Immune Thrombocytopenia (ITP)Phase 2/3Advance JrNCT07194850
A Study of Efgartigimod IV in Participants From 12 Years to Less Than 18 Years of Age With Chronic Immune Thrombocytopenia (ITP)
A Phase 2/3 study testing Efgartigimod IV in people with Immune Thrombocytopenia (ITP) and ITP - Immune Thrombocytopenia, run by argenx at 26 sites in 10 countries.
Trial summary
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 more about this study
- Who is it for?
- Ages 12 to 17 years
- Conditions the study is for
- Immune Thrombocytopenia (ITP), ITP - Immune Thrombocytopenia, ITP
- What is being tested?
- Efgartigimod IV
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 24 participants
- Where
- 26 sitesin 10 countries
- Study dates
- 20 Oct 2025to Oct 2030
- Phase
- Phase 2/3
- Are there results?
- Not yet: the study is under way
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 12 to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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 join
- Secondary ITP according to the following definition by the International Working Group (IWG): all forms of immune-mediated thrombocytopenia except primary ITP
- Nonimmune thrombocytopenia
- ITP-associated critical or severe bleeding
- History of hereditary thrombocytopenia
Trial locations and contacts
26 sites in 10 countries. Contact the study team or a site near you to ask about taking part.
26 sites in 10 countries
Spain5 sites, 5 recruiting
- Recruiting
Hospital Universitari Vall d'Hebron
Barcelona, 08035
Thais Murciano Carrillo, MD, 0034934893093, thais.murciano@vallhebron.cat
- Recruiting
Hospital Sant Joan de Deu Barcelona
Esplugues de Llobregat, 08950
Ruben Berrueco Moreno, MD, 0034936009733, ruben.berrueco@sjd.es
- Recruiting
Hospital Infantil Universitario Nino Jesus (HIUNJS)
Madrid, 28009
Julian Sevilla Navarro, MD, 0034915036900, julian.sevilla@salud.madrid.org
- Recruiting
Hospital Materno-Infantil Universitario Gregorio Maranon
Madrid, 28009
David Diaz Perez, MD, 0037915290037, ddiazp@salud.madrid.org
- Recruiting
Hospital Universitario 12 de Octubre
Madrid, 28041
Maria del Carmen Garrido Colino, MD, 0034913908000, cgarridoc@salud.madrid.org
Italy4 sites, 4 recruiting
- Recruiting
Gaslini Children's Hospital
Genoa, 16147
Maurizio Miano, MD, +3901056363683, mauriziomiano@gaslini.org
- Recruiting
Fondazione IRCCS San Gerardo dei Tintori
Monza, 20900
Marco Spinelli, MD, +390392336816, marco.spinelli@irccs-sangerardo.it
- Recruiting
Bambino Gesu Children's Hospital
Roma, 165
Giuseppe Palumbo, MD, +390668594182, Giuseppe.palumbo@opbg.net
- Recruiting
A.O.U. Citta della Salute e della Scienza di Torino - Ospedale Infantile Regina Margherita
Torino, 10126
Franca Fagioli, MD, +390113135230, Franca.fagioli@unito.it
France3 sites, 3 recruiting
- Recruiting
Centre Hospitalier Universitaire de Bordeaux Groupe Hospitalier Pellegrin Hopital des Enfants
Bordeaux, 33000
Nathalie Aladjidi, MD, +33557820261, nathalie.aladjidi@chu-bordeaux.fr
- Recruiting
Centre Hospitalier Universitaire de Grenoble - Hopital Couple - Enfant
La Tronche, 38700
Cecile Perret, 04 76 76 72 50, cperret1@chu-grenoble.fr
- Recruiting
CHU de Nancy Hopitaux de Brabois
Vandœuvre-lès-Nancy, 54500
Helene Deutsch, 857-350-4834, h.deutsch@chru-nancy.fr
Japan3 sites, 3 recruiting
- Recruiting
Hyogo Prefectural Kobe Children's Hospital
Kobe, Hyōgo, 650-0047
Daiichiro Hasegawa, +81 787326961, hasegawa_kch@hp.pref.hyogo.jp
- Recruiting
Local Incorporated Administrative Agency Osaka City Hospital Organization Osaka City General Hospital
Osaka, Osaka, 534-0021
Hiroyuki Fujisaki, MD, +1 857-350-4834, h-fujisaki@med.osakacity-hp.or.jp
- Recruiting
Hokkaido University Hospital
Sapporo, Hokkaido, 060-8648
Shinsuke Hirabayashi, hirashin@huhp.hokudai.ac.jp
United Kingdom3 sites, 3 recruiting
- Recruiting
Cardiff and Vale NHS Trust - University Hospital of Wales (UHW)
Cardiff, CF14 4XW
Philip Connor, MD, +442020744829, philip.connor@wales.nhs.uk
- Recruiting
The Royal Hospital for Children - Glasgow Health Board
Glasgow, G51 4TF
Sarah Clarke, MD, sarah.clarke2@nhs.scot
- Recruiting
Royal Manchester Children's Hospital - Manchester University NHS Foundation Trust
Manchester, M139WL
John Grainger, MD, +441617018416, john.grainger@mft.nhs.uk
Germany2 sites, 2 recruiting
- Recruiting
Charite - Universitaetsmedizin Berlin - Campus Virchow-Klinikum (CVK)
Berlin, 13353
