Immune System Diseases

All Conditions

Neoplasms

Symptoms and General Pathology

Diseases and Abnormalities at or Before Birth

Behaviors and Mental Disorders

Autoimmune Diseases

Neoplasm Metastasis

Immunologic Deficiency Syndromes

Primary Immunodeficiency Diseases

Syndrome

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Genetic Diseases, Inborn

Quality of Life

All Drugs and Chemicals

Antibodies

Immunoglobulins

Immunoglobulin G

Immunoglobulins, Intravenous

Immunologic Factors

Any marketed SC or IV IgG can be documented

Not applicable. All interventions are at the discretion of the investigator. All marketed IgG formulations can be documented.

Immunoglobulin G (IgG)

Wilhelm Kirch, MD, PhD

David Pittrow, MD, PhD

Michael Borte, MD, PhD

Ulrich Baumann, MD, PhD

Manfred Hensel, MD, PhD

Dörte Huscher

Marcel Reiser, MD, PhD

Martin Stangel, MD, PhD

Ralph Gold, MD, PhD

Claudia Sommer, MD, PhD

This non-interventional, epidemiological study assesses long-term outcomes in subjects receiving immunoglobulins (IgG) for any treatment purpose, irrespective of the regimen prescribed by the treating physician, under routine clinical conditions in Germany.

Long-term outcome data are collected on patient characteristics in the various indications, drug utilization of intravenous and subcutaneous IgG (e.g. treatment and dosing patterns), effectiveness (i.e. number of infections), tolerability, health related quality of life, and economic variables (number of hospitalizations, sick-leave days etc.) with the possibility to estimate direct costs.

In view of the broad range of indications in immunodeficiency and immunomodulation, it is of interest to document the use of IgG under the conditions of everyday practice and to analyze the endpoints (outcomes). A prospective cohort study such as this is an important evidence source for such rare diseases as those mentioned above. The aim of this outcome study is to fill the gap of the lack of long-term data in these rare diseases treated with IgG.

Assessment of Immunoglobulins (IgG) in a Long-term Non-interventional Study

An Open, Uncontrolled, Non-interventional Observational Cohort Outcome Study of Immunoglobulins in 3 Indications: Primary and Secondary Immunodeficiencies and Neurological Auto-immune Diseases

Dosage of immunoglobulins (IgG); frequency of IgG administrations; days of treatment with IgG; duration of infusion of IgG.

For immunodeficiencies (primary PID and secondary SID):

frequency of infections; degree of severity of infections (SBIs); duration of antibiotic treatment; necessity of antibiotic treatment.

Grip strength (dynamometer) Electrophysiology (EMG, ENG); Inflammatory Neuropathy Cause and Treatment (INCAT) disability score; EDSS, annual relapse rate; Myasthenia Score.

Number of sick-leave days Number of medical visits Days of hospitalisation due to infections or due to disability or loss of function Degree of disability

GWT-TUD GmbH

Technische Universität Dresden

Heart and Blood Diseases

Blood and Lymph Conditions

Rare Diseases

Multiple Myeloma

Neoplasms, Plasma Cell

Neoplasms by Histologic Type

Hemostatic Disorders

Blood Coagulation Disorders

Vascular Diseases

Paraproteinemias

Blood Protein Disorders

Hematologic Diseases

Hemorrhagic Disorders

Lymphoproliferative Disorders

Immunoproliferative Disorders

Participants with MM diagnosed with SID will be treated with HyQvia as part of routine clinical care and will be followed prospectively from the date of starting HyQvia treatment through 12 months of follow-up.

As this is an observational study, no intervention will be administered in this study.

No Intervention

Takeda Contact

Site Contact

Emanuel Leonardo Aguilera

Alexandra Jungova

Jan Straub

Clement Gourguechon

Marie-Lorraine Chretien

Katell Le Du

Omar Benbrahim

Jean-Francois Viallard

Richard Schabath

Efstathios Kastritis

Ioanna Sakellari

Angelo Vacca

Alessandra Romano

Tamara Berno

Francesco Cinetto

Jacek Rolinski

Sorina Badeelita

Ciprian Tomuleasa

Luminita Ocroteala

Alina Camelia Catana

Silvia Sanchez-Ramon

Joaquin Martinez-Lopez

Albert Perez-Montana

Goran Jonson

Karlis Pauksens

Selami Toprak

Volkan Karakus

Muhlis Cem Ar

Omur Sevindik

Study Director

In this study, multiple myeloma participants with secondary immunodeficiency (SID) will be treated with HyQvia according to their clinic's standard practice. The study's main aim is to look into infusion parameters of HyQvia administration.

