Real-world CANadian CUvitru Non-Interventional Study in Subjects Transitioning From Subcutaneous Immunoglobulin (CANCUN)

Study Overview
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Brief Summary
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This study will provide insights on the infusion parameters, dosing, and experience of participants transitioning to CUVITRU in a real-world setting.

Official Title
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Real-world CANadian CUvitru Non-Interventional Study in Subjects Transitioning From Subcutaneous Immunoglobulin (CANCUN)

Conditions
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Primary Immunodeficiency Diseases (PID)

Intervention / Treatment
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* Biological: CUVITRU


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria (The participant will not be considered eligible for the study without meeting all of the criteria below): Voluntarily provide written, signed, and dated (personally or via a legally authorized representative) informed consent or assent as applicable to participate in the study; Participant > 2 years of age with a documented diagnosis of PID or SID requiring IgG replacement therapy, as defined by the International Union of Immunological Societies Scientific Committee 2009 and by diagnostic criteria according to Conley et al., 1999; Participant has received subcutaneous immunoglobulin (SCIG) therapy previous to CUVITRU for at least 3 months; and Participant has received CUVITRU in line with the product specification (CUVITRU Product Monograph (Baxalta Canada Corporation, 2018) at start of study Exclusion Criteria (Participants are excluded from the study if any of the following criteria are met): Participation in any interventional clinical study within the last 30 days Participant participates in a clinical study in parallel during the observation period; and Participant had a dose change 30 days prior to transition to CUVITRU for Cohort 1

Ages Eligible for Study
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Minimum Age: 3 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Cohort 1<br>Cohort1 will include the participants who have been transitioned to CUVITRU at the time of enrollment in the study. | Biological: CUVITRU<br>* CUVITRU<br>* Other names: Immune Globulin Subcutaneous (Human);|
| Cohort 2<br>Cohort 2 will include participants 6 months (±2 weeks) after CUVITRU initiation. | Biological: CUVITRU<br>* CUVITRU<br>* Other names: Immune Globulin Subcutaneous (Human);|
| Cohort 3<br>Cohort 3 will include participants 12 months (-1 or +2 months) after CUVITRU initiation. | Biological: CUVITRU<br>* CUVITRU<br>* Other names: Immune Globulin Subcutaneous (Human);|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Infusion parameter 1: Cohort 1-Start of data collection | Median infusion volume per site | Baseline | 
| Infusion parameter 1: Cohort 1- Month 3 | Median infusion volume per site | Month 3 | 
| Infusion parameter 1: Cohort 1- Month 6 | Median infusion volume per site | Month 6 | 
| Infusion parameter 1: Cohort 1- 12 Month final follow-up | Median infusion volume per site | 12 Month final follow-up | 
| Infusion parameter 1: Cohort 2- Start of data collection | Median infusion volume per site | Baseline | 
| Infusion parameter 1: Cohort 2- 12 Month final follow-up | Median infusion volume per site | 12 Month final follow-up | 
| Infusion parameter 1.1: Cohort 1- Start of data collection | Median infusion volume per infusion | Baseline | 
| Infusion parameter 1.1: Cohort 1- Month 3 | Median infusion volume per infusion | Month 3 | 
| Infusion parameter 1.1: Cohort 1- Month 6 | Median infusion volume per infusion | Month 6 | 
| Infusion parameter 1.1: Cohort 1- 12 Month final follow-up | Median infusion volume per infusion | 12 Month final follow-up | 
| Infusion parameter 1.1: Cohort 2- Start of data collection | Median infusion volume per infusion | Baseline | 
| Infusion parameter 1.1: Cohort 2- 12 Month final follow-up | Median infusion volume per infusion | 12 Month final follow-up | 
| Infusion parameter 2: Cohort 1- Start of data collection | Median number of infusion sites | Baseline | 
| Infusion parameter 2: Cohort 1- Month 3 | Median number of infusion sites | Month 3 | 
| Infusion parameter 2: Cohort 1- Month 6 | Median number of infusion sites | Month 6 | 
| Infusion parameter 2: Cohort 1- 12 Month final follow-up | Median number of infusion sites | 12 Month final follow-up | 
| Infusion parameter 2: Cohort 2- Start of data collection | Median number of infusion sites | Baseline | 
| Infusion parameter 2: Cohort 2- 12 Month final follow-up | Median number of infusion sites | 12 Month final follow-up | 
| Infusion parameter 3: Cohort 1- Start of data collection | Median infusion duration | Baseline | 
| Infusion parameter 3: Cohort 1- Month 3 | Median infusion duration | Month 3 | 
| Infusion parameter 3: Cohort 1- Month 6 | Median infusion duration | Month 6 | 
| Infusion parameter 3: Cohort 1- 12 Month final follow-up | Median infusion duration | 12 Month final follow-up | 
| Infusion parameter 3: Cohort 2- Start of data collection | Median infusion duration | Baseline | 
| Infusion parameter 3: Cohort 2- 12 Month final follow-up | Median infusion duration | 12 Month final follow-up | 
| Infusion parameter 3.1: Cohort 1- Month 3 Follow-up | Median number of infusions to reach participant's maximum infusion volume | Month 3 | 
| Infusion parameter 3.1: Cohort 1- Month 6 Follow-up | Median number of infusions to reach participant's maximum infusion volume | Month 6 | 
| Infusion parameter 3.1: Cohort 1- 12 Month final follow-up | Median number of infusions to reach participant's maximum infusion volume | 12 Month final follow-up | 
| Infusion parameter 3.1: Cohort 2- 12 Month final follow-up | Median number of infusions to reach participant's maximum infusion volume | 12 Month final follow-up | 
| Infusion parameter 3.2: Cohort 1- Start of data collection | Median number of infusions per month per participant | Baseline | 
| Infusion parameter 3.2: Cohort 1- Month 3 | Median number of infusions per month per participant | Month 3 | 
| Infusion parameter 3.2: Cohort 1- Month 6 | Median number of infusions per month per participant | Month 6 | 
| Infusion parameter 3.2: Cohort 1- 12 Month final follow-up | Median number of infusions per month per participant | 12 Month final follow-up | 
| Infusion parameter 3.2: Cohort 2- Start of data collection | Median number of infusions per month per participant | Baseline | 
| Infusion parameter 3.2: Cohort 2- 12 Month final follow-up | Median number of infusions per month per participant | 12 Month final follow-up | 
| Infusion parameter 3.3: Cohort 1- Start of data collection | Median number of infusions to reach final dose interval per participant | Baseline | 
| Infusion parameter 3.3: Cohort 1- Month 3 | Median number of infusions to reach final dose interval per participant | Month 3 | 
| Infusion parameter 3.3: Cohort 1- Month 6 | Median number of infusions to reach final dose interval per participant | Month 6 | 
| Infusion parameter 3.3: Cohort 1- 12 Month final follow-up | Median number of infusions to reach final dose interval per participant | 12 Month final follow-up | 
| Infusion parameter 3.3: Cohort 2- Start of data collection | Median number of infusions to reach final dose interval per participant | Baseline | 
| Infusion parameter 3.3: Cohort 2- 12 Month final follow-up | Median number of infusions to reach final dose interval per participant | 12 Month final follow-up | 
| Infusion parameter 1: Cohort 3- 12 Month Final Follow-up | Median infusion volume per site. | 12 Month final follow-up | 
| Infusion parameter 1.1: Cohort 3- 12 Month Final Follow-up | Median infusion volume per infusion. | 12 Month final follow-up | 
| Infusion parameter 2: Cohort 3- 12 Month final follow-up | Median number of infusion sites. | 12 Month final follow-up | 
| Infusion parameter 3: Cohort 3- 12 Month final follow-up | Median infusion duration. | 12 Month final follow-up | 
| Infusion parameter 3.1: Cohort 3- 12 Month final follow-up | Median number of infusions to reach participant's maximum infusion volume (infusion rate) | 12 Month final follow-up | 
| Infusion parameter 3.2: Cohort 3- 12 Month final follow-up | Median number of infusions per month per participant | 12 Month final follow-up | 
| Infusion parameter 3.3: Cohort 3- 12 Month final follow-up | Median number of infusions to reach final dose interval per participant | 12 Month final follow-up | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Infusion parameter 4.1: Cohort 1- Start of data collection | Median maximal infusion rate per site | Baseline | 
| Infusion parameter 4.1: Cohort 1- Month 3 | Median maximal infusion rate per site | Month 3 | 
| Infusion parameter 4.1: Cohort 1- Month 6 | Median maximal infusion rate per site | Month 6 | 
| Infusion parameter 4.1: Cohort 1- 12 Month final follow-up | Median maximal infusion rate per site | 12 Month final follow-up | 
| Infusion parameter 4.1: Cohort 2- Start of data collection | Median maximal infusion rate per site | Baseline | 
| Infusion parameter 4.1: Cohort 2- 12 Month final follow-up | Median maximal infusion rate per site | 12 Month final follow-up | 
