Primary Immunodeficiency Diseases (PID)
Conditions
Brief summary
This study will provide insights on the infusion parameters, dosing, and experience of participants transitioning to CUVITRU in a real-world setting.
Interventions
BIOLOGICALCUVITRU
CUVITRU
Sponsors
Baxalta Innovations GmbH, now part of Shire
Baxalta now part of Shire
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
3 Years to No maximum
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Infusion parameter 1: Cohort 3- 12 Month Final Follow-up | 12 Month final follow-up | Median infusion volume per site. |
| Infusion parameter 1.1: Cohort 3- 12 Month Final Follow-up | 12 Month final follow-up | Median infusion volume per infusion. |
| Infusion parameter 1: Cohort 1-Start of data collection | Baseline | Median infusion volume per site |
| Infusion parameter 1: Cohort 1- Month 3 | Month 3 | Median infusion volume per site |
| Infusion parameter 1: Cohort 1- Month 6 | Month 6 | Median infusion volume per site |
| Infusion parameter 1: Cohort 1- 12 Month final follow-up | 12 Month final follow-up | Median infusion volume per site |
| Infusion parameter 1: Cohort 2- Start of data collection | Baseline | Median infusion volume per site |
| Infusion parameter 1: Cohort 2- 12 Month final follow-up | 12 Month final follow-up | Median infusion volume per site |
| Infusion parameter 1.1: Cohort 1- Start of data collection | Baseline | Median infusion volume per infusion |
| Infusion parameter 1.1: Cohort 1- Month 3 | Month 3 | Median infusion volume per infusion |
| Infusion parameter 1.1: Cohort 1- Month 6 | Month 6 | Median infusion volume per infusion |
| Infusion parameter 3.3: Cohort 2- 12 Month final follow-up | 12 Month final follow-up | Median number of infusions to reach final dose interval per participant |
| Infusion parameter 1.1: Cohort 1- 12 Month final follow-up | 12 Month final follow-up | Median infusion volume per infusion |
| Infusion parameter 1.1: Cohort 2- Start of data collection | Baseline | Median infusion volume per infusion |
| Infusion parameter 1.1: Cohort 2- 12 Month final follow-up | 12 Month final follow-up | Median infusion volume per infusion |
| Infusion parameter 2: Cohort 1- Start of data collection | Baseline | Median number of infusion sites |
| Infusion parameter 2: Cohort 1- Month 3 | Month 3 | Median number of infusion sites |
| Infusion parameter 2: Cohort 1- Month 6 | Month 6 | Median number of infusion sites |
| Infusion parameter 2: Cohort 1- 12 Month final follow-up | 12 Month final follow-up | Median number of infusion sites |
| Infusion parameter 2: Cohort 2- Start of data collection | Baseline | Median number of infusion sites |
| Infusion parameter 2: Cohort 2- 12 Month final follow-up | 12 Month final follow-up | Median number of infusion sites |
| Infusion parameter 3: Cohort 1- Start of data collection | Baseline | Median infusion duration |
| Infusion parameter 3: Cohort 1- Month 3 | Month 3 | Median infusion duration |
| Infusion parameter 3: Cohort 1- Month 6 | Month 6 | Median infusion duration |
| Infusion parameter 3: Cohort 1- 12 Month final follow-up | 12 Month final follow-up | Median infusion duration |
| Infusion parameter 3: Cohort 2- Start of data collection | Baseline | Median infusion duration |
| Infusion parameter 3: Cohort 2- 12 Month final follow-up | 12 Month final follow-up | Median infusion duration |
| Infusion parameter 3.1: Cohort 1- Month 3 Follow-up | Month 3 | Median number of infusions to reach participant's maximum infusion volume |
| Infusion parameter 3.1: Cohort 1- Month 6 Follow-up | Month 6 | Median number of infusions to reach participant's maximum infusion volume |
| Infusion parameter 3.1: Cohort 1- 12 Month final follow-up | 12 Month final follow-up | Median number of infusions to reach participant's maximum infusion volume |
| Infusion parameter 3.1: Cohort 2- 12 Month final follow-up | 12 Month final follow-up | Median number of infusions to reach participant's maximum infusion volume |
| Infusion parameter 3.2: Cohort 1- Start of data collection | Baseline | Median number of infusions per month per participant |
