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

Primary Immunodeficiency Diseases (PID) Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03716700
• Other study ID #: SHP664-402
• Status: Completed
• Start date: September 24, 2018
• Est. completion date: July 23, 2020

Study information

• Verified date: March 2021
• Source: Takeda
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

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

Recruitment information / eligibility

• Status: Completed
• Enrollment: 126
• Est. completion date: July 23, 2020
• Est. primary completion date: July 23, 2020
• Accepts healthy volunteers: No
• Gender: All
• Age group: 3 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Immunologic Deficiency Syndromes
• Primary Immunodeficiency Diseases (PID)

Intervention

Biological:
• CUVITRU
CUVITRU

Locations

Country	Name	City	State
Canada	University of McMaster	Hamilton	Ontario
Canada	Grand River Allergy	Kitchener	Ontario
Canada	Toronto Allergists	Toronto	Ontario

Sponsors (2)

Lead Sponsor	Collaborator
Baxalta now part of Shire	Baxalta Innovations GmbH, now part of Shire

Country where clinical trial is conducted

Canada, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Infusion parameter 1: Cohort 1-Start of data collection	Median infusion volume per site	Baseline
Primary	Infusion parameter 1: Cohort 1- Month 3	Median infusion volume per site	Month 3
Primary	Infusion parameter 1: Cohort 1- Month 6	Median infusion volume per site	Month 6
Primary	Infusion parameter 1: Cohort 1- 12 Month final follow-up	Median infusion volume per site	12 Month final follow-up
Primary	Infusion parameter 1: Cohort 2- Start of data collection	Median infusion volume per site	Baseline
Primary	Infusion parameter 1: Cohort 2- 12 Month final follow-up	Median infusion volume per site	12 Month final follow-up
Primary	Infusion parameter 1.1: Cohort 1- Start of data collection	Median infusion volume per infusion	Baseline
Primary	Infusion parameter 1.1: Cohort 1- Month 3	Median infusion volume per infusion	Month 3
Primary	Infusion parameter 1.1: Cohort 1- Month 6	Median infusion volume per infusion	Month 6
Primary	Infusion parameter 1.1: Cohort 1- 12 Month final follow-up	Median infusion volume per infusion	12 Month final follow-up
Primary	Infusion parameter 1.1: Cohort 2- Start of data collection	Median infusion volume per infusion	Baseline
Primary	Infusion parameter 1.1: Cohort 2- 12 Month final follow-up	Median infusion volume per infusion	12 Month final follow-up
Primary	Infusion parameter 2: Cohort 1- Start of data collection	Median number of infusion sites	Baseline
Primary	Infusion parameter 2: Cohort 1- Month 3	Median number of infusion sites	Month 3
Primary	Infusion parameter 2: Cohort 1- Month 6	Median number of infusion sites	Month 6
Primary	Infusion parameter 2: Cohort 1- 12 Month final follow-up	Median number of infusion sites	12 Month final follow-up
Primary	Infusion parameter 2: Cohort 2- Start of data collection	Median number of infusion sites	Baseline
Primary	Infusion parameter 2: Cohort 2- 12 Month final follow-up	Median number of infusion sites	12 Month final follow-up
Primary	Infusion parameter 3: Cohort 1- Start of data collection	Median infusion duration	Baseline
Primary	Infusion parameter 3: Cohort 1- Month 3	Median infusion duration	Month 3
Primary	Infusion parameter 3: Cohort 1- Month 6	Median infusion duration	Month 6
Primary	Infusion parameter 3: Cohort 1- 12 Month final follow-up	Median infusion duration	12 Month final follow-up
Primary	Infusion parameter 3: Cohort 2- Start of data collection	Median infusion duration	Baseline
Primary	Infusion parameter 3: Cohort 2- 12 Month final follow-up	Median infusion duration	12 Month final follow-up
Primary	Infusion parameter 3.1: Cohort 1- Month 3 Follow-up	Median number of infusions to reach participant's maximum infusion volume	Month 3
Primary	Infusion parameter 3.1: Cohort 1- Month 6 Follow-up	Median number of infusions to reach participant's maximum infusion volume	Month 6
Primary	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
Primary	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
Primary	Infusion parameter 3.2: Cohort 1- Start of data collection	Median number of infusions per month per participant	Baseline
Primary	Infusion parameter 3.2: Cohort 1- Month 3	Median number of infusions per month per participant	Month 3
Primary	Infusion parameter 3.2: Cohort 1- Month 6	Median number of infusions per month per participant	Month 6
Primary	Infusion parameter 3.2: Cohort 1- 12 Month final follow-up	Median number of infusions per month per participant	12 Month final follow-up
Primary	Infusion parameter 3.2: Cohort 2- Start of data collection	Median number of infusions per month per participant	Baseline
Primary	Infusion parameter 3.2: Cohort 2- 12 Month final follow-up	Median number of infusions per month per participant	12 Month final follow-up
Primary	Infusion parameter 3.3: Cohort 1- Start of data collection	Median number of infusions to reach final dose interval per participant	Baseline
Primary	Infusion parameter 3.3: Cohort 1- Month 3	Median number of infusions to reach final dose interval per participant	Month 3
Primary	Infusion parameter 3.3: Cohort 1- Month 6	Median number of infusions to reach final dose interval per participant	Month 6
Primary	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
Primary	Infusion parameter 3.3: Cohort 2- Start of data collection	Median number of infusions to reach final dose interval per participant	Baseline