| Infusion parameter 4.2: Cohort 1- Start of data collection | Number of infusions that are discontinued, slowed, or interrupted | Baseline | 
| Infusion parameter 4.2: Cohort 1- Month 3 | Number of infusions that are discontinued, slowed, or interrupted | Month 3 | 
| Infusion parameter 4.2: Cohort 1- Month 6 | Number of infusions that are discontinued, slowed, or interrupted | Month 6 | 
| Infusion parameter 4.2: Cohort 1- 12 Month final follow-up | Number of infusions that are discontinued, slowed, or interrupted | 12 Month final follow-up | 
| Infusion parameter 4.2: Cohort 2- Start of data collection | Number of infusions that are discontinued, slowed, or interrupted | Baseline | 
| Infusion parameter 4.2: Cohort 2- 12 Month final follow-up | Number of infusions that are discontinued, slowed, or interrupted | 12 Month final follow-up | 
| Infusion parameter 4.3: Cohort 1- Month 3 | Median number of infusions to reach participant's maximum infusion rate | Month 3 | 
| Infusion parameter 4.3: Cohort 1- Month 6 | Median number of infusions to reach participant's maximum infusion rate | Month 6 | 
| Infusion parameter 4.3: Cohort 1- 12 Month final follow-up | Median number of infusions to reach participant's maximum infusion rate | 12 Month final follow-up | 
| Infusion parameter 4.3: Cohort 2- 12 Month final follow-up | Median number of infusions to reach participant's maximum infusion rate | 12 Month final follow-up | 
| Infusion parameter 5.1: Cohort 1- Start of data collection | Mean dose | Baseline | 
| Infusion parameter 5.1: Cohort 1- Month 3 | Mean dose | Month 3 | 
| Infusion parameter 5.1: Cohort 1- Month 6 | Mean dose | Month 6 | 
| Infusion parameter 5.1: Cohort 1- 12 Month final follow-up | Mean dose | 12 Month final follow-up | 
| Infusion parameter 5.1: Cohort 2- Start of data collection | Mean dose | Baseline | 
| Infusion parameter 5.1: Cohort 2- 12 Month final follow-up | Mean dose | 12 Month final follow-up | 
| Infusion parameter 5.2: Cohort 1- Start of data collection | Mean dosing interval | Baseline | 
| Infusion parameter 5.2: Cohort 1- Month 3 | Mean dosing interval | Month 3 | 
| Infusion parameter 5.2: Cohort 1- Month 6 | Mean dosing interval | Month 6 | 
| Infusion parameter 5.2: Cohort 1- 12 Month final follow-up | Mean dosing interval | 12 Month final follow-up | 
| Infusion parameter 5.2: Cohort 2- Start of data collection | Mean dosing interval | Baseline | 
| Infusion parameter 5.2: Cohort 2- 12 Month final follow-up | Mean dosing interval | 12 Month final follow-up | 
| Infusion parameter 5.3: Cohort 1- Start of data collection | Mean number of dose adjustments | Baseline | 
| Infusion parameter 5.3: Cohort 1- Month 3 | Mean number of dose adjustments | Month 3 | 
| Infusion parameter 5.3: Cohort 1- Month 6 | Mean number of dose adjustments | Month 6 | 
| Infusion parameter 5.3: Cohort 1- 12 Month final follow-up | Mean number of dose adjustments | 12 Month final follow-up | 
| Infusion parameter 5.3: Cohort 2- Start of data collection | Mean number of dose adjustments | Baseline | 
| Infusion parameter 5.3: Cohort 2- 12 Month final follow-up | Mean number of dose adjustments | 12 Month final follow-up | 
| Infusion parameter 4.1: Cohort 3- 12 Month final follow-up | Median maximal infusion rate per site | 12 Month final follow-up | 
| Infusion parameter 4.2: Cohort 3- 12 Month final follow-up | Number of infusions that are discontinued, slowed, or interrupted | 12 Month final follow-up | 
| Infusion parameter 4.3: Cohort 3- 12 Month final follow-up | Median number of infusions to reach participant's maximum infusion rate | 12 Month final follow-up | 
| Infusion parameter 5.1: Cohort 3- 12 Month final follow-up | Mean dose | 12 Month final follow-up | 
| Infusion parameter 5.2: Cohort 3- 12 Month final follow-up | Mean dosing interval | 12 Month final follow-up | 
| Infusion parameter 5.3: Cohort 3- 12 Month final follow-up | Mean number of dose adjustments | 12 Month final follow-up | 
| Treatment Satisfaction Questionnaire for Medication-9 (TSQM-9): Cohort 1 | TSQM-9 is a 9-item, validated, self-administered instrument used to assess participant's satisfaction with medication. The three domains assessed are effectiveness (3 items), convenience (3 items), and global satisfaction (3 items). Scores for each domain are calculated by adding up the items in each domain and then transforming the composite score into a value ranging from 0 to 100. Higher score indicated greater satisfaction in that domain. | 12 Month final follow-up | 
| Treatment Satisfaction Questionnaire for Medication-9 (TSQM-9): Cohort 2 | TSQM-9 is a 9-item, validated, self-administered instrument used to assess participant's satisfaction with medication. The three domains assessed are effectiveness (3 items), convenience (3 items), and global satisfaction (3 items). Scores for each domain are calculated by adding up the items in each domain and then transforming the composite score into a value ranging from 0 to 100. Higher score indicated greater satisfaction in that domain. | 12 Month final follow-up | 
| Treatment Satisfaction Questionnaire for Medication-9 (TSQM-9): Cohort 3 | TSQM-9 is a 9-item, validated, self-administered instrument used to assess participant's satisfaction with medication. The three domains assessed are effectiveness (3 items), convenience (3 items), and global satisfaction (3 items). Scores for each domain are calculated by adding up the items in each domain and then transforming the composite score into a value ranging from 0 to 100. Higher score indicated greater satisfaction in that domain. | 12 Month final follow-up | 
| Life Quality Index (LQI): Cohort 1 | The LQI is a self-administered questionnaire developed specifically for participants/legal guardians involved in IVIG treatments. It consists of 15-items, divided into four domains: treatment interferences (6 items), therapy-related problems (4 items), therapy setting (3 items), and treatment costs (2 items). Items are rated on a 7-point Likert-type scale ranging from 1: Extremely bad to 7: Extremely good. Total scores range from 15 to 105, with higher scores indicating the highest possible satisfaction with factors such as independence, therapy convenience, social/school/work activities, and health and travel costs. | 12 Month final follow-up | 
| Life Quality Index (LQI): Cohort 2 | The LQI is a self-administered questionnaire developed specifically for participants/legal guardians involved in IVIG treatments. It consists of 15-items, divided into four domains: treatment interferences (6 items), therapy-related problems (4 items), therapy setting (3 items), and treatment costs (2 items). Items are rated on a 7-point Likert-type scale ranging from 1: Extremely bad to 7: Extremely good. Total scores range from 15 to 105, with higher scores indicating the highest possible satisfaction with factors such as independence, therapy convenience, social/school/work activities, and health and travel costs | 12 Month final follow-up | 
| Life Quality Index (LQI): Cohort 3 | The LQI is a self-administered questionnaire developed specifically for participants/legal guardians involved in IVIG treatments. It consists of 15-items, divided into four domains: treatment interferences (6 items), therapy-related problems (4 items), therapy setting (3 items), and treatment costs (2 items). Items are rated on a 7-point Likert-type scale ranging from 1: Extremely bad to 7: Extremely good. Total scores range from 15 to 105, with higher scores indicating the highest possible satisfaction with factors such as independence, therapy convenience, social/school/work activities, and health and travel costs | 12 Month final follow-up | 
| Treatment Preference Questionnaire (TPQ): Cohort 1 | The TPQ is a self-administered questionnaire developed to assess participants' preference towards the administration of new subcutaneous immunoglobulin G (SCIG) therapy. There are 4-items on the questionnaire, which investigate a participant's reference on the clinic/hospital/home setting of receiving the immunoglobulin therapy, the participant's rating on the frequency and method of administration, and the participant's preference to continue receiving the SCIG treatment. | 12 Month final follow-up | 
| Treatment Preference Questionnaire (TPQ): Cohort 2 | The TPQ is a self-administered questionnaire developed to assess participants' preference towards the administration of new subcutaneous immunoglobulin G (SCIG) therapy. There are 4-items on the questionnaire, which investigate a participant's preference on the clinic/hospital/home setting of receiving the immunoglobulin therapy, the participant's rating on the frequency and method of administration, and the participant's preference to continue receiving the SCIG treatment. | 12 Month final follow-up | 
| Treatment Preference Questionnaire (TPQ): Cohort 3 | The TPQ is a self-administered questionnaire developed to assess participants' preference towards the administration of new subcutaneous immunoglobulin G (SCIG) therapy. There are 4-items on the questionnaire, which investigate a participant's preference on the clinic/hospital/home setting of receiving the immunoglobulin therapy, the participant's rating on the frequency and method of administration, and the participant's preference to continue receiving the SCIG treatment. | 12 Month final follow-up |