A Study of Immune Globulin Subcutaneous (Human), 20% Solution (IGSC, 20%) in Japanese Participants With Primary Immunodeficiency Diseases (PID)
Primary Immunodeficiency Diseases (PID)
About this trial
This is an interventional treatment trial for Primary Immunodeficiency Diseases (PID)
Eligibility Criteria
Inclusion Criteria:
- Be of Japanese descent, defined as born in Japan and having Japanese parents and Japanese maternal and paternal grandparents.
- Participants must have a documented diagnosis of a form of primary humoral immunodeficiency involving antibody formation and requiring gammaglobulin replacement. The diagnosis must be confirmed by the medical director prior to treatment with IGIV.
- Participant is 2 years or older at the time of screening.
- Written informed consent is obtained from either the participants or the participants legally authorized representative prior to any study-related procedures and study product administration.
- Participant has been receiving a consistent dose of IGIV over a period of at least 3 months prior to screening equivalent to approximately 200-600 mg/kg-body weight (BW) per 3- 4 week period, as according to the product package insert
- Participant has a serum trough level of IgG greater than or equal to (>=) 5 gram per liter (g/L) at screening.
- Participant has not had a serious bacterial infection within the 3 months prior to screening.
- Participant is willing and able to comply with the requirements of the protocol.
Exclusion Criteria:
- Participant has a known history of or is positive at screening for one or more of the following: hepatitis B surface antigen (HBsAg), polymerase chain reaction (PCR) for hepatitis C virus (HCV), PCR for human immunodeficiency virus (HIV) Type 1/2.
Abnormal laboratory values at screening meeting any one of the following criteria (abnormal tests may be repeated once to determine if they are persistent):
- Persistent alanine aminotransferase (ALT) and aspartate amino transferase (AST) greater than (>) 2.5 times the upper limit of normal (ULN) for the testing laboratory
- Persistent severe neutropenia (defined as an absolute neutrophil count [ANC] lesser than or equal to (<=) 500/milli cubic meter [mm^3]).
- Participant has presence of renal function impairment defined by estimated glomerular filtration rate (eGFR) is less than (<) 60 milliliter per minute/ 1.73 square meter (mL/min/1.73m^2).
- Participant has been diagnosed with or has a malignancy (other than adequately treated basal cell or squamous cell carcinoma of the skin or carcinoma in situ of the cervix), unless the disease-free period prior to screening exceeds 5 years.
- Participant is receiving anti-coagulation therapy or has a history of thrombotic episodes (including deep vein thrombosis, myocardial infarction, cerebrovascular accident, pulmonary embolism) within 12 months prior to screening or a history of thrombophilia.
- Participant has abnormal protein loss (protein losing enteropathy, nephrotic syndrome).
- Participant has anemia that would preclude phlebotomy for laboratory studies according to standard practice at the site.
- Participant has an ongoing history of hypersensitivity or persistent reactions (urticaria, breathing difficulty, severe hypotension, or anaphylaxis) following IV immunoglobulin, SC immunoglobulin, and/or Immune Serum Globulin (ISG) infusions.
- Participant has immunoglobulin A (IgA) deficiency (IgA less than 0.07 g/L), known anti IgA antibodies, and a history of hypersensitivity.
- Participant is on preventative (prophylactic) systemic antibacterial antibiotics at doses sufficient to treat or prevent bacterial infections, and cannot stop these antibiotics at the time of screening.
- Participant has active infection and is receiving antibiotic therapy for the treatment of infection at the time of screening.
- Participant has a bleeding disorder, or a platelet count less than 20,000/ microliter (mcL), or, in the opinion of the investigator, would be at significant risk of increased bleeding or bruising as a result of subcutaneous therapy.
- Participant has total protein > 9 gram per deciliter (g/dL) or myeloma, or macroglobulinemia (IgM) or paraproteinemia.
Women of childbearing potential meeting any one of the following criteria:
- Participant presents with a positive pregnancy test.
- Participant is breast feeding.
- Participant intends to begin nursing during the course of the study.
- Participant does not agree to employ adequate birth-control measures (e.g. intrauterine device, diaphragm or condom [for male partner] with spermicidal jelly or foam, or birth control pills/patches) throughout the course of the study.
- Participant has participated in another clinical study and has been exposed to an IP or device within 30 days prior to study enrollment.
- Participant is scheduled to participate in another non-observational (interventional) clinical study involving an IP or device during the course of the study.
- Participant has severe dermatitis that would preclude adequate sites for safe product administration.
Sites / Locations
- Nagoya University Hospital
- Kyushu University Hospital
- Kurume University Hospital
- Gifu University Hospital
- Hiroshima University Hospital
- Kanazawa University Hospital
- National Defense Medical College Hospital
- Tokyo Medical Dental University Hospital
Arms of the Study
Arm 1
Arm 2
Arm 3
Experimental
Experimental
Experimental
Epoch 1: Immune Globulin Intravenous (IGIV)
Epoch 2: Immune Globulin Subcutaneous 20% Solution (IGSC)
Epoch 3: Immune Globulin Subcutaneous 20% Solution (IGSC)
Participants will receive approximately 200 to 600 milligrams per kilogram (mg/kg) of Immunoglobulin Globulin Intravenous (IGIV) infusion for every 3 or 4 weeks for a total of 13 weeks.
Participants will receive approximately 50 to 200 mg/kg of IGSC infusion, 20 percent (%) once a week for a total of 24 weeks.
Participants will receive approximately 100 to 400 mg/kg of IGSC infusion, 20% once every two weeks in a sub-set of 7 participants for a total of 12 weeks.