Back to resultsAn Extension Study of TAK-664 for Japanese People With Primary Immunodeficiency Disease
Primary Purpose
Primary Immunodeficiency Disease
Status
Active
Phase
Phase 3
Locations
Japan
Study Type
Interventional
Intervention
IGSC 20% infusion
Sponsored by
About this trial
This is an interventional treatment trial for Primary Immunodeficiency Disease
Eligibility Criteria
Inclusion Criteria:
Participant has completed or is about to complete Takeda Clinical Study TAK-664-3001.
A participant is considered to have completed Study TAK-664-3001 successfully if they fulfill the following criterion: Completed Epoch 2, in which IGSC, 20% is administered weekly (completion of Epoch 3, in which IGSC, 20% is administered biweekly, is not mandatory for participation in TAK-664-3002 study).
- Written informed consent is obtained from either the Participant or the Participant's legally authorized representative prior to any study-related procedures and study product administration.
- Participant is willing and able to comply with the requirements of the protocol.
Exclusion Criteria:
- Participant has developed a new serious medical condition during participation in Study TAK-664-3001 such that the Participant's safety or medical care would be impacted by participation in the extension study TAK-664-3002.
- Participant is scheduled to participate in another non-Takeda clinical study involving an Investigational Product or investigational device in the course of this study.
- If a female of childbearing potential, Participant is pregnant or has a negative pregnancy test but does not agree to employ adequate birth control measures for the duration of the study.
Sites / Locations
- Nagoya University Hospital
- Kurume University Hospital
- Kanazawa University Hospital
- National defense medical college Hospital
- Kyushu University Hospital
- Gifu University Hospital
- Hiroshima University Hospital
- Tokyo Medical Dental University Hospital
Arms of the Study
Arm 1
Arm 2
Arm Type
Experimental
Experimental
Arm Label
Epoch 2 in previous study: Immune Globulin Subcutaneous 20% Solution (IGSC)
Epoch 3 in previous study: Immune Globulin Subcutaneous 20% Solution (IGSC)
Arm Description
Participants will receive between 50 and 200 mg/kg of Immunoglobulin Globulin subcutaneous (IGSC) infusion, 20 percent (%) once a week until the study drug becomes commercially available (approximately 3 years). All participants of this arm will have assigned Epoch 2 of previous study (TAK-664-3001). The dose of IGSC will be established in previous study.
Participants will receive between 100 and 400 mg/kg of Immunoglobulin Globulin subcutaneous (IGSC) infusion, 20 percent (%) once every 2 weeks until the study drug becomes commercially available (approximately 3 years). All participants of this arm will have assigned Epoch 3 of previous study (TAK-664-3001).The dose of IGSC will be established in previous study (TAK-664-3001). For participants who discontinue Epoch 3 and enter Study TAK-664-3002, the dose regimen will be determined on a case-by-case basis.
Outcomes
Primary Outcome Measures
Number of Participants with Treatment Emergent Adverse Events (TEAEs)
Treatment emergent adverse events are defined as AEs with onset after date/time of first dose of study drug in Study-TAK-664-3001, or medical conditions present prior to the start of study drug Study-TAK-664-3001 but increased in severity or relationship after date/time of first dose of study drug in Study-TAK-664-3001. An AE is defined as any untoward medical occurrence in a clinical investigation subject administered a drug; it does not necessarily have to have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (eg, a clinically significant abnormal laboratory value), symptom, or disease temporally associated with the use of a drug whether or not it is considered related to the drug. Number of participants with TEAEs will be assessed.
Secondary Outcome Measures
Serum Trough Levels of Immune Globulin G (IgG) Antibodies Following Weekly Administration of IGSC, 20%
Serum trough levels of IgG antibodies measured during treatment after Epoch 2 of the previous study will be assessed.
Serum Trough Levels of IgG Subclasses Following Weekly Administration of IGSC, 20%
Serum trough levels of IgG subclasses measured during treatment after Epoch 2 of the previous study will be assessed.
Serum Trough Levels of IgG Antibodies Following Biweekly Administration of IGSC, 20%
Serum trough levels of IgG antibodies measured during treatment after Epoch 3 of the previous study will be assessed.
Serum Trough Levels of IgG Subclasses Following Biweekly Administration of IGSC, 20%
Serum trough levels of IgG subclasses measured during treatment after Epoch 3 of the previous study will be assessed.
Annual Rate of Validated Acute Serious Bacterial Infections (ASBI)
The ASBI rate will be calculated as the mean number of ASBI per participants per year. Annual rate of validated ASBI per participant will be assessed.
Annual Rate of All Infections
Annual rate is the number of participants reporting any infection per year.
Number of Days Participants not Able to Attend School or Work to Perform Normal Daily Activities due to Illness/Infection
Number of days not able to attend school or work to perform normal daily activities due to illness/infection will be assessed.
