Status:
TERMINATED
Study to Evaluate the Efficacy and Safety of PANZYGA in Pediatric Patients With Chronic Immune Thrombocytopenia (ITP)
Lead Sponsor:
Octapharma
Conditions:
Chronic Immune Thrombocytopenia
Eligibility:
All Genders
1-18 years
Phase:
PHASE4
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.
Eligibility Criteria
Inclusion
- 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
- 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.
Key Trial Info
Start Date :
January 21 2020
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
May 17 2024
Estimated Enrollment :
6 Patients enrolled
Trial Details
Trial ID
NCT03866798
Start Date
January 21 2020
End Date
May 17 2024
Last Update
September 24 2024
Active Locations (8)
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1
Octapharma Research Site
Sacramento, California, United States, 95817
2
Octapharma Research Site
Minneapolis, Minnesota, United States, 55404
3
Octapharma Research Site
Rochester, Minnesota, United States, 55905
4
Octapharma Research Site
Columbus, Ohio, United States, 43205