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

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

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 Overview

• Status: Active, not recruiting
• Conditions: Chronic Immune Thrombocytopenia
• Intervention / Treatment: Biological: Panzyga

• Study Type: Interventional
• Enrollment (Actual): 20
• Phase: Phase 4

Contacts and Locations

• Study Contact: Name: Patrick Murphy; Phone Number: 866-337-1868; Email: ctgov@clinicalresearchmgt.com

Study Locations

• United States
  • California
    • Sacramento, California, United States, 95817
      • Octapharma Research Site
  • Minnesota
    • Minneapolis, Minnesota, United States, 55404
      • Octapharma Research Site
    • Rochester, Minnesota, United States, 55905
      • Octapharma Research Site
  • Ohio
    • Columbus, Ohio, United States, 43205
      • Octapharma Research Site
    • Toledo, Ohio, United States, 43606
      • Octapharma Research Site
  • Pennsylvania
    • Philadelphia, Pennsylvania, United States, 19104
      • Octapharma Research Site
  • Rhode Island
    • Providence, Rhode Island, United States, 02903
      • Octapharma Research Site
  • Texas
    • Houston, Texas, United States, 77030
      • Octapharma Research Site

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 1 year to 18 years (Child, Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

1. Females and males aged from ≥1 year to <18 years old
2. Confirmed diagnosis of Chronic Immune Thrombocytopenia (ITP) according to American Society of Hematology (ASH) 2019 guidelines
3. Platelets count <30x10^9/L at the Baseline Visit
4. 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])
5. 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.
6. 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:

1. Thrombocytopenia secondary to other diseases (such as Acquired Immunodeficiency Syndrome [AIDS] or systemic lupus erythematosus [SLE]), drug-related thrombocytopenia, or congenital thrombocytopenia
2. Administration of intravenous immunoglobulin (IGIV) or anti-D immunoglobulin within 3 weeks (+/- 3 days) before enrollment
3. 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
4. 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)
5. 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
6. Nonresponsive to previous treatment with IGIV or anti-D immunoglobulin
7. Evidence of an active major bleeding episode at Screening
8. Splenectomy in the previous 3 months or planned splenectomy throughout the study period
9. Evans syndrome (experiencing active disease with 2 out of 3 of the following: autoimmune thrombocytopenia, autoimmune hemolytic anemia, and/or autoimmune neutropenia)
10. Known or suspected human immunodeficiency virus (HIV), hepatitis B virus (HBV), and/or hepatitis C virus (HCV) infections
11. Emergency surgery in the previous 4 weeks
12. 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)
13. History of severe hypersensitivity to blood or plasma derived products, or any component of the PANZYGA
14. Known immunoglobulin A (IgA) deficiency and antibodies against IgA
15. History of, or suspected alcohol or drug abuse in the previous year
16. Females who are pregnant or nursing
17. Unable or unwilling to comply with the study protocol
18. Receipt of any other investigational medicinal product within 3 months before study entry
19. Risk factors* for thromboembolic events in whom the risks outweigh the potential benefit of PANZYGA treatment.

20. 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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Panzyga	Biological: Panzyga; Immune Globulin, intravenous, human-ifas

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Increasing the platelet count in pediatric patients with chronic ITP	8 Days

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Time to Reach Platelet Count of at least 50x10^9/L	defined as the number of days for subjects to reach Platelet Count of at least 50x10^9/L	32 Days
Duration of Platelet Response	defined as the number of days the platelet count remains above at least 50x10^9/L	32 days
Maximum platelet count recorded during the study		39 days
Adverse Events	Adverse Events	39 days
Blood Pressure	Blood Pressure	39 days
Physical Examinations	Physical Examinations	39 days
Heart Rate	Heart Rate	39 days
Temperature	Temperature	39 days
Respiratory Rate	Respiratory Rate	39 days
Complete Blood Count	Complete Blood Count	39 days
White Blood Cell Differential	White Blood Cell Differential	39 days
Hematocrit	Hematocrit	39 days
Hemoglobin	Hemoglobin	39 days
Platelet Counts	Platelet Counts	39 days
Reticulocytes	Reticulocytes	39 days
Bilirubin Levels	Total, direct, and indirect bilirubin	39 days
ALT (Alanine Aminotransferase)	ALT	39 days
AST (Aspartate Aminotransferase)	AST	39 days
Creatinine	Creatinine	39 days
Sodium	Sodium	39 days
Calcium	Calcium	39 days
Potassium	Potassium	39 days
BUN (blood urea nitrogen)	BUN	39 days
LDH (lactase dehydrogenase)	LDH	39 days

Collaborators and Investigators

• Sponsor: Octapharma

Study record dates

Study Major Dates

• Study Start (Actual): January 21, 2020
• Primary Completion (Estimated): April 1, 2024
• Study Completion (Estimated): April 1, 2024

Study Registration Dates

• First Submitted: March 6, 2019
• First Submitted That Met QC Criteria: March 6, 2019
• First Posted (Actual): March 7, 2019

Study Record Updates

• Last Update Posted (Actual): January 11, 2024
• Last Update Submitted That Met QC Criteria: January 10, 2024
• Last Verified: January 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Pathologic Processes; Immune System Diseases; Autoimmune Diseases; Hematologic Diseases; Hemorrhage; Hemorrhagic Disorders; Blood Coagulation Disorders; Skin Manifestations; Blood Platelet Disorders; Thrombotic Microangiopathies; Purpura, Thrombocytopenic; Purpura; Purpura, Thrombocytopenic, Idiopathic; Thrombocytopenia
• Other Study ID Numbers: NGAM-10

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No