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
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
101
Intravenous infusion of efgartigimod
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
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
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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
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