A Phase 3, Open-label, Non-controlled, Multi-dose Study to Evaluate the Pharmacokinetics, Safety and Tolerability, and Efficacy of Immune Globulin Subcutaneous (Human), 20% Solution (IGSC, 20%) in Japanese Subjects With Primary Immunodeficiency Diseases (PID)

TAK-664-3001

In this study, Japanese participants with primary immunodeficiency diseases were treated with Immune Globulin Subcutaneous (Human), 20% solution, (IGSC, 20%). This study will be in 3 parts: Part 1: Infusions with Immunoglobulin Intravenous (IGIV) every 3 or 4 weeks for 13 weeks. Part 2: Participants will switch to weekly subcutaneous infusions with IGSC, 20% for 24 weeks. Part 3: A subset will receive biweekly subcutaneous infusions with IGSC, 20% for 12 weeks. The main aim of the study is to assess base levels of Immunoglobulin globulin G (IgG) levels in the blood of the participants after weekly and biweekly treatment with IGSC, 20% (in Parts 2 and 3 of the study). Their PID will be treated by their doctor according to their doctor's usual clinical practice.

2020-08-11

2021-12-22

2021-12-22

Completed

Early phase I

17

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 \>= 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) \> 2.5 times the upper limit of normal (ULN) for the testing laboratory * Persistent severe neutropenia (defined as an absolute neutrophil count \[ANC\] \<= 500/milli cubic meter \[mm\^3\]). * Participant has presence of renal function impairment defined by estimated glomerular filtration rate (eGFR) is \<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.

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2024-03-22