Susanne Holzhauer, MD, +4930450666215, susanne.holzhauer@charite.de
- Recruiting
Universitaetsklinikum Freiburg Kinder - Kinder- und Jugendklinik
Freiburg im Breisgau, 79106
Sarah Salou, MD, +49 (0)761-270-43829, sarah.salou@uniklinik-freiburg.de
Poland2 sites, 2 recruiting
- Recruiting
Samodzielny Publiczny Zaklad Opieki Zdrowotnej Uniwersytecki Szpital Kliniczny nr 4
Lodz, 92-213
Wojciech Mlynarski, MD, +48660732794, wojciech.mlynarski@umed.lodz.pl
- Recruiting
Uniwersytecki Szpital Dzieciecy w Lublinie
Lublin, 20-093
Katarzyna Drabko, MD, +48606729876, katarzyna.drabko@umlub.pl
Serbia2 sites, 2 recruiting
- Recruiting
Institute Of Mother And Child Healthcare Of Serbia Dr Vukan Cupic
Belgrade, 11070
Dragana Aleksic, MD, aleksic.dragana9@gmail.com
- Recruiting
University Clinical Centre of Kragujevac
Kragujevac, 34000
Zoran Igrutinovic, MD, +381505177, igzor@fmn.kg.ac.rs
Lithuania1 site, 1 recruiting
- Recruiting
Vilnius University Hospital Santaros Klinikos, Children's Hospital
Vilnius, 8406
Sonata Trakymiene, MD, +37052720412, sonata.saulytetrakymiene@santa.lt
Romania1 site, 1 recruiting
- Recruiting
Institutul Clinic Fundeni
Bucharest, 022328
Anca Colita, MD, +40213172194, secretariat@icfundeni.ro
About this study
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
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).
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
- Conditions
- Immune Thrombocytopenia (ITP), ITP - Immune Thrombocytopenia, ITP, Immune Thrombocytopenic Purpura, Immune Thrombocytopenic Purpura ( ITP ), Idiopathic Thrombocytopenic Purpura
- Being tested
- Efgartigimod IV, Placebo IV
- 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.
Efgartigimod IVBiological
Intravenous infusion of efgartigimod
Placebo IVOther
Intravenous infusion of placebo
| Study group | Role | What they receive |
|---|---|---|
| Efgartigimod IV | Experimental | Participants receiving efgartigimod IV during the double-blinded treatment period and the open-label treatment period(s) Receives: Biological: Efgartigimod IV |
| Placebo IV | Placebo comparator | Participants receiving placebo IV during the double-blinded treatment period and receiving efgartigimod IV during the open-label treatment period(s) Receives: Biological: Efgartigimod IV; Other: Placebo IV |
What is being measured
The primary outcome is the main question the study is designed to answer.
Efgartigimod serum concentrations in the DBTP
Measured: Up to 24 weeks
Total IgG levels in the DBTP
Measured: Up to 24 weeks
Secondary outcomes (11)
Efgartigimod serum concentrations over time during the DBTP
Measured: Up to 24 weeks
Percent change from baseline in total IgG levels in serum over time during the DBTP
Measured: Up to 24 weeks
Incidence of AEs, SAEs and AEs leading to IMP discontinuation
SAE: Serious adverse event; AE: adverse event
Measured: Up to 136 weeks
Sustained platelet count response between study weeks 19 and 24 of the DBTP and in OLTP1 for participants receiving placebo in the DBTP
Sustained platelet count defined as achieving platelet counts of ≥50 × 10^9/L for at least 4 of the 6 study visits
Measured: Up to 48 weeks
Extent of disease control during the DBTP and during the first 24 weeks of OLTP1 for those participants receiving placebo in the DBTP
Extend of disease defined as the number of cumulative weeks with a platelet count of ≥50 × 10^9/L
Measured: Up to 48 weeks
Changes from baseline for platelet counts over time
Measured: Up to 76 weeks
Incidence of bleeding, assessed by the Modified Buchanan and Adix Bleeding Score for pediatric ITP
The Modified Buchanan and Adix Bleeding Score for pediatric ITP is a semiquantitative assessment tool that measures bleeding signs and symptoms, comprising a score based on a scale of 0 to 5, each representing a different level of severity (0 = no risk; 5 = highest severity).
Measured: Up to 76 weeks
Incidence of ADA and Nab against efgartigimod in serum
ADA: anti-drug antibodies; Nab: neutralizing antibodies
Measured: Up to 76 weeks
Change from baseline in EQ-5D-5L
The European Quality of Life 5 Dimensions 5 Level (EQ-5D-5L) is a questionnaire comprised of 5 dimensions. Participants are asked to select the statement in each dimension which best describes their health on the day they complete the questionnaire. Responses in each dimension are coded as a 1-digit number ranging from 1 (no problems) to 5 (extreme problems).
