Back to resultsEfficacy and Safety of Immunoglobulin Intravenous (Human) 10% (NewGam) in Primary Immune Thrombocytopenia
Primary Purpose
Primary Thrombocytopenia
Status
Completed
Phase
Phase 3
Locations
Germany
Study Type
Interventional
Intervention
NewGam
Sponsored by
About this trial
This is an interventional treatment trial for Primary Thrombocytopenia focused on measuring ITP, Primary immune thrombocytopenia
Eligibility Criteria
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.
Sites / Locations
- Abdulgabar Salama
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
NewGam
Arm Description
Each participant received 1 g/kg NewGam intravenously on 2 consecutive days.
Outcomes
Primary Outcome Measures
Percentage of Responders
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
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
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
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
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
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
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
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
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
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
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
The platelet count at each study visit are presented.
Maximum Platelet Count
The maximum platelet count achieved during the study is presented.
Percentage of Responders Who Achieved a Normal Platelet Count
The percentage of responders who achieved a normal platelet count is presented.
Bleeding Intensity
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.
Percentage of Participants Who Achieved a Platelet Count > 30x10^9/L
The percentage of participants who achieved a platelet count > 30x10^9/L within 1 and 2 days after infusion is reported.
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT01349790
Brief Title
Efficacy and Safety of Immunoglobulin Intravenous (Human) 10% (NewGam) in Primary Immune Thrombocytopenia
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
Study Type
Interventional
2. Study Status
Record Verification Date
May 2017
Overall Recruitment Status
Completed
Study Start Date
October 2011 (undefined)
Primary Completion Date
July 2013 (Actual)
Study Completion Date
July 2013 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Octapharma
4. Oversight
Data Monitoring Committee
Yes
5. Study Description
Brief Summary
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.
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Primary Thrombocytopenia
Keywords
ITP, Primary immune thrombocytopenia
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
40 (Actual)
8. Arms, Groups, and Interventions
Arm Title
NewGam
Arm Type
Experimental
Arm Description
Each participant received 1 g/kg NewGam intravenously on 2 consecutive days.
Intervention Type
Drug
Intervention Name(s)
NewGam
Intervention Description
NewGam is a solution of human normal immunoglobulin 10% treated with solvent/detergent and nanofiltered for intravenous administration.
Primary Outcome Measure Information:
Title
Percentage of Responders
Description
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.
Time Frame
Day 1 to Day 8
Secondary Outcome Measure Information:
Title
Percentage of Alternative Responders
Description
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.
Time Frame
Day 1 to Day 22
Title
Percentage of Complete Responders
Description
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.
Time Frame
Day 1 to Day 22
Title
Percentage of Alternative Responders Who Lost the Response
Description
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.
Time Frame
Day 1 to Day 22
Title
Percentage of Complete Responders Who Lost the Response
Description
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 Frame
Day 1 to Day 22
Title
Time to a Response
Description
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 Frame
Day 1 to Day 8
Title
Time to an Alternative Response
Description
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 Frame
Day 1 to Day 22
Title
Time to a Complete Response
Description
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.
Time Frame
Day 1 to Day 22
Title
Duration of a Response
Description
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.
Time Frame
Day 1 to Day 22
Title
Duration of an Alternative Response
Description
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.
Time Frame
Day 1 to Day 22
Title
Duration of a Complete Response
Description
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.
Time Frame
Day 1 to Day 22
Title
Platelet Count by Visit
Description
The platelet count at each study visit are presented.
Time Frame
Day 1 to Day 22
Title
Maximum Platelet Count
Description
The maximum platelet count achieved during the study is presented.
Time Frame
Day 1 to Day 22
Title
Percentage of Responders Who Achieved a Normal Platelet Count
Description
The percentage of responders who achieved a normal platelet count is presented.
Time Frame
Day 1 to Day 22
Title
Bleeding Intensity
Description
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.
Time Frame
Day 1 to Day 22
Title
Percentage of Participants Who Achieved a Platelet Count > 30x10^9/L
Description
The percentage of participants who achieved a platelet count > 30x10^9/L within 1 and 2 days after infusion is reported.
Time Frame
Day 1 to Day 2
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
65 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Abdulgabar Salama, MD
Organizational Affiliation
Universitätsklinikum Charite, Med. Fakultät der Humboldt-Universität Berlin
Official's Role
Principal Investigator
First Name & Middle Initial & Last Name & Degree
Wolfgang Frenzel, MD
Organizational Affiliation
Octapharma AG
Official's Role
Study Director
Facility Information:
Facility Name
Abdulgabar Salama
City
Berlin
Country
Germany
12. IPD Sharing Statement
Citations:
PubMed Identifier
30687970
Citation
Arbach O, Taumberger AB, Wietek S, Cervinek L, Salama A. Efficacy and safety of a new intravenous immunoglobulin (Panzyga(R) ) in chronic immune thrombocytopenia. Transfus Med. 2019 Feb;29(1):48-54. doi: 10.1111/tme.12573. Epub 2019 Jan 27.
Results Reference
derived
Learn more about this trial
Efficacy and Safety of Immunoglobulin Intravenous (Human) 10% (NewGam) in Primary Immune Thrombocytopenia
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