High Infusion Rate Study of Immunoglobulin Intravenous (Human) 10% (NewGam)

Study Overview
=================
Brief Summary
-----------------
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.

Detailed Description
-----------------
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.

Official Title
-----------------
Clinical Study to Evaluate the Safety and Tolerability of Immunoglobulin Intravenous (Human) 10% (NewGam) Administered at High Infusion Rates to Patients With Primary Immunodeficiency Diseases (Extension of Study NGAM-01)

Conditions
-----------------
Primary Immunodeficiency Disease

Intervention / Treatment
-----------------
* Biological: NewGam


Participation Criteria
=================
Eligibility Criteria
-----------------
Inclusion Criteria: Completion of the main study NGAM-01. At each of the last 3 infusions in the main study NGAM-01, administration of NewGam at the maximum infusion rate of 0.08 mL/kg/min and without the need for premedication. Exclusion Criteria: Any condition or circumstance that would have led to the exclusion of the subject from the NGAM-01 study. Administration of any immunoglobulin infusion other than NewGam between conclusion of the NGAM-01 study and the beginning of the present study. A deviation of the subject's treatment interval of more than 7 days between the last infusion of NewGam in the NGAM-01 study and the first infusion of NewGam in the present study.

Ages Eligible for Study
-----------------
Minimum Age: 2 Years
Maximum Age: 75 Years

Sexes Eligible for Study
-----------------
All

Accepts Healthy Volunteers
-----------------
No

Study Plan
=================
How is the study designed?
-----------------

Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: NewGam<br>Participants received NewGam 200-800 mg/kg intravenously every 3 or 4 weeks for 3 months (5 or 4 total infusions, respectively). | Biological: NewGam<br>* 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.<br>|

What is the study measuring?
-----------------
Primary Outcome Measures

| Outcome Measure | 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) | 

Secondary Outcome Measures

| Outcome Measure | 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. | 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) | 

 Terms related to the study
=================
Keywords Provided by Centre Hospitalier Valida
-----------------
PID, Primary Immunodeficiency Disease