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

argenx101 enrolled

Overview

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.

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

101

Conditions

Primary Immune Thrombocytopenia

Interventions

efgartigimodBIOLOGICAL

Intravenous infusion of efgartigimod

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

Inclusion criteria: 1. 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). 2. Patients enrolled in the ARGX-113-1801 trial who completed the 24-weeks trial period. 3. Women of childbearing potential must have a negative urine pregnancy test at baseline before trial medication (infusion) can be administered. 4. 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) 6\. 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). 7\. Patient has completed a 52-week treatment period. Exclusion criteria: 1. 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). 2. Pregnant or lactating women, and those intending to become pregnant during the trial or within 90 days after the last dosing. 3. Patients with known medical history of hypersensitivity to any of the ingredients of efgartigimod. 4. Use of any other investigational drug or participation in any other investigational trial.

Locations (87)

Investigator Site 0010045

Washington D.C., District of Columbia, United States

Investigator Site 0010037

Ocala, Florida, United States

Investigator Site 0010042

Iowa City, Iowa, United States

Investigator Site 0010040

Columbus, Ohio, United States

Investigator Site 0430002

Vienna, Austria

Investigator Site 0430003

Vienna, Austria

Investigator Site 0320012

Brasschaat, Belgium

Investigator Site 0320011

Bruges, Belgium

Investigator Site 0320014

Turnhout, Belgium

Investigator Site 0320002

Yvoir, Belgium

...and 77 more locations

Outcomes

Primary Outcomes

Frequency and severity of Adverse Events

Time frame: Up to 60 weeks

Frequency and severity of vital signs

Time frame: Up to 60 weeks

Frequency and severity of laboratory assessments

Time frame: Up to 60 weeks

Secondary Outcomes

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

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

Mean change from baseline in platelet count at each visit.

Time frame: Up to 60 weeks, at each visit

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

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

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

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

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

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

Time frame: Up to 60 weeks, at each visit

Reduction in concurrent ITP therapy.

Time frame: Up to 60 weeks, at each visit

Incidence and severity of the WHO-classified bleeding events.

Time frame: Up to 60 weeks, at each visit

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

Time frame: Up to 52 weeks

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

Time frame: Up to 52 weeks

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

Time frame: Up to 52 weeks

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

Time frame: Up to 216 weeks

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

Time frame: Up to 60 weeks

Pharmacodynamics markers: total IgG.

Time frame: Up to 60 weeks

Data from ClinicalTrials.gov

This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.

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

Phase 3CompletedNCT04225156

argenx101 enrolled

Overview

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.

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

101

Conditions

Primary Immune Thrombocytopenia

Interventions

efgartigimodBIOLOGICAL

Intravenous infusion of efgartigimod

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

Locations (87)

Investigator Site 0010045

Washington D.C., District of Columbia, United States

Investigator Site 0010037

Ocala, Florida, United States

Investigator Site 0010042

Iowa City, Iowa, United States

Investigator Site 0010040

Columbus, Ohio, United States

Investigator Site 0430002

Vienna, Austria

Investigator Site 0430003

Vienna, Austria

Investigator Site 0320012

Brasschaat, Belgium

Investigator Site 0320011

Bruges, Belgium

Investigator Site 0320014

Turnhout, Belgium

Investigator Site 0320002

Yvoir, Belgium

...and 77 more locations

Outcomes

Primary Outcomes

Frequency and severity of Adverse Events

Time frame: Up to 60 weeks

Frequency and severity of vital signs

Time frame: Up to 60 weeks

Frequency and severity of laboratory assessments

Time frame: Up to 60 weeks

Secondary Outcomes

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

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

Mean change from baseline in platelet count at each visit.

Time frame: Up to 60 weeks, at each visit

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

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

Time frame: Up to 5 weeks, between visit 19 and 24 of the trial

Time frame: Up to 7 weeks, between visit 17 and 24 of the trial

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

Time frame: Up to 60 weeks, at each visit

Reduction in concurrent ITP therapy.

Time frame: Up to 60 weeks, at each visit

Incidence and severity of the WHO-classified bleeding events.

Time frame: Up to 60 weeks, at each visit

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

Time frame: Up to 52 weeks

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

Time frame: Up to 52 weeks

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

Time frame: Up to 52 weeks

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

Time frame: Up to 216 weeks

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

Time frame: Up to 60 weeks

Pharmacodynamics markers: total IgG.

Time frame: Up to 60 weeks

Data from ClinicalTrials.gov

This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.