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Primary Immunodeficiency Diseases (PID)NCT03716700

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

An observational study of Primary Immunodeficiency Diseases (PID), run by Baxalta now part of Shire at 3 sites in Canada.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

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

Who is it for?
Ages 3 years and older
Conditions the study is for
Primary Immunodeficiency Diseases (PID)
What is being tested?
CUVITRU
Type of treatment
Injection or infusion
How many people
126 participants
Where
3 sitesCanada
Study dates
24 Sep 2018to 23 Jul 2020
Phase
Observational
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 3 years and older
  • All sexes
  • No healthy volunteers
  • Child, Adult, Older adult

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.

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

Trial locations and contacts

3 sites in 1 country.

Canada3 sites

  • University of McMaster

    Hamilton, Ontario, L8S 4L8

  • Grand River Allergy

    Kitchener, Ontario, N2M 5E2

  • Toronto Allergists

    Toronto, Ontario, M5G 1E2

Other trials in Canada

About this study

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

Being tested
CUVITRU
Medical subject headings
Primary Immunodeficiency Diseases

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • CUVITRUBiological

    Also called 20% Solution, IGSC, Immune Globulin Subcutaneous (Human)

    CUVITRU

Study groups in NCT03716700
Study groupRoleWhat they receive
Cohort 1Cohort1 will include the participants who have been transitioned to CUVITRU at the time of enrollment in the study.
Receives: Biological: CUVITRU
Cohort 2Cohort 2 will include participants 6 months (±2 weeks) after CUVITRU initiation.
Receives: Biological: CUVITRU
Cohort 3Cohort 3 will include participants 12 months (-1 or +2 months) after CUVITRU initiation.
Receives: Biological: CUVITRU

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Infusion parameter 1: Cohort 1-Start of data collection

