Primary Immune Thrombocytopenia MedDRA version: 20.1 Level: LLT Classification code 10074678 Term: Primary immune thrombocytopenic purpura System Organ Class: 100000004851
Conditions
Interventions
Product Name: ARGX-113
Product Code: ARGX-113
Pharmaceutical Form: Concentrate for solution for infusion
INN or Proposed INN: N/A
CAS Nu
Sponsors
Argenx BVBA
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: Main study 1. Ability to understand the requirements of the study, and comply withthe 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 < 30 × 10E9/L and who have not experienced major bleeding in the last 4 weeks prior to Screening. 5. Women of childbearing potential must have a negative serum pregnancy test at Screening and a negative urine pregnancy test at Baseline prior to administration of IMP. 6. Female participants of childbearing potential must agree to use a highly effective method of birth control (i.e., pregnancy rate of less than 1% per year) during the study and for 90 days after the discontinuation of IMP. 7. Non-sterilized male patients who are sexually active with a female partner of childbearing potential must use effective double contraception Additional Inclusion Criteria for the Extended Follow-up Period - Sign the amended ICF of the main study including its extended followup period - Completed Visit 16 of the FU period of the protocol with a platelet count = 30 × 10E9/L and/or at least doubling of the Baseline platelet count and absence of bleeding and remained on the same SoC Eligibility criteria for the open-label treatment period (first treatment cycle) 1. Please refer to inclusion criteria 5, 6 and 7 from the main study. 2. Provide written informed consent 3. Received at least 3 doses of the IMP and had at least 2 weeks of follow-up in the main study. 4. Patient is at the same SoC as in the main study. Dose and/or frequency increase is allowed, changing or stopping the SoC is not allowed. 5. During up to 21 weeks of FU, the patient is relapsing i.e. platelet count decreases to below 30 x 10E9/L or the patient's platelets never went up to 30 x 10E9/L and are still below 30 x 10E9/L, and absence of bleeding. Eligibility criteria for subsequent open-label retreatment cycles The patient has the right to receive more than 1 retreatment cycle if: 1. Patient reached a platelet count of at least twice the platelet count measured on the day of the first IMP administration during the previous (re)treatment cycle, confirmed on at least 2 separate consecutive occasions (at least 1 day apart but with maximum 7 days in between the 2 measurements), and measured during the treatment period up to minimum 4 weeks of follow-up. 2. Patient
Exclusion criteria
Exclusion criteria: 1. Use of anticoagulants, or any drug with antiplatelet effect (e.g., acetylsalicylic acid [aspirin] or other salicylate containing medications, cyclooxygenase inhibitors, adenosine diphosphate (ADP) receptor inhibitors, phosphodiesterase inhibitors, glycoprotein IIB/IIIA inhibitors, adenosine reuptake inhibitors, thromboxane inhibitors, antimalarials, and prostacyclins) 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 at any time. 5. Use of rituximab within 6 months prior to Screening. Use of any anti-CD20 other than rituximab at any time is not permitted. 6. Use of corticosteroids which has not been stable for at least 4 weeks prior to Screening. 7. Use of immunosuppressants is not permitted within 4 weeks prior to Screening, with the exception of the following oral immunosuppressants: azathioprine [up to 2.5 mg/kg/day], danazol [up to 15 mg/kg/day], mycophenolate mofetil [up to 3 g/day], mycophenolate sodium [up to 2160 mg/day]) 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 given as below: a. Aspartate aminotransferase (AST) or ALT > 3 × upper limit of normal (ULN). b. Total serum bilirubin of > 1.5 x ULN (except for Grade 1 hyperbilirubinemia as defined by the National Cancer Institute [NCI] Common Terminology Criteria for Adverse Events [CTCAE], due solely to a documented medical diagnosis of Gilbert’s syndrome). c. Serum creatinine of > 1.5 mg/dL or creatinine clearance 1.5 or activated partial thromboplastin time > 1.5 × ULN. g. Total immunoglobulin G (IgG) level 1+ proteinuria Note: A patient with isolated laboratory values that meet the thresholds listed above may be considered eligible. The agreed upon medical rationale, discussed between the investigator and sponsor, must be documented in the patient's chart prior to patient enrolment in the study 11. History of myeloproliferative or lymphoproliferative disorders at any time; or have a history of malignancy at any time unless deemed cured by adequate treatment with no evidence of recurrence for = 5 years prior to Screening. Patients with completely excised nonmelanoma skin cancers or cervical carcinoma in situ would be permitted at any time. 12. History of cerebrovascular accident or myocardial infarction within the last 12 months, before Screening, or current severe/unstable angina, ar
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 × 109/L and/or at least doubling of the Baseline count and absence of bleeding. - To evaluate the clinical effect of ARGX-113 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.To assess the PK of ARGX-113. To assess the PD effects of ARGX-113. To evaluate the immunogenicity of ARGX-113. ; Primary end point(s): -Incidence and severity of 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 × 109/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 5,1: o Complete response (CR): Platelet count = 100×109/L, confirmed on at least 2 separate consecutive occasions = 7 days apart, and the absence of bleeding; o Response (R): Platelet count = 30 and < 100×109/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 × 109/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 | — |
Countries
Austria, Belgium, Czech Republic, France, Germany, Hungary, Poland, Spain, Ukraine, United Kingdom
Contacts
Public ContactRegulatory
Argenx BVBA
Outcome results
None listed