Primary immune thrombocytopenia (ITP) MedDRA version: 23.0 Level: LLT Classification code 10083843 Term: Primary immune thrombocytopenia System Organ Class: 100000004851
Conditions
Sponsors
argenx BV
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: Participants are eligible to be included in the trial only if all of the following criteria apply: 1. Ability to understand the requirements of the trial and provide written informed consent (including consent for the use and disclosure of research-related health information), willing and able to comply with the trial protocol procedures (including attending the required trial visits). 2. Participants enrolled in the ARGX-113-2004 trial who completed the 24-week trial period. Note: If a participant has had an SAE during the ARGX-113-2004 trial, their eligibility should be evaluated by the investigator and the sponsor’s trial physician. The decision of enrolling the participant will be evaluated case by case. 3a. Agree to use contraceptives consistent with local regulations regarding the methods of contraception for those participating in clinical trials and the following: Females participants of childbearing potential must have a negative urine pregnancy test at baseline before receiving IMP. Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 136 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 20
Exclusion criteria
Exclusion criteria: Participants are eligible to be included in the trial only if none of the following criteria apply: 1. Introduction or continuation of nonpermitted medications during the ARGX-113-2004 trial (such as anti-CD20 therapy, romiplostim, monoclonal antibodies, Fc fusion proteins, or live/live-attenuated vaccines) 2. Use of any other investigational drug or participation in any other investigational trial 3. Known hypersensitivity reaction to efgartigimod PH20 SC or any of its excipients 4. Pregnant or lactating females and those who intend to become pregnant during the trial or within 90 days after last dose of efgartigimod PH20 SC
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To evaluate the safety and tolerability of efgartigimod PH20 SC in adult patients with primary ITP;Secondary Objective: • To evaluate the efficacy of efgartigimod PH20 SC on overall platelet count response • To evaluate the use of rescue treatment and reduction in concurrent ITP therapy while receiving treatment with efgartigimod PH20 SC • To evaluate the incidence and severity of bleeding events while receiving treatment with efgartigimod PH20 SC • To assess the PK of efgartigimod PH20 SC • To evaluate the effects of efgartigimod PH20 SC treatment on quality-of-life (QoL) measures and patient-reported outcomes (PRO) • To assess the PD effects of efgartigimod PH20 SC • To evaluate the feasibility of self-administration of efgartigimod PH20 SC • To assess the immunogenicity of efgartigimod ;Primary end point(s): 1. Incidence, frequency, and severity of adverse events (AEs), AEs of special interest (AESIs), and serious AEs (SAEs) 2. Vital signs, electrocardiogram (ECG), and laboratory safety evaluations;Timepoint(s) of evaluation of this end point: 1. At all visits 2. At planned visits per schedule of assessment | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): 1. Extent of disease control defined as the percentage of weeks in the trial with platelet counts of =50×10^9/L 2. Proportion 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 3. Mean change from baseline in platelet count at each visit 4. For patients rolling over from the ARGX-113-2004 trial with a platelet count of <30×10^9/L: time to response defined as the time to achieve 2 consecutive platelet counts of =50×10^9/L 5. The percentage of weeks in the trial with platelet counts of =30×10^9/L and =20×10^9/L above baseline 6. In patients with a baseline platelet count of <15×10^9/L in the current trial (ARGX-113-2005), the percentage of weeks in the trial with platelet counts of =30×10^9/L and =20×10^9/L above baseline 7. In patients with the first exposure to efgartigimod PH20 SC, the proportion of patients who achieve a sustained platelet response defined as achieving platelet counts of =50×10^9/L for at least 4 of the 6 visits between week 19 and week 24 8. In patients with the first exposure to efgartigimod PH20 SC, the proportion of patients achieving platelet counts of =50×10^9/L for at least 6 of the 8 visits between week 17 and week 24 9. Proportion of patients for whom dose and/or frequency of concurrent ITP therapies have been reduced compared to baseline 10. Rate of receipt of rescue therapy (rescue per patient per month) 11. Incidence and severity of the World Health Organization (WHO)-classified bleeding events 12. Serum efgartigimod concentration observed predose (Ctrough) 13. Change from baseline in PRO (Functional Assessment of Chronic Illness Therapy Fatigue Scale [FACIT-fatigue], Functional Assessment of Cancer Therapy questionnaire-Th6 [FACT-Th6]) and QoL (Short Form-36 [SF-36]) and ITP- Patient Assessment Questionnaire (ITP- PAQ) at planned visits 14. Pharmacodynamics markers: total IgG 15. Number and | — |
Countries
Argentina, Australia, Bulgaria, Chile, China, Colombia, Denmark, France, Georgia, Germany, Greece, Ireland, Israel, Italy, Japan, Jordan, Korea, Republic of, Latvia, Mexico, New Zealand, Norway, Peru, Poland, Portugal, Romania, Russian Federation, Serbia, South Africa, Spain, Taiwan, Thailand, Tunisia, Turkey, United Kingdom, United States
Contacts
Public ContactRegulatory
argenx BV
Outcome results
None listed