Back to resultsA Study About the Long-Term Safety of TAK-881 in People With Primary Immunodeficiency Diseases
Primary Purpose
Primary Immunodeficiency Diseases (PID)
Status
Not yet recruiting
Phase
Phase 3
Locations
Study Type
Interventional
Intervention
TAK-881
Sponsored by
About this trial
This is an interventional treatment trial for Primary Immunodeficiency Diseases (PID)
Eligibility Criteria
Inclusion Criteria Participants who meet ALL of the following criteria are eligible for this study: Participant must have completed Study TAK-881-3001 (NCT05755035). If female of childbearing potential, the participant presents with a negative pregnancy test at screening and agrees to employ an acceptable form of contraception for the duration of the study. Participant/legally authorized representative is willing and able to comply with the requirements of the protocol. Participant/Participant's legally authorized representative has provided informed consent/assent, if applicable, (that is, in writing, documented via a signed and dated informed consent form (ICF) and/or eConsent, if available), and any required privacy authorization prior to the initiation of any study procedures. Exclusion Criteria Participant has a serious medical condition such that the Participant's safety or medical care would be impacted by participation in this long-term follow-up study. New medical condition that developed during participation in Study TAK-881-3001 (NCT05755035). that, in the judgment of the investigator, could increase risk to the Participant or interfere with the evaluation of TAK-881 and/or conduct of the study. Participant has participated in another clinical study involving an investigational product (IP) or investigational device within 30 days prior to screening (with the exception of Study TAK-881-3001 [NCT05755035]). Participant is scheduled to participate in another clinical study involving an IP or investigational device during the course of this study. Participant is a family member or employee of the investigator or the investigator's site staff. If female, Participant is pregnant or lactating at the time of screening or intends to become pregnant or begin lactating during the study.
Sites / Locations
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
TAK-881
Arm Description
Epoch 1: Participants with anti-rHuPH20 antibody titer <1:160 at all-time points during study TAK-881-3001 and study TAK-881-3002 will receive TAK-881 subcutaneous (SC) infusion at the same dose and same treatment interval as at the last infusion in TAK-881-3001 (NCT05755035) at Week 1 and at Weeks 13 and Week 25 of study TAK-881-3002. Epoch 2: Participants with anti-rHuPH20 antibody titer >=1:160 at any time point during Study TAK-881-3001 (NCT05755035) and/or Study TAK-881-3002 Epoch 1, will continue receiving TAK-881 every 12 weeks for up to Week 121. After the first TAK-881 infusion in study TAK-881-3002, the interval and/or the dose may be adjusted only at scheduled site visits at the investigator's discretion.
Outcomes
Primary Outcome Measures
Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
Number of Participants With Infusion Withdrawals, Interruptions, and Infusion Rate Reductions due to TAK-881-related TEAEs
Secondary Outcome Measures
Annualized Rate of all Infections
Annualized Rate of Acute Serious Bacterial Infections (ASBIs)
Annualized Rate of Episodes of Fever
Time to First ASBI
Duration of Infections
Number of Participants With Positive Binding Antibodies and Neutralizing Antibodies to rHuPH20
Trough Level of Total IgG
Doses of TAK-881
Treatment Interval of TAK-881
Number of Infusions Per Month With TAK-881
Number of Infusions Sites (Needle Sticks) per Month With TAK-881
Number of Infusions Sites (Needle Sticks) per infusion With TAK-881
Duration of Infusions (minutes) With TAK-881
Monthly Infusion Time (minutes/month) With TAK-881
Maximum Infusion Rate per Site (milliliter/hour/site) With TAK-881
Infusion Volume per Site (milliliter/site) With TAK-881
Treatment Satisfaction Questionnaire for Medication (TSQM-9) Scores in Participants Aged >=16 Years
TSQM-9 is a 9-item, validated, self-administered instrument to assess participants satisfaction with medication. It consists of 3 subscales: effectiveness, convenience and global satisfaction. The item scores of each of the 3 domains are summed and transformed to create a total score of 0 (extremely dissatisfied) to 100 (extremely satisfied). Higher score indicates greater satisfaction in that domain.