    Median infusion volume per site

    Measured: Baseline

  2. Infusion parameter 1: Cohort 1- Month 3

    Median infusion volume per site

    Measured: Month 3

  3. Infusion parameter 1: Cohort 1- Month 6

    Median infusion volume per site

    Measured: Month 6

  4. Infusion parameter 1: Cohort 1- 12 Month final follow-up

    Median infusion volume per site

    Measured: 12 Month final follow-up

  5. Infusion parameter 1: Cohort 2- Start of data collection

    Median infusion volume per site

    Measured: Baseline

  6. Infusion parameter 1: Cohort 2- 12 Month final follow-up

    Median infusion volume per site

    Measured: 12 Month final follow-up

  7. Infusion parameter 1.1: Cohort 1- Start of data collection

    Median infusion volume per infusion

    Measured: Baseline

  8. Infusion parameter 1.1: Cohort 1- Month 3

    Median infusion volume per infusion

    Measured: Month 3

  9. Infusion parameter 1.1: Cohort 1- Month 6

    Median infusion volume per infusion

    Measured: Month 6

  10. Infusion parameter 1.1: Cohort 1- 12 Month final follow-up

    Median infusion volume per infusion

    Measured: 12 Month final follow-up

  11. Infusion parameter 1.1: Cohort 2- Start of data collection

    Median infusion volume per infusion

    Measured: Baseline

  12. Infusion parameter 1.1: Cohort 2- 12 Month final follow-up

    Median infusion volume per infusion

    Measured: 12 Month final follow-up

  13. Infusion parameter 2: Cohort 1- Start of data collection

    Median number of infusion sites

    Measured: Baseline

  14. Infusion parameter 2: Cohort 1- Month 3

    Median number of infusion sites

    Measured: Month 3

  15. Infusion parameter 2: Cohort 1- Month 6

    Median number of infusion sites

    Measured: Month 6

  16. Infusion parameter 2: Cohort 1- 12 Month final follow-up

    Median number of infusion sites

    Measured: 12 Month final follow-up

  17. Infusion parameter 2: Cohort 2- Start of data collection

    Median number of infusion sites

    Measured: Baseline

  18. Infusion parameter 2: Cohort 2- 12 Month final follow-up

    Median number of infusion sites

    Measured: 12 Month final follow-up

  19. Infusion parameter 3: Cohort 1- Start of data collection

    Median infusion duration

    Measured: Baseline

  20. Infusion parameter 3: Cohort 1- Month 3

    Median infusion duration

    Measured: Month 3

  21. Infusion parameter 3: Cohort 1- Month 6

    Median infusion duration

    Measured: Month 6

  22. Infusion parameter 3: Cohort 1- 12 Month final follow-up

    Median infusion duration

    Measured: 12 Month final follow-up

  23. Infusion parameter 3: Cohort 2- Start of data collection

    Median infusion duration

    Measured: Baseline

  24. Infusion parameter 3: Cohort 2- 12 Month final follow-up

    Median infusion duration

    Measured: 12 Month final follow-up

  25. Infusion parameter 3.1: Cohort 1- Month 3 Follow-up

    Median number of infusions to reach participant's maximum infusion volume

    Measured: Month 3

  26. Infusion parameter 3.1: Cohort 1- Month 6 Follow-up

    Median number of infusions to reach participant's maximum infusion volume

    Measured: Month 6

  27. Infusion parameter 3.1: Cohort 1- 12 Month final follow-up

    Median number of infusions to reach participant's maximum infusion volume

    Measured: 12 Month final follow-up

  28. Infusion parameter 3.1: Cohort 2- 12 Month final follow-up

    Median number of infusions to reach participant's maximum infusion volume

    Measured: 12 Month final follow-up

  29. Infusion parameter 3.2: Cohort 1- Start of data collection

    Median number of infusions per month per participant

    Measured: Baseline

  30. Infusion parameter 3.2: Cohort 1- Month 3

    Median number of infusions per month per participant

    Measured: Month 3

  31. Infusion parameter 3.2: Cohort 1- Month 6

    Median number of infusions per month per participant

    Measured: Month 6

  32. Infusion parameter 3.2: Cohort 1- 12 Month final follow-up

    Median number of infusions per month per participant

    Measured: 12 Month final follow-up

  33. Infusion parameter 3.2: Cohort 2- Start of data collection

    Median number of infusions per month per participant

    Measured: Baseline

  34. Infusion parameter 3.2: Cohort 2- 12 Month final follow-up

    Median number of infusions per month per participant

    Measured: 12 Month final follow-up

  35. Infusion parameter 3.3: Cohort 1- Start of data collection

    Median number of infusions to reach final dose interval per participant

    Measured: Baseline

  36. Infusion parameter 3.3: Cohort 1- Month 3

    Median number of infusions to reach final dose interval per participant

    Measured: Month 3

  37. Infusion parameter 3.3: Cohort 1- Month 6

    Median number of infusions to reach final dose interval per participant

    Measured: Month 6

  38. Infusion parameter 3.3: Cohort 1- 12 Month final follow-up

    Median number of infusions to reach final dose interval per participant

    Measured: 12 Month final follow-up

  39. Infusion parameter 3.3: Cohort 2- Start of data collection

    Median number of infusions to reach final dose interval per participant

    Measured: Baseline

  40. Infusion parameter 3.3: Cohort 2- 12 Month final follow-up

    Median number of infusions to reach final dose interval per participant

    Measured: 12 Month final follow-up

  41. Infusion parameter 1: Cohort 3- 12 Month Final Follow-up

    Median infusion volume per site.

    Measured: 12 Month final follow-up

  42. Infusion parameter 1.1: Cohort 3- 12 Month Final Follow-up

    Median infusion volume per infusion.

    Measured: 12 Month final follow-up

  43. Infusion parameter 2: Cohort 3- 12 Month final follow-up

    Median number of infusion sites.

    Measured: 12 Month final follow-up

  44. Infusion parameter 3: Cohort 3- 12 Month final follow-up

    Median infusion duration.

    Measured: 12 Month final follow-up

  45. Infusion parameter 3.1: Cohort 3- 12 Month final follow-up

    Median number of infusions to reach participant's maximum infusion volume (infusion rate)

    Measured: 12 Month final follow-up

  46. Infusion parameter 3.2: Cohort 3- 12 Month final follow-up

    Median number of infusions per month per participant

    Measured: 12 Month final follow-up

  47. Infusion parameter 3.3: Cohort 3- 12 Month final follow-up

    Median number of infusions to reach final dose interval per participant

    Measured: 12 Month final follow-up

Secondary outcomes (49)

  1. Infusion parameter 4.1: Cohort 1- Start of data collection

    Median maximal infusion rate per site

    Measured: Baseline

  2. Infusion parameter 4.1: Cohort 1- Month 3

    Median maximal infusion rate per site

    Measured: Month 3

  3. Infusion parameter 4.1: Cohort 1- Month 6

    Median maximal infusion rate per site

    Measured: Month 6

  4. Infusion parameter 4.1: Cohort 1- 12 Month final follow-up

    Median maximal infusion rate per site

    Measured: 12 Month final follow-up

  5. Infusion parameter 4.1: Cohort 2- Start of data collection

    Median maximal infusion rate per site

    Measured: Baseline

  6. Infusion parameter 4.1: Cohort 2- 12 Month final follow-up

    Median maximal infusion rate per site

    Measured: 12 Month final follow-up

  7. Infusion parameter 4.2: Cohort 1- Start of data collection

    Number of infusions that are discontinued, slowed, or interrupted

    Measured: Baseline

  8. Infusion parameter 4.2: Cohort 1- Month 3

    Number of infusions that are discontinued, slowed, or interrupted

    Measured: Month 3

  9. Infusion parameter 4.2: Cohort 1- Month 6

    Number of infusions that are discontinued, slowed, or interrupted

    Measured: Month 6

  10. Infusion parameter 4.2: Cohort 1- 12 Month final follow-up

    Number of infusions that are discontinued, slowed, or interrupted

    Measured: 12 Month final follow-up

  11. Infusion parameter 4.2: Cohort 2- Start of data collection

    Number of infusions that are discontinued, slowed, or interrupted

    Measured: Baseline

  12. Infusion parameter 4.2: Cohort 2- 12 Month final follow-up

    Number of infusions that are discontinued, slowed, or interrupted

    Measured: 12 Month final follow-up

  13. Infusion parameter 4.3: Cohort 1- Month 3

    Median number of infusions to reach participant's maximum infusion rate

    Measured: Month 3

  14. Infusion parameter 4.3: Cohort 1- Month 6

    Median number of infusions to reach participant's maximum infusion rate

    Measured: Month 6

  15. Infusion parameter 4.3: Cohort 1- 12 Month final follow-up

    Median number of infusions to reach participant's maximum infusion rate

    Measured: 12 Month final follow-up

  16. Infusion parameter 4.3: Cohort 2- 12 Month final follow-up

    Median number of infusions to reach participant's maximum infusion rate

    Measured: 12 Month final follow-up

  17. Infusion parameter 5.1: Cohort 1- Start of data collection

    Mean dose

    Measured: Baseline

  18. Infusion parameter 5.1: Cohort 1- Month 3

    Mean dose

    Measured: Month 3

  19. Infusion parameter 5.1: Cohort 1- Month 6

    Mean dose

    Measured: Month 6

  20. Infusion parameter 5.1: Cohort 1- 12 Month final follow-up

    Mean dose

    Measured: 12 Month final follow-up

  21. Infusion parameter 5.1: Cohort 2- Start of data collection

    Mean dose

    Measured: Baseline

  22. Infusion parameter 5.1: Cohort 2- 12 Month final follow-up

    Mean dose

    Measured: 12 Month final follow-up

  23. Infusion parameter 5.2: Cohort 1- Start of data collection

    Mean dosing interval

    Measured: Baseline

  24. Infusion parameter 5.2: Cohort 1- Month 3

    Mean dosing interval

    Measured: Month 3

  25. Infusion parameter 5.2: Cohort 1- Month 6

    Mean dosing interval

    Measured: Month 6

  26. Infusion parameter 5.2: Cohort 1- 12 Month final follow-up

    Mean dosing interval

    Measured: 12 Month final follow-up

  27. Infusion parameter 5.2: Cohort 2- Start of data collection

