Clinical trial NCT04842643: 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
Overall status: COMPLETED
Phase: PHASE3
Conditions: Primary Immunodeficiency Disease
Lead sponsor: Takeda
Sponsor class: INDUSTRY
First posted: 2021-04-13
Enrollment: 12
Sex: ALL
Minimum age (years): 2
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.


Eligibility criteria: Inclusion Criteria:

1. 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).
2. 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.
3. Participant is willing and able to comply with the requirements of the protocol.

Exclusion Criteria:

1. 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.
2. 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.
3. 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.

Primary outcomes:
measure: Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs and Non-serious TEAEs
timeFrame: From the first dose of the study drug in the current extension study TAK-664-3002, up to 3 years post-dose
measure: Number of Participants With Drug-related and Non-related TEAEs
timeFrame: From the first dose of the study drug in the current extension study TAK-664-3002, up to 3 years post-dose
measure: Number of Participants With Severe, Local and Systemic TEAEs
timeFrame: From the first dose of the study drug in the current extension study TAK-664-3002, up to 3 years post-dose
measure: Number of Participants With TEAEs Leading to Premature Discontinuation From Study and Infusion-associated TEAEs
timeFrame: From the first dose of the study drug in the current extension study TAK-664-3002, up to 3 years post-dose
Secondary outcomes:
measure: Serum Trough Levels of Total Immune Globulin G (IgG) and IgG1, IgG2, IgG3, IgG4 Antibodies Subclasses Following Weekly Administration of IGSC, 20%
timeFrame: At end of treatment in the current extension study TAK-664-3002 (i.e. 3 years)
measure: Serum Trough Levels of IgG and IgG1, IgG2, IgG3, IgG4 Antibodies Subclasses Following Biweekly Administration of IGSC, 20%
timeFrame: At end of treatment in the current extension study TAK-664-3002 (i.e. 3 years)
measure: Annual Rate of Validated Acute Serious Bacterial Infections (ASBI)
timeFrame: From first dose of study drug in core study TAK-664-3001 up to end of current extension study TAK-664-3002 (up to approximately 4.5 years)
measure: Annual Rate of All Infections
timeFrame: From first dose of study drug in core study TAK-664-3001 up to end of current extension study TAK-664-3002 (up to approximately 4.5 years)
measure: Number of Days Participants Not Able to Attend School or Work to Perform Normal Daily Activities Due to Illness/Infection
timeFrame: From first dose of study drug in core study TAK-664-3001 up to end of current extension study TAK-664-3002 (up to approximately 4.5 years)
measure: Number of Days Participants on Antibiotics
timeFrame: From first dose of study drug in core study TAK-664-3001 up to end of current extension study TAK-664-3002 (up to approximately 4.5 years)
measure: Number of Hospitalizations Due to Illness or Infection
timeFrame: From first dose of study drug in core study TAK-664-3001 up to end of current extension study TAK-664-3002 (up to approximately 4.5 years)
measure: Length of Hospital Stay Due to Illness or Infection
timeFrame: From first dose of study drug in core study TAK-664-3001 up to end of current extension study TAK-664-3002 (up to approximately 4.5 years)
measure: Number of Acute Physician Visits Due to Illness/Infection
timeFrame: From first dose of study drug in core study TAK-664-3001 up to end of current extension study TAK-664-3002 (up to approximately 4.5 years)
measure: Number of Participants With Their Response for Treatment Preference Questionnaire
timeFrame: From the first dose of the study drug in the current extension study TAK-664-3002, up to 3 years post-dose
measure: Number of Participants With Tolerability Events Related to the Infusion of Study Drug
timeFrame: From the first dose of the study drug in the current extension study TAK-664-3002, up to 3 years post-dose
Other outcomes:
Locations:
city: Nagoya
country: Japan
facility: Nagoya University Hospital
state: Aichi
city: Kurume
country: Japan
facility: Kurume University Hospital
state: Fukuoka
city: Kanazawa
country: Japan
facility: Kanazawa University Hospital
state: Ishikawa
city: Tokorozawa
country: Japan
facility: National defense medical college Hospital
state: Saitama
city: Fukuoka
country: Japan
facility: Kyushu University Hospital
city: Gifu
country: Japan
facility: Gifu University Hospital
city: Hiroshima
country: Japan
facility: Hiroshima University Hospital
city: Tokyo
country: Japan
facility: Tokyo Medical Dental University Hospital
Study officials:
affiliation: Takeda
name: Study Director
role: STUDY_DIRECTOR
References: