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

Phase 3

Completed

• Conditions: Primary Immunodeficiency Disease
• Interventions: Biological: IGSC 20% infusion

• Registration Number: NCT04842643
• Lead Sponsor: Takeda

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2021-04-11
• Study First Submitted QC: 2021-04-11
• Study First Posted: 2021-04-13
• Study Start: 2021-04-27
• Primary Completion: 2024-04-25
• Study Completion: 2024-04-25
• Status Verified: 2024-05
• Last Update Submitted: 2024-05-08
• Last Update Posted: 2024-05-10

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 12

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.

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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.

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Epoch 3 in previous study: Immune Globulin Subcutaneous 20% Solution (IGSC)	IGSC 20% infusion	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.
Epoch 2 in previous study: Immune Globulin Subcutaneous 20% Solution (IGSC)	IGSC 20% infusion	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.

Primary Outcome Measures

Name	Time	Method
Number of Participants with Treatment Emergent Adverse Events (TEAEs)	Up to 3 years	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

Name	Time	Method
Serum Trough Levels of IgG Antibodies Following Biweekly Administration of IGSC, 20%	Baseline, Every 12 weeks after first dose up to 3 years	Serum trough levels of IgG antibodies measured during treatment after Epoch 3 of the previous study will be assessed.
Annual Rate of Validated Acute Serious Bacterial Infections (ASBI)	Baseline, Up to 3 years	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	Baseline, Up to 3 years	Annual rate is the number of participants reporting any infection per year.
Treatment Preference	Baseline, End of study (3 years)	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.
Serum Trough Levels of Immune Globulin G (IgG) Antibodies Following Weekly Administration of IGSC, 20%	Baseline, Every 12 weeks after first dose up to 3 years	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%	Baseline, Every 12 weeks after first dose up to 3 years	Serum trough levels of IgG subclasses measured during treatment after Epoch 2 of the previous study will be assessed.
Number of Hospitalizations due to Illness or Infection	Baseline, Up to 3 years	Number of hospitalizations due to illness or infection will be assessed.
Serum Trough Levels of IgG Subclasses Following Biweekly Administration of IGSC, 20%	Baseline, Every 12 weeks after first dose up to 3 years	Serum trough levels of IgG subclasses measured during treatment after Epoch 3 of the previous study will be assessed.
Length of Hospital Stay due to Illness or Infection	Baseline, Up to 3 years	Number of days due to illness or infection will be assessed.
Number of Days Participants not Able to Attend School or Work to Perform Normal Daily Activities due to Illness/Infection	Baseline, Up to 3 years	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	Baseline, Up to 3 years	Number of days participants on antibiotics will be assessed.
Number of Acute Physician Visits due to Illness/Infection	Baseline, Up to 3 years	Number of acute (urgent or unscheduled) physician visits due to illness/infection will be reported.

Trial Locations

Locations (8)

Kurume University Hospital; 🇯🇵 Kurume, Fukuoka, Japan

Gifu University Hospital; 🇯🇵 Gifu, Japan

Tokyo Medical Dental University Hospital; 🇯🇵 Tokyo, Japan

Nagoya University Hospital; 🇯🇵 Nagoya, Aichi, Japan

Hiroshima University Hospital; 🇯🇵 Hiroshima, Japan

Kyushu University Hospital; 🇯🇵 Fukuoka, Japan

Kanazawa University Hospital; 🇯🇵 Kanazawa, Ishikawa, Japan

National defense medical college Hospital; 🇯🇵 Tokorozawa, Saitama, Japan