| Infusion parameter 3.2: Cohort 1- Month 3 | Month 3 | Median number of infusions per month per participant |
| Infusion parameter 3.2: Cohort 1- Month 6 | Month 6 | Median number of infusions per month per participant |
| Infusion parameter 3.2: Cohort 1- 12 Month final follow-up | 12 Month final follow-up | Median number of infusions per month per participant |
| Infusion parameter 3.2: Cohort 2- Start of data collection | Baseline | Median number of infusions per month per participant |
| Infusion parameter 3.2: Cohort 2- 12 Month final follow-up | 12 Month final follow-up | Median number of infusions per month per participant |
| Infusion parameter 3.3: Cohort 1- Start of data collection | Baseline | Median number of infusions to reach final dose interval per participant |
| Infusion parameter 3.3: Cohort 1- Month 3 | Month 3 | Median number of infusions to reach final dose interval per participant |
| Infusion parameter 3.3: Cohort 1- Month 6 | Month 6 | Median number of infusions to reach final dose interval per participant |
| Infusion parameter 3.3: Cohort 1- 12 Month final follow-up | 12 Month final follow-up | Median number of infusions to reach final dose interval per participant |
| Infusion parameter 3.3: Cohort 2- Start of data collection | Baseline | Median number of infusions to reach final dose interval per participant |
| Infusion parameter 2: Cohort 3- 12 Month final follow-up | 12 Month final follow-up | Median number of infusion sites. |
| Infusion parameter 3: Cohort 3- 12 Month final follow-up | 12 Month final follow-up | Median infusion duration. |
| Infusion parameter 3.1: Cohort 3- 12 Month final follow-up | 12 Month final follow-up | Median number of infusions to reach participant's maximum infusion volume (infusion rate) |
| Infusion parameter 3.2: Cohort 3- 12 Month final follow-up | 12 Month final follow-up | Median number of infusions per month per participant |
| Infusion parameter 3.3: Cohort 3- 12 Month final follow-up | 12 Month final follow-up | Median number of infusions to reach final dose interval per participant |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Treatment Preference Questionnaire (TPQ): Cohort 2 | 12 Month final follow-up | 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. |
| Infusion parameter 4.1: Cohort 3- 12 Month final follow-up | 12 Month final follow-up | Median maximal infusion rate per site |
| Treatment Preference Questionnaire (TPQ): Cohort 3 | 12 Month final follow-up | 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. |
| Infusion parameter 4.2: Cohort 3- 12 Month final follow-up | 12 Month final follow-up | Number of infusions that are discontinued, slowed, or interrupted |
| Infusion parameter 4.1: Cohort 1- Start of data collection | Baseline | Median maximal infusion rate per site |
| Infusion parameter 4.1: Cohort 1- Month 3 | Month 3 | Median maximal infusion rate per site |
| Infusion parameter 4.1: Cohort 1- Month 6 | Month 6 | Median maximal infusion rate per site |
| Infusion parameter 4.1: Cohort 1- 12 Month final follow-up | 12 Month final follow-up | Median maximal infusion rate per site |
| Infusion parameter 4.1: Cohort 2- Start of data collection | Baseline | Median maximal infusion rate per site |
| Infusion parameter 4.1: Cohort 2- 12 Month final follow-up | 12 Month final follow-up | Median maximal infusion rate per site |
| Infusion parameter 4.2: Cohort 1- Start of data collection | Baseline | Number of infusions that are discontinued, slowed, or interrupted |
| Infusion parameter 4.2: Cohort 1- Month 3 | Month 3 | Number of infusions that are discontinued, slowed, or interrupted |
| Infusion parameter 4.2: Cohort 1- Month 6 | Month 6 | Number of infusions that are discontinued, slowed, or interrupted |
| Infusion parameter 4.2: Cohort 1- 12 Month final follow-up | 12 Month final follow-up | Number of infusions that are discontinued, slowed, or interrupted |
| Infusion parameter 4.2: Cohort 2- Start of data collection | Baseline | Number of infusions that are discontinued, slowed, or interrupted |