This is a prospective, observational study of adults having multiple myeloma (MM) with SID treated with HyQvia as part of routine clinical care. This study will characterize the real-world infusion parameters of HyQvia administration.

The study will enroll approximately 100 participants.

Study data will be requested through participants' routine clinic visits and patient-reported outcome (PRO)s are voluntary.

This multi-center trial will be conducted in selected European and Latin American countries. The overall time of this study is 38 months. Participants will make multiple visits to the clinic 12 days after last dose of study drug for a follow-up assessment.

Real-world Usage of HyQvia in Multiple Myeloma Adults With Secondary Immunodeficiency

A Prospective, Observational Study to Assess the Real-World Usage and Outcomes of HyQvia in Patients With Multiple Myeloma (MM) Diagnosed With Secondary Immunodeficiency (SID)

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.

Dose of HyQvia will be calculated as grams per kilogram (g/kg) body weight per 4 weeks.

Absolute dose of HyQvia will be calculated as dose per infusion in milliliters (mL).

Treatment interval and ramp up dosing intervals will be reported as Weekly, every 2 weeks, 3 weeks, 4 weeks and other administration according to the schedule defined by physician.

Infusion rate will be reported in milliliters per hour (mL/h).

The duration between start and end of infusion will be reported.

Location of infusion sites will include right upper abdomen, left upper abdomen, right lower abdomen, left lower abdomen, right or left thigh, right or left arm, and other (specified).

Site of care will be categorized as home, hospital, doctor's office, other (specified).

Type of pump will include peristaltic infusion pump and syringe driver pump.

Availability of caregiver support will be collected as yes, no, and unknown. If yes, caregiver relationship will be specified.

Reasons for discontinued, or interrupted HyQvia treatment will include local adverse events (AE)/discomfort, systemic AE, administration complexity, lack of efficacy, insurance/reimbursement-related, low immunoglobulin trough level, lack of effectiveness, inability to tolerate large volumes and other (specified). If switch, type of treatment after switch (e.g., antibiotic treatment, intravenous immunoglobulin \[IVIg\], subcutaneous immunoglobulin \[SCIg\], etc.) or switches to other treatment will be included.

Clinical characteristics of participants will include type of MM, and MM treatment/procedures, comorbidities, concomitant medications, laboratory tests and imaging.

Type of hemato-oncological treatment will list type, switches, interruptions and discontinuation with reasons of hemato-oncological treatment or procedure.

MM disease status and outcome will include response and criteria used to assess response (if known).

OS will be summarized as the frequency and percentage of participants surviving to each follow-up visit. Additionally, time-to death, defined as the time from HyQvia initiation to death occurring before the end of the study period, will be assessed.

Number of participants with HCRU including annualized rates and emergency room visits, urgent care visits, outpatient visits, telemedicine visits, and other physician visits, with stratification of hospitalizations and visits due to infection, visit type and provider type.

An Adverse Event (AE) is defined as any untoward medical occurrence in a clinical investigation participant administered a drug; it does not necessarily have to have a causal relationship with this treatment. An SAE is defined as an untoward medical occurrence that at any dose is fatal, life-threatening, requires inpatient hospitalisation or results in prolongation of an existing hospitalization, results in persistent or significant disability/incapacity, is a congenital anomaly/birth defect, is a medically important event. AEs will be reported as local or systemic AEs.

Premedication use for HyQvia infusions will include start date, stop date and medication name. Technical problems with infusion will include problems with the handling of the pump, problems with the preparation of the infusion, problems with the infusion itself, infusion site leakage, and other (specified).

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization
Assessment of Immunoglobulins (IgG) in a Long-term Non-interventional Study	NCT01287689	Primary Immunod... Secondary Immun... Neurological Au...	Immunoglobulin ...	-	Technische Universität Dresden
Assessment of Immunoglobulins (IgG) in a Long-term Non-interventional Study	NCT01287689	Primary Immunod... Secondary Immun... Neurological Au...	Immunoglobulin ...	-	Technische Universität Dresden
Real-world Usage of HyQvia in Multiple Myeloma Adults With Secondary Immunodeficiency	NCT05879757	Multiple Myelom... Secondary Immun...	No Intervention	18 Years -	Takeda

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