Phase 3
N=17
A Study of Immune Globulin Subcutaneous (Human), 20% Solution (IGSC, 20%) in Japanese Participants With Primary Immunodeficiency Diseases (PID)
Primary Immunodeficiency Diseases (PID)
Bottom Line
View on ClinicalTrials.gov: NCT04346108 ↗Enrolled (actual)
17
Serious AEs
0.0%
Results posted
Mar 2024
Primary outcome: Primary: Epoch 2: Total Serum Trough Levels of Immune Globulin G (IgG) Antibodies During Period 2 — 8.56 grams per liter (g/L)
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- Immune Globulin Intravenous (IGIV) (Biological); Immune Globulin Subcutaneous, 20% Solution (IGSC, 20%) (Biological)
- Age
- Pediatric, Adult, Older Adult · 2+ yrs
- Sex
- All
- Sponsor
- Baxalta now part of Shire
- Primary completion
- Dec 2021
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Epoch 2: Total Serum Trough Levels of Immune Globulin G (IgG) Antibodies During Period 2 |
8.56 | — |
| PRIMARY Epoch 3: Total Serum Trough Levels of IgG Antibodies |
8.39 | — |
| SECONDARY Epoch 1: Total Serum Trough Levels of IgG Antibodies |
8.18 | — |
| SECONDARY Epoch 2: Area Under the Curve From Time 0 to Last Interval (AUC0-last) for Total Serum Levels of IgG |
58.9 | — |
| SECONDARY Epoch 2: AUC0-last for Total Serum Levels of IgG Subclasses |
35.8; 23.4; 1.32; 1.67 | — |
| SECONDARY Epoch 2: Apparent Clearance (CL/F) for Total Serum Levels of IgG |
1.93 | — |
| SECONDARY Epoch 2: CL/F for Total Serum Levels of IgG Subclasses |
3.18; 4.86; 99.5; 68.3 | — |
| SECONDARY Epoch 2: Maximum Concentration (Cmax) for Total Serum Levels of IgG |
9.08 | — |
| SECONDARY Epoch 2: Cmax for Total Serum Levels of IgG Subclasses |
5.40; 3.51; 0.202; 0.256 | — |
| SECONDARY Epoch 2: Minimum Concentration (Cmin) for Total Serum Levels of IgG |
7.45 | — |
| SECONDARY Epoch 2: Cmin for Total Serum Levels of IgG Subclasses |
4.89; 3.15; 0.181; 0.218 | — |
| SECONDARY Epoch 2: Time to Maximum Concentration (Tmax) for Total Serum Levels of IgG |
71.08 | — |
| SECONDARY Epoch 2: Tmax for Total Serum Levels of IgG Subclasses |
71.08; 71.08; 72.92; 71.17 | — |
| SECONDARY Trough Levels of Specific Antibodies to Clinically Relevant Pathogens: Clostridium Tetani Toxoid and Hepatitis B Virus (HBV) |
0.524; 0.602; 0.674; 1.52; 1.97; 1.80 | — |
| SECONDARY Trough Levels of Specific Antibodies to Clinically Relevant Pathogen: Haemophilus Influenzae (HIB) |
1.58; 1.55; 2.50; 1.77; 2.04; 1.73 | — |
| SECONDARY Health Related Quality of Life: Treatment Preference |
0; 0; 0; 1; 3; 1 | — |
| SECONDARY Number of Participants With Treatment Emergent Adverse Events (TEAEs) |
11; 16; 3 | — |
| SECONDARY Number of Participants With Tolerability Events Related to the Infusion of Study Drug |
0; 0; 0; 0; 1; 0 | — |
| SECONDARY Annual Rate of Validated Acute Serious Bacterial Infections (ASBI) |
0.0; 0.25; 0.0 | — |
| SECONDARY Annual Rate of All Infections Per Year |
1.65; 2.78; 0.00 | — |
| SECONDARY Number of Days Participants Not Able to Attend School or Work to Perform Normal Daily Activities Due to Illness/Infection |
0.00; 0.00; 4.30 | — |
| SECONDARY Number of Days Participants Were on Antibiotics |
0.00; 0.00; 0.00 | — |
| SECONDARY Number of Participants Hospitalized Due to Illness or Infection |
0; 0; 0 | — |
| SECONDARY Length of Hospital Stay |
0.00; 0.00; 0.00 | — |
| SECONDARY Number of Acute Physician Visits Due to Illness/Infection |
1.18; 3.30; 1.624 | — |
| SECONDARY Health-related Quality of Life (HRQoL): Pediatric Quality of Life Inventory (PedsQL) Total Scale Score |
88.04; 87.23; 9.78; -6.79; 14.13; 20.65 | — |
| SECONDARY EuroQoL (Quality of Life)-5 Dimensions 3 Levels (EQ-5D-3L) Total Scale Score |
0.8080; 0.8217; 87.5; 77.1; -0.0270; 0.0016 | — |
| SECONDARY Health-related Quality of Life (HRQoL): Short Form-36 Health Survey (SF-36) Score |
50.21; 50.59; 0.10; -0.30; 1.81; 3.60 | — |
| SECONDARY Health Related Quality of Life: Treatment Satisfaction Questionnaire for Medication-9 (TSQM-9) Score |
69.44; 75.25; 62.96; 65.66; 75.00; 74.68 | — |
| SECONDARY Health Related Quality of Life: Treatment Satisfaction Questionnaire for Life Quality Index (LQI) Score |
81.48; 77.02; 81.94; 79.17; 77.78; 74.75 | — |
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.
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 >= 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] 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.
Data sourced from ClinicalTrials.gov (NCT04346108). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.