Number of Days Participants on Antibiotics
Number of days participants on antibiotics will be assessed.
Number of Hospitalizations due to Illness or Infection
Number of hospitalizations due to illness or infection will be assessed.
Length of Hospital Stay due to Illness or Infection
Number of days due to illness or infection will be assessed.
Number of Acute Physician Visits due to Illness/Infection
Number of acute (urgent or unscheduled) physician visits due to illness/infection will be reported.
Treatment Preference
Treatment preference questionnaire is a self-administered questionnaire developed to assess participants' preference towards the administration of new IGSC therapy. There are 4-items on the questionnaire, which investigate a participant's preference on the clinic/hospital/home setting of receiving the immunoglobulin therapy, the participant's rating on the frequency and method of administration, and the participant's preference to continue receiving the IGSC treatment.
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT04842643
Brief Title
An Extension Study of TAK-664 for Japanese People With Primary Immunodeficiency Disease
Official Title
A Phase 3, Open-label, Non-controlled, Multi-dose, Extension Study to Evaluate the Long-term Safety and Tolerability of IGSC, 20% in Japanese Subjects With Primary Immunodeficiency Disease (PID)
Study Type
Interventional
2. Study Status
Record Verification Date
April 2023
Overall Recruitment Status
Active, not recruiting
Study Start Date
April 27, 2021 (Actual)
Primary Completion Date
April 30, 2024 (Anticipated)
Study Completion Date
April 30, 2024 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Takeda
4. Oversight
Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No
5. Study Description
Brief Summary
This study is an extension study for participants with primary immunodeficiency disorders who were previously treated with IGSC, 20% in the TAK-664-3001 study. They must have completed that study or be about to complete it before joining this study. Participants will continue treatment with IGCS, 20% in this study.
The main aim of this study is to check for side effects from long-term treatment with IGSC, 20% . This medicine is not yet licensed in Japan, so participants will be treated with IGSC, 20% until it becomes commercially available.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Primary Immunodeficiency Disease
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Parallel Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
12 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Epoch 2 in previous study: Immune Globulin Subcutaneous 20% Solution (IGSC)
Arm Type
Experimental
Arm Description
Participants will receive between 50 and 200 mg/kg of Immunoglobulin Globulin subcutaneous (IGSC) infusion, 20 percent (%) once a week until the study drug becomes commercially available (approximately 3 years). All participants of this arm will have assigned Epoch 2 of previous study (TAK-664-3001). The dose of IGSC will be established in previous study.
Arm Title
Epoch 3 in previous study: Immune Globulin Subcutaneous 20% Solution (IGSC)
Arm Type
Experimental
Arm Description
Participants will receive between 100 and 400 mg/kg of Immunoglobulin Globulin subcutaneous (IGSC) infusion, 20 percent (%) once every 2 weeks until the study drug becomes commercially available (approximately 3 years). All participants of this arm will have assigned Epoch 3 of previous study (TAK-664-3001).The dose of IGSC will be established in previous study (TAK-664-3001).
For participants who discontinue Epoch 3 and enter Study TAK-664-3002, the dose regimen will be determined on a case-by-case basis.
Intervention Type
Biological
Intervention Name(s)
IGSC 20% infusion
Other Intervention Name(s)
Immune Globulin Infusion (Human)
Intervention Description
IGSC 20% infusion, The dose of IGSC will be established in previous study (TAK-664-3001)
Primary Outcome Measure Information:
Title
Number of Participants with Treatment Emergent Adverse Events (TEAEs)
Description
Treatment emergent adverse events are defined as AEs with onset after date/time of first dose of study drug in Study-TAK-664-3001, or medical conditions present prior to the start of study drug Study-TAK-664-3001 but increased in severity or relationship after date/time of first dose of study drug in Study-TAK-664-3001. An AE is defined as any untoward medical occurrence in a clinical investigation subject administered a drug; it does not necessarily have to have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (eg, a clinically significant abnormal laboratory value), symptom, or disease temporally associated with the use of a drug whether or not it is considered related to the drug. Number of participants with TEAEs will be assessed.
Time Frame
Up to 3 years
Secondary Outcome Measure Information:
Title
Serum Trough Levels of Immune Globulin G (IgG) Antibodies Following Weekly Administration of IGSC, 20%
Description
Serum trough levels of IgG antibodies measured during treatment after Epoch 2 of the previous study will be assessed.
Time Frame
Baseline, Every 12 weeks after first dose up to 3 years
Title
Serum Trough Levels of IgG Subclasses Following Weekly Administration of IGSC, 20%
Description
Serum trough levels of IgG subclasses measured during treatment after Epoch 2 of the previous study will be assessed.
