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Primary Immune ThrombocytopeniaPhase 3ADVANCENCT04188379

A Study to Assess the Efficacy and Safety 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 121 sites in 18 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
Search all trials

Trial summary

This is a randomized, double-blind placebo-controlled multicenter phase 3 trial to evaluate the efficacy and safety of ARGX-113 in participants 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?
Yes, some participants may receive a placebo
How many people
131 participants
Where
121 sitesin 18 countries
Study dates
16 Dec 2019to 3 Feb 2022
Phase
Phase 3
Are there results?
Yes, posted in the registrySummary in the doctors' view

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).
  • Male or female patient aged ≥18 years.
  • Confirmed ITP diagnosis, at least 3 months before randomization and according to the American Society of Hematology Criteria, and no known other etiology for thrombocytopenia.
  • Diagnosis supported by a response to a prior ITP therapy (other than thrombopoietin receptor agonists [TPO-RAs]), in the opinion of the investigator.
  • Mean platelet count of <30×10E9/L from 2 counts: 1 platelet count collected during the screening period and the predose platelet count on the day of randomization.
  • At the start of the trial, the patient is either on concurrent ITP treatment(s) and has received at least 1 prior therapy for ITP in the past, or the patient is not on treatment for ITP but has received at least 2 prior treatments for ITP. Patients receiving permitted concurrent ITP treatment(s) at baseline, must have been stable in dose and frequency for at least 4 weeks prior to randomization.
  • Women of childbearing potential must have a negative serum pregnancy test at the screening visit and 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.

Who cannot join

  • ITP/thrombocytopenia associated with another condition, eg, lymphoma, chronic lymphocytic leukemia, viral infection, hepatitis, induced or alloimmune thrombocytopenia, or thrombocytopenia associated with myeloid dysplasia.
  • Use of certain medications before the start of the studies (more details in the protocol)
  • Patients who have a history of malignancy, including malignant thymoma, or myeloproliferative or lymphoproliferative disorders, unless deemed cured by adequate treatment with no evidence of recurrence for ≥3 years before screening. Patients with completely excised non-melanoma skin cancer (such as basal cell carcinoma or squamous cell carcinoma) or cervical carcinoma in situ would be permitted at any time.
  • Uncontrolled hypertension, defined as a repeated elevated blood pressure exceeding 160 mmHg (systolic) and/or 100 mmHg (diastolic) despite appropriate treatments.
  • History of any major thrombotic or embolic event within 12 months prior to randomization.
  • History of coagulopathy or hereditary thrombocytopenia or a family history of thrombocytopenia.
Show all 12Show fewer
  • History of a recent or planned major surgery (that involves major organs eg, brain, heart, lung, liver, bladder, or gastrointestinal tract) within 4 weeks of randomization.
  • Positive serum test at screening for an active viral infection with any of the following conditions: Hepatitis B virus (HBV), Hepatitis C virus (HCV), Human immunodeficiency virus (HIV)
  • Clinical evidence of significant unstable or uncontrolled acute or chronic diseases other than ITPdespite appropriate treatments which could put the patient at undue risk.
  • Patients with known medical history of hypersensitivity to any of the ingredients of the IMP.
  • Patients who previously participated in a clinical trial with efgartigimod and have received at least 1 administration of the IMP.
  • Pregnant or lactating females. More details in the protocol

Trial locations and contacts

121 sites in 18 countries.

121 sites in 18 countries

Turkey (Türkiye)16 sites

  • Investigator Site 0900002

    Adana

  • Investigator Site 0900007

    Adapazarı

  • Investigator Site 0900003

    Ankara

  • Investigator Site 0900006

    Ankara

  • Investigator Site 0900008

    Ankara

  • Investigator Site 0900015

    Ankara

  • Investigator Site 0900016

    Edirne

  • Investigator Site 0900013

    Istanbul

  • Investigator Site 0900004

    Izmir

  • Investigator Site 0900014

    Kocaeli

  • Investigator Site 0900018

    Malatya

  • Investigator Site 0900005

    Manisa

  • Investigator Site 0900010

    Mersin

  • Investigator Site 0900009

    Samsun

  • Investigator Site 0900017

    Tekirdağ

  • Investigator Site 0900019

    Trabzon

Other trials in Turkey (Türkiye)

Japan13 sites

  • Investigator Site 0810024

    Bunkyō City

  • Investigator Site 0810015

    Hirakata

  • Investigator Site 0810010

    Hiroshima

  • Investigator site 0810017

    Iruma

  • Investigator site 0810011

    Isehara

  • Investigator Site 0810022

    Kashiwa

  • Investigator site 0810018

    Maebashi

  • Investigator site 0810020

    Minatoku

  • Investigator Site 0810021

    Niigata

  • Investigator site 0810014

    Sapporo

  • Investigator site 0810016

    Shibukawa

  • Investigator Site 0810023

    Shimotsuke

  • Investigator Site 0810025

    Shinjuku-Ku

Other trials in Japan

Italy12 sites

  • Investigator Site 0390012

    Campobasso

  • 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 0390021

    Roma

  • Investigator Site 0390009

    Siena

  • Investigator Site 0390017

    Torino

  • Investigator Site 0390016

    Trieste

Other trials in Italy

Poland10 sites

  • Investigator Site 0480030

    Bialystok

  • Investigator Site 0480015

    Brzozów

  • Investigator Site 0480010

    Bydgoszcz

  • Investigator Site 0480013

    Chorzów

  • Investigator Site 0480012

    Gdansk

  • Investigator Site 0480008

    Katowice

  • Investigator Site 0480011

    Lodz

  • Investigator Site 0480014

    Lublin

  • Investigator site 0480026

    Nowy Sącz

  • Investigator Site 0480016

    Wroclaw

Other trials in Poland

Spain10 sites

  • Investigator Site 0340006

    Barcelona

  • Investigator Site 0340007

    Barcelona

  • Investigator Site 0340030

    Burgos

  • Investigator Site 0340009

    Madrid

  • Investigator Site 0340014

    Madrid

  • Investigator Site 0340015

    Madrid

  • Investigator site 0340012

    Palma de Mallorca

  • Investigator Site 0340013

    Seville

  • Investigator Site 0340004

    Valencia

  • Investigator Site 0340011

    Valencia

Other trials in Spain

United States9 sites

  • Investigator Site 0010049

    Cleveland, Ohio, 44106

  • Investigator Site 0010040

    Columbus, Ohio, 44106

  • Investigator Site 0010046

    Greenville, North Carolina, 27834

  • Investigator Site 0010042

    Iowa City, Iowa, 52242

  • Investigator Site 0010034

    Jacksonville, Florida, 32204

  • Investigator site 0010037

    Ocala, Florida, 34474

  • Investigator Site 0010041

    Philadelphia, Pennsylvania, 19104

  • Investigator Site 0010038

    Tucson, Arizona, 85711

  • Investigator Site 0010045

    Washington D.C., District of Columbia, 20007

Other trials in United States

France7 sites

  • Investigator Site 0330019

    Clermont-Ferrand

  • Investigator Site 0330009

    Créteil

  • Investigator Site 0330015

    Le Mans

  • Investigator Site 0330018

    Montpellier

  • Investigator Site 0330016

    Périgueux

  • Investigator Site 0330008

    Pessac

  • Investigator Site 0330017

    Rouen

Other trials in France

Belgium6 sites

  • Investigator Site 0320012

    Brasschaat

  • Investigator Site 0320011

    Bruges

  • Investigator Site 0320015

    Leuven

  • Investigator Site 0320014

    Turnhout

  • Investigator Site 0320020

    Verviers

  • Investigator site 0320002

    Yvoir

Other trials in Belgium

Georgia6 sites

  • Investigator Site 9950006

    Tbilisi

  • Investigator Site 9950007

    Tbilisi

  • Investigator Site 9950008

    Tbilisi

  • Investigator Site 9950009

    Tbilisi

  • Investigator Site 9950011

    Tbilisi

  • Investigator Site 9950012

    Tbilisi

Other trials in Georgia

Russia6 sites

  • Investigator site 0070006

    Kaluga

  • Investigator Site 0070007

    Petrozavodsk

  • Investigator Site 0070013

    Rostov-on-Don

  • Investigator Site 0070015

    Syktyvkar

  • Investigator Site 0070012

    Tula

  • Investigator site 0070010

    Ufa

Other trials in Russia

Hungary5 sites

  • Investigator Site 0360004

    Budapest

  • Investigator Site 0360006

    Debrecen

  • Investigator Site 0360015

    Győr

  • Investigator site 0360010

    Nyíregyháza

  • Investigator Site 0360014

    Szombathely

Other trials in Hungary

Czechia4 sites

  • Investigator Site 4200001

    Brno

  • Investigator Site 4200008

    Olomouc

  • Investigator Site 4200006

    Ostrava

  • Investigator Site 4200007

    Prague

Other trials in Czechia

United Kingdom4 sites

  • Investigator Site 0440008

    London

  • Investigator Site 0440010

    Rhyl

  • Investigator Site 0440012

    Southampton

  • Investigator Site 0440014

    Truro

Other trials in United Kingdom

Austria3 sites

  • Investigator Site 0430004

    Linz

  • Investigator Site 0430002

    Vienna

  • Investigator Site 0430003

    Vienna

Other trials in Austria

Germany3 sites

  • Investigator Site 0490010

    Düsseldorf

  • Investigator Site 0490008

    Essen

  • Investigator Site 0490012

    Giessen

Other trials in Germany

Netherlands3 sites

  • Investigator Site 0310005

    Rotterdam

  • Investigator Site 0310007

    Rotterdam

  • Investigator site 0310006

    The Hague

Other trials in Netherlands

Bulgaria2 sites

  • Investigator Site 3590001

    Pleven

  • Investigator Site 3590002

    Sofia

Other trials in Bulgaria

Ukraine2 sites

  • Investigator Site 3800022

    Kharkiv

  • Investigator site 3800006

    Mykolayiv

Other trials in Ukraine

About this study

Official title: A Phase 3, Multicenter, Randomized, Double-Blinded, Placebo-Controlled Trial to Evaluate the Efficacy and Safety of Efgartigimod (ARGX 113) 10 mg/kg Intravenous in Adult Patients With Primary Immune Thrombocytopenia

This is a randomized, double-blind placebo-controlled multicenter phase 3 trial to evaluate the efficacy and safety of ARGX-113 in participants with primary ITP.

Being tested
efgartigimod, Placebo
Keywords
immune thrombocytopenia, sustained platelet count response, bleeding events
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

  • PlaceboOther

    Intravenous infusion of placebo

Study groups in NCT04188379
Study groupRoleWhat they receive
efgartigimodExperimentalPatient receiving efgartigimod
Receives: Biological: efgartigimod
PlaceboPlacebo comparatorPatients receiving placebo
Receives: Other: Placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Percentage of Participants With Chronic ITP With a Sustained Platelet Count 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.

    Percentage of participants with chronic ITP with a sustained platelet count response was 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 study.

    Measured: From Week 19 up to Week 24

Secondary outcomes (4)

  1. Extent of Disease Control Defined as the Number of Cumulative Weeks Over the Planned 24-week Treatment Period With Platelet Counts of ≥50×10^9/L in the Chronic ITP Population

    Extent of disease control, defined as the cumulative number of weeks over the planned 24-week treatment period with platelet counts of ≥50 × 10^9/L in the chronic ITP population.

    Measured: From Week 1 up to Week 24

  2. Percentage of Participants With a Sustained Platelet Count Response for at Least 4 of the 6 Visits Between Week 19 and 24 of the Study

    Percentage of participants in the overall population (chronic and persistent ITP) with a sustained platelet count response, defined as achieving platelet counts of at least 50 × 10^9/L for at least 4 of the 6 visits between weeks 19 and 24 of the study.

    Measured: From Week 19 up to Week 24

  3. Incidence and Severity of the WHO-classified Bleeding Events

    Incidence of the World Health Organization (WHO)-classified bleeding events in the overall population.

    This secondary endpoint used the WHO-classified bleeding scale. Bleeding was the predominant clinical manifestation of ITP and was typically related to platelet count. Accordingly, measuring bleeding was important for monitoring this participant population.

    Measured: From Week 1 to Week 24

  4. Percentage of Participants in the Overall Population Achieving Platelet Counts of at Least 50×10^9/L for at Least 6 of the 8 Visits Between Week 17 and Week 24

    Percentage of participants in the overall population achieving platelet counts of at least 50 × 10^9/L for at least 6 of the 8 visits between Week 17 and 24 of the study.

    Measured: From Week 17 up to Week 24

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted5 Dec 2019
  2. Study start16 Dec 2019
  3. Primary completion3 Feb 2022
  4. Study completion3 Feb 2022

Resources

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
Randomised
Intervention model
Parallel groups
Masking
Quadruple blind
Who is masked
Participant, Care provider, Investigator, Outcomes assessor
Enrolment
131 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04188379
Sponsor's protocol ID
ARGX-113-1801
Other IDs
2019-002100-41, 2019
Acronym
ADVANCE
Status last verified
Feb 2025
First submitted
4 Dec 2019
Last updated
13 Mar 2025
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Publications

  • Broome CM, McDonald V, Miyakawa Y, Carpenedo M, Kuter DJ, Al-Samkari H, Bussel JB, Godar M, Ayguasanosa J, De Beuf K, Rodeghiero F, Michel M, Newland A; ADVANCE Investigator Study Group. Efficacy and safety of the neonatal Fc receptor inhibitor efgartigimod in adults with primary immune thrombocytopenia (ADVANCE IV): a multicentre, randomised, placebo-controlled, phase 3 trial. Lancet. 2023 Nov 4;402(10413):1648-1659. doi: 10.1016/S0140-6736(23)01460-5. Epub 2023 Sep 28.

    PubMed 37778358

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 121 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 8 inclusion and 12 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 13 Mar 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 121 sites in Turkey (Türkiye), Japan, Italy, Poland, Spain 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. Some participants in this study may receive a placebo. 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?

Yes. Results are posted in the registry (first posted 13 Mar 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

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?

Percentage of Participants With Chronic ITP With a Sustained Platelet Count 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: From Week 19 up to Week 24). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT04188379, which also lists 1 linked publication.

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 13 Mar 2025.

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