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

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

Study Overview

• Status: Completed
• Conditions: Primary Immunodeficiency Diseases (PID)
• Intervention / Treatment: Biological: CUVITRU

• Study Type: Observational
• Enrollment (Actual): 126

Contacts and Locations

Study Locations

• Canada
  • Ontario
    • Hamilton, Ontario, Canada, L8S 4L8
      • University of McMaster
    • Kitchener, Ontario, Canada, N2M 5E2
      • Grand River Allergy
    • Toronto, Ontario, Canada, M5G 1E2
      • Toronto Allergists

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 3 years and older (ADULT, OLDER_ADULT, CHILD)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

• Sampling Method: Non-Probability Sample

Study Population

Data will be collected on approximately 500 participants with primary immunodeficiency (PID) and secondary immunodeficiency (SID) requiring Immunoglobulin G (IgG) replacement therapy across 8-10 sites in Canada (excluding Quebec). Any specific number of participants in each cohort is not a requirement.

Description

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

Study Plan

How is the study designed?

Number of groups / cohorts

3

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Cohort 1; Cohort1 will include the participants who have been transitioned to CUVITRU at the time of enrollment in the study.	Biological: CUVITRU; CUVITRU; Other Names: IGSC; 20% Solution; Immune Globulin Subcutaneous (Human)
Cohort 2; Cohort 2 will include participants 6 months (±2 weeks) after CUVITRU initiation.	Biological: CUVITRU; CUVITRU; Other Names: IGSC; 20% Solution; Immune Globulin Subcutaneous (Human)
Cohort 3; Cohort 3 will include participants 12 months (-1 or +2 months) after CUVITRU initiation.	Biological: CUVITRU; CUVITRU; Other Names: IGSC; 20% Solution; Immune Globulin Subcutaneous (Human)

What is the study measuring?

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

Collaborators and Investigators

• Sponsor: Baxalta now part of Shire
• Collaborators: Baxalta Innovations GmbH, now part of Shire

Publications and helpful links

General Publications

• Keith PK, Cowan J, Kanani A, Kim H, Lacuesta G, Lee JK, Chen J, Park M, Gladiator A. Transitioning subcutaneous immunoglobulin 20% therapies in patients with primary and secondary immunodeficiencies: Canadian real-world study. Allergy Asthma Clin Immunol. 2022 Aug 7;18(1):70. doi: 10.1186/s13223-022-00709-8.

Helpful Links

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

Study record dates

Study Major Dates

• Study Start (ACTUAL): September 24, 2018
• Primary Completion (ACTUAL): July 23, 2020
• Study Completion (ACTUAL): July 23, 2020

Study Registration Dates

• First Submitted: August 31, 2018
• First Submitted That Met QC Criteria: October 19, 2018
• First Posted (ACTUAL): October 23, 2018

Study Record Updates

• Last Update Posted (ACTUAL): March 5, 2021
• Last Update Submitted That Met QC Criteria: March 3, 2021
• Last Verified: March 1, 2021

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Immune System Diseases; Genetic Diseases, Inborn; Immunologic Deficiency Syndromes; Primary Immunodeficiency Diseases; Physiological Effects of Drugs; Immunologic Factors; Immunoglobulins; Immunoglobulins, Intravenous; gamma-Globulins; Rho(D) Immune Globulin
• Other Study ID Numbers: SHP664-402

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.
• IPD Sharing Access Criteria: IPD from eligible studies will be shared with qualified researchers according to the criteria and process described on https://vivli.org/ourmember/takeda/. For approved requests, the researchers will be provided access to anonymized data (to respect patient privacy in line with applicable laws and regulations) and with information necessary to address the research objectives under the terms of a data sharing agreement.
• IPD Sharing Supporting Information Type: STUDY_PROTOCOL; SAP; ICF; CSR

Drug and device information, study documents

• Studies a U.S. FDA-regulated device product: No