Primary Immunodeficiency Disease, Secondary Immunodeficiency
Conditions
Brief 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).
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.
Interventions
DRUG16,5% Cutaquig
Participants with primary or secondary immunodefiiciney disease and who do not tolerate other immunoglobulin treatments will be asked to use 16.5% Cutaquig
Sponsors
Octapharma
Ottawa Hospital Research Institute
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* 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.
Exclusion criteria
* Pregnant Women
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Incidence of Treatment-Emergent Adverse Events per participant per study visit of 16.5% Subcutaneous Immunoglobulin (Cutaquig®) Treatment | 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 | 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) | 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) | 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 | 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 |
Countries
Canada
Contacts
Primary ContactJuthaporn Cowan, MD, PhD, FRCPC
Outcome results
None listed