Study to Evaluate the Safety and Efficacy of Efgartigimod (ARGX-113) PH20 Subcutaneous in Adult Patients With Primary Immune Thrombocytopenia

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 - ADVANCE SC+

Status

Active, not recruiting

Phases

Phase 3

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2020-004033-20-PT

Enrollment

156

Registered

2021-05-20

Start date

2021-09-03

Completion date

Unknown

Last updated

2024-07-22

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Primary immune thrombocytopenia (ITP) MedDRA version: 23.0 Level: LLT Classification code 10083843 Term: Primary immune thrombocytopenia System Organ Class: 10005329 - Blood and lymphatic system disorders

Interventions

Product Name: efgartigimod PH20 SC Pharmaceutical Form: Solution for injection INN or Proposed INN: efgartigimod alfa CAS Number: 1821402-21-4 Current Sponsor code: ARGX-113 Concentration unit: mg/ml

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: Participants are eligible to be included in the trial only if all of the following criteria apply: 1. 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). 2. 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. The decision of enrolling the participant will be evaluated case by case. 3a. Agree to use contraceptives measures consistent with local regulations and the following: Female participants of childbearing potential must have a negative urine pregnancy test at baseline before receiving IMP. 4. 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). 6. Participant has completed a 52-week treatment period. Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 136 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 20

Exclusion criteria

Exclusion criteria: Participants are eligible to be included in the trial only if none of the following criteria apply: 1. 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) 2. Use of any other investigational drug or participation in any other investigational trial 3. Known hypersensitivity reaction to efgartigimod PH20 SC or any of its excipients 4. 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

Design outcomes

Secondary

Measure	Time frame
Secondary end point(s): First 52-week treatment period only 1. Extent of disease control defined as the percentage of weeks in the trial with platelet counts of =50×10^9/L 2. Proportion of participants 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 3. Mean change from baseline in platelet count at each visit 4. For participants rolling over from the ARGX-113-2004 trial with a platelet count of <30×10^9/L: time to response defined as the time to achieve 2 consecutive platelet counts of =50×10^9/L 5. The percentage of weeks in the trial with platelet counts of =30×10^9/L and =20×10^9/L above baseline 6. In patients with a baseline platelet count of <15×10^9/L in the current trial (ARGX-113-2005), the percentage of weeks in the trial with platelet counts of =30×10^9/L and =20×10^9/L above baseline 7. In participants with the first exposure to efgartigimod PH20 SC, the proportion of patients who achieve a sustained platelet response defined as achieving platelet counts of =50×10^9/L for at least 4 of the 6 visits between week 19 and week 24 8. In participants with the first exposure to efgartigimod PH20 SC, the proportion of participants achieving platelet counts of =50×10^9/L for at least 6 of the 8 visits between week 17 and week 24 9. Proportion of participants for whom dose and/or frequency of concurrent ITP therapies have been reduced compared to baseline 10. Rate of receipt of rescue therapy (rescue per participant per month) 11. Incidence and severity of the World Health Organization (WHO)-classified bleeding events 12. Serum efgartigimod concentration observed predose (Ctrough) 13. Change from baseline in PRO (Functional Assessment of Chronic Illness Therapy Fatigue Scale [FACIT-fatigue], Functional Assessment of Cancer Therapy questionnaire-Th6 [FACT-Th6]), QoL (Short Form-36 [SF-36]), and ITP-Patient Assessment Questionnaire [ITP-PAQ] at planned vi	—

Measure

Time frame

Secondary end point(s): First 52-week treatment period only 1. Extent of disease control defined as the percentage of weeks in the trial with platelet counts of =50×10^9/L 2. Proportion of participants 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 3. Mean change from baseline in platelet count at each visit 4. For participants rolling over from the ARGX-113-2004 trial with a platelet count of <30×10^9/L: time to response defined as the time to achieve 2 consecutive platelet counts of =50×10^9/L 5. The percentage of weeks in the trial with platelet counts of =30×10^9/L and =20×10^9/L above baseline 6. In patients with a baseline platelet count of <15×10^9/L in the current trial (ARGX-113-2005), the percentage of weeks in the trial with platelet counts of =30×10^9/L and =20×10^9/L above baseline 7. In participants with the first exposure to efgartigimod PH20 SC, the proportion of patients who achieve a sustained platelet response defined as achieving platelet counts of =50×10^9/L for at least 4 of the 6 visits between week 19 and week 24 8. In participants with the first exposure to efgartigimod PH20 SC, the proportion of participants achieving platelet counts of =50×10^9/L for at least 6 of the 8 visits between week 17 and week 24 9. Proportion of participants for whom dose and/or frequency of concurrent ITP therapies have been reduced compared to baseline 10. Rate of receipt of rescue therapy (rescue per participant per month) 11. Incidence and severity of the World Health Organization (WHO)-classified bleeding events 12. Serum efgartigimod concentration observed predose (Ctrough) 13. Change from baseline in PRO (Functional Assessment of Chronic Illness Therapy Fatigue Scale [FACIT-fatigue], Functional Assessment of Cancer Therapy questionnaire-Th6 [FACT-Th6]), QoL (Short Form-36 [SF-36]), and ITP-Patient Assessment Questionnaire [ITP-PAQ] at planned vi

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Primary

Measure	Time frame
Main Objective: To evaluate the safety and tolerability of efgartigimod PH20 SC in adult patients with primary ITP;Secondary Objective: First 52-week treatment period only • To evaluate the efficacy of efgartigimod PH20 SC on overall platelet count response • To evaluate the use of rescue treatment and reduction in concurrent ITP therapy while receiving treatment with efgartigimod PH20 SC • To evaluate the incidence and severity of bleeding events while receiving treatment with efgartigimod PH20 SC • To assess the PK of efgartigimod PH20 SC • To evaluate the effects of efgartigimod PH20 SC treatment on quality-of-life (QoL) measures and patient-reported outcomes (PRO) • To assess the PD effects of efgartigimod PH20 SC • To evaluate the feasibility of self-administration of efgartigimod PH20 SC First 52-week treatment period and additional 52-week treatment periods • To assess the immunogenicity of efgartigimod;Primary end point(s): 1. Incidence, frequency, and severity of adverse events (AEs), AEs of special interest (AESIs), and serious AEs (SAEs) 2. Vital signs, electrocardiogram (ECG), and laboratory safety evaluations;Timepoint(s) of evaluation of this end point: 1. At all visits 2. At planned visits per schedule of assessment	—

Measure

Time frame

Main Objective: To evaluate the safety and tolerability of efgartigimod PH20 SC in adult patients with primary ITP;Secondary Objective: First 52-week treatment period only • To evaluate the efficacy of efgartigimod PH20 SC on overall platelet count response • To evaluate the use of rescue treatment and reduction in concurrent ITP therapy while receiving treatment with efgartigimod PH20 SC • To evaluate the incidence and severity of bleeding events while receiving treatment with efgartigimod PH20 SC • To assess the PK of efgartigimod PH20 SC • To evaluate the effects of efgartigimod PH20 SC treatment on quality-of-life (QoL) measures and patient-reported outcomes (PRO) • To assess the PD effects of efgartigimod PH20 SC • To evaluate the feasibility of self-administration of efgartigimod PH20 SC First 52-week treatment period and additional 52-week treatment periods • To assess the immunogenicity of efgartigimod;Primary end point(s): 1. Incidence, frequency, and severity of adverse events (AEs), AEs of special interest (AESIs), and serious AEs (SAEs) 2. Vital signs, electrocardiogram (ECG), and laboratory safety evaluations;Timepoint(s) of evaluation of this end point: 1. At all visits 2. At planned visits per schedule of assessment

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Countries

Argentina, Australia, Bulgaria, Chile, China, Colombia, Denmark, France, Georgia, Germany, Greece, Ireland, Israel, Italy, Japan, Jordan, Korea, Republic of, Latvia, Mexico, New Zealand, Norway, Peru, Poland, Portugal, Romania, Russian Federation, Serbia, South Africa, Spain, Taiwan, Thailand, Tunisia, Türkiye, United Kingdom, United States

Contacts

Public ContactRegulatory

argenx BV

regulatory@argenx.com+32 9 310 3400

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026