An Extension Study of TAK-664 for Japanese People With Primary Immunodeficiency Disease

NCT04842643 | Completed Phase 3 Results

• 3 other identifiers: TAK-664-3002U1111-1265-9447jRCT2041210006
• Study Type: interventional
• Target: 12
• Locations: 1 country
• Sites: 8

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.

Conditions

Primary Immunodeficiency Disease

Outcome Measures

Primary Outcomes (4)

• Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs and Non-serious TEAEs

  TEAEs were defined as adverse events (AEs) with onset after date-time of first dose of study drug (intravenous immunoglobulin \[IGIV\] or IGSC), or medical conditions present prior to the start of study drug (IGIV or IGSC) but increased in severity or relationship after date-time of first dose of study drug (IGIV or IGSC). A serious TEAE was an AE that resulted in any of the following outcomes: death; life threatening; persistent/significant disability/incapacity; initial or prolonged inpatient hospitalization; congenital anomaly/birth defect or was otherwise considered medically important.

  From the first dose of the study drug in the current extension study TAK-664-3002, up to 3 years post-dose

• Number of Participants With Drug-related and Non-related TEAEs

  TEAEs were defined as adverse events (AEs) with onset after date-time of first dose of study drug (intravenous immunoglobulin \[IGIV\] or IGSC), or medical conditions present prior to the start of study drug (IGIV or IGSC) but increased in severity or relationship after date-time of first dose of study drug (IGIV or IGSC). Any TEAE that was recorded by the investigator as "probably related" or "possibly related" to study drug was considered as study drug related AE, and any AE recorded as "unlikely related" or "not related" was considered as unrelated AE.

  From the first dose of the study drug in the current extension study TAK-664-3002, up to 3 years post-dose

• Number of Participants With Severe, Local and Systemic TEAEs

  A severe TEAE was an AE that caused considerable interference with the participant's usual activities. Local TEAEs were defined as AEs that were included in the MedDRA Higher Level Group Term "administration site reactions" or contained the phrase "injection site" or "infusion site". Systemic TEAEs were defined as AEs that were not included in the Medical Dictionary for Regulatory Activities (MedDRA) Higher Level Group Term "administration site reactions" and did not contain the phrase "injection site" or "infusion site".

  From the first dose of the study drug in the current extension study TAK-664-3002, up to 3 years post-dose

• Number of Participants With TEAEs Leading to Premature Discontinuation From Study and Infusion-associated TEAEs

  Infusion associated TEAEs were defined as any TEAE that began during study drug infusion or within 72 hours of completion of study drug infusion. TEAEs leading to premature discontinuation from study and infusion-associated TEAEs were reported.

  From the first dose of the study drug in the current extension study TAK-664-3002, up to 3 years post-dose

Secondary Outcomes (11)

• Serum Trough Levels of Total Immune Globulin G (IgG) and IgG1, IgG2, IgG3, IgG4 Antibodies Subclasses Following Weekly Administration of IGSC, 20%

  At end of treatment in the current extension study TAK-664-3002 (i.e. 3 years)

• Serum Trough Levels of IgG and IgG1, IgG2, IgG3, IgG4 Antibodies Subclasses Following Biweekly Administration of IGSC, 20%

  At end of treatment in the current extension study TAK-664-3002 (i.e. 3 years)

• Annual Rate of Validated Acute Serious Bacterial Infections (ASBI)

  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)

• Annual Rate of All Infections

  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)

• Number of Days Participants Not Able to Attend School or Work to Perform Normal Daily Activities Due to Illness/Infection

  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)

Study Arms (1)

IGSC, 20%

EXPERIMENTAL

Participants who completed Epoch 2 of core study TAK-664-3001 (NCT04346108), received between 50 to 200 mg/kg of Immune globulin subcutaneous (IGSC) infusion, 20% infusion once a week (Epoch 2 regimen) and 100 to 400 mg/kg of IGSC infusion, 20% biweekly (Epoch 3 regimen) until the study drug becomes commercially available.

Biological: IGSC 20% infusion

Interventions

IGSC 20% infusion BIOLOGICAL

IGSC 20% infusion,

Also known as: Immune Globulin Infusion (Human)

IGSC, 20%

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:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Takeda: lead

Study Sites (8)

Nagoya University Hospital

Nagoya, Aichi-ken, Japan

Location

Kurume University Hospital

Kurume, Fukuoka, Japan

Location

Kanazawa University Hospital

Kanazawa, Ishikawa-ken, Japan

Location

National defense medical college Hospital

Tokorozawa, Saitama, Japan

Location

Kyushu University Hospital

Fukuoka, Japan

Location

Gifu University Hospital

Gifu, Japan

Location

Hiroshima University Hospital

Hiroshima, Japan

Location

Tokyo Medical Dental University Hospital

Tokyo, Japan

Location

Related Links

• To obtain more information on the study, click here/on this link

MeSH Terms

Conditions

Primary Immunodeficiency Diseases

Condition Hierarchy (Ancestors)

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

Results Point of Contact

• Title: Study Director
• Organization: Takeda

TrialDisclosures@takeda.com

+1-877-825-3327

Study Officials

• Study Director

  Takeda

  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: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 11, 2021
• First Posted: April 13, 2021
• Study Start: April 27, 2021
• Primary Completion: April 25, 2024
• Study Completion: April 25, 2024
• Last Updated: January 20, 2025
• Results First Posted: January 20, 2025

Record last verified: 2024-12

Data Sharing

• IPD Sharing: Will not share

Locations

JP (8)