Post-Marketing Study to Evaluate the Efficacy and Safety of PANZYGA in Pediatric Patients With Chronic Immune Thrombocytopenia (ITP)

Octapharma1 site in 1 country6 target enrollmentJanuary 21, 2020

ConditionsChronic Immune Thrombocytopenia

InterventionsPanzyga

DrugsPanzyga

Overview

Phase: Phase 4
Intervention: Panzyga
Conditions: Chronic Immune Thrombocytopenia
Sponsor: Octapharma
Enrollment: 6
Locations: 1
Primary Endpoint: Platelet Count Increase
Status: Terminated
Last Updated: last year

Overview Investigators Eligibility Criteria Arms & Interventions Outcomes Sites Similar Trials

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.

Registry

clinicaltrials.gov

Start Date

January 21, 2020

End Date

May 17, 2024

Last Updated

last year

Study Type

Interventional

Study Design

Single Group

Sex

All

Investigators

Sponsor

Octapharma

Responsible Party

Sponsor

Eligibility Criteria

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

Arms & Interventions

Panzyga

Intervention: Panzyga

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)