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| ID | Type | Description | Link |
|---|---|---|---|
| 2009-014589-24 | EudraCT Number |
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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.
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| NewGam | Experimental | Each participant received 1 g/kg NewGam intravenously on 2 consecutive days. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| NewGam | Drug | NewGam is a solution of human normal immunoglobulin 10% treated with solvent/detergent and nanofiltered for intravenous administration. |
|
| Measure | Description | Time Frame |
|---|---|---|
| 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. | Day 1 to Day 8 |
| Measure | Description | Time Frame |
|---|---|---|
| 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. | Day 1 to Day 22 |
| Percentage of Complete Responders |
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Inclusion Criteria:
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:
Unresponsive to previous treatment with IGIV or anti-D immunoglobulin.
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| Name | Affiliation | Role |
|---|---|---|
| Abdulgabar Salama, MD | Universitätsklinikum Charite, Med. Fakultät der Humboldt-Universität Berlin | Principal Investigator |
| Wolfgang Frenzel, MD | Octapharma AG | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Abdulgabar Salama | Berlin | Germany |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 30687970 | Derived | 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. |
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| ID | Title | Description |
|---|---|---|
| FG000 | NewGam | Each participant received 1 g/kg NewGam intravenously on 2 consecutive days. |
| Title | Milestones | Reasons Not Completed | ||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
|
Safety set: All participants enrolled in the study who received at least part of 1 dose of NewGam.
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| ID | Title | Description |
|---|---|---|
| BG000 | NewGam | Each participant received 1 g/kg NewGam intravenously on 2 consecutive days. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | 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. | Full analysis set: All participants enrolled in the study who received at least part of 1 dose of NewGam and who had at least 1 post-baseline measurement of platelet concentration. | Posted | Number | 95% Confidence Interval | Percentage of participants | Day 1 to Day 8 |
|
|
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Safety set: All participants enrolled in the study who received at least part of 1 dose of NewGam.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | NewGam | Each participant received 1 g/kg NewGam intravenously on 2 consecutive days. |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Autoimmune thrombocytopenia | Blood and lymphatic system disorders | MedDRA (Unspecified) | Systematic Assessment |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Headache | Nervous system disorders | MedDRA (Unspecified) | Systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Michael Eppolito, Director, Clinical Operations Immunology and ICU Medicine | Octapharma USA | 201 604-1155 | michael.eppolito@octapharma.com |
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| ID | Term |
|---|---|
| D016553 | Purpura, Thrombocytopenic, Idiopathic |
| ID | Term |
|---|---|
| D011696 | Purpura, Thrombocytopenic |
| D011693 | Purpura |
| D001778 | Blood Coagulation Disorders |
| D006402 | Hematologic Diseases |
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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. |
| Day 1 to Day 22 |
| 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. | Day 1 to Day 22 |
| 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. | Day 1 to Day 22 |
| 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. | Day 1 to 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. | Day 1 to Day 22 |
| 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. | Day 1 to Day 22 |
| 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. | Day 1 to Day 22 |
| 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. | Day 1 to Day 22 |
| 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. | Day 1 to Day 22 |
| Platelet Count by Visit | The platelet count at each study visit are presented. | Day 1 to Day 22 |
| Maximum Platelet Count | The maximum platelet count achieved during the study is presented. | Day 1 to Day 22 |
| Percentage of Responders Who Achieved a Normal Platelet Count | The percentage of responders who achieved a normal platelet count is presented. | Day 1 to Day 22 |
| 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. | Day 1 to Day 22 |
| 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. | Day 1 to Day 2 |
| Adverse Event |
|
| Lost to Follow-up |
|
| years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
|
|
| Secondary | 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. | Full analysis set: All participants enrolled in the study who received at least part of 1 dose of NewGam and who had at least 1 post-baseline measurement of platelet concentration. | Posted | Number | 95% Confidence Interval | Percentage of participants | Day 1 to Day 22 |
|
|
|
| Secondary | 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. | Full analysis set: All participants enrolled in the study who received at least part of 1 dose of NewGam and who had at least 1 post-baseline measurement of platelet concentration. | Posted | Number | 95% Confidence Interval | Percentage of participants | Day 1 to Day 22 |
|
|
|
| Secondary | 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. | Full analysis set: All participants enrolled in the study who received at least part of 1 dose of NewGam and who had at least 1 post-baseline measurement of platelet concentration. Only participants with an alternative response were included in the analysis. | Posted | Number | 95% Confidence Interval | Percentage of participants | Day 1 to Day 22 |
|
|
|
| Secondary | 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. | Full analysis set: All participants enrolled in the study who received at least part of 1 dose of NewGam and who had at least 1 post-baseline measurement of platelet concentration. Only participants with a complete response were included in the analysis. | Posted | Number | 95% Confidence Interval | Percentage of participants | Day 1 to Day 22 |
|
|
|
| Secondary | 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. | Full analysis set: All participants enrolled in the study who received at least part of 1 dose of NewGam who had at least 1 post-baseline measurement of platelet concentration. Only responders were included in the analysis. | Posted | Median | 95% Confidence Interval | Days | Day 1 to Day 8 |
|
|
|
| Secondary | 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. | Full analysis set: All participants enrolled in the study who received at least part of 1 dose of NewGam who had at least 1 post-baseline measurement of platelet concentration. Only participants with an alternative response were included in the analysis. | Posted | Median | 95% Confidence Interval | Days | Day 1 to Day 22 |
|
|
|
| Secondary | 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. | Full analysis set: All participants enrolled in the study who received at least part of 1 dose of NewGam who had at least 1 post-baseline measurement of platelet concentration. Only participants with a complete response were included in the analysis. | Posted | Median | 95% Confidence Interval | Days | Day 1 to Day 22 |
|
|
|
| Secondary | 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. | Full analysis set: All participants enrolled in the study who received at least part of 1 dose of NewGam who had at least 1 post-baseline measurement of platelet concentration. Only responders were included in the analysis. | Posted | Median | 95% Confidence Interval | Days | Day 1 to Day 22 |
|
|
|
| Secondary | 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. | Full analysis set: All participants enrolled in the study who received at least part of 1 dose of NewGam who had at least 1 post-baseline measurement of platelet concentration. Only participants with an alternative response were included in the analysis. | Posted | Median | 95% Confidence Interval | Days | Day 1 to Day 22 |
|
|
|
| Secondary | 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. | Full analysis set: All participants enrolled in the study who received at least part of 1 dose of NewGam who had at least 1 post-baseline measurement of platelet concentration. Only participants with a complete response were included in the analysis. | Posted | Median | 95% Confidence Interval | Days | Day 1 to Day 22 |
|
|
|
| Secondary | Platelet Count by Visit | The platelet count at each study visit are presented. | Full analysis set: All participants enrolled in the study who received at least part of 1 dose of NewGam who had at least 1 post-baseline measurement of platelet concentration. | Posted | Mean | Standard Deviation | 10^9 platelets/L | Day 1 to Day 22 |
|
|
|
| Secondary | Maximum Platelet Count | The maximum platelet count achieved during the study is presented. | Full analysis set: All participants enrolled in the study who received at least part of 1 dose of NewGam who had at least 1 post-baseline measurement of platelet concentration. | Posted | Mean | Standard Deviation | 10^9 platelets/L | Day 1 to Day 22 |
|
|
|
| Secondary | Percentage of Responders Who Achieved a Normal Platelet Count | The percentage of responders who achieved a normal platelet count is presented. | Full analysis set: All participants enrolled in the study who received at least part of 1 dose of NewGam who had at least 1 post-baseline measurement of platelet concentration. Only responders were included in the analysis. | Posted | Number | 95% Confidence Interval | Percentage of participants | Day 1 to Day 22 |
|
|
|
| Secondary | 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. | Full analysis set: All participants enrolled in the study who received at least part of 1 dose of NewGam who had at least 1 post-baseline measurement of platelet concentration. | Posted | Number | Percentage of participants | Day 1 to Day 22 |
|
|
|
| Secondary | 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. | Safety set: All participants enrolled in the study who received at least part of 1 dose of NewGam. | Posted | Number | Percentage of participants | Day 1 to Day 2 |
|
|
|
| 6 |
| 40 |
| 30 |
| 40 |
| Pneumonitis | Respiratory, thoracic and mediastinal disorders | MedDRA (Unspecified) | Systematic Assessment |
|
| Dysphagia | Gastrointestinal disorders | MedDRA (Unspecified) | Systematic Assessment |
|
| Cerebral haematoma | Nervous system disorders | MedDRA (Unspecified) | Systematic Assessment |
|
| Meningitis aseptic | Nervous system disorders | MedDRA (Unspecified) | Systematic Assessment |
|
| Pneumonia | Infections and infestations | MedDRA (Unspecified) | Systematic Assessment |
|
| Respiratory failure | Respiratory, thoracic and mediastinal disorders | MedDRA (Unspecified) | Systematic Assessment |
|
| Sepsis | Infections and infestations | MedDRA (Unspecified) | Systematic Assessment |
|
| Dizziness | Nervous system disorders | MedDRA (Unspecified) | Systematic Assessment |
|
| Autoimmune thrombocytopenia | Blood and lymphatic system disorders | MedDRA (Unspecified) | Systematic Assessment |
|
| Anaemia | Blood and lymphatic system disorders | MedDRA (Unspecified) | Systematic Assessment |
|
| Idiopathic thrombocytopenic purpura | Blood and lymphatic system disorders | MedDRA (Unspecified) | Systematic Assessment |
|
| Nausea | Gastrointestinal disorders | MedDRA (Unspecified) | Systematic Assessment |
|
| Vomiting | Gastrointestinal disorders | MedDRA (Unspecified) | Systematic Assessment |
|
| Asthenia | General disorders | MedDRA (Unspecified) | Systematic Assessment |
|
| Chills | General disorders | MedDRA (Unspecified) | Systematic Assessment |
|
| Pyrexia | General disorders | MedDRA (Unspecified) | Systematic Assessment |
|
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| D006425 |
| Hemic and Lymphatic Diseases |
| D057049 | Thrombotic Microangiopathies |
| D013921 | Thrombocytopenia |
| D001791 | Blood Platelet Disorders |
| D000095542 | Cytopenia |
| D006474 | Hemorrhagic Disorders |
| D001327 | Autoimmune Diseases |
| D007154 | Immune System Diseases |
| D006470 | Hemorrhage |
| D010335 | Pathologic Processes |
| D013568 | Pathological Conditions, Signs and Symptoms |
| D012877 | Skin Manifestations |
| D012816 | Signs and Symptoms |
| Title |
|---|
| Measurements |
|---|
|
| Day 4 |
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| Day 5 |
|
| Day 6 |
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| Day 7 |
|
| Day 8 |
|
| Day 15 |
|
| Day 22/End of treatment |
|
| Title | Measurements |
|---|---|
|
| Overall - None - Baseline |
|
| Overall - None - Day 8 |
|
| Overall - None - Day 22 |
|
| Overall - Minor - Baseline |
|
| Overall - Minor - Day 8 |
|
| Overall - Minor - Day 22 |
|
| Overall - Mild - Baseline |
|
| Overall - Mild - Day 8 |
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| Overall - Mild - Day 22 |
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| Overall - Moderate - Baseline |
|
| Overall - Moderate - Day 8 |
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| Overall - Moderate - Day 22 |
|
| Overall - Severe - Baseline |
|
| Overall - Severe - Day 8 |
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| Overall - Severe - Day 22 |
|
| Epistaxis - Total - Baseline |
|
| Epistaxis - Total - Day 22 |
|
| Epistaxis - None - Baseline |
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| Epistaxis - None - Day 22 |
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| Epistaxis - Minor - Baseline |
|
| Epistaxis - Minor - Day 22 |
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| Epistaxis - Mild - Baseline |
|
| Epistaxis - Mild - Day 22 |
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| Epistaxis - Moderate - Baseline |
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| Epistaxis - Moderate - Day 22 |
|
| Epistaxis - Severe - Baseline |
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| Epistaxis - Severe - Day 22 |
|
| Oral bleeding - Total - Baseline |
|
| Oral bleeding - Total - Day 22 |
|
| Oral bleeding - None - Baseline |
|
| Oral bleeding - None - Day 22 |
|
| Oral bleeding - Minor - Baseline |
|
| Oral bleeding - Minor - Day 22 |
|
| Oral bleeding - Mild - Baseline |
|
| Oral bleeding - Mild - Day 22 |
|
| Oral bleeding - Moderate - Baseline |
|
| Oral bleeding - Moderate - Day 22 |
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| Oral bleeding - Severe - Baseline |
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| Oral bleeding - Severe - Day 22 |
|
| Skin bleeding - Total - Baseline |
|
| Skin bleeding - Total - Day 22 |
|
| Skin bleeding - None - Baseline |
|
| Skin bleeding - None - Day 22 |
|
| Skin bleeding - Minor - Baseline |
|
| Skin bleeding - Minor - Day 22 |
|
| Skin bleeding - Mild - Baseline |
|
| Skin bleeding - Mild - Day 22 |
|
| Skin bleeding - Moderate - Baseline |
|
| Skin bleeding - Moderate - Day 22 |
|
| Skin bleeding - Severe - Baseline |
|
| Skin bleeding - Severe - Day 22 |
|