Time Frame
Baseline, Every 12 weeks after first dose up to 3 years
Title
Serum Trough Levels of IgG Antibodies Following Biweekly Administration of IGSC, 20%
Description
Serum trough levels of IgG antibodies measured during treatment after Epoch 3 of the previous study will be assessed.
Time Frame
Baseline, Every 12 weeks after first dose up to 3 years
Title
Serum Trough Levels of IgG Subclasses Following Biweekly Administration of IGSC, 20%
Description
Serum trough levels of IgG subclasses measured during treatment after Epoch 3 of the previous study will be assessed.
Time Frame
Baseline, Every 12 weeks after first dose up to 3 years
Title
Annual Rate of Validated Acute Serious Bacterial Infections (ASBI)
Description
The ASBI rate will be calculated as the mean number of ASBI per participants per year. Annual rate of validated ASBI per participant will be assessed.
Time Frame
Baseline, Up to 3 years
Title
Annual Rate of All Infections
Description
Annual rate is the number of participants reporting any infection per year.
Time Frame
Baseline, Up to 3 years
Title
Number of Days Participants not Able to Attend School or Work to Perform Normal Daily Activities due to Illness/Infection
Description
Number of days not able to attend school or work to perform normal daily activities due to illness/infection will be assessed.
Time Frame
Baseline, Up to 3 years
Title
Number of Days Participants on Antibiotics
Description
Number of days participants on antibiotics will be assessed.
Time Frame
Baseline, Up to 3 years
Title
Number of Hospitalizations due to Illness or Infection
Description
Number of hospitalizations due to illness or infection will be assessed.
Time Frame
Baseline, Up to 3 years
Title
Length of Hospital Stay due to Illness or Infection
Description
Number of days due to illness or infection will be assessed.
Time Frame
Baseline, Up to 3 years
Title
Number of Acute Physician Visits due to Illness/Infection
Description
Number of acute (urgent or unscheduled) physician visits due to illness/infection will be reported.
Time Frame
Baseline, Up to 3 years
Title
Treatment Preference
Description
Treatment preference questionnaire is a self-administered questionnaire developed to assess participants' preference towards the administration of new IGSC therapy. There are 4-items on the questionnaire, which investigate a participant's preference on the clinic/hospital/home setting of receiving the immunoglobulin therapy, the participant's rating on the frequency and method of administration, and the participant's preference to continue receiving the IGSC treatment.
Time Frame
Baseline, End of study (3 years)
10. Eligibility
Sex
All
Minimum Age & Unit of Time
2 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Participant has completed or is about to complete Takeda Clinical Study TAK-664-3001.
A participant is considered to have completed Study TAK-664-3001 successfully if they fulfill the following criterion: Completed Epoch 2, in which IGSC, 20% is administered weekly (completion of Epoch 3, in which IGSC, 20% is administered biweekly, is not mandatory for participation in TAK-664-3002 study).
Written informed consent is obtained from either the Participant or the Participant's legally authorized representative prior to any study-related procedures and study product administration.
Participant is willing and able to comply with the requirements of the protocol.
Exclusion Criteria:
Participant has developed a new serious medical condition during participation in Study TAK-664-3001 such that the Participant's safety or medical care would be impacted by participation in the extension study TAK-664-3002.
Participant is scheduled to participate in another non-Takeda clinical study involving an Investigational Product or device-used-in-clinical-trial in the course of this study.
If a female of childbearing potential, Participant is pregnant or has a negative pregnancy test but does not agree to employ adequate birth control measures for the duration of the study.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Study Director
Organizational Affiliation
Takeda
Official's Role
Study Director
Facility Information:
Facility Name
Nagoya University Hospital
City
Nagoya
State/Province
Aichi
Country
Japan
Facility Name
Kurume University Hospital
City
Kurume
State/Province
Fukuoka
Country
Japan
Facility Name
Kanazawa University Hospital
City
Kanazawa
State/Province
Ishikawa
Country
Japan
Facility Name
National defense medical college Hospital
City
Tokorozawa
State/Province
Saitama
Country
Japan
Facility Name
Kyushu University Hospital
City
Fukuoka
Country
Japan
Facility Name
Gifu University Hospital
City
Gifu
Country
Japan
Facility Name
Hiroshima University Hospital
City
Hiroshima
Country
Japan
Facility Name
Tokyo Medical Dental University Hospital
City
Tokyo
Country
Japan
12. IPD Sharing Statement
Plan to Share IPD
No
IPD Sharing Plan Description
De-identified individual participant data from this particular study will not be shared as there is a reasonable likelihood that individual patients could be re-identified (due to the limited number of study participants/study sites).
Links:
URL
https://clinicaltrials.takeda.com/study-detail/60769026688ad8001f42fb97
Description
To obtain more information on the study, click here/on this link
Learn more about this trial
An Extension Study of TAK-664 for Japanese People With Primary Immunodeficiency Disease
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