Health-Related Quality of Life (HRQoL): EuroQol 5 Dimension 5 Level Questionnaire (EQ-5D-5L) Score in Participants Aged >=12 Years
The EQ-5D-5L is a validated, self-administered assessment of overall health designed by the EuroQol Group. The instrument consists of two components: the EQ-5D descriptive system and the standard vertical EQ visual analogue scale (EQ-VAS). The descriptive system consists of 5 dimensions (mobility, self-care, usual activities, pain/discomfort, and anxiety/depression). Each dimension has five response categories: no problems, slight problems, some or moderate problems, severe problems, or extreme problems. Participants will be asked to describe their health state that day by choosing one of 5 responses that reflect the levels of severity for each of the 5 dimensions. The EQ-VAS measures one's self-perceived health today on a vertical scale from 0 (worst imaginable health) to 100 (best imaginable health) on which participants have to indicate their current health. Higher score indicates best health status.
Treatment Preference Measured by a Disease-specific Questionnaire in Participants Aged >=12 Years
The disease-specific treatment preference questionnaire is a self-administered, non-validated scale to assess participant preference for various attributes of IgG therapy and accompanying devices, such as ease of use of administration of TAK-881 complexity of administration, and convenience. The questionnaire consists of two components. The first component asks participants to indicate the extent to which they like or dislike aspects of TAK-881, including treatment frequency, time, ease, convenience, and complexity. Each item has five response categories: like very much, like, no preference, dislike, and dislike very much. The second component is a single question asking whether they would choose to continue receiving treatment after the trial ends.
Number of Days Lost From School, Work, Daycare, or to Perform Normal Daily Activities Due To Infection and/or their treatment or other Illnesses (non-Infection)
Number of Days on Antibiotics
Number of Hospitalizations With Indications (Infection or other Illnesses)
Number of Days of Hospitalization
Number of Acute Physician Visits (Office and Emergency Room) Due to Infection or Other Illnesses
Infusion Preparation Time For TAK-881 by Nurse/Pharmacist in Participant Aged >=16 Years
Full Information
NCT ID
NCT06076642
First Posted
October 4, 2023
Last Updated
October 4, 2023
Sponsor
Takeda
Collaborators
Takeda Development Center Americas, Inc.
1. Study Identification
Unique Protocol Identification Number
NCT06076642
Brief Title
A Study About the Long-Term Safety of TAK-881 in People With Primary Immunodeficiency Diseases
Official Title
Long-term Safety and Tolerability of TAK-881 in Subjects With Primary Immunodeficiency Diseases (PIDD)
Study Type
Interventional
2. Study Status
Record Verification Date
October 2023
Overall Recruitment Status
Not yet recruiting
Study Start Date
December 6, 2024 (Anticipated)
Primary Completion Date
May 7, 2027 (Anticipated)
Study Completion Date
May 7, 2027 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Takeda
Collaborators
Takeda Development Center Americas, Inc.
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes
5. Study Description
Brief Summary
The main aim of this study is to learn if TAK-881 is safe and well tolerated during long-term use in adults and children with PIDD who are eligible and completed study TAK-881-3001 (NCT05755035).
The participants will receive the first dose of TAK-881 immunoglobulin (IgG) infusion at the same dosage as the last dose administered in study TAK-881-3001 (NCT05755035). After the first TAK-881 infusion in study TAK-881-3002, the dosing interval and/or the dose of TAK-881 can be adjusted by the study doctor to every 2, 3 or 4 weeks at scheduled site visits,
Participants will visit the clinic every 12 weeks until the End of Study (EOS) visit.
Detailed Description
The study consists of two Epochs: Epoch 1 and Epoch 2. In Epoch 1 eligible participants will have the opportunity to receive TAK-881 for a minimum of 6 months. After 6 months in study TAK-881-3002, the results of the anti-rHuPH20 binding antibody assay from either study TAK-881-3001 (NCT05755035) or study TAK-881-3002 (Epoch 1) will be used to determine the next steps in the study:
Epoch 1: Participants with an anti-rHuPH20 antibody titer less than [<] 1:160 will complete the EOS visit at the next scheduled IgG infusion following study visit Week 25.
Epoch 2: Participants with an anti-rHuPH20 antibody titer greater than or equal to [>=] 1:160 at any time point during Study TAK-881-3001 (NCT05755035) and/or study TAK-881-3002 (Epoch 1), will continue with an additional two years of TAK-881 treatment and undergo safety follow-up and will complete the EOS visit at the next scheduled IgG infusion following study Visit Week 121.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Primary Immunodeficiency Diseases (PID)
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
39 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
TAK-881
Arm Type
Experimental
Arm Description
Epoch 1: Participants with anti-rHuPH20 antibody titer <1:160 at all-time points during study TAK-881-3001 and study TAK-881-3002 will receive TAK-881 subcutaneous (SC) infusion at the same dose and same treatment interval as at the last infusion in TAK-881-3001 (NCT05755035) at Week 1 and at Weeks 13 and Week 25 of study TAK-881-3002.
Epoch 2: Participants with anti-rHuPH20 antibody titer >=1:160 at any time point during Study TAK-881-3001 (NCT05755035) and/or Study TAK-881-3002 Epoch 1, will continue receiving TAK-881 every 12 weeks for up to Week 121.
After the first TAK-881 infusion in study TAK-881-3002, the interval and/or the dose may be adjusted only at scheduled site visits at the investigator's discretion.
Intervention Type
Biological
Intervention Name(s)
TAK-881
Other Intervention Name(s)
Immune Globulin Subcutaneous (Human), 20% Solution with Recombinant Human Hyaluronidase (rHuPH20).
Intervention Description
Participants will receive SC infusion of TAK-881.
Primary Outcome Measure Information:
Title
Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
Time Frame
Up to Week 121
Title
Number of Participants With Infusion Withdrawals, Interruptions, and Infusion Rate Reductions due to TAK-881-related TEAEs
Time Frame
Up to Week 121
Secondary Outcome Measure Information:
Title
Annualized Rate of all Infections
Time Frame
Up to Week 121
Title
Annualized Rate of Acute Serious Bacterial Infections (ASBIs)
Time Frame
Up to Week 121
Title
Annualized Rate of Episodes of Fever
Time Frame
Up to Week 121
Title
Time to First ASBI
Time Frame
Up to Week 121
Title
Duration of Infections
Time Frame
Up to Week 121
Title
Number of Participants With Positive Binding Antibodies and Neutralizing Antibodies to rHuPH20
Time Frame
Up to Week 121
Title
Trough Level of Total IgG
Time Frame
Up to Week 121
Title
Doses of TAK-881
Time Frame
Up to Week 121
Title
Treatment Interval of TAK-881
Time Frame
Up to Week 121
Title
Number of Infusions Per Month With TAK-881
Time Frame
Up to Week 121
Title
Number of Infusions Sites (Needle Sticks) per Month With TAK-881
Time Frame
Up to Week 121
Title
Number of Infusions Sites (Needle Sticks) per infusion With TAK-881
Time Frame
Up to Week 121
Title
Duration of Infusions (minutes) With TAK-881
Time Frame
Up to Week 121
Title
Monthly Infusion Time (minutes/month) With TAK-881
Time Frame
Up to Week 121
Title
Maximum Infusion Rate per Site (milliliter/hour/site) With TAK-881
Time Frame
Up to Week 121
Title
Infusion Volume per Site (milliliter/site) With TAK-881
Time Frame
Up to Week 121
Title
Treatment Satisfaction Questionnaire for Medication (TSQM-9) Scores in Participants Aged >=16 Years
Description
TSQM-9 is a 9-item, validated, self-administered instrument to assess participants satisfaction with medication. It consists of 3 subscales: effectiveness, convenience and global satisfaction. The item scores of each of the 3 domains are summed and transformed to create a total score of 0 (extremely dissatisfied) to 100 (extremely satisfied). Higher score indicates greater satisfaction in that domain.
Time Frame
From Week 13 up to Week 121
Title
Health-Related Quality of Life (HRQoL): EuroQol 5 Dimension 5 Level Questionnaire (EQ-5D-5L) Score in Participants Aged >=12 Years
Description
The EQ-5D-5L is a validated, self-administered assessment of overall health designed by the EuroQol Group. The instrument consists of two components: the EQ-5D descriptive system and the standard vertical EQ visual analogue scale (EQ-VAS). The descriptive system consists of 5 dimensions (mobility, self-care, usual activities, pain/discomfort, and anxiety/depression). Each dimension has five response categories: no problems, slight problems, some or moderate problems, severe problems, or extreme problems. Participants will be asked to describe their health state that day by choosing one of 5 responses that reflect the levels of severity for each of the 5 dimensions. The EQ-VAS measures one's self-perceived health today on a vertical scale from 0 (worst imaginable health) to 100 (best imaginable health) on which participants have to indicate their current health. Higher score indicates best health status.
