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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 : September 4, 2020
Sponsor:
Information provided by (Responsible Party):
Octapharma

Tracking Information
First Submitted Date  ICMJE March 6, 2019
First Posted Date  ICMJE March 7, 2019
Last Update Posted Date September 4, 2020
Actual Study Start Date  ICMJE January 21, 2020
Estimated Primary Completion Date March 2022   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: March 6, 2019)
Platelet Count [ Time Frame: 8 Days ]
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: March 8, 2019)
  • Time to Reach Platelet Count of at least 50x10^9/L [ Time Frame: 32 Days39 days ]
  • Duration of Platelet Response [ Time Frame: 39 days ]
    defined as the number of days the platelet count remains above at least 50x10^9/L
  • Maximum Platelet Count [ Time Frame: 39 days ]
    Maximum platelet count recorded during the study
  • Adverse Events [ Time Frame: 39 days ]
    Adverse Events
  • Blood Pressure [ Time Frame: 39 days ]
    Blood Pressure
  • Heart Rate [ Time Frame: 39 days ]
    Heart Rate
  • Temperature [ Time Frame: 39 days ]
    Temperature
  • Respiratory Rate [ Time Frame: 39 days ]
    Respiratory Rate
  • Complete Blood Count [ Time Frame: 39 days ]
    Complete Blood Count
  • White Blood Cell Differential [ Time Frame: 39 days ]
    White Blood Cell Differential
  • Hematocrit [ Time Frame: 39 days ]
    Hematocrit
  • Hemoglobin [ Time Frame: 39 days ]
    Hemoglobin
  • Platelet Counts [ Time Frame: 39 days ]
    Platelet Counts
  • Reticulocytes [ Time Frame: 39 days ]
    Reticulocytes
  • Bilirubin Levels [ Time Frame: 39 days ]
    Total, direct, and indirect bilirubin
  • ALT (Alanine Aminotransferase) [ Time Frame: 39 days ]
    ALT
  • AST (Aspartate Aminotransferase) [ Time Frame: 39 days ]
    AST
  • Creatinine [ Time Frame: 39 days ]
    Creatinine
  • Sodium [ Time Frame: 39 days ]
    Sodium
  • Calcium [ Time Frame: 39 days ]
    Calcium
  • Potassium [ Time Frame: 39 days ]
    Potassium
  • BUN (blood urea nitrogen) [ Time Frame: 39 days ]
    BUN
  • LDH (lactase dehydrogenase) [ Time Frame: 39 days ]
    LDH
Original Secondary Outcome Measures  ICMJE
 (submitted: March 6, 2019)
  • Time to Reach Platelet Count of at least 50x10^9/L [ Time Frame: 32 Days39 days ]
  • Duration of Platelet Response [ Time Frame: 39 days ]
    defined as the number of days the platelet count remains above at least 50x10^9/L
  • Maximum Platelet Count [ Time Frame: 39 days ]
    Maximum platelet count recorded during the study
  • Adverse Events [ Time Frame: 39 days ]
    Adverse Events
  • Blood Pressure [ Time Frame: 39 days ]
    Blood Pressure
  • Heart Rate [ Time Frame: 39 days ]
    Heart Rate
  • Temperature [ Time Frame: 39 days ]
    Temperature
  • Respiratory Rate [ Time Frame: 39 days ]
    Respiratory Rate
  • Complete Blood Count [ Time Frame: 39 days ]
    Complete Blood Count
  • White Blood Cell Differential [ Time Frame: 39 days ]
    White Blood Cell Differential
  • Hematocrit [ Time Frame: 39 days ]
    Hematocrit
  • Hemoglobin [ Time Frame: 39 days ]
    Hemoglobin
  • Platelet Counts [ Time Frame: 39 days ]
    Platelet Counts
  • Reticulocytes [ Time Frame: 39 days ]
    Reticulocytes
  • Bilirubin Levels [ Time Frame: 39 days ]
    Total, direct, and indirect bilirubin
  • ALT [ Time Frame: 39 days ]
    ALT
  • AST [ Time Frame: 39 days ]
    AST
  • Creatinine [ Time Frame: 39 days ]
    Creatinine
  • Sodium [ Time Frame: 39 days ]
    Sodium
  • Calcium [ Time Frame: 39 days ]
    Calcium
  • Potassium [ Time Frame: 39 days ]
    Potassium
  • BUN [ Time Frame: 39 days ]
    BUN
  • LDH [ Time Frame: 39 days ]
    LDH
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Study to Evaluate the Efficacy and Safety of PANZYGA in Pediatric Patients With Chronic Immune Thrombocytopenia (ITP)
Official Title  ICMJE Post-Marketing Study to Evaluate the Efficacy and Safety of PANZYGA in Pediatric Patients With Chronic Immune Thrombocytopenia (ITP)
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.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 4
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Chronic Immune Thrombocytopenia
Intervention  ICMJE Biological: Panzyga
Immune Globulin, intravenous, human-ifas
Study Arms  ICMJE Experimental: Panzyga
Panzyga
Intervention: Biological: Panzyga
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: March 6, 2019)
20
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE March 2022
Estimated Primary Completion Date March 2022   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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 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 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.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 1 Year to 18 Years   (Child, Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Mikaela Raymond 866-337-1868 ctgov@clinicalresearchmgt.com
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03866798
Other Study ID Numbers  ICMJE NGAM-10
Has Data Monitoring Committee Not Provided
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party Octapharma
Study Sponsor  ICMJE Octapharma
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account Octapharma
Verification Date September 2020

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP