A Study of Immune Globulin Subcutaneous (Human), 20% Solution (IGSC, 20%) in Japanese Participants With Primary Immunodeficiency Diseases (PID)

NCT04346108 | Completed Phase 3 Results

• 2 other identifiers: TAK-664-3001JapicCTI-205162
• Study Type: interventional
• Target: 17
• Locations: 1 country
• Sites: 8

Brief Summary

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.

Conditions

Primary Immunodeficiency Diseases (PID)

Outcome Measures

Primary Outcomes (2)

• Epoch 2: Total Serum Trough Levels of Immune Globulin G (IgG) Antibodies During Period 2

  Total serum trough levels of IgG antibodies measured during period 2 of Epoch 2 were assessed.

  Epoch 2 (period 2): Up to 24 weeks

• Epoch 3: Total Serum Trough Levels of IgG Antibodies

  Total serum trough levels of IgG antibodies measured during Epoch 3 were assessed.

  Epoch 3: Up to Week 12

Secondary Outcomes (28)

• Epoch 1: Total Serum Trough Levels of IgG Antibodies

  Epoch 1: Up to Week 13

• Epoch 2: Area Under the Curve From Time 0 to Last Interval (AUC0-last) for Total Serum Levels of IgG

  Epoch 2: Week 21

• Epoch 2: AUC0-last for Total Serum Levels of IgG Subclasses

  Epoch 2: Week 21

• Epoch 2: Apparent Clearance (CL/F) for Total Serum Levels of IgG

  Epoch 2: Week 21

• Epoch 2: CL/F for Total Serum Levels of IgG Subclasses

  Epoch 2: Week 21

Study Arms (3)

Epoch 1: IGIV 200-600 mg/kg

EXPERIMENTAL

Participants received 200 to 600 mg/kg of Immunoglobulin Intravenous (IGIV) infusion for every 3 or 4 weeks for up to 13 weeks.

Biological: Immune Globulin Intravenous (IGIV)

Epoch 2: IGSC (20%) 50-200 mg/kg

EXPERIMENTAL

Participants who entered to Epoch 2 from Epoch 1 received 50-200 mg/kg of Immune Globulin Subcutaneous (Human) 20% infusion once a week up to approximately 24 weeks after Epoch 1.

Biological: Immune Globulin Subcutaneous, 20% Solution (IGSC, 20%)

Epoch 3: IGSC (20%) 100-400 mg/kg

EXPERIMENTAL

Participants who entered to Epoch 3 from Epoch 1 received 100-400 mg/kg of Immune Globulin Subcutaneous (Human) 20% infusion biweekly up to approximately 12 weeks after Epoch 2.

Biological: Immune Globulin Subcutaneous, 20% Solution (IGSC, 20%)

Interventions

Immune Globulin Intravenous (IGIV) BIOLOGICAL

Participants will receive IGIV infusion.

Also known as: Immune Globulin Infusion (Human)

Epoch 1: IGIV 200-600 mg/kg

Immune Globulin Subcutaneous, 20% Solution (IGSC, 20%) BIOLOGICAL

Participants will receive IGSC, 20% SC infusion.

Also known as: Immune Globulin Infusion (Human)

Epoch 2: IGSC (20%) 50-200 mg/kg; Epoch 3: IGSC (20%) 100-400 mg/kg

Eligibility Criteria

• Age: 2 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Baxalta now part of Shire: lead

Study Sites (8)

Nagoya University Hospital

Nagoya, Aichi-ken, 466-8560, Japan

Location

Kyushu University Hospital

Fukuoka, Fukuoka, 812-8582, Japan

Location

Kurume University Hospital

Kurume-shi, Fukuoka, 830-0011, Japan

Location

Gifu University Hospital

Gifu, Gifu, 501-1194, Japan

Location

Hiroshima University Hospital

Hiroshima, Hiroshima, 734-8551, Japan

Location

Kanazawa University Hospital

Kanazawa, Ishikawa-ken, 920-8641, Japan

Location

National Defense Medical College Hospital

Tokorozawa-shi, Saitama, 359-8513, Japan

Location

Tokyo Medical Dental University Hospital

Bunkyo-ku, Tokyo, 113-8519, Japan

Location

MeSH Terms

Conditions

Primary Immunodeficiency Diseases

Interventions

gamma-Globulins; Solutions

Condition Hierarchy (Ancestors)

Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Immunologic Deficiency Syndromes; Immune System Diseases

Intervention Hierarchy (Ancestors)

Immunoglobulins; Immunoproteins; Blood Proteins; Proteins; Amino Acids, Peptides, and Proteins; Serum Globulins; Globulins; Pharmaceutical Preparations

Results Point of Contact

• Title: Study Director
• Organization: Shire

ClinicalTransparency@shire.com

+1 866 842 5335

Study Officials

• Study Director

  Shire

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 10, 2020
• First Posted: April 15, 2020
• Study Start: August 11, 2020
• Primary Completion: December 22, 2021
• Study Completion: December 22, 2021
• Last Updated: March 22, 2024
• Results First Posted: March 22, 2024

Record last verified: 2023-09

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF, CSR
• Access Criteria: IPD from eligible studies will be shared with qualified researchers according to the criteria and process described on https://vivli.org/ourmember/takeda/. For approved requests, the researchers will be provided access to anonymized data (to respect patient privacy in line with applicable laws and regulations) and with information necessary to address the research objectives under the terms of a data sharing agreement.

Locations

JP (8)