Ensayos clínicos

Inclusion Criteria:

• Females and males aged from ≥ 1 year to < 18 years old.
• Confirmed diagnosis of Chronic Immune Thrombocytopenia (ITP) according to American Society of Hematology (ASH) 2011 guidelines.
• Platelets count < 30x10^9/L at the Baseline Visit.
• Voluntarily given written informed consent (provided by patient's parent or legal guardian) and assent (provided by patient [if age-appropriate per IRB (Institutional Review Board) requirements]).
• Females of childbearing potential have been using at least 1 acceptable form of birth control for a minimum of 30 days (or a minimum of 3 months for hormonal contraceptives) prior to the Screening visit, and must agree to use at least 1 acceptable method of contraception for 30 days after the last dose of PANZYGA. Acceptable methods of birth control for this study include: intrauterine device (IUD), hormonal contraception, male or female condom, spermicide gel, diaphragm, sponge, or cervical cap. Abstinence is not considered an acceptable method of birth control.
• Parent or legal guardian must agree and be willing to assist the participant attend study visits, and to follow all protocol requirements and instructions of the study doctor.

Exclusion Criteria: 

• Thrombocytopenia secondary to other diseases (such as Acquired Immunodeficiency Syndrome [AIDS] or systemic lupus erythematosus [SLE]), drug-related thrombocytopenia, or congenital thrombocytopenia.
• Administration of intravenous immunoglobulin (IGIV) or anti-D immunoglobulin within 3 weeks before enrollment.
• Administration of thrombopoietin receptor agonists when the dose has NOT been stable within 3 weeks before enrollment and a dosage change is planned before Day 32.
• Administration of oral immunosuppressants when the dose has NOT been stable during the preceding 2 months (2 weeks for long-term corticosteroid therapy) and a dosage change is planned before Day 32.
  • Note: topical agents and inhaled corticosteroid therapy use is permitted.
• Administration of long-term anti-prolific agents or attenuated androgen therapy when the dose has NOT been stable during the preceding 2 months and a dosage change is planned before Day 32.
• Non-responsive to previous treatment with IGIV or anti-D immunoglobulin.
• Evidence of an active major bleeding episode at Screening.
• Splenectomy in the previous 4 weeks or planned splenectomy throughout the study period.
• Evans syndrome (experiencing active disease with 2 out of 3 of the following: autoimmune thrombocytopenia, autoimmune hemolytic anemia, and/or autoimmune neutropenia).
• Known or suspected human immunodeficiency virus (HIV), hepatitis B virus (HBV), and/or hepatitis C virus (HCV) infections.
• Emergency surgery in the previous 4 weeks.
• Severe liver and/or kidney disease (alanine aminotransferase [ALT] >3 x upper limit of normal (ULN), aspartate aminotransferase [AST] > 3 x upper limit of normal (ULN), and/or creatinine > 120 µmol/L).
• History of severe hypersensitivity to blood or plasma derived products, or any component of the PANZYGA.
• Known immunoglobulin A (IgA) deficiency and antibodies against IgA. History of, or suspected alcohol or drug abuse in the previous year.
• Females who are pregnant or nursing.
• Unable or unwilling to comply with the study protocol.
• Receipt of any other investigational medicinal product within 3 months before study entry.
• Risk factors* for thromboembolic events in whom the risks outweigh the potential benefit of PANZYGA treatment.
• Any other condition(s), that in the Investigator's opinion, make it undesirable for the patient to participate in the study or may interfere with protocol compliance.

*Risk factors include, but are not limited to:

• obesity, advanced age, hypertension, diabetes, a history of atherosclerosis/vascular disease or thrombotic events, hyperlipidemia, multiple cardiovascular risk factors, acquired or inherited thrombophilic disorders, prolonged periods of immobilization, severe hypovolemia, central venous catheterization, active malignancy and/or known or suspected hyperviscosity.