    Mean dosing interval

    Measured: Baseline

  28. Infusion parameter 5.2: Cohort 2- 12 Month final follow-up

    Mean dosing interval

    Measured: 12 Month final follow-up

  29. Infusion parameter 5.3: Cohort 1- Start of data collection

    Mean number of dose adjustments

    Measured: Baseline

  30. Infusion parameter 5.3: Cohort 1- Month 3

    Mean number of dose adjustments

    Measured: Month 3

  31. Infusion parameter 5.3: Cohort 1- Month 6

    Mean number of dose adjustments

    Measured: Month 6

  32. Infusion parameter 5.3: Cohort 1- 12 Month final follow-up

    Mean number of dose adjustments

    Measured: 12 Month final follow-up

  33. Infusion parameter 5.3: Cohort 2- Start of data collection

    Mean number of dose adjustments

    Measured: Baseline

  34. Infusion parameter 5.3: Cohort 2- 12 Month final follow-up

    Mean number of dose adjustments

    Measured: 12 Month final follow-up

  35. Infusion parameter 4.1: Cohort 3- 12 Month final follow-up

    Median maximal infusion rate per site

    Measured: 12 Month final follow-up

  36. Infusion parameter 4.2: Cohort 3- 12 Month final follow-up

    Number of infusions that are discontinued, slowed, or interrupted

    Measured: 12 Month final follow-up

  37. Infusion parameter 4.3: Cohort 3- 12 Month final follow-up

    Median number of infusions to reach participant's maximum infusion rate

    Measured: 12 Month final follow-up

  38. Infusion parameter 5.1: Cohort 3- 12 Month final follow-up

    Mean dose

    Measured: 12 Month final follow-up

  39. Infusion parameter 5.2: Cohort 3- 12 Month final follow-up

    Mean dosing interval

    Measured: 12 Month final follow-up

  40. Infusion parameter 5.3: Cohort 3- 12 Month final follow-up

    Mean number of dose adjustments

    Measured: 12 Month final follow-up

  41. 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.

    Measured: 12 Month final follow-up

  42. 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.

    Measured: 12 Month final follow-up

  43. 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.

    Measured: 12 Month final follow-up

  44. 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.

    Measured: 12 Month final follow-up

  45. 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

    Measured: 12 Month final follow-up

  46. 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

    Measured: 12 Month final follow-up

  47. 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.

    Measured: 12 Month final follow-up

  48. 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.

    Measured: 12 Month final follow-up

  49. 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.

    Measured: 12 Month final follow-up

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted23 Oct 2018
  2. Study start24 Sep 2018
  3. Primary completion23 Jul 2020
  4. Study completion23 Jul 2020

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Observational
Observational model
Cohort
Time perspective
Prospective
Enrolment
126 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03716700
Sponsor's protocol ID
SHP664-402
Status last verified
Mar 2021
First submitted
31 Aug 2018
Last updated
5 Mar 2021
Results posted
No
Data monitoring committee
No
FDA-regulated device
No
Individual data sharing
Yes: 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.

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.

    PubMed 35934726

Questions about this trial

What is this clinical trial studying?

An observational study of Primary Immunodeficiency Diseases (PID), run by Baxalta now part of Shire at 3 sites in Canada.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 5 Mar 2021. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 3 sites in Canada. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Infusion parameter 1: Cohort 1-Start of data collection (time frame: Baseline). The study lists 47 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT03716700, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 5 Mar 2021; AzTrials last refreshed its copy on 6 Nov 2026.

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