Primary Immune ThrombocytopeniaPhase 3ADVANCE+NCT04225156
A Long-term Study to Assess the Safety and Efficacy of Efgartigimod in Adult Patients With Primary Immune Thrombocytopenia (ITP).
A Phase 3 study testing efgartigimod in people with Primary Immune Thrombocytopenia, run by argenx at 87 sites in 18 countries.
Trial summary
This is an open-label long-term multicenter phase 3 trial to evaluate the efficacy and safety of ARGX-113 in adult patients with primary ITP. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Primary Immune Thrombocytopenia
- What is being tested?
- efgartigimod
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 101 participants
- Where
- 87 sitesin 18 countries
- Study dates
- 2 Jun 2020to 11 Mar 2026
- Phase
- Phase 3
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Ability to understand the requirements of the trial, to provide written informed consent (including consent for the use and disclosure of research-related health information), and to comply with the trial protocol procedures (including required trial visits).
- Patients enrolled in the ARGX-113-1801 trial who completed the 24-weeks trial period.
- Women of childbearing potential must have a negative urine pregnancy test at baseline before trial medication (infusion) can be administered.
- Women of childbearing potential should use a highly effective or acceptable method of contraception during the trial and for 90 days after the last administration of the IMP. They must be on a stable regimen, for at least 1 month (as listed in the protocol)
- 6. Ability to understand the requirements of the additional 52-week treatment period of the trial, to provide written informed consent (including consent for the use and disclosure of research-related health information), and to comply with the trial protocol procedures (including required trial visits).
- 7. Patient has completed a 52-week treatment period.
Who cannot join
- Introduction or continuation of non-permitted medications during the ARGX-113-1801 trial (such as anti-CD20 therapy, romiplostim, monoclonal antibodies, Fc fusion proteins or live/live-attenuated vaccines).
- Pregnant or lactating women, and those intending to become pregnant during the trial or within 90 days after the last dosing.
- Patients with known medical history of hypersensitivity to any of the ingredients of efgartigimod.
- Use of any other investigational drug or participation in any other investigational trial.
Trial locations and contacts
87 sites in 18 countries.
87 sites in 18 countries
Turkey (Türkiye)11 sites
Investigator Site 0900003
Ankara
Investigator Site 0900006
Ankara
Investigator Site 0900015
Ankara
Investigator Site 0900016
Edirne
Investigator Site 0900013
Istanbul
Investigator Site 0900004
Izmir
Investigator Site 0900010
Mersin
Investigator Site 0900007
Sakarya
Investigator Site 0900009
Samsun
Investigator Site 0900017
Tekirdağ
Investigator Site 0900019
Trabzon
Italy9 sites
Investigator Site 0390014
Milan
Investigator Site 0390020
Monza
Investigator Site 0390015
Novara
Investigator Site 0390010
Ravenna
Investigator Site 0390011
Reggio Calabria
Investigator Site 0390018
Reggio Emilia
Investigator Site 0390019
Rimini
Investigator Site 0390009
Siena
Investigator Site 0390016
Trieste
Japan9 sites
Investigator Site 0810015
Hirakata
Investigator Site 0810010
Hiroshima
Investigator Site 0810017
Iruma
Investigator Site 0810022
Kashiwa
Investigator Site 0810018
Maebashi
Investigator Site 0810021
Niigata
Investigator Site 0810014
Sapporo
Investigator Site 0810016
Shibukawa
Investigator Site 0810023
Shimotsuke
Spain9 sites
Investigator Site 0340006
Barcelona
Investigator Site 0340007
Barcelona
Investigator Site 0340009
Madrid
Investigator Site 0340014
Madrid
Investigator Site 0340012
Palma de Mallorca
Investigator Site 0340015
Pozuelo de Alarcón
Investigator Site 0340013
Seville
Investigator Site 0340004
Valencia
Investigator Site 0340011
Valencia
Russia7 sites
Investigator Site 0070006
Kaluga
Investigator Site 0070008
Moscow
Investigator Site 0070007
Petrozavodsk
Investigator Site 0070013
Rostov-on-Don
Investigator Site 0070015
Syktyvkar
Investigator Site 0070012
Tula
Investigator Site 0070010
Ufa
Georgia5 sites
Investigator site 9950007
Tbilisi
Investigator site 9950008
Tbilisi
Investigator site 9950009
Tbilisi
Investigator site 9950011
Tbilisi
Investigator site 9950012
Tbilisi
Hungary5 sites
Investigator Site 0360004
Budapest
Investigator Site 0360006
Debrecen
Investigator Site 0360015
Győr
Investigator Site 0360010
Nyíregyháza
Investigator Site 0360014
Szombathely
Poland5 sites
Investigator Site 0480012
Gdansk
Investigator Site 0480013
Katowice
Investigator Site 0480011
Lodz
Investigator Site 0480014
Lublin
Investigator Site 0480026
Nowy Sącz
United States4 sites
Investigator Site 0010040
Columbus, Ohio, 43210
Investigator Site 0010042
Iowa City, Iowa, 52242
Investigator Site 0010037
Ocala, Florida, 34474
Investigator Site 0010045
Washington D.C., District of Columbia, 20007
Belgium4 sites
Investigator Site 0320012
Brasschaat
Investigator Site 0320011
Bruges
Investigator Site 0320014
Turnhout
Investigator Site 0320002
Yvoir
Czechia4 sites
Investigator Site 4200001
Brno
Investigator Site 4200008
Olomouc
Investigator Site 4200006
Ostrava
Investigator Site 4200007
Prague
France4 sites
Investigator Site 0330009
Créteil
Investigator Site 0330018
Montpellier
Investigator Site 0330016
Périgueux
Investigator Site 0330008
Pessac
Austria2 sites
Investigator Site 0430002
Vienna
Investigator Site 0430003
Vienna
Bulgaria2 sites
Investigator Site 3590001
Pleven
Investigator Site 3590002
Sofia
Germany2 sites
Investigator Site 0490010
Düsseldorf
Investigator Site 0490008
Essen
Netherlands2 sites
Investigator Site 0310005
Rotterdam
Investigator Site 0310006
The Hague
United Kingdom2 sites
Investigator Site 0440008
London
Investigator Site 0440012
Southampton
About this study
Official title: A Phase 3, Multicenter, Open-label, Long-term Trial to Evaluate the Safety and Efficacy of Efgartigimod (ARGX 113) 10 mg/kg Intravenous in Adult Patients With Primary Immune Thrombocytopenia.
This is an open-label long-term multicenter phase 3 trial to evaluate the efficacy and safety of ARGX-113 in adult patients with primary ITP.
- Conditions
- Primary Immune Thrombocytopenia
- Being tested
- efgartigimod
- Medical subject headings
- Purpura, Thrombocytopenic, Idiopathic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
efgartigimodBiological
Also called ARGX-113
Intravenous infusion of efgartigimod
| Study group | Role | What they receive |
|---|---|---|
| efgartigimod | Experimental | patients receiving efgartigimod Receives: Biological: efgartigimod |
What is being measured
The primary outcome is the main question the study is designed to answer.
Frequency and severity of Adverse Events
Measured: Up to 60 weeks
Frequency and severity of vital signs
Measured: Up to 60 weeks
Frequency and severity of laboratory assessments
Measured: Up to 60 weeks
Secondary outcomes (17)
Extent of disease control defined as the percentage of weeks in the trial with platelet counts of ≥50×10E9/L.
Measured: Over the 52 weeks of treatment
Percentage of patients with overall platelet count response defined as achieving a platelet count of ≥50×10^9/L on at least 4 occasions at any time during the 52-week treatment period.
Measured: Over the 52 weeks of treatment
Mean change from baseline in platelet count at each visit.
Measured: Up to 60 weeks, at each visit
For patients rolling-over from the ARGX-113-1801 trial with a platelet count of <30×10^9/L: time to response is defined as the time to achieve 2 consecutive platelet counts of ≥50×10^9/L
Measured: Up to 60 weeks, at each visit
The percentage of weeks in the trial with platelet counts of ≥30×109/L and at least 20×10E9/L above baseline.
Measured: Over the 52 weeks of treatment
In patients with baseline platelet count of <15×10E9/L in the current trial (ARGX-113-1803), the percentage of weeks in the trial with platelet counts of ≥30×10E9/L and at least 20×10E9/L above baseline.
Measured: Over the 52 weeks of treatment
In patients with first exposure to efgartigimod: proportion of patients who achieve a sustained platelet response defined as achieving platelet counts of at least 50×10^9/L for at least 4 of the 6 visits between week 19 and 24 of the trial.
