Neoplasms

Symptoms and General Pathology

All Conditions

Immune System Diseases

Diseases and Abnormalities at or Before Birth

Neoplasm Metastasis

Immunologic Deficiency Syndromes

Primary Immunodeficiency Diseases

Syndrome

Neoplastic Processes

Genetic Diseases, Inborn

All Drugs and Chemicals

Pharmaceutical Solutions

Antibodies

Immunoglobulins

Immunoglobulins, Intravenous

Immunologic Factors

Immunoglobulin replacement therapy with subcutaneous injections of Cutaquig® 165 mg/mL at home.

Cutaquig®

Vicki Voong, BScN

Bruce Ritchie, MD

This study will assess the relative safety, tolerability, and participant satisfaction in participants using the rapid manual push method with Cutaquig®. The hypothesis being that treatment with Cutaquig® by rapid manual push method will improve the safety, tolerability and patient satisfaction of participants with PID or SID.

Cutaquig® by rapid push is already approved in Canada and has proven to be efficacious in preventing significant infection. However, relative safety, tolerability, and patient satisfaction have not been studied in these patients. The information gained from this study will improve the safety and tolerability knowledge database and will support the optimal use of Cutaquig® - thus benefitting both physicians and patients.

Primary and secondary immunodeficiency diseases (PID and SID, respectively) affect the development and/or function of the immune system, resulting in increased frequency of infection. Treatment of these disorders using Cutaquig® (a subcutaneous immunoglobulin infusion (SCIG)) was licensed in Canada in 2018 and has proven to be effective in preventing significant infection. Other brands of SCIG can be given by the participant using a syringe (called rapid manual push method), which shows the same efficacy as administration by a programmable pump. The rapid manual push method has the potential to improve quality of life, reduce infusion time, and reduce the cost of administration. This study will evaluate the safety, tolerability, and patient satisfaction of Cutaquig® by the rapid manual push method in participants with PID or SID. It will also compare the efficacy of Cutaquig with prior SCIG treatment, if applicable, in participants with PID or SID.

At the infusion visits, blood samples will be collected for laboratory evaluation.

Observational Study of Subcutaneous Immunoglobulin (Cutaquig) in Patients With Primary and Secondary Immunodeficiency.

Safety, Tolerability, and Patient Satisfaction of 16.5% Subcutaneous Immunoglobulin (Cutaquig®) Treatment by Rapid Push in Patients With Primary or Secondary Immunodeficiency (PID or SID)

1. The primary objective is to assess safety of cutaquig® in patients with PID or SID by rapid push at infusion volumes ranging from 15 mL/infusion site to a maximum of 60 mL/infusion site into a maximum of 4 infusion sites and
2. assess safety of a maximum infusion speed of 120 mL/h for all sites combined (2 mL/min)

1. The primary objective is to assess tolerability of cutaquig® in patients with PID or SID by rapid push at infusion volumes ranging from 15 mL/infusion site to a maximum of 60 mL/infusion site into a maximum of 4 infusion sites and
2. assess tolerability of a maximum infusion speed of 120 mL/h for all sites combined (2 mL/min)

Key secondary objectives are to assess by a questionnaire

1. time to infuse cutaquig®
2. total volume infused per infusion site
3. total volume infused per infusion
4. frequency of infusion
5. number of injection sites
6. ease of infusion (injectability) Other secondary objectives are to
7. assess patient quality of life (QoL) and patient satisfaction by using the EQ-5D-5L (descriptive system compromises five dimensions: mobility, self-care, usual activities, pain/discomfort, and anxiety and depression).
8. compare the injectability of cutaquig® with prior SCIG administration if applicable

Octapharma

University of Alberta

Infections

Communicable Diseases

Epstein-Barr Virus Infections

Virus Diseases

Disease Attributes

Herpesviridae Infections

DNA Virus Infections

Tumor Virus Infections

Patients with refractory EBV will get one dose of EBV specific CTLs. If they don't show a response based on EBV PCRs, patients may get up to another 4 doses of EBV-CTLs (5 doses maximum)

EBV specific CTLs will be generated from HLA related matched and mismatched donors in a GMP facility and administered to the patient with refractory CTLs.

cytotoxic t-lymphocytes

Mitchell Cairo, MD

Lauren Harrison

Neena Kapoor, MD

Julia Chu, MD

Kenneth Cooke, MD

Shalini Shenoy, MD

Mitchell S Cairo, MD

Mitchell S. Cairo, MD

Dean Lee, MD, PhD

Nancy Bunin, MD

Julie A Talano, MD

Meredith Beversdorf, RN

Related donor Epstein-Barr Virus (EBV) specific cytotoxic T cells (CTLs) manufactured with the Miltenyi CliniMACS Prodigy Cytokine Capture System will be administered in children, adolescents and young adults with refractory EBV infection post Allogeneic Hematopoietic Stem Cell Transplantation (AlloHSCT), with primary immunodeficiencies (PID) or post solid organ transplant.

Funding Source: FDA OOPD

All patients given identical treatment with CTLs for refractory EBV.

EBV-specific Cytotoxic T-lymphocytes (CTLs) for Refractory EBV Infection

A Pilot Study in the Treatment of Refractory Epstein-Barr Virus (EBV) Infection With Related Donor EBV Cytotoxic T-Lymphocytes in Children, Adolescents and Young Adult Recipients

Patients will be monitored for adverse events related to the infusion of EBV CTLs

Patients will be followed for improvement in viral infection by monitoring EBV PCR weekly for response to treatment with CTLs

Children's Hospital of Philadelphia

Medical College of Wisconsin

Nationwide Children's Hospital

Indiana University

Washington University School of Medicine

University of California, Los Angeles

University of California, San Francisco

New York Medical College

Principal Investigator

Respiratory Tract (Lung and Bronchial) Diseases

Urinary Tract, Sexual Organs, and Pregnancy Conditions

COVID-19

Acquired Immunodeficiency Syndrome

HIV Infections

Coronavirus Infections

Pneumonia

Pneumonia, Viral

Respiratory Tract Infections

Coronaviridae Infections

Nidovirales Infections

RNA Virus Infections

Lung Diseases

Respiratory Tract Diseases

Immunosuppressive Agents

Patients with acquired immunodeficiency associated with other immunosuppressive therapy.

Mark Polizzotto, MD

CORIA is an observational cohort study of immunosuppressed populations who test positive for COVID-19. This includes people living with HIV, cancer, acquired immunodeficiency associated with other immunosuppressive therapy, primary immunodeficiency and recipients of a solid organ transplant. Participants will have routine clinical data collected with optional baseline collection and storage of a blood sample for storage . The study will be conducted in up to 30 sites within Australia.

Coronavirus (COVID-19) Outcomes Registries in Immunocompromised Individuals Australia (CORIA)

Coronavirus Outcomes Registries in Immunocompromised Individuals Australia (CORIA): a Multisite Registry and Optional Biorepository in People With COVID-19 and Selected Conditions Affecting Immune Function

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization
Observational Study of Subcutaneous Immunoglobulin (Cutaquig) in Patients With Primary and Secondary Immunodeficiency.	NCT04354129	Primary Immune ... Secondary Immun...	Cutaquig®	18 Years -	University of Alberta
EBV-specific Cytotoxic T-lymphocytes (CTLs) for Refractory EBV Infection	NCT03266653	Epstein-Barr Vi... Primary Immune ...	cytotoxic t-lym...	1 Month - 79 Years	New York Medical College
Coronavirus (COVID-19) Outcomes Registries in Immunocompromised Individuals Australia (CORIA)	NCT04354818	HIV-1-infection Cancer Primary Immune ... Immunosuppressi... COVID-19		18 Years -	Kirby Institute
Observational Study of Subcutaneous Immunoglobulin (Cutaquig) in Patients With Primary and Secondary Immunodeficiency.	NCT04354129	Primary Immune ... Secondary Immun...	Cutaquig®	18 Years -	University of Alberta

Spots Global Cancer Trial Database for primary immune deficiency disorder