Primary	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
Primary	Infusion parameter 1: Cohort 3- 12 Month Final Follow-up	Median infusion volume per site.	12 Month final follow-up
Primary	Infusion parameter 1.1: Cohort 3- 12 Month Final Follow-up	Median infusion volume per infusion.	12 Month final follow-up
Primary	Infusion parameter 2: Cohort 3- 12 Month final follow-up	Median number of infusion sites.	12 Month final follow-up
Primary	Infusion parameter 3: Cohort 3- 12 Month final follow-up	Median infusion duration.	12 Month final follow-up
Primary	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
Primary	Infusion parameter 3.2: Cohort 3- 12 Month final follow-up	Median number of infusions per month per participant	12 Month final follow-up
Primary	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	Infusion parameter 4.1: Cohort 1- Start of data collection	Median maximal infusion rate per site	Baseline
Secondary	Infusion parameter 4.1: Cohort 1- Month 3	Median maximal infusion rate per site	Month 3
Secondary	Infusion parameter 4.1: Cohort 1- Month 6	Median maximal infusion rate per site	Month 6
Secondary	Infusion parameter 4.1: Cohort 1- 12 Month final follow-up	Median maximal infusion rate per site	12 Month final follow-up
Secondary	Infusion parameter 4.1: Cohort 2- Start of data collection	Median maximal infusion rate per site	Baseline
Secondary	Infusion parameter 4.1: Cohort 2- 12 Month final follow-up	Median maximal infusion rate per site	12 Month final follow-up
Secondary	Infusion parameter 4.2: Cohort 1- Start of data collection	Number of infusions that are discontinued, slowed, or interrupted	Baseline
Secondary	Infusion parameter 4.2: Cohort 1- Month 3	Number of infusions that are discontinued, slowed, or interrupted	Month 3
Secondary	Infusion parameter 4.2: Cohort 1- Month 6	Number of infusions that are discontinued, slowed, or interrupted	Month 6
Secondary	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
Secondary	Infusion parameter 4.2: Cohort 2- Start of data collection	Number of infusions that are discontinued, slowed, or interrupted	Baseline
Secondary	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
Secondary	Infusion parameter 4.3: Cohort 1- Month 3	Median number of infusions to reach participant's maximum infusion rate	Month 3
Secondary	Infusion parameter 4.3: Cohort 1- Month 6	Median number of infusions to reach participant's maximum infusion rate	Month 6
Secondary	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
Secondary	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
Secondary	Infusion parameter 5.1: Cohort 1- Start of data collection	Mean dose	Baseline
Secondary	Infusion parameter 5.1: Cohort 1- Month 3	Mean dose	Month 3
Secondary	Infusion parameter 5.1: Cohort 1- Month 6	Mean dose	Month 6
Secondary	Infusion parameter 5.1: Cohort 1- 12 Month final follow-up	Mean dose	12 Month final follow-up
Secondary	Infusion parameter 5.1: Cohort 2- Start of data collection	Mean dose	Baseline
Secondary	Infusion parameter 5.1: Cohort 2- 12 Month final follow-up	Mean dose	12 Month final follow-up
Secondary	Infusion parameter 5.2: Cohort 1- Start of data collection	Mean dosing interval	Baseline
Secondary	Infusion parameter 5.2: Cohort 1- Month 3	Mean dosing interval	Month 3
Secondary	Infusion parameter 5.2: Cohort 1- Month 6	Mean dosing interval	Month 6
Secondary	Infusion parameter 5.2: Cohort 1- 12 Month final follow-up	Mean dosing interval	12 Month final follow-up
Secondary	Infusion parameter 5.2: Cohort 2- Start of data collection	Mean dosing interval	Baseline
Secondary	Infusion parameter 5.2: Cohort 2- 12 Month final follow-up	Mean dosing interval	12 Month final follow-up
Secondary	Infusion parameter 5.3: Cohort 1- Start of data collection	Mean number of dose adjustments	Baseline
Secondary	Infusion parameter 5.3: Cohort 1- Month 3	Mean number of dose adjustments	Month 3
Secondary	Infusion parameter 5.3: Cohort 1- Month 6	Mean number of dose adjustments	Month 6
Secondary	Infusion parameter 5.3: Cohort 1- 12 Month final follow-up	Mean number of dose adjustments	12 Month final follow-up
Secondary	Infusion parameter 5.3: Cohort 2- Start of data collection	Mean number of dose adjustments	Baseline
Secondary	Infusion parameter 5.3: Cohort 2- 12 Month final follow-up	Mean number of dose adjustments	12 Month final follow-up
Secondary	Infusion parameter 4.1: Cohort 3- 12 Month final follow-up	Median maximal infusion rate per site	12 Month final follow-up
Secondary	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
Secondary	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
Secondary	Infusion parameter 5.1: Cohort 3- 12 Month final follow-up	Mean dose	12 Month final follow-up
Secondary	Infusion parameter 5.2: Cohort 3- 12 Month final follow-up	Mean dosing interval	12 Month final follow-up
Secondary	Infusion parameter 5.3: Cohort 3- 12 Month final follow-up	Mean number of dose adjustments	12 Month final follow-up
Secondary	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
Secondary	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
Secondary	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
Secondary	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
Secondary	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
Secondary	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
Secondary	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
Secondary	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
Secondary	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

External Links

•   To obtain more information on the study, click here/on this link