Time Frame
From Baseline up to Week 121
Title
Treatment Preference Measured by a Disease-specific Questionnaire in Participants Aged >=12 Years
Description
The disease-specific treatment preference questionnaire is a self-administered, non-validated scale to assess participant preference for various attributes of IgG therapy and accompanying devices, such as ease of use of administration of TAK-881 complexity of administration, and convenience. The questionnaire consists of two components. The first component asks participants to indicate the extent to which they like or dislike aspects of TAK-881, including treatment frequency, time, ease, convenience, and complexity. Each item has five response categories: like very much, like, no preference, dislike, and dislike very much. The second component is a single question asking whether they would choose to continue receiving treatment after the trial ends.
Time Frame
At Weeks 25 and 73
Title
Number of Days Lost From School, Work, Daycare, or to Perform Normal Daily Activities Due To Infection and/or their treatment or other Illnesses (non-Infection)
Time Frame
Up to Week 121
Title
Number of Days on Antibiotics
Time Frame
Up to Week 121
Title
Number of Hospitalizations With Indications (Infection or other Illnesses)
Time Frame
Up to Week 121
Title
Number of Days of Hospitalization
Time Frame
Up to Week 121
Title
Number of Acute Physician Visits (Office and Emergency Room) Due to Infection or Other Illnesses
Time Frame
Up to Week 121
Title
Infusion Preparation Time For TAK-881 by Nurse/Pharmacist in Participant Aged >=16 Years
Time Frame
Up to Week 121
10. Eligibility
Sex
All
Minimum Age & Unit of Time
2 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria
Participants who meet ALL of the following criteria are eligible for this study:
Participant must have completed Study TAK-881-3001 (NCT05755035).
If female of childbearing potential, the participant presents with a negative pregnancy test at screening and agrees to employ an acceptable form of contraception for the duration of the study.
Participant/legally authorized representative is willing and able to comply with the requirements of the protocol.
Participant/Participant's legally authorized representative has provided informed consent/assent, if applicable, (that is, in writing, documented via a signed and dated informed consent form (ICF) and/or eConsent, if available), and any required privacy authorization prior to the initiation of any study procedures.
Exclusion Criteria
Participant has a serious medical condition such that the Participant's safety or medical care would be impacted by participation in this long-term follow-up study.
New medical condition that developed during participation in Study TAK-881-3001 (NCT05755035). that, in the judgment of the investigator, could increase risk to the Participant or interfere with the evaluation of TAK-881 and/or conduct of the study.
Participant has participated in another clinical study involving an investigational product (IP) or investigational device within 30 days prior to screening (with the exception of Study TAK-881-3001 [NCT05755035]).
Participant is scheduled to participate in another clinical study involving an IP or investigational device during the course of this study.
Participant is a family member or employee of the investigator or the investigator's site staff.
If female, Participant is pregnant or lactating at the time of screening or intends to become pregnant or begin lactating during the study.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Takeda Contact
Phone
+1-877-825-3327
Email
medinfoUS@takeda.com
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Study Director
Organizational Affiliation
Takeda
Official's Role
Study Director
12. IPD Sharing Statement
Plan to Share IPD
Yes
IPD Sharing Plan Description
Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.
IPD Sharing Access Criteria
IPD from eligible studies will be shared with qualified researchers according to the criteria and process described on https://vivli.org/ourmember/takeda/ For approved requests, the researchers will be provided access to anonymized data (to respect patient privacy in line with applicable laws and regulations) and with information necessary to address the research objectives under the terms of a data sharing agreement.
IPD Sharing URL
https://vivli.org/ourmember/takeda/
Learn more about this trial
A Study About the Long-Term Safety of TAK-881 in People With Primary Immunodeficiency Diseases
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