Skip to main content
OpenTrials
Active, Not Recruiting

NCT Number: NCT04812925

A Phase 3 Study to Evaluate the Safety and Efficacy of Efgartigimod PH20 Subcutaneous in Adult Patients With Primary Immune Thrombocytopenia

A Phase 3 study to evaluate the safety and efficacy of efgartigimod PH20 subcutaneous in adult patients with primary immune thrombocytopenia

Active, Not Recruiting

This study is active but is not currently recruiting participants.

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 0540001, Buenos Aires, Argentina

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 and provide written informed consent (including consent for the use and disclosure of research-related health information), willing and able to comply with the trial protocol procedures (including attending the required trial visits).
  • Participants enrolled in the ARGX-113-2004 trial who completed the 24-week trial period.

Note: If a participant has had an SAE during the ARGX-113-2004 trial, their eligibility should be evaluated by the investigator and the sponsor's trial physician. The decision of enrolling the participant will be evaluated case by case.

3a. Agree to use contraceptives consistent with local regulations and the following:

  • Female participants of childbearing potential must have a negative urine pregnancy test at baseline before receiving IMP.

In addition to the above criteria, for participants who want to continue receiving efgartigimod during an additional 52-week treatment period (only applicable in case efgartigimod is not yet commercially available for patients with primary ITP or available through another patient program for patients with primary ITP), the following criteria apply:

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

Exclusion criteria

  • Introduction or continuation of nonpermitted medications during the ARGX-113-2004 trial (such as anti-CD20 therapy, romiplostim, monoclonal antibodies, Fc fusion proteins, or live/live-attenuated vaccines)
  • Use of any other investigational drug or participation in any other investigational trial
  • Known hypersensitivity reaction to efgartigimod PH20 SC or any of its excipients
  • Pregnant or lactating females and those who intend to become pregnant during the trial or within 90 days after last dose of efgartigimod PH20 SC

Treatment and study plan

efgartigimod PH20 SC

Biological

Subcutaneous injection with efgartigimod PH20 SC

Other names: ARGX-113 PH20 SC

Primary outcomes

  1. Incidence, frequency, and severity of adverse events (AEs), AEs of special interest (AESIs), and serious AEs (SAEs)

    Time frame: 216 weeks

  2. Vital sign measurement: blood pressure in the overall population

    Time frame: 216 weeks

  3. ECG: PR, QT and QRS interval in the overall population

    Time frame: 216 weeks

  4. Laboratory safety evaluations: CRP analysis in the overall population

    Time frame: 216 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: 52 weeks

  2. Proportion of patients with overall platelet count response defined as achieving a platelet count of ≥50×10E9/L on at least 4 occasions at any time during the 52-week treatment period

    Time frame: 52 weeks

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

    Time frame: 52 weeks

  4. For patients rolling over from the ARGX-113-2004 trial with a platelet count of <30×10E9/L: time to response defined as the time to achieve 2 consecutive platelet counts of ≥50×10E9/L

    Time frame: 52 weeks

  5. The percentage of weeks in the trial with platelet counts of ≥30×10E9/L and ≥20×10E9/L above baseline

    Time frame: 52 weeks

  6. In patients with a baseline platelet count of <15×10E9/L in the current trial (ARGX-113-2005), the percentage of weeks in the trial with platelet counts of ≥30×10E9/L and ≥20×10E9/L above baseline

    Time frame: 52 weeks

  7. In patients with the first exposure to efgartigimod PH20 SC, the proportion of patients who achieve a sustained platelet response defined as achieving platelet counts of ≥50×10E9/L for at least 4 of the 6 visits between week 19 and week 24

    Time frame: 5 weeks (week 19-24)

  8. In patients with the first exposure to efgartigimod PH20 SC, the proportion of patients achieving platelet counts of ≥50×10E9/L for at least 6 of the 8 visits between week 17 and week 24

    Time frame: 7 weeks (week 17-24)

  9. Proportion of patients for whom dose and/or frequency of concurrent ITP therapies have been reduced compared to baseline

    Time frame: 52 weeks

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

    Time frame: 52 weeks

  11. Incidence of the World Health Organization (WHO)-classified bleeding events

    Time frame: 52 weeks

  12. Severity of the World Health Organization (WHO)-classified bleeding events

    Time frame: 52 weeks

  13. Serum efgartigimod concentration observed predose (Ctrough)

    Time frame: 52 weeks

  14. Change from baseline in PRO (Functional Assessment of Chronic Illness Therapy Fatigue Scale [FACIT-fatigue]) at planned visits

    Time frame: 52 weeks

  15. Change from baseline in PRO (Functional Assessment of Cancer Therapy questionnaire-Th6 [FACT-Th6]) at planned visits

    Time frame: 52 weeks

  16. Change from baseline in PRO (QoL (Short Form-36 [SF-36]) at planned visits

    Time frame: 52 weeks

  17. Pharmacodynamics markers: total IgG

    Time frame: 52 weeks

  18. Number of patients who performed self-administration at home over time

    Time frame: 52 weeks

  19. Percentage of patients who performed self-administration at home over time

    Time frame: 52 weeks

  20. Number of caregivers who administered the injection to the patient at home over time

    Time frame: 52 weeks

  21. Percentage of caregivers who administered the injection to the patient at home over time

    Time frame: 52 weeks

  22. Number of training visits needed for the participant or caregiver to be competent to start administering efgartigimod PH20 SC

    Time frame: 52 weeks

  23. Number of self- or caregiver-supported administrations at home

    Time frame: 52 weeks

  24. Percentage of self- or caregiver-supported administrations at home

    Time frame: 52 weeks

  25. Incidence and prevalence of antibodies to efgartigimod

    Time frame: 216 weeks

  26. Titers of antibodies to efgartigimod

    Time frame: 216 weeks

  27. Presence of neutralizing antibodies (NAb) against efgartigimod

    Time frame: 216 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) PH20 Subcutaneous in Adult Patients With Primary Immune Thrombocytopenia

Acronym: ADVANCE SC+

Important dates

Study start
2021
Primary completion
2026
Study completion
2026
First posted
Mar 24, 2021
Registry last updated
Jan 12, 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.