Skip to main content
OpenTrials
Completed

NCT Number: NCT04225156

A Long-term Study to Assess the Safety and Efficacy of Efgartigimod in Adult Patients With Primary Immune Thrombocytopenia (ITP).

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.

Completed

Looking for future studies?

Notify Me

Key information

Conditions

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

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

Investigator Site 0430002, Vienna, Austria

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

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

Inclusion criteria

  • 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)
  • 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).
  • Patient has completed a 52-week treatment period.

Exclusion criteria

  • 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.

Treatment and study plan

Efgartigimod

Biological

Intravenous infusion of efgartigimod

Other names: ARGX-113

Primary outcomes

  1. Frequency and severity of Adverse Events

    Time frame: Up to 60 weeks

  2. Frequency and severity of vital signs

    Time frame: Up to 60 weeks

  3. Frequency and severity of laboratory assessments

    Time frame: Up to 60 weeks

Secondary outcomes

  1. 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

  2. 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

  3. Mean change from baseline in platelet count at each visit.

    Time frame: Up to 60 weeks, at each visit

  4. 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

  5. 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

  6. 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

  7. 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

  8. 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

  9. Rate of receipt of rescue therapy (rescue per patient per month).

    Time frame: Up to 60 weeks, at each visit

  10. Reduction in concurrent ITP therapy.

    Time frame: Up to 60 weeks, at each visit

  11. Incidence and severity of the WHO-classified bleeding events.

    Time frame: Up to 60 weeks, at each visit

  12. Change from baseline in Patient reported Outcomes (FACIT-Fatigue) at planned visits.

    Time frame: Up to 52 weeks

  13. Change from baseline in Patient reported Outcomes (Fact-Th6) at planned visits.

    Time frame: Up to 52 weeks

  14. Change from baseline in Quality of Life (SF-36) at planned visits.

    Time frame: Up to 52 weeks

  15. Incidence of anti-drug antibodies (ADA) to efgartigimod.

    Time frame: Up to 216 weeks

  16. Pharmacokinetic parameter of efgartigimod: serum concentration observed predose (Ctrough).

    Time frame: Up to 60 weeks

  17. Pharmacodynamics markers: total IgG.

    Time frame: Up to 60 weeks

Sponsors and collaborators

Lead sponsor

argenx

Industry

Registry information

Official study 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.

Acronym: ADVANCE+

Important dates

Study start
2020
Primary completion
2026
Study completion
2026
First posted
Jan 13, 2020
Registry last updated
Mar 16, 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.

Related clinical trials

Published trials that share one or more normalized conditions with this study.