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This was a prospective, open-label, non-controlled, non-randomized multicenter Phase III study of 2 multiple-dose intravenous NewGam regimens (every 3 weeks or every 4 weeks, continuing the patient's infusion interval in the main study NCT01012323 [NGAM-01]) for 3 months. The primary objective of the study was to assess the safety and tolerability of high infusion rates of NewGam.
Patients received NewGam via an infusion pump to control precise infusion rates. All NewGam infusions started at a rate of 0.01 mL/kg/min (60 mg/kg/h) for the first 30 minutes followed by 0.03 mL/kg/min (180 mg/kg/h) for the next 15 minutes. If tolerated, further increments were made at predefined patterns with the following maximum rates: 0.10 mL/kg/min (600 mg/kg/h) in the first infusion; if this was tolerated, 0.12 mL/kg/min (720 mg/kg/h) in the second infusion; if this was tolerated, 0.14 mL/kg/min (840 mg/kg/h) in all subsequent infusions.
If an adverse event occurred during an infusion, the rate was reduced to half the rate at which the event occurred or the infusion was interrupted until symptoms subsided. The infusion was then resumed at a rate tolerated by the patient.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| NewGam | Experimental | Participants received NewGam 200-800 mg/kg intravenously every 3 or 4 weeks for 3 months (5 or 4 total infusions, respectively). |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| NewGam | Biological | The initial dose and dosing interval for each participant was the dose and dosing interval the participant received at the end of the NGAM-01 study. The dose of NewGam, solvent/detergent treated human normal immunoglobulin 10%, remained the same throughout the study, as long as the minimum trough level of serum immunoglobulin G (IgG) was above 5 g/L. If the serum IgG trough level dropped to 5 g/L or less, the dose was to be adjusted at the investigator's discretion. NewGam was supplied as a solution for infusion. |
| Measure | Description | Time Frame |
|---|---|---|
| Percentage of Participants Who Experienced at Least 1 Adverse Event Causally Related to the Administration of the Study Drug | An adverse event was considered to be causally related to the administration of the study drug if it judged to be probably or possibly related to the study drug, as assessed by the investigator. | Baseline (follow-up visit of study NGAM-01) to the end of the study (follow-up visit of study NGAM-05) (up to 4 months) |
| Percentage of Participants Who Experienced at Least 1 Adverse Event Temporally Related to the Study Drug | An adverse event was considered to be temporally related to the study drug if it started during an infusion or within 72 hours after the end of an infusion. | Baseline (follow-up visit of study NGAM-01) to the end of the study (follow-up visit of study NGAM-05) (up to 4 months) |
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline in the Quality of Life (QoL) at the End of the Study | QoL was assessed with the Child Health Questionnaire-Parent Form (CHQ-PF50), completed by a parent or guardian, in participants < 14 years of age at the start of the previous study NGAM-01 and with the Short Form-36 Health Survey (SF-36-HS) in participants ≥ 14 years of age. The CHQ-PF50 consists of 50 items organized into 15 subscales.The 15 subscales could be combined into 2 summary scores, physical and psychosocial. The calculated scores were transformed so that each scale had a range of 0-100. A higher score indicates better health. The SF-36-HS is composed of 36 items. Responses to the 36 items were combined to create 8 scales. The 8 scales could be further combined into 2 scores: Physical component summary and mental component summary. The item and scale scores were transformed to a range of 0-100 with a mean of 50 and a standard deviation of 10 in the general US population. A higher score indicates better health. For both instruments, a positive change indicates improvement. |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| James N Moy, MD | Rush Medical Center | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of California Irvine | Irvine | California | United States | |||
| Immunoe Research Center |
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| ID | Title | Description |
|---|---|---|
| FG000 | NewGam | Participants received NewGam 200-800 mg/kg intravenously every 3 or 4 weeks for 3 months (5 or 4 total infusions, respectively). |
| Title | Milestones | Reasons Not Completed | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
Safety analysis set: All participants who received at least 1 dose of NewGam.
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| ID | Title | Description |
|---|---|---|
| BG000 | NewGam | Participants received NewGam 200-800 mg/kg intravenously every 3 or 4 weeks for 3 months (5 or 4 total infusions, respectively). |
| 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 |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Secondary | Change From Baseline in the Quality of Life (QoL) at the End of the Study | QoL was assessed with the Child Health Questionnaire-Parent Form (CHQ-PF50), completed by a parent or guardian, in participants < 14 years of age at the start of the previous study NGAM-01 and with the Short Form-36 Health Survey (SF-36-HS) in participants ≥ 14 years of age. The CHQ-PF50 consists of 50 items organized into 15 subscales.The 15 subscales could be combined into 2 summary scores, physical and psychosocial. The calculated scores were transformed so that each scale had a range of 0-100. A higher score indicates better health. The SF-36-HS is composed of 36 items. Responses to the 36 items were combined to create 8 scales. The 8 scales could be further combined into 2 scores: Physical component summary and mental component summary. The item and scale scores were transformed to a range of 0-100 with a mean of 50 and a standard deviation of 10 in the general US population. A higher score indicates better health. For both instruments, a positive change indicates improvement. | Safety analysis set: All participants who received at least 1 dose of NewGam. | Posted | Mean | Standard Deviation | Units on a scale | Baseline (follow-up visit of study NGAM-01) to the end of the study (follow-up visit of study NGAM-05) (up to 4 months) |
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Safety analysis set: All participants who received at least 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 | Participants received NewGam 200-800 mg/kg intravenously every 3 or 4 weeks for 3 months (5 or 4 total infusions, respectively). |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Abdominal pain | Gastrointestinal disorders | MedDRA (14.0) | 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 |
|---|---|
| D000081207 | Primary Immunodeficiency Diseases |
| ID | Term |
|---|---|
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D007153 | Immunologic Deficiency Syndromes |
| D007154 | Immune System Diseases |
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|
| Baseline (follow-up visit of study NGAM-01) to the end of the study (follow-up visit of study NGAM-05) (up to 4 months) |
| Centennial |
| Colorado |
| United States |
| Rush Universtity Medical Center | Chicago | Illinois | United States |
| Cardinal Glennon Children's Hospital | St Louis | Missouri | United States |
| Midlands Pediatrics | Papillion | Nebraska | United States |
| Seattle Children's Hospital | Seattle | Washington | United States |
| Years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| ID | Title | Description |
|---|---|---|
| OG000 | NewGam | Participants received NewGam 200-800 mg/kg intravenously every 3 or 4 weeks for 3 months (5 or 4 total infusions, respectively). |
|
|
| Primary | Percentage of Participants Who Experienced at Least 1 Adverse Event Causally Related to the Administration of the Study Drug | An adverse event was considered to be causally related to the administration of the study drug if it judged to be probably or possibly related to the study drug, as assessed by the investigator. | Safety analysis set: All participants who received at least 1 dose of NewGam. | Posted | Number | Percentage of participants | Baseline (follow-up visit of study NGAM-01) to the end of the study (follow-up visit of study NGAM-05) (up to 4 months) |
|
|
|
| Primary | Percentage of Participants Who Experienced at Least 1 Adverse Event Temporally Related to the Study Drug | An adverse event was considered to be temporally related to the study drug if it started during an infusion or within 72 hours after the end of an infusion. | Safety analysis set: All participants who received at least 1 dose of NewGam. | Posted | Number | Percentage of participants | Baseline (follow-up visit of study NGAM-01) to the end of the study (follow-up visit of study NGAM-05) (up to 4 months) |
|
|
|
| 0 |
| 21 |
| 12 |
| 21 |
| Diarrhoea | Gastrointestinal disorders | MedDRA (14.0) | Systematic Assessment |
|
| Nausea | Gastrointestinal disorders | MedDRA (14.0) | Systematic Assessment |
|
| Vomiting | Gastrointestinal disorders | MedDRA (14.0) | Systematic Assessment |
|
| Chest pain | General disorders | MedDRA (14.0) | Systematic Assessment |
|
| Pyrexia | General disorders | MedDRA (14.0) | Systematic Assessment |
|
| Sinusitis | Infections and infestations | MedDRA (14.0) | Systematic Assessment |
|
| Headache | Nervous system disorders | MedDRA (14.0) | Systematic Assessment |
|
| Nasopharyngitis | Infections and infestations | MedDRA (14.0) | Systematic Assessment |
|
| Contusion | Injury, poisoning and procedural complications | MedDRA (14.0) | Systematic Assessment |
|
Octapharma agreements may vary with individual investigators, but will not prohibit any investigator from publishing. Octapharma supports the publication of results from all centers of a multi-center trial but requests that reports based on single-site data not precede the primary publication of the entire clinical trial. Octapharma also reserves the right to review data prior to publishing and provide comments/changes within a certain time period.