NCT03866798TerminatedPhase 4
Post-Marketing Study to Evaluate the Efficacy and Safety of PANZYGA in Pediatric Patients With Chronic Immune Thrombocytopenia (ITP)
Conditions
Interventions
Drugs
Trial Snapshot
- Phase
- Phase 4
- Status
- Terminated
- Sponsor
- Octapharma
- Enrollment
- 6
- Locations
- 1
- Primary Endpoint
- Platelet Count Increase
Study Overview
Brief Summary
This is a prospective, open-label, single-arm, multicenter, Phase 4 study evaluating the efficacy and safety of PANZYGA in pediatric patients with chronic ITP.
Study Design
- Study Type
- Interventional
- Allocation
- Na
- Intervention Model
- Single Group
- Primary Purpose
- Treatment
- Masking
- None
Eligibility Criteria
- Ages
- 1 Year to 18 Years (Child, Adult)
- Sex
- All
- Accepts Healthy Volunteers
- No
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) 2019 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])
- •Sexually active females who 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 throughout the study and 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. For non-sexually active females who have begun menstruating, abstinence is 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 (+/- 3 days) 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
- •Nonresponsive to previous treatment with IGIV or anti-D immunoglobulin
- •Evidence of an active major bleeding episode at Screening
- •Splenectomy in the previous 3 months 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] >3x upper limit of normal (ULN), aspartate aminotransferase [AST] >3x 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.
Arms & Interventions
Panzyga
Experimental
Panzyga
Intervention: Panzyga (Biological)
Outcomes
Primary Outcomes
Platelet Count Increase
Time Frame: 8 Days
Number of subjects with an increase in platelet count at least once to ≥50 × 10\^9/L within 7 days after the first infusion, i.e., by Day 8 (increase must have occurred at least once on any day up to and including Day 8).
Secondary Outcomes
- Days to Reach Platelet Count of at Least 50x10^9/L(Through study completion, up to 37 days)
- Duration of Platelet Response(Through study completion, up to 37 days)
- Maximum Platelet Count(Through study completion, up to 37 days)
Investigators
Study Sites (1)
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