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NCT Number: NCT07194850

A Study of Efgartigimod IV in Participants From 12 Years to Less Than 18 Years of Age With Chronic Immune Thrombocytopenia (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

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Key information

Conditions

Immune Thrombocytopenia (ITP) Autoimmune Diseases Blood Coagulation Disorders Blood Platelet Disorders Cytopenia Hematologic Diseases Hemic and Lymphatic Diseases Hemorrhage Hemorrhagic Disorders ITP ITP - Immune Thrombocytopenia Idiopathic Thrombocytopenic Purpura Idiopathic Thrombocytopenic Purpura (ITP) Immune System Diseases Immune Thrombocytopenic Purpura Immune Thrombocytopenic Purpura ( ITP ) Pathologic Processes Pathological Conditions, Signs and Symptoms Purpura Purpura, Thrombocytopenic Purpura, Thrombocytopenic, Idiopathic Signs and Symptoms Skin Manifestations Thrombocytopenia Thrombotic Microangiopathies

Age range

12 year–17 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2 / Phase 3

Primary location

Centre Hospitalier Universitaire de Bordeaux Groupe Hospitalier Pellegrin Hopital des Enfants, Bordeaux, France

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • 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

Exclusion criteria

  • 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

Treatment and study plan

efgartigimod IV

Biological

Intravenous infusion of efgartigimod

Placebo IV

Other

Intravenous infusion of placebo

Primary outcomes

  1. Efgartigimod serum concentrations in the DBTP

    Time frame: Up to 24 weeks

  2. Total IgG levels in the DBTP

    Time frame: Up to 24 weeks

Secondary outcomes

  1. Efgartigimod serum concentrations over time during the DBTP

    Time frame: Up to 24 weeks

  2. Percent change from baseline in total IgG levels in serum over time during the DBTP

    Time frame: Up to 24 weeks

  3. Incidence of AEs, SAEs and AEs leading to IMP discontinuation

    Time frame: Up to 136 weeks

    SAE: Serious adverse event; AE: adverse event

  4. 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

    Sustained platelet count defined as achieving platelet counts of ≥50 × 10^9/L for at least 4 of the 6 study visits

  5. 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

    Extend of disease defined as the number of cumulative weeks with a platelet count of ≥50 × 10^9/L

  6. Changes from baseline for platelet counts over time

    Time frame: Up to 76 weeks

  7. Incidence of bleeding, assessed by the Modified Buchanan and Adix Bleeding Score for pediatric ITP

    Time frame: Up to 76 weeks

    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).

  8. Incidence of ADA and Nab against efgartigimod in serum

    Time frame: Up to 76 weeks

    ADA: anti-drug antibodies; Nab: neutralizing antibodies

  9. Change from baseline in EQ-5D-5L

    Time frame: Up to 76 weeks

    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).

  10. Change from baseline in KIT Child Self-Report and KIT Parent Impact Report

    Time frame: Up to 76 weeks

    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).

  11. Change from baseline in peds FACIT-F

    Time frame: Up to 76 weeks

    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.

Study contacts

Contact information is provided by the study sponsor or research team.

Sabine Coppieters, MD

CONTACT

[email protected]

857-350-4834

Sponsors and collaborators

Lead sponsor

argenx

Industry

Registry information

Official study 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

Acronym: Advance Jr

Important dates

Study start
2025
Primary completion
2028
Study completion
2030
First posted
Sep 26, 2025
Registry last updated
Jul 9, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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