Clinical trial NCT03677557: Safety, Tolerability, Patient Satisfaction and Cost of 16.5% Subcutaneous Immunoglobulin (Cutaquig®) Treatment
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)
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE4
Conditions: Primary Immunodeficiency Disease
Secondary Immunodeficiency
Lead sponsor: Ottawa Hospital Research Institute
Sponsor class: OTHER
Collaborators: name: Octapharma
First posted: 2018-09-19
Enrollment: 30
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.

Eligibility criteria: 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

Primary outcomes:
measure: Incidence of Treatment-Emergent Adverse Events per participant per study visit of 16.5% Subcutaneous Immunoglobulin (Cutaquig®) Treatment
timeFrame: The outcome measure will be assessed through study completion, an average of 1 year
measure: Retention of participants who are able to tolerate the study intervention at 12 months
timeFrame: Cumulative proportion of participants who are on Cutaquig at 12 months.
measure: Quality of Life (Patient Satisfaction)
timeFrame: The outcome measure will be assessed through study completion, an average of 1 year
measure: Patient satisfaction (Quality of life)
timeFrame: The outcome measure will be assessed through study completion, an average of 1 year
measure: Treatment associated cost
timeFrame: The outcome measure will be assessed through study completion, an average of 1 year
Secondary outcomes:
Other outcomes:
Locations:
city: Ottawa
country: Canada
facility: The Ottawa Hospital, General Campus
state: Ontario
Study officials:
affiliation: The Ottawa Hospital
name: Juthaporn Cowan, MD, PhD, FRCPC
role: PRINCIPAL_INVESTIGATOR
References: