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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 : Not yet recruiting
First Posted : March 7, 2019
Last Update Posted : March 12, 2019
Sponsor:
Information provided by (Responsible Party):
Octapharma

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

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 20 participants
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)
Estimated Study Start Date : July 2019
Estimated Primary Completion Date : March 2022
Estimated Study Completion Date : March 2022


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




Primary Outcome Measures :
  1. Platelet Count [ Time Frame: 8 Days ]

Secondary Outcome Measures :
  1. Time to Reach Platelet Count of at least 50x10^9/L [ Time Frame: 32 Days39 days ]
  2. Duration of Platelet Response [ Time Frame: 39 days ]
    defined as the number of days the platelet count remains above at least 50x10^9/L

  3. Maximum Platelet Count [ Time Frame: 39 days ]
    Maximum platelet count recorded during the study

  4. Adverse Events [ Time Frame: 39 days ]
    Adverse Events

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

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

  7. Temperature [ Time Frame: 39 days ]
    Temperature

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

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

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

  11. Hematocrit [ Time Frame: 39 days ]
    Hematocrit

  12. Hemoglobin [ Time Frame: 39 days ]
    Hemoglobin

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

  14. Reticulocytes [ Time Frame: 39 days ]
    Reticulocytes

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

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

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

  18. Creatinine [ Time Frame: 39 days ]
    Creatinine

  19. Sodium [ Time Frame: 39 days ]
    Sodium

  20. Calcium [ Time Frame: 39 days ]
    Calcium

  21. Potassium [ Time Frame: 39 days ]
    Potassium

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

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



Information from the National Library of Medicine

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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) 2011 guidelines
  3. Platelets count <30x109/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. Females of childbearing potential 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 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. Abstinence is not 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 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 no dosage change is planned until 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 4 weeks 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.

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: Mikaela Raymond 866-337-1868 ctgov@clinicalresearchmgt.com

Sponsors and Collaborators
Octapharma

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Responsible Party: Octapharma
ClinicalTrials.gov Identifier: NCT03866798     History of Changes
Other Study ID Numbers: NGAM-10
First Posted: March 7, 2019    Key Record Dates
Last Update Posted: March 12, 2019
Last Verified: March 2019
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
Purpura, Thrombocytopenic, Idiopathic
Blood Platelet Disorders
Hematologic Diseases
Purpura, Thrombocytopenic
Purpura
Blood Coagulation Disorders
Thrombotic Microangiopathies
Hemorrhagic Disorders
Autoimmune Diseases
Immune System Diseases
Hemorrhage
Pathologic Processes
Skin Manifestations
Signs and Symptoms