Measured: Up to 5 weeks, between visit 19 and 24 of the trial
In patients with first exposure to efgartigimod: proportion of patients in the overall population achieving platelet counts of at least 50x10^9/L for at least 6 of the 8 visits between week 17 and 24 of the trial.
Measured: Up to 7 weeks, between visit 17 and 24 of the trial
Rate of receipt of rescue therapy (rescue per patient per month).
Measured: Up to 60 weeks, at each visit
Reduction in concurrent ITP therapy.
Measured: Up to 60 weeks, at each visit
Incidence and severity of the WHO-classified bleeding events.
Measured: Up to 60 weeks, at each visit
Change from baseline in Patient reported Outcomes (FACIT-Fatigue) at planned visits.
Measured: Up to 52 weeks
Change from baseline in Patient reported Outcomes (Fact-Th6) at planned visits.
Measured: Up to 52 weeks
Change from baseline in Quality of Life (SF-36) at planned visits.
Measured: Up to 52 weeks
Incidence of anti-drug antibodies (ADA) to efgartigimod.
Measured: Up to 216 weeks
Pharmacokinetic parameter of efgartigimod: serum concentration observed predose (Ctrough).
Measured: Up to 60 weeks
Pharmacodynamics markers: total IgG.
Measured: Up to 60 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Jan 2020
- Study start2 Jun 2020
- Primary completion11 Mar 2026
- Study completion11 Mar 2026
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Primary Immune ThrombocytopeniaStudies of the same condition, open and under way.
- More trials run by argenxThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 101 participants (actual)
Sponsor and investigators
- Lead sponsor
- argenx
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04225156
- Sponsor's protocol ID
- ARGX-113-1803
- Other IDs
- 2019-002101-21, 2019
- Acronym
- ADVANCE+
- Status last verified
- Mar 2026
- First submitted
- 6 Jan 2020
- Last updated
- 16 Mar 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 16 Mar 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 3, Multicenter, Open-label, Long-term Trial to Evaluate the Safety and Efficacy of Efgartigimod (ARGX 113) 10 mg/kg Intravenous in Adult Patients With Primary Immune Thrombocytopenia.
- ClinicalTrials.gov ID
- NCT04225156
- Acronym / sponsor ID
- ADVANCE+ / ARGX-113-1803
- Conditions
- Primary Immune Thrombocytopenia
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 101 participants (actual)
- Lead sponsor
- argenxIndustry (pharma, biotech, devices)
- Study start
- 2 Jun 2020 (actual)
- Primary completion
- 11 Mar 2026 (actual)
- Study completion
- 11 Mar 2026 (actual)
- First posted
- 13 Jan 2020 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is an open-label long-term multicenter phase 3 trial to evaluate the efficacy and safety of ARGX-113 in adult patients with primary ITP.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| efgartigimod | Experimental | Biological: efgartigimodpatients receiving efgartigimod |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- Ability to understand the requirements of the trial, to provide written informed consent (including consent for the use and disclosure of research-related health information), and to comply with the trial protocol procedures (including required trial visits).
- Patients enrolled in the ARGX-113-1801 trial who completed the 24-weeks trial period.
- Women of childbearing potential must have a negative urine pregnancy test at baseline before trial medication (infusion) can be administered.
- Women of childbearing potential should use a highly effective or acceptable method of contraception during the trial and for 90 days after the last administration of the IMP. They must be on a stable regimen, for at least 1 month (as listed in the protocol)
- 6. Ability to understand the requirements of the additional 52-week treatment period of the trial, to provide written informed consent (including consent for the use and disclosure of research-related health information), and to comply with the trial protocol procedures (including required trial visits).
Key exclusion criteria (4 listed)
- Introduction or continuation of non-permitted medications during the ARGX-113-1801 trial (such as anti-CD20 therapy, romiplostim, monoclonal antibodies, Fc fusion proteins or live/live-attenuated vaccines).
- Pregnant or lactating women, and those intending to become pregnant during the trial or within 90 days after the last dosing.
- Patients with known medical history of hypersensitivity to any of the ingredients of efgartigimod.
