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

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Octapharma
ClinicalTrials.gov Identifier:
NCT01349790
First received: May 5, 2011
Last updated: November 28, 2016
Last verified: November 2016
  Purpose
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.

Condition Intervention Phase
Primary Thrombocytopenia
Drug: NewGam
Phase 3

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official 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

Resource links provided by NLM:


Further study details as provided by Octapharma:

Primary Outcome Measures:
  • Percentage of responders [ Time Frame: Day 1 to Day 8 ] [ Designated as safety issue: No ]
    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 Outcome Measures:
  • Percentage of alternative responders [ Time Frame: Day 1 to Day 22 ] [ Designated as safety issue: No ]
    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.

  • Percentage of complete responders [ Time Frame: Day 1 to Day 22 ] [ Designated as safety issue: No ]
    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.

  • Percentage of alternative responders who lost the response [ Time Frame: Day 1 to Day 22 ] [ Designated as safety issue: No ]
    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.

  • Percentage of complete responders who lost the response [ Time Frame: Day 1 to Day 22 ] [ Designated as safety issue: No ]
    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.

  • Time to a response [ Time Frame: Day 1 to Day 8 ] [ Designated as safety issue: No ]
    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.

  • Time to an alternative response [ Time Frame: Day 1 to Day 22 ] [ Designated as safety issue: No ]
    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.

  • Time to a complete response [ Time Frame: Day 1 to Day 22 ] [ Designated as safety issue: No ]
    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.

  • Duration of a response [ Time Frame: Day 1 to Day 22 ] [ Designated as safety issue: No ]
    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.

  • Duration of an alternative response [ Time Frame: Day 1 to Day 22 ] [ Designated as safety issue: No ]
    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.

  • Duration of a complete response [ Time Frame: Day 1 to Day 22 ] [ Designated as safety issue: No ]
    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.

  • Platelet count by Visit [ Time Frame: Day 1 to Day 22 ] [ Designated as safety issue: No ]
    The platelet count at each study visit are presented.

  • Maximum platelet count [ Time Frame: Day 1 to Day 22 ] [ Designated as safety issue: No ]
    The maximum platelet count achieved during the study is presented.

  • Percentage of responders who achieved a normal platelet count [ Time Frame: Day 1 to Day 22 ] [ Designated as safety issue: No ]
    The percentage of responders who achieved a normal platelet count is presented.

  • Bleeding intensity [ Time Frame: Day 1 to Day 22 ] [ Designated as safety issue: No ]
    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, Day 8, and Day 22 are reported.

  • Percentage of participants who achieved a platelet count > 30x10^9/L [ Time Frame: Day 1 to Day 2 ] [ Designated as safety issue: No ]
    The percentage of participants who achieved a platelet count > 30x10^9/L within 1 and 2 days after infusion is reported.


Enrollment: 40
Study Start Date: October 2011
Study Completion Date: July 2013
Primary Completion Date: July 2013 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: NewGam
Each participant received 1 g/kg NewGam intravenously on 2 consecutive days.
Drug: NewGam
NewGam is a solution of human normal immunoglobulin 10% treated with solvent/detergent and nanofiltered for intravenous administration.

Detailed Description:
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.
  Eligibility

Ages Eligible for Study:   18 Years to 80 Years   (Adult, Senior)
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Age of ≥ 18 and ≤ 80 years.
  2. Confirmed diagnosis of chronic primary immune thrombocytopenia (ITP) with a threshold platelet count < 100x10^9/L) of at least 12 months duration.
  3. Platelet count of no more than 20x10^9/L with or without bleeding manifestations.
  4. Freely given written informed consent from patient.
  5. Women of childbearing potential must have a negative result on a pregnancy test (human chorionic gonadotropine [HCG]-based assay) and need to practice contraception using a method of proven reliability for the duration of the study.

Exclusion Criteria:

  1. Thrombocytopenia secondary to other diseases (such as acquired immunodeficiency syndrome [AIDS] or systemic lupus erythematosus [SLE]) or drug-related thrombocytopenia.
  2. Administration of intravenous immunoglobulin (IGIV), anti-D immunoglobulin or thrombopoetin 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.
  3. Unresponsive to previous treatment with IGIV or anti-D immunoglobulin.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01349790

Locations
Germany
Abdulgabar Salama
Berlin, Germany
Sponsors and Collaborators
Octapharma
Investigators
Principal Investigator: Abdulgabar Salama, MD Universitätsklinikum Charite, Med. Fakultät der Humboldt-Universität Berlin
Study Director: Wolfgang Frenzel, MD Octapharma
  More Information

Responsible Party: Octapharma
ClinicalTrials.gov Identifier: NCT01349790     History of Changes
Other Study ID Numbers: NGAM-02  2009-014589-24 
Study First Received: May 5, 2011
Last Updated: November 28, 2016
Health Authority: United States: Food and Drug Administration
Germany: Paul-Ehrlich-Institut

Keywords provided by Octapharma:
ITP
Primary immune thrombocytopenia

Additional relevant MeSH terms:
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
Immunoglobulins
Antibodies
Immunologic Factors
Physiological Effects of Drugs

ClinicalTrials.gov processed this record on December 02, 2016