Primary Immune Thrombocytopenia MedDRA version: 20.1 Level: LLT Classification code 10074678 Term: Primary immune thrombocytopenic purpura System Organ Class: 100000004851
Conditions
Sponsors
Argenx BVBA
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: Main study 1. Ability to understand the requirements of the study, and comply with the study protocol procedures (including required study visits). 2. Male or female patients aged = 18 to = 85 years. 3. Eligible patients must receive standard-of-care treatment for ITP following the ASH guidelines and International Working Group (IWG) stable in dose and frequency for at least 4 weeks prior to Screening. 4. Confirmed diagnosis of ITP according to the American Society of Hematology Criteria 2011 with (average) blood platelet counts =65 years) yes F.1.3.1 Number of subjects for this age range 14
Exclusion criteria
Exclusion criteria: 1. Use of anticoagulants, or any drug with antiplatelet effect during the study and within 3 weeks prior to Screening 2. Patients who have received any blood support or transfusion within 4 weeks prior to Screening 3. Use of IVIg or anti-D immunoglobulin treatment within 4 weeks prior to Screening 4. Use of recombinant thrombopoietin 5. Use of rituximab within 6 months prior to Screening. other anti-CD20s not permitted 6. Use of corticosteroids not been stable for at least 4 weeks prior to Screening 7. Use of immunosuppressants not permitted within 4 weeks prior to Screening except azathioprine, danazol, mycophenolate mofetil, mycophenolate sodium which must have been stable for at least 4 weeks prior to Screening 8. Use of any other biological therapy or investigational drug than those previously indicated within 3 months or 5 half-lives of the drug (whichever is longer) prior to Screening 9. Received vaccinations within 4 weeks prior to Screening or planned during the study 10. At Screening, have clinically significant laboratory abnormalities 11. History of myeloproliferative or lymphoproliferative disorders at any time; or history of malignancy at any time unless deemed cured 12. History of cerebrovascular accident or myocardial infarction within the last 12 months, before Screening, or current severe/unstable angina, arrhythmia, or at risk of ventricular arrhythmia, symptomatic congestive heart failure 13. History of any thrombotic or embolic event within 12 months prior to Screening 14. History of coagulopathy or hereditary thrombocytopenia or a family history of thrombocytopenia 15. Known history or symptoms of systemic lupus erythematosus, antiphospholipid antibody syndrome or any other clinically documented auto-immune disease other than ITP 16. Prior history or symptoms suggestive of untreated Helicobacter pylori infection 17. History of a recent major surgery 18. Active infection; a recent serious infection within the 8 weeks prior to Screening. 19. Clinical evidence of significant unstable or uncontrolled acute or chronic diseases other than ITP, uncontrolled diabetes 20. History of alcoholism or drug/chemical/substance abuse within the past 2 years prior to Screening per investigator’s opinion 21. Body Mass Index (BMI) at Screening = 35 kg/m2 22. Female patient who is pregnant or lactating or have been lactating within 3 months of Screening Additional Exclusion Criterion for the Extended Follow-up Period 23. At the moment of start of extended FU period, being enrolled in a clinical study with another investigational drug or device Exclusion criteria for the open-label treatment period (first treatment cycle) 1. At the moment of start of the open-label treatment period, being enrolled in a clinical study with another investigational drug or device 2. Please refer to the following exclusion criteria from the main study: 1, 4, 19, 21, and 22 3. Use of IVIg or anti-D immunoglobulin treatment as rescue therapy at any time during the main study or extended FU period 4. Received vaccinations within 4 weeks prior to any (re)treatment cycle 5. At Treatment Evaluation Visit, have clinically significant laboratory abnormalities 6. Patient has a history of severe and/or serious hypersensitivity reaction to IMP in ARGX- 113-1603 main study Exclusion criteria for subsequent open-label retreatment cycles 1. Please refer to the exclusion criteria for the first treatment cycle of the open-label treatment period 2. Patient has a histor
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To evaluate the safety and tolerability of ARGX-113 (short and long term) To evaluate the safety of repeated use of ARGX-113;Secondary Objective: To evaluate the patients with initial response i.e., platelet count = 30 × 10E9/L and/or at least doubling of the Baseline platelet count and absence of bleeding. - To evaluate the clinical effect of ARGX-113 (short term and long term) on: ?- platelet counts; ? - use of rescue treatment; ?- bleeding events. - To evaluate the efficacy of repeated use of ARGX-113. - To assess the effect of ARGX-113 on quality of life. - To assess the PK of ARGX-113. - To assess the PD effects of ARGX-113. - To evaluate the immunogenicity of ARGX-113. Specifically for the extended FU period: To assess the duration of the treatment effect for all patients who did not relapse during the treatment period. This will be based on: - local platelet counts; - use of rescue treatment; - bleeding events. ;Primary end point(s): -Incidence and severity of treatment-emergent AEs (TEAEs) and serious AEs (SAEs). - Changes from Baseline (mean, median, minimum and maximum values, shifts) in vital signs, electrocardiogram parameters (ECGs), physical examination abnormalities, and clinical laboratory assessments.;Timepoint(s) of evaluation of this end point: Various time points throughout the study | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): -Frequency and proportion of patients with initial response i.e., platelet count = 30 × 10E9/L and/or at least doubling of the Baseline count and absence of bleeding at any time during the study. - Mean change in platelet counts compared to Baseline. -Frequency and proportion of patients with following response at any time during the study: o Complete response (CR): Platelet count = 100×10E9/L, confirmed on at least 2 separate consecutive occasions = 7 days apart, and the absence of bleeding; o Response (R): Platelet count = 30 and < 100×10E9/L, and a greater than 2-fold increase in platelet count from Baseline, confirmed on at least 2 separate consecutive occasions = 7 days apart, and the absence of bleeding; o No response (NR): Platelet count < 30 × 10E9/L or less than doubling of the Baseline count or bleeding; -Time to initial response: time from starting treatment to time to reach CR or R; -Duration of response: time from the achievement of CR or R to loss of CR or R; -Frequency and proportion of patients with response to = 50×10E9/L: Platelet count increase to at least = 50×10E9/L at any time during the study. - Frequency and proportion of patients requiring rescue therapy. -General bleeding assessment by the World Health Organization (WHO) bleeding scale and SMOG index of the ITP specific bleeding assessment tool (ITP-BAT).* - Change from Baseline in the Short Form-36 item Health Status Questionnaire (SF-36) and the Functional Assessment of Cancer Therapy (FACT-Th6).* - PK parameters of ARGX-113 including maximum observed concentration (Cmax), time of maximum concentration (tmax), plasma concentration prior to dosing (Ctrough), apparent terminal half-life (t1/2,?z), and accumulation ratio (Rac).* - Evaluation of pharmacodynamic markers*: Total IgG, IgG isotypes** IgG1, IgG2, IgG3, IgG4; and antiplatelet antibody levels. In addition, IgA, IgD, IgE, and IgM will also be assessed.** - Evaluate the incidence of antidrug antibodies (ADA) to | — |
Countries
Austria, Belgium, Czech Republic, France, Germany, Hungary, Poland, Spain, Ukraine, United Kingdom
Contacts
Public ContactRegulatory
Argenx BVBA
Outcome results
None listed