Primary Immunodeficiency DiseasePhase 4NCT03677557
Safety, Tolerability, Patient Satisfaction and Cost of 16.5% Subcutaneous Immunoglobulin (Cutaquig®) Treatment
A Phase 4 study testing 16,5% Cutaquig in people with Primary Immunodeficiency Disease and Secondary Immunodeficiency, run by Ottawa Hospital Research Institute at 1 site in Canada.
Trial summary
Patients with primary or secondary immunodeficiency disease who have developed adverse reactions to products available on the market such as Cuvitru® (Shire), Hizentra® (CSL Behring) or 10% Gammunex® (Grifols), may benefit from utilizing 16.5% Cutaquig® (Octapharma). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Primary Immunodeficiency Disease, Secondary Immunodeficiency
- What is being tested?
- 16,5% Cutaquig
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 30 participants
- Where
- 1 siteCanada
- Study dates
- 19 Sep 2018to 31 Aug 2021
- Phase
- Phase 4
- 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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Patients aged 18 years and older
- Patients with primary or secondary immunodeficiency disease who are currently on subcutaneous immunoglobulin treatment but have developed adverse events and are willing to change the treatment product.
Who cannot join
- Pregnant Women
Trial locations and contacts
1 site in 1 country.
Canada1 site, 1 recruiting
- Recruiting
The Ottawa Hospital, General Campus
Ottawa, Ontario, K1H 8L6
Andrea Trinidad, 613-737-8899 ext. 77204, atrinidad@ohri.ca
About this study
Official title: Safety, Tolerability, Patient Satisfaction and Cost of 16.5% Subcutaneous Immunoglobulin (Cutaquig®) Treatment in Patients Who Did Not Tolerate Other 20% Subcutaneous Immunoglobulin Product(s)
Patients with primary or secondary immunodeficiency disease who have developed adverse reactions to products available on the market such as Cuvitru® (Shire), Hizentra® (CSL Behring) or 10% Gammunex® (Grifols), may benefit from utilizing 16.5% Cutaquig® (Octapharma).
Read the detailed description
This is a prospective interventional study before and after clinically driven change in treatment formulation.
Polyvalent immunoglobulin treatment is used in patients with primary or secondary antibody deficiency diseases to prevent and lower the risk of infection. There are multiple products available in the market. Most products are administered via intravenous route such as Privigen® (CSL Behring), Gammunex® (Grifols), and Panzyga® (Octapharma). Up until recently, there have been only two products that are licensed for subcutaneous administration - 20% Hizentra® (CSL Behring) and 10% Gammunex® (Grifols).
In our clinical experience, approximately 10% of patients treated with 20% Hizentra® developed adverse reactions. Some are mild and tolerable. Some are moderate to severe and required alteration of treatment plan: For example, changing the product from 20% Hizentra® to 10% Gammunex®. However, this results in a 100% increase in the injection volume due to the lesser concentration of the product, but a decrease in viscosity - both of which might alter overall tolerance. Likewise, any new treatment may bring new adverse events such as rash.
In 2018, there will be two additional subcutaneous immunoglobulin products available in Canada - 16.5% Cutaquig® (Octapharma) and 20% Cuvitru® (Shire).
Even though both new products are licensed and proven to be efficacious regarding preventing significant infection (1,2), the relative safety, tolerability, patient satisfaction, treatment-associated cost has not been studied in patients using the 16.5% Cutaquig®. The study product will be provided through the Canadian Blood Service (CBS) on a special request basis which is a standard procedure for any patients who are intolerable to inventory products.
- Being tested
- 16,5% Cutaquig
- 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.
16,5% CutaquigDrug
Participants with primary or secondary immunodefiiciney disease and who do not tolerate other immunoglobulin treatments will be asked to use 16.5% Cutaquig
| Study group | Role | What they receive |
|---|---|---|
| Cutaquig Intervention | Other | Participants with primary or secondary immunodeficiency disease who are currently on subcutaneous immunoglobulin treatment but have developed adverse events including allergic reaction and are willing to change the treatment product to 16.5% Cutaquig. Receives: Drug: 16,5% Cutaquig |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of Treatment-Emergent Adverse Events per participant per study visit of 16.5% Subcutaneous Immunoglobulin (Cutaquig®) Treatment
The safety will be measured by the number of adverse events per participant per study visit
Measured: The outcome measure will be assessed through study completion, an average of 1 year
Retention of participants who are able to tolerate the study intervention at 12 months
Retention of participants who are able to tolerate the study intervention will be calculated as the number of participants enrolled between the 6 and 12 month visits
Measured: Cumulative proportion of participants who are on Cutaquig at 12 months.
Quality of Life (Patient Satisfaction)
This will be measured by quality of life questionnaire (SF-36) before and after change in treatment at 6 and 12 months
Measured: The outcome measure will be assessed through study completion, an average of 1 year
Patient satisfaction (Quality of life)
This will be measured by quality of life questionnaire (Euroquol 5D-5L) before and after change in treatment at 6 and 12 months
Measured: The outcome measure will be assessed through study completion, an average of 1 year
Treatment associated cost
This will measure the cost of nursing time and will be reported as dollars/patient/year
Measured: The outcome measure will be assessed through study completion, an average of 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Sep 2018
- Study start19 Sep 2018
- Primary completion31 Aug 2021 (estimated)
- Study completion31 Aug 2021 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Primary Immunodeficiency DiseaseStudies of the same condition, open and under way.
