Active — Not RecruitingPhase 3

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

United States, Argentina, Australia173 participantsStarted 2021-11-17

Plain-language summary

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

Who can participate

Age range

18 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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.

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

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

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

Timeframe: 216 weeks

Vital sign measurement: blood pressure in the overall population

Timeframe: 216 weeks

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

Timeframe: 216 weeks

Laboratory safety evaluations: CRP analysis in the overall population

Timeframe: 216 weeks

Trial details

NCT IDNCT04812925

Sponsorargenx

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2026-10-01

View on ClinicalTrials.gov More Primary Immune Thrombocytopenia trials

Who can participate

Age range

18 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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.

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

What they're measuring

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

Timeframe: 216 weeks

Vital sign measurement: blood pressure in the overall population

Timeframe: 216 weeks

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

Timeframe: 216 weeks

Laboratory safety evaluations: CRP analysis in the overall population

Timeframe: 216 weeks