Measured: Up to 76 weeks
Change from baseline in KIT Child Self-Report and KIT Parent Impact Report
The Kids' ITP Tools (KIT) comprises two disease- specific tools: a self-report form for children aged 12 and older, and a parent impact form. Respondents provide insights into their disease experience using a 1-week recall period. The instrument generates a total score, calculated by summing the items and converting them to a scale of 0 to 100, where higher scores reflect a better disease-specific quality of life (QoL).
Measured: Up to 76 weeks
Change from baseline in peds FACIT-F
In all participants, the pediatric Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-F) will use to assess health-related quality of life (HRQoL) before and after treatment. The FACIT-F questionnaire has a score range of 0 to 52, where 0 represents the worst possible fatigue and 52 indicates no fatigue.
Measured: Up to 76 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Sep 2025
- Study start20 Oct 2025
- Primary completionOct 2028 (estimated)
- Study completionOct 2030 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Immune Thrombocytopenia (ITP)Studies 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 2/3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 24 participants (estimated)
Sponsor and investigators
- Lead sponsor
- argenx
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07194850
- Sponsor's protocol ID
- ARGX-113-2409
- Other IDs
- 2025-521055-23-00, 2025
- Acronym
- Advance Jr
- Status last verified
- Sep 2026
- First submitted
- 19 Sep 2025
- Last updated
- 16 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 16 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT07194850
- Acronym / sponsor ID
- Advance Jr / ARGX-113-2409
- Conditions
- Immune Thrombocytopenia (ITP), ITP - Immune Thrombocytopenia, ITP, Immune Thrombocytopenic Purpura, Immune Thrombocytopenic Purpura ( ITP ), Idiopathic Thrombocytopenic Purpura
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 24 participants (estimated)
- Lead sponsor
- argenxIndustry (pharma, biotech, devices)
- Study start
- 20 Oct 2025 (actual)
- Primary completion
- Oct 2028 (estimated)
- Study completion
- Oct 2030 (estimated)
- First posted
- 26 Sep 2025 (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.
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).
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
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Efgartigimod IV | Experimental | Biological: Efgartigimod IVParticipants receiving efgartigimod IV during the double-blinded treatment period and the open-label treatment period(s) |
| Placebo IV | Placebo comparator | Biological: Efgartigimod IV, Other: Placebo IVParticipants receiving placebo IV during the double-blinded treatment period and receiving efgartigimod IV during the open-label treatment period(s) |
Who may qualify
- Ages
- 12 to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (6 listed)
- 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
Key exclusion criteria (4 listed)
- Secondary ITP according to the following definition by the International Working Group (IWG): all forms of immune-mediated thrombocytopenia except primary ITP
- Nonimmune thrombocytopenia
- ITP-associated critical or severe bleeding
- History of hereditary thrombocytopenia
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 |
|---|---|---|
| Efgartigimod IVIntravenous infusion of efgartigimod | Biological | Efgartigimod IV, Placebo IV |
| Placebo IVIntravenous infusion of placebo | Other | Placebo IV |
Primary outcome
Efgartigimod serum concentrations in the DBTP
Time frame: Up to 24 weeks
Total IgG levels in the DBTP
Time frame: Up to 24 weeks
11 secondary outcomes
Efgartigimod serum concentrations over time during the DBTP
Time frame: Up to 24 weeks
Percent change from baseline in total IgG levels in serum over time during the DBTP
Time frame: Up to 24 weeks
Incidence of AEs, SAEs and AEs leading to IMP discontinuation
Time frame: Up to 136 weeks
Sustained platelet count response between study weeks 19 and 24 of the DBTP and in OLTP1 for participants receiving placebo in the DBTP
Time frame: Up to 48 weeks
Extent of disease control during the DBTP and during the first 24 weeks of OLTP1 for those participants receiving placebo in the DBTP
Time frame: Up to 48 weeks
Changes from baseline for platelet counts over time
Time frame: Up to 76 weeks
Incidence of bleeding, assessed by the Modified Buchanan and Adix Bleeding Score for pediatric ITP
Time frame: Up to 76 weeks
Incidence of ADA and Nab against efgartigimod in serum
Time frame: Up to 76 weeks
Change from baseline in EQ-5D-5L
Time frame: Up to 76 weeks
Change from baseline in KIT Child Self-Report and KIT Parent Impact Report
Time frame: Up to 76 weeks
Change from baseline in peds FACIT-F
Time frame: Up to 76 weeks
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Oct 2026.
- Study websiteLink provided by the sponsor.
- Individual participant dataSharing planned: No
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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.
26 sites in 10 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing Efgartigimod IV in people with Immune Thrombocytopenia (ITP) and ITP - Immune Thrombocytopenia, run by argenx at 26 sites in 10 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: looking for participants now, as of the sponsor's last update on 16 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 26 sites in Spain, Italy, France, Japan, United Kingdom and 5 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 combined phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Sabine Coppieters, MD (857-350-4834, clinicaltrials@argenx.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Efgartigimod serum concentrations in the DBTP (time frame: Up to 24 weeks). The study lists 2 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 Oct 2026; AzTrials last refreshed its copy on 7 Nov 2026.