- More trials run by Ottawa Hospital Research InstituteThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 4
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 30 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Ottawa Hospital Research Institute
- Sponsor type
- University, hospital or other
- Collaborators
- Octapharma
- Responsible party
- Sponsor
- Investigators
- Juthaporn Cowan, MD, PhD, FRCPC, Principal investigator, The Ottawa Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03677557
- Sponsor's protocol ID
- 2018 0388-01H
- Status last verified
- Mar 2020
- First submitted
- 6 Jul 2018
- Last updated
- 4 Mar 2020
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 4 Mar 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Safety, Tolerability, Patient Satisfaction and Cost of 16.5% Subcutaneous Immunoglobulin (Cutaquig®) Treatment in Patients Who Did Not Tolerate Other 20% Subcutaneous Immunoglobulin Product(s)
- ClinicalTrials.gov ID
- NCT03677557
- Acronym / sponsor ID
- 2018 0388-01H
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 30 participants (estimated)
- Lead sponsor
- Ottawa Hospital Research InstituteUniversity, hospital or other
- Collaborators
- Octapharma
- Study start
- 19 Sep 2018 (actual)
- Primary completion
- 31 Aug 2021 (estimated)
- Study completion
- 31 Aug 2021 (estimated)
- First posted
- 19 Sep 2018 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Patients with primary or secondary immunodeficiency disease who have developed adverse reactions to products available on the market such as Cuvitru® (Shire), Hizentra® (CSL Behring) or 10% Gammunex® (Grifols), may benefit from utilizing 16.5% Cutaquig® (Octapharma).
Detailed description
This is a prospective interventional study before and after clinically driven change in treatment formulation.
Polyvalent immunoglobulin treatment is used in patients with primary or secondary antibody deficiency diseases to prevent and lower the risk of infection. There are multiple products available in the market. Most products are administered via intravenous route such as Privigen® (CSL Behring), Gammunex® (Grifols), and Panzyga® (Octapharma). Up until recently, there have been only two products that are licensed for subcutaneous administration - 20% Hizentra® (CSL Behring) and 10% Gammunex® (Grifols).
In our clinical experience, approximately 10% of patients treated with 20% Hizentra® developed adverse reactions. Some are mild and tolerable. Some are moderate to severe and required alteration of treatment plan: For example, changing the product from 20% Hizentra® to 10% Gammunex®. However, this results in a 100% increase in the injection volume due to the lesser concentration of the product, but a decrease in viscosity - both of which might alter overall tolerance. Likewise, any new treatment may bring new adverse events such as rash.
In 2018, there will be two additional subcutaneous immunoglobulin products available in Canada - 16.5% Cutaquig® (Octapharma) and 20% Cuvitru® (Shire).
Even though both new products are licensed and proven to be efficacious regarding preventing significant infection (1,2), the relative safety, tolerability, patient satisfaction, treatment-associated cost has not been studied in patients using the 16.5% Cutaquig®. The study product will be provided through the Canadian Blood Service (CBS) on a special request basis which is a standard procedure for any patients who are intolerable to inventory products.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Cutaquig Intervention | Other | Drug: 16,5% CutaquigParticipants with primary or secondary immunodeficiency disease who are currently on subcutaneous immunoglobulin treatment but have developed adverse events including allergic reaction and are willing to change the treatment product to 16.5% Cutaquig. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (2 listed)
- Patients aged 18 years and older
- Patients with primary or secondary immunodeficiency disease who are currently on subcutaneous immunoglobulin treatment but have developed adverse events and are willing to change the treatment product.
Key exclusion criteria (1 listed)
- Pregnant Women
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| 16,5% CutaquigParticipants with primary or secondary immunodefiiciney disease and who do not tolerate other immunoglobulin treatments will be asked to use 16.5% Cutaquig | Drug | Cutaquig Intervention |
Primary outcome
Incidence of Treatment-Emergent Adverse Events per participant per study visit of 16.5% Subcutaneous Immunoglobulin (Cutaquig®) Treatment
Time frame: The outcome measure will be assessed through study completion, an average of 1 year
The safety will be measured by the number of adverse events per participant per study visit
Retention of participants who are able to tolerate the study intervention at 12 months
Time frame: Cumulative proportion of participants who are on Cutaquig at 12 months.
Retention of participants who are able to tolerate the study intervention will be calculated as the number of participants enrolled between the 6 and 12 month visits
Quality of Life (Patient Satisfaction)
Time frame: The outcome measure will be assessed through study completion, an average of 1 year
This will be measured by quality of life questionnaire (SF-36) before and after change in treatment at 6 and 12 months
Patient satisfaction (Quality of life)
Time frame: The outcome measure will be assessed through study completion, an average of 1 year
This will be measured by quality of life questionnaire (Euroquol 5D-5L) before and after change in treatment at 6 and 12 months
Treatment associated cost
Time frame: The outcome measure will be assessed through study completion, an average of 1 year
This will measure the cost of nursing time and will be reported as dollars/patient/year
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 Mar 2020.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Juthaporn Cowan, MD, PhD, FRCPC principal investigator
The Ottawa Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing 16,5% Cutaquig in people with Primary Immunodeficiency Disease and Secondary Immunodeficiency, run by Ottawa Hospital Research Institute at 1 site in Canada.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 1 exclusion criteria. 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: status not confirmed, as of the sponsor's last update on 4 Mar 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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.
What does this phase mean?
Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Juthaporn Cowan, MD, PhD, FRCPC (613-737-8899 ext. 73954, jcowan@toh.ca), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of Treatment-Emergent Adverse Events per participant per study visit of 16.5% Subcutaneous Immunoglobulin (Cutaquig®) Treatment (time frame: The outcome measure will be assessed through study completion, an average of 1 year). The study lists 5 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 4 Mar 2020.