to Assess the Efficacy and Safety of Efgartigimod IV in Adult Participants With Primary Immune Thrombocytopenia
Key facts
- Who runs it
- argenx
- Where
- Bassano Del Grappa, Florence, Milan, Naples, Novara, Ravenna, Reggio Emilia, Trieste, Turin, and Varese, ItalySee the 12 sitesAlso in 17 other countries, 86 sites
- Trial code
- 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
- Age
- 18 years and older
- Sex
- Women and men
- Healthy volunteers
- Cannot take part
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 Italy
Also in 17 other countries, 86 sites
- Regione Del Veneto Azienda ULSS N 7 PedemontanaBassano Del Grappa, ItalyTaking people
- Careggi University HospitalFlorence, ItalyTaking people
- ASST Grande Ospedale Metropolitano NiguardaMilan, ItalyTaking people
- Fondazione IRCCS Cà Granda Ospedale Maggiore PoliclinicoMilan, ItalyTaking people
- Azienda Ospedaliera Universitaria Federico II Di NapoliNaples, ItalyTaking people
- Azienda Ospedaliero Universitaria Maggiore della CaritàNovara, ItalyTaking people
- Azienda Unità Sanitaria Locale della RomagnaRavenna, ItalyTaking people
- Azienda USL IRCCS di Reggio EmiliaReggio Emilia, ItalyTaking people
- Azienda Sanitaria Universitaria Giuliano IsontinaTrieste, ItalyTaking people
- Azienda Ospedaliera Ordine Mauriziano di TorinoTurin, ItalyTaking people
- Azienda Ospedaliera Universitaria Citta Della Salute E Della Scienza Di TorinoTurin, ItalyTaking people
- Azienda Socio Sanitaria Territoriale Dei Sette LaghiVarese, ItalyTaking 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.
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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
- 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 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
- Record updated
- 1 May 2026