Study to Evaluate the Efficacy and Safety of PANZYGA in Pediatric Patients With Chronic Immune Thrombocytopenia (ITP)
Study Details
Study Description
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.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
|
Phase 4 |
Study Design
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| Experimental: Panzyga Panzyga |
Biological: Panzyga
Immune Globulin, intravenous, human-ifas
|
Outcome Measures
Primary Outcome Measures
- Increasing the platelet count in pediatric patients with chronic ITP [8 Days]
Secondary Outcome Measures
- Time to Reach Platelet Count of at least 50x10^9/L [32 Days]
defined as the number of days for subjects to reach Platelet Count of at least 50x10^9/L
- Duration of Platelet Response [32 days]
defined as the number of days the platelet count remains above at least 50x10^9/L
- Maximum platelet count recorded during the study [39 days]
- Adverse Events [39 days]
Adverse Events
- Blood Pressure [39 days]
Blood Pressure
- Physical Examinations [39 days]
Physical Examinations
- Heart Rate [39 days]
Heart Rate
- Temperature [39 days]
Temperature
- Respiratory Rate [39 days]
Respiratory Rate
- Complete Blood Count [39 days]
Complete Blood Count
- White Blood Cell Differential [39 days]
White Blood Cell Differential
- Hematocrit [39 days]
Hematocrit
- Hemoglobin [39 days]
Hemoglobin
- Platelet Counts [39 days]
Platelet Counts
- Reticulocytes [39 days]
Reticulocytes
- Bilirubin Levels [39 days]
Total, direct, and indirect bilirubin
- ALT (Alanine Aminotransferase) [39 days]
ALT
- AST (Aspartate Aminotransferase) [39 days]
AST
- Creatinine [39 days]
Creatinine
- Sodium [39 days]
Sodium
- Calcium [39 days]
Calcium
- Potassium [39 days]
Potassium
- BUN (blood urea nitrogen) [39 days]
BUN
- LDH (lactase dehydrogenase) [39 days]
LDH
Eligibility Criteria
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
-
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.
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 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 |
| 5 | Octapharma Research Site | Toledo | Ohio | United States | 43606 |
| 6 | Octapharma Research Site | Philadelphia | Pennsylvania | United States | 19104 |
| 7 | Octapharma Research Site | Providence | Rhode Island | United States | 02903 |
| 8 | Octapharma Research Site | Houston | Texas | United States | 77030 |
Sponsors and Collaborators
- Octapharma
Investigators
None specified.Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- NGAM-10