This is a randomized, double-blind placebo-controlled multicenter phase 3 trial to evaluate the efficacy and safety of ARGX-113 in patients with primary ITP.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Intravenous infusion of efgartigimod
Intravenous infusion of placebo
Number of cumulative weeks over the planned 24 week treatment period with platelet counts of ≥50×10^9/L in patients with chronic ITP.
Time frame: Up to 24 weeks
Number of cumulative weeks over the planned 24-week treatment period with platelet counts of ≥50×10^09/L in the overall population (chronic and persistent ITP).
Time frame: Up to 24 weeks
Proportion of patients in the overall population with a sustained platelet count response defined as achieving platelet counts of ≥50×10^9/L for at least 4 of the 6 visits between week 19 and 24 of the trial
Time frame: Up to 6 weeks
Proportion of patients in the overall population achieving platelet counts of ≥50×10^9/L for at least 6 of the 8 visits between week 17 and 24 of the trial
Time frame: Up to 8 weeks
Proportion of patients in the overall population 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 treatment period
Time frame: Up to 24 weeks
Extent of disease control defined as the number of cumulative weeks until week 12, with platelet counts of ≥50×10^9/L in the overall population
Time frame: Up to 12 weeks
Proportion of patients in the overall population with overall platelet count response defined as achieving a platelet count of ≥50×10^9/L on at least 4 occasions at any time until week 12
Time frame: Up to 12 weeks
Mean change from baseline in platelet count at each visit in the overall population.
Time frame: Up to 31 weeks
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
Time to response defined as the time to achieve 2 consecutive platelet counts of ≥50×10^9/L in the overall population
Time frame: Up to 31 weeks
The number of cumulative weeks over the treatment period with platelet counts of ≥30×10^9/L and ≥20×10^9/L above baseline in the overall population
Time frame: Up to 24 weeks
In patients with baseline platelet count of <15×10^9/L, the number of cumulative weeks over the treatment period with platelet counts of ≥30×10^9/L and ≥20×10^9/L above baseline in the overall population
Time frame: Up to 31 weeks
Incidence and severity of the World Health Organization (WHO)-classified bleeding events in the overall population
Time frame: Up to 31 weeks
Rate of receipt of rescue therapy (rescue per patient per month)
Time frame: Up to 31 weeks
Proportion of patients for whom dose and/or frequency of concurrent ITP therapies have increased at week 12 or later
Time frame: Up to 24 weeks
Incidence and severity of adverse events (AEs), AEs of special interest (AESIs)and serious AEs (SAEs).
Time frame: Up to 31 weeks
Vital signs, electrocardiogram (ECG), and laboratory assessments
Time frame: Up to 31 weeks
Change from baseline in PRO (FACIT-Fatigue, Fact-Th6) at planned visits
Time frame: Up to 31 weeks
Change from baseline in QoL (SF-36) at planned visits
Time frame: Up to 31 weeks
Incidence of anti-drug antibodies (ADA) to efgartigimod
Time frame: Up to 31 weeks
Pharmacokinetic parameters of efgartigimod : maximum observed serum concentration (Cmax)
Time frame: Up to 31 weeks
Pharmacokinetic parameters of efgartigimod : serum concentration observed predose (Cthrough)
Time frame: Up to 31 weeks
Pharmacodynamics markers: total immunoglobulin (Ig) G, IgG isotypes (IgG1, IgG2, IgG3, IgG4), antiplatelet antibody levels
Time frame: Up to 31 weeks