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NCT Number: NCT01349790

Efficacy and Safety of Immunoglobulin Intravenous (Human) 10% (NewGam) in Primary Immune Thrombocytopenia

NewGam is a newly developed human normal immunoglobulin solution for intravenous administration (IGIV). This study will evaluate the safety and efficacy of NewGam 10% in patients with primary immune thrombocytopenia.

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Key information

Conditions

Primary Thrombocytopenia Autoimmune Diseases Blood Coagulation Disorders Blood Platelet Disorders Cytopenia Hematologic Diseases Hemic and Lymphatic Diseases Hemorrhage Hemorrhagic Disorders Immune System Diseases Pathologic Processes Pathological Conditions, Signs and Symptoms Purpura Purpura, Thrombocytopenic Purpura, Thrombocytopenic, Idiopathic Signs and Symptoms Skin Manifestations Thrombocytopenia Thrombotic Microangiopathies

Age range

18 year–65 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

Abdulgabar Salama

Berlin, Germany

About this study

The primary objective of the study is to assess the efficacy of NewGam in correcting the platelet count. The secondary objective of the study is to evaluate the safety of NewGam. Safety will be assessed by monitoring vital signs, physical examination, evaluation of adverse events (AE) and laboratory parameters, and viral safety testing.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Age of ≥ 18 and ≤ 65 years.
  • Confirmed diagnosis of chronic primary immune thrombocytopenia (ITP) (diagnosed with a threshold platelet count < 100x10^9/L) of at least 12 months duration.
  • Platelet count of no more than 20x10^9/L with or without bleeding manifestations.
  • Freely given written informed consent from patient.
  • Women of childbearing potential must have a negative result on a pregnancy test (human chorionic gonadotropin [HCG]-based assay) and need to practice contraception using a method of proven reliability for the duration of the study.

Exclusion criteria

  • Thrombocytopenia secondary to other diseases (such as acquired immunodeficiency syndrome [AIDS] or systemic lupus erythematosus [SLE]), or drug-related thrombocytopenia.
  • Administration of intravenous immunoglobulin (IGIV), anti-D immunoglobulin or thrombopoietin receptor agonists, or other platelet enhancing drugs (including immunosuppressive or other immunomodulatory drugs) within 3 weeks before enrollment, except for:
  • Long-term corticosteroid therapy when the dose has been stable during the preceding 3 weeks and no dosage change is planned until study Day 22.
  • Long-term azathioprine, cyclophosphamide, or attenuated androgen therapy when the dose has been stable during the preceding 3 months and no dosage change is planned until study Day 22.
  • Unresponsive to previous treatment with IGIV or anti-D immunoglobulin.

Treatment and study plan

NewGam

Drug

NewGam is a solution of human normal immunoglobulin 10% treated with solvent/detergent and nanofiltered for intravenous administration.

Primary outcomes

  1. Percentage of Responders

    Time frame: Day 1 to Day 8

    A responder is a study participant with an increase in platelets to ≥ 50x10^9/L within 7 days after the first infusion, ie, by study Day 8.

Secondary outcomes

  1. Percentage of Alternative Responders

    Time frame: Day 1 to Day 22

    An alternative responder is a study participant with an increase in platelets to ≥ 30x10^9/L and to at least double the baseline platelet count, confirmed on at least 2 occasions at least 7 days apart, and absence of bleeding.

  2. Percentage of Complete Responders

    Time frame: Day 1 to Day 22

    A complete responder is a study participant with an increase in platelets to ≥ 100x10^9/L, confirmed on at least 2 occasions at least 7 days apart, and absence of bleeding.

  3. Percentage of Alternative Responders Who Lost the Response

    Time frame: Day 1 to Day 22

    An alternative responder who lost the response is a study participant who met the criterion for an alternative response but who then deteriorated, ie, their platelet count decreased to < 30x10^9/L, their platelet count decreased to less than double the baseline count, or bleeding occurred.

  4. Percentage of Complete Responders Who Lost the Response

    Time frame: Day 1 to Day 22

    A complete responder who lost the response is a study participant who met the criterion for a complete response but who then deteriorated, ie, their platelet count decreased to < 100x10^9/L or bleeding occurred.

  5. Time to a Response

    Time frame: Day 1 to Day 8

    A study participant had a response if their platelets increased to ≥ 50x10^9/L within 7 days after the first infusion, ie, by study Day 8.

  6. Time to an Alternative Response

    Time frame: Day 1 to Day 22

    A study participant had a response if their platelets increased to ≥ 30x10^9/L and to at least double the baseline platelet count, confirmed on at least 2 occasions at least 7 days apart, and absence of bleeding.

  7. Time to a Complete Response

    Time frame: Day 1 to Day 22

    A study participant had a complete response if their platelets increased to ≥ 100x10^9/L, confirmed on at least 2 occasions at least 7 days apart, and absence of bleeding.

  8. Duration of a Response

    Time frame: Day 1 to Day 22

    The duration of a response was defined as the time from when a response was achieved until the platelet count fell below 50x10^9/L.

  9. Duration of an Alternative Response

    Time frame: Day 1 to Day 22

    The duration of an alternative response was defined as the time from when an alternative response was achieved until the platelet count fell below 50x10^9/L.

  10. Duration of a Complete Response

    Time frame: Day 1 to Day 22

    The duration of a complete response was defined as the time from when a complete response was achieved until the platelet count fell below 50x10^9/L.

  11. Platelet Count by Visit

    Time frame: Day 1 to Day 22

    The platelet count at each study visit are presented.

  12. Maximum Platelet Count

    Time frame: Day 1 to Day 22

    The maximum platelet count achieved during the study is presented.

  13. Percentage of Responders Who Achieved a Normal Platelet Count

    Time frame: Day 1 to Day 22

    The percentage of responders who achieved a normal platelet count is presented.

  14. Bleeding Intensity

    Time frame: Day 1 to Day 22

    The percentage of participants with various intensities of overall bleeding, epistaxis (bleeding of the nose), oral bleeding, and skin bleeding graded as none, minor, mild, moderate, or severe at Baseline and Day 22 are reported.

  15. Percentage of Participants Who Achieved a Platelet Count > 30x10^9/L

    Time frame: Day 1 to Day 2

    The percentage of participants who achieved a platelet count > 30x10^9/L within 1 and 2 days after infusion is reported.

Sponsors and collaborators

Lead sponsor

Octapharma

Industry

Registry information

Official study title

Prospective, Open-label, Non-controlled, Multicenter, Phase III Clinical Study to Evaluate the Efficacy and Safety of Immunoglobulin Intravenous (Human) 10% (NewGam) in Primary Immune Thrombocytopenia

Important dates

Study start
2011
Primary completion
2013
Study completion
2013
First posted
May 9, 2011
Registry last updated
Jun 7, 2017

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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