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

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ClinicalTrials.gov Identifier: NCT03866798
Recruitment Status : Recruiting
First Posted : March 7, 2019
Last Update Posted : December 21, 2021
Sponsor:
Information provided by (Responsible Party):
Octapharma

Study Description
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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
Chronic Immune Thrombocytopenia Biological: Panzyga Phase 4

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 20 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Post-Marketing Study to Evaluate the Efficacy and Safety of PANZYGA in Pediatric Patients With Chronic Immune Thrombocytopenia (ITP)
Actual Study Start Date : January 21, 2020
Estimated Primary Completion Date : April 2024
Estimated Study Completion Date : April 2024

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: Panzyga
Panzyga
 Biological: Panzyga
Immune Globulin, intravenous, human-ifas


Outcome Measures
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Primary Outcome Measures :
  1. Increasing the platelet count in pediatric patients with chronic ITP [ Time Frame: 8 Days ]

Secondary Outcome Measures :
  1. Time to Reach Platelet Count of at least 50x10^9/L [ Time Frame: 32 Days ]
    defined as the number of days for subjects to reach Platelet Count of at least 50x10^9/L

  2. Duration of Platelet Response [ Time Frame: 32 days ]
    defined as the number of days the platelet count remains above at least 50x10^9/L

  3. Maximum platelet count recorded during the study [ Time Frame: 39 days ]
  4. Adverse Events [ Time Frame: 39 days ]
    Adverse Events

  5. Blood Pressure [ Time Frame: 39 days ]
    Blood Pressure

  6. Physical Examinations [ Time Frame: 39 days ]
    Physical Examinations

  7. Heart Rate [ Time Frame: 39 days ]
    Heart Rate

  8. Temperature [ Time Frame: 39 days ]
    Temperature

  9. Respiratory Rate [ Time Frame: 39 days ]
    Respiratory Rate

  10. Complete Blood Count [ Time Frame: 39 days ]
    Complete Blood Count

  11. White Blood Cell Differential [ Time Frame: 39 days ]
    White Blood Cell Differential

  12. Hematocrit [ Time Frame: 39 days ]
    Hematocrit

  13. Hemoglobin [ Time Frame: 39 days ]
    Hemoglobin

  14. Platelet Counts [ Time Frame: 39 days ]
    Platelet Counts

  15. Reticulocytes [ Time Frame: 39 days ]
    Reticulocytes

  16. Bilirubin Levels [ Time Frame: 39 days ]
    Total, direct, and indirect bilirubin

  17. ALT (Alanine Aminotransferase) [ Time Frame: 39 days ]
    ALT

  18. AST (Aspartate Aminotransferase) [ Time Frame: 39 days ]
    AST

  19. Creatinine [ Time Frame: 39 days ]
    Creatinine

  20. Sodium [ Time Frame: 39 days ]
    Sodium

  21. Calcium [ Time Frame: 39 days ]
    Calcium

  22. Potassium [ Time Frame: 39 days ]
    Potassium

  23. BUN (blood urea nitrogen) [ Time Frame: 39 days ]
    BUN

  24. LDH (lactase dehydrogenase) [ Time Frame: 39 days ]
    LDH


Eligibility Criteria
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Ages Eligible for Study:   1 Year to 18 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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.
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03866798


Contacts
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Contact: Patrick Murphy 866-337-1868 ctgov@clinicalresearchmgt.com

Locations
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United States, California
Octapharma Research Site Recruiting
Sacramento, California, United States, 95817
United States, Minnesota
Octapharma Research Site Recruiting
Minneapolis, Minnesota, United States, 55404
Octapharma Research Site Recruiting
Rochester, Minnesota, United States, 55905
United States, Ohio
Octapharma Research Site Recruiting
Columbus, Ohio, United States, 43205
Octapharma Research Site Recruiting
Toledo, Ohio, United States, 43606
United States, Pennsylvania
Octapharma Research Site Recruiting
Philadelphia, Pennsylvania, United States, 19104
United States, Rhode Island
Octapharma Research Site Recruiting
Providence, Rhode Island, United States, 02903
United States, Texas
Octapharma Research Site Recruiting
Houston, Texas, United States, 77030
Sponsors and Collaborators
Octapharma
More Information
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Responsible Party: Octapharma
ClinicalTrials.gov Identifier: NCT03866798    
Other Study ID Numbers: NGAM-10
First Posted: March 7, 2019    Key Record Dates
Last Update Posted: December 21, 2021
Last Verified: December 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Thrombocytopenia
Immune System Diseases
Purpura, Thrombocytopenic, Idiopathic
Blood Platelet Disorders
Hematologic Diseases
Purpura, Thrombocytopenic
Purpura
 Blood Coagulation Disorders
Thrombotic Microangiopathies
Hemorrhagic Disorders
Autoimmune Diseases
Hemorrhage
Pathologic Processes
Skin Manifestations