- Use of any other investigational drug or participation in any other investigational trial.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| efgartigimodAlso: ARGX-113Intravenous infusion of efgartigimod | Biological | efgartigimod |
Primary outcome
Frequency and severity of Adverse Events
Time frame: Up to 60 weeks
Frequency and severity of vital signs
Time frame: Up to 60 weeks
Frequency and severity of laboratory assessments
Time frame: Up to 60 weeks
17 secondary outcomes
Extent of disease control defined as the percentage of weeks in the trial with platelet counts of ≥50×10E9/L.
Time frame: Over the 52 weeks of treatment
Percentage of patients with overall platelet count response defined as achieving a platelet count of ≥50×10^9/L on at least 4 occasions at any time during the 52-week treatment period.
Time frame: Over the 52 weeks of treatment
Mean change from baseline in platelet count at each visit.
Time frame: Up to 60 weeks, at each visit
For patients rolling-over from the ARGX-113-1801 trial with a platelet count of <30×10^9/L: time to response is defined as the time to achieve 2 consecutive platelet counts of ≥50×10^9/L
Time frame: Up to 60 weeks, at each visit
The percentage of weeks in the trial with platelet counts of ≥30×109/L and at least 20×10E9/L above baseline.
Time frame: Over the 52 weeks of treatment
In patients with baseline platelet count of <15×10E9/L in the current trial (ARGX-113-1803), the percentage of weeks in the trial with platelet counts of ≥30×10E9/L and at least 20×10E9/L above baseline.
Time frame: Over the 52 weeks of treatment
In patients with first exposure to efgartigimod: proportion of patients who achieve a sustained platelet response defined as achieving platelet counts of at least 50×10^9/L for at least 4 of the 6 visits between week 19 and 24 of the trial.
Time frame: Up to 5 weeks, between visit 19 and 24 of the trial
In patients with first exposure to efgartigimod: proportion of patients in the overall population achieving platelet counts of at least 50x10^9/L for at least 6 of the 8 visits between week 17 and 24 of the trial.
Time frame: Up to 7 weeks, between visit 17 and 24 of the trial
Rate of receipt of rescue therapy (rescue per patient per month).
Time frame: Up to 60 weeks, at each visit
Reduction in concurrent ITP therapy.
Time frame: Up to 60 weeks, at each visit
Incidence and severity of the WHO-classified bleeding events.
Time frame: Up to 60 weeks, at each visit
Change from baseline in Patient reported Outcomes (FACIT-Fatigue) at planned visits.
Time frame: Up to 52 weeks
Change from baseline in Patient reported Outcomes (Fact-Th6) at planned visits.
Time frame: Up to 52 weeks
Change from baseline in Quality of Life (SF-36) at planned visits.
Time frame: Up to 52 weeks
Incidence of anti-drug antibodies (ADA) to efgartigimod.
Time frame: Up to 216 weeks
Pharmacokinetic parameter of efgartigimod: serum concentration observed predose (Ctrough).
Time frame: Up to 60 weeks
Pharmacodynamics markers: total IgG.
Time frame: Up to 60 weeks
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Mar 2026.
- Individual participant dataSharing planned: Undecided
Related trials for Primary Immune Thrombocytopenia
RecruitingPhase 3
A Study of Mezagitamab in Adults With Chronic Primary Immune Thrombocytopenia
Immune Thrombocytopenic Purpura (ITP)
RecruitingPhase 1/2
A Study of Pirtobrutinib in Participants With Immune Thrombocytopenia
Immune Thrombocytopenia (ITP)
RecruitingPhase 2
A Study to Assess the Tolerability of Ianalumab (VAY736) With Investigator's Choice Thrombopoietin Receptor Agonist (IC TPO-RA) in Patients With Primary Immune Thrombocytopenia (ITP)
Primary Immune Thrombocytopenia (ITP), Primary Evans Syndrome (ES)
RecruitingPhase 2
Study to Assess the Safety and Tolerability of Tafasitamab in Adult Participants With Primary Autoimmune Blood Cell Disorders
Immune Thrombocytopenia
RecruitingPhase 3
A Follow-up Study of Mezagitamab in Adults With Chronic Primary Immune Thrombocytopenia
Immune Thrombocytopenic Purpura (ITP)
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
87 sites in 18 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing efgartigimod in people with Primary Immune Thrombocytopenia, run by argenx at 87 sites in 18 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 16 Mar 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 87 sites in Turkey (Türkiye), Italy, Japan, Spain, Russia and 13 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Frequency and severity of Adverse Events (time frame: Up to 60 weeks). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 16 Mar 2026.