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

JRCT ID: jRCT2041210006

Registered date:09/04/2021

Basic Information

• Recruitment status: Not Recruiting
• Health condition(s) or Problem(s) studied: Primary Immunodeficiency Disease
• Date of first enrollment: 27/04/2021
• Target sample size: 12
• Study type: Interventional

Intervention(s)

Epoch 2 in previous study: Immune Globulin Subcutaneous 20% Solution (IGSC)
Participants will receive between 50 and 200 mg/kg of Immunoglobulin Globulin subcutaneous (IGSC) infusion, 20 percent (%) once a week for 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.

Epoch 3 in previous study: Immune Globulin Subcutaneous 20% Solution (IGSC)
Participants will receive between 100 and 400 mg/kg of Immunoglobulin Globulin subcutaneous (IGSC) infusion, 20 percent (%) once every 2 weeks for 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.

Outcome(s)

Primary Outcome

1.Number of Participants with Treatment Emergent Adverse Events (TEAEs)
Time Frame: 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

1.Serum Trough Levels of Immune Globulin G (IgG) Antibodies Following Weekly Administration of IGSC, 20%
Timeframe: 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.

2.Serum Trough Levels of IgG Subclasses Following Weekly Administration of IGSC, 20%
Timeframe: 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.

3.Serum Trough Levels of IgG Antibodies Following Biweekly Administration of IGSC, 20%
Timeframe: 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.

4.Serum Trough Levels of IgG Subclasses Following Biweekly Administration of IGSC, 20%
Timeframe: 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.

5.Annual Rate of Validated Acute Serious Bacterial Infections (ASBI)
Time Frame: Baseline, Up to 3years
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.

6.Annual Rate of All Infections
Time Frame: Baseline, Up to 3 years
Annual rate is the number of participants reporting any infection per year.

7.Number of Days Participants not Able to Attend School or Work to Perform Normal Daily Activities due to Illness/Infection
Time Frame: 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.

8.Number of Days Participants on Antibiotics
Time Frame: Baseline, Up to 3 years
Number of days participants on antibiotics will be assessed.

9.Number of Hospitalizations due to Illness or Infection
Time Frame: Baseline, Up to 3 years
Number of hospitalizations due to illness or infection will be assessed.

10.Length of Hospital Stay due to Illness or Infection
Time Frame: Baseline, Up to 3 years
Number of days due to illness or infection will be assessed.

11.Number of Acute Physician Visits due to Illness/Infection
Time Frame: Baseline, up to 3 years
Number of acute (urgent or unscheduled) physician visits due to illness/infection will be reported.

12.Treatment Preference
Time Frame: 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.

Key inclusion & exclusion criteria

• Age minimum: >= 2age old
• Age maximum: Not applicable
• Gender: Both

Include 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.

Exclude 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.

Related Information

• Primary Sponsor: Nishizawa Atsushi
• Secondary ID(s): U1111-1265-9447,NCT04842643