Trial Outcomes & Findings for High Infusion Rate Study of Immunoglobulin Intravenous (Human) 10% (NewGam) (NCT NCT01313507)
NCT ID: NCT01313507
Last Updated: 2017-03-29
Results Overview
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.
COMPLETED
PHASE3
21 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)
2017-03-29
Participant Flow
Participant milestones
| Measure |
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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Overall Study
STARTED
|
21
|
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Overall Study
COMPLETED
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21
|
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Overall Study
NOT COMPLETED
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0
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Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
High Infusion Rate Study of Immunoglobulin Intravenous (Human) 10% (NewGam)
Baseline characteristics by cohort
| Measure |
NewGam
n=21 Participants
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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|---|---|
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Age, Continuous
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23.8 Years
STANDARD_DEVIATION 19.78 • n=5 Participants
|
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Sex: Female, Male
Female
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8 Participants
n=5 Participants
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Sex: Female, Male
Male
|
13 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 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)Population: Safety analysis set: All participants who received at least 1 dose of NewGam.
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.
Outcome measures
| Measure |
NewGam
n=21 Participants
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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|---|---|
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Percentage of Participants Who Experienced at Least 1 Adverse Event Causally Related to the Administration of the Study Drug
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19.0 Percentage of participants
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PRIMARY outcome
Timeframe: 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)Population: Safety analysis set: All participants who received at least 1 dose of NewGam.
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.
Outcome measures
| Measure |
NewGam
n=21 Participants
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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|---|---|
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Percentage of Participants Who Experienced at Least 1 Adverse Event Temporally Related to the Study Drug
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38.1 Percentage of participants
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SECONDARY outcome
Timeframe: 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)Population: Safety analysis set: All participants who received at least 1 dose of NewGam.
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.
Outcome measures
| Measure |
NewGam
n=21 Participants
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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|---|---|
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Change From Baseline in the Quality of Life (QoL) at the End of the Study
CHQ-PF50 - Physical component summary
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-2.63 Units on a scale
Standard Deviation 5.22
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Change From Baseline in the Quality of Life (QoL) at the End of the Study
CHQ-PF50 - Psychosocial component summary
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-2.01 Units on a scale
Standard Deviation 5.78
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Change From Baseline in the Quality of Life (QoL) at the End of the Study
SF-36 - Physical component summary
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0.21 Units on a scale
Standard Deviation 6.00
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Change From Baseline in the Quality of Life (QoL) at the End of the Study
SF-36 - Mental component summary
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0.36 Units on a scale
Standard Deviation 7.33
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Adverse Events
NewGam
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
NewGam
n=21 participants at risk
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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|---|---|
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Gastrointestinal disorders
Abdominal pain
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9.5%
2/21
Safety analysis set: All participants who received at least 1 dose of NewGam.
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Gastrointestinal disorders
Diarrhoea
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9.5%
2/21
Safety analysis set: All participants who received at least 1 dose of NewGam.
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Gastrointestinal disorders
Nausea
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14.3%
3/21
Safety analysis set: All participants who received at least 1 dose of NewGam.
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|
Gastrointestinal disorders
Vomiting
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14.3%
3/21
Safety analysis set: All participants who received at least 1 dose of NewGam.
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|
General disorders
Chest pain
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9.5%
2/21
Safety analysis set: All participants who received at least 1 dose of NewGam.
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General disorders
Pyrexia
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9.5%
2/21
Safety analysis set: All participants who received at least 1 dose of NewGam.
|
|
Infections and infestations
Sinusitis
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19.0%
4/21
Safety analysis set: All participants who received at least 1 dose of NewGam.
|
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Nervous system disorders
Headache
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9.5%
2/21
Safety analysis set: All participants who received at least 1 dose of NewGam.
|
|
Infections and infestations
Nasopharyngitis
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9.5%
2/21
Safety analysis set: All participants who received at least 1 dose of NewGam.
|
|
Injury, poisoning and procedural complications
Contusion
|
9.5%
2/21
Safety analysis set: All participants who received at least 1 dose of NewGam.
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Additional Information
Michael Eppolito, Director, Clinical Operations Immunology and ICU Medicine
Octapharma USA
Results disclosure agreements
- Principal investigator is a sponsor employee 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.
- Publication restrictions are in place
Restriction type: OTHER