| Infusion parameter 4.2: Cohort 2- 12 Month final follow-up | 12 Month final follow-up | Number of infusions that are discontinued, slowed, or interrupted |
| Infusion parameter 4.3: Cohort 1- Month 3 | Month 3 | Median number of infusions to reach participant's maximum infusion rate |
| Infusion parameter 4.3: Cohort 1- Month 6 | Month 6 | Median number of infusions to reach participant's maximum infusion rate |
| Infusion parameter 4.3: Cohort 1- 12 Month final follow-up | 12 Month final follow-up | Median number of infusions to reach participant's maximum infusion rate |
| Infusion parameter 4.3: Cohort 2- 12 Month final follow-up | 12 Month final follow-up | Median number of infusions to reach participant's maximum infusion rate |
| Infusion parameter 5.1: Cohort 1- Start of data collection | Baseline | Mean dose |
| Infusion parameter 5.1: Cohort 1- Month 3 | Month 3 | Mean dose |
| Infusion parameter 5.1: Cohort 1- Month 6 | Month 6 | Mean dose |
| Infusion parameter 5.1: Cohort 1- 12 Month final follow-up | 12 Month final follow-up | Mean dose |
| Infusion parameter 5.1: Cohort 2- Start of data collection | Baseline | Mean dose |
| Infusion parameter 5.1: Cohort 2- 12 Month final follow-up | 12 Month final follow-up | Mean dose |
| Infusion parameter 5.2: Cohort 1- Start of data collection | Baseline | Mean dosing interval |
| Infusion parameter 5.2: Cohort 1- Month 3 | Month 3 | Mean dosing interval |
| Infusion parameter 5.2: Cohort 1- Month 6 | Month 6 | Mean dosing interval |
| Infusion parameter 5.2: Cohort 1- 12 Month final follow-up | 12 Month final follow-up | Mean dosing interval |
| Infusion parameter 5.2: Cohort 2- Start of data collection | Baseline | Mean dosing interval |
| Infusion parameter 5.2: Cohort 2- 12 Month final follow-up | 12 Month final follow-up | Mean dosing interval |
| Infusion parameter 5.3: Cohort 1- Start of data collection | Baseline | Mean number of dose adjustments |
| Infusion parameter 5.3: Cohort 1- Month 3 | Month 3 | Mean number of dose adjustments |
| Infusion parameter 5.3: Cohort 1- Month 6 | Month 6 | Mean number of dose adjustments |
| Infusion parameter 5.3: Cohort 1- 12 Month final follow-up | 12 Month final follow-up | Mean number of dose adjustments |
| Infusion parameter 5.3: Cohort 2- Start of data collection | Baseline | Mean number of dose adjustments |
| Infusion parameter 5.3: Cohort 2- 12 Month final follow-up | 12 Month final follow-up | Mean number of dose adjustments |
| Infusion parameter 4.3: Cohort 3- 12 Month final follow-up | 12 Month final follow-up | Median number of infusions to reach participant's maximum infusion rate |
| Infusion parameter 5.1: Cohort 3- 12 Month final follow-up | 12 Month final follow-up | Mean dose |
| Infusion parameter 5.2: Cohort 3- 12 Month final follow-up | 12 Month final follow-up | Mean dosing interval |
| Infusion parameter 5.3: Cohort 3- 12 Month final follow-up | 12 Month final follow-up | Mean number of dose adjustments |
| Treatment Satisfaction Questionnaire for Medication-9 (TSQM-9): Cohort 1 | 12 Month final follow-up | 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. |
| Treatment Satisfaction Questionnaire for Medication-9 (TSQM-9): Cohort 2 | 12 Month final follow-up | 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. |
| Treatment Satisfaction Questionnaire for Medication-9 (TSQM-9): Cohort 3 | 12 Month final follow-up | 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. |
| Life Quality Index (LQI): Cohort 1 | 12 Month final follow-up | 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. |
| Life Quality Index (LQI): Cohort 2 | 12 Month final follow-up | 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 |
| Life Quality Index (LQI): Cohort 3 | 12 Month final follow-up | 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 |
| Treatment Preference Questionnaire (TPQ): Cohort 1 | 12 Month final follow-up | 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. |
Countries
Canada
Outcome results
None listed