Primary Immunodeficiency Diseases (PID)
Conditions
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 (can be adjusted by participant/caregiver) 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 on the day of the first non-investigational product (IP) 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 on the day of the first non-IP infusion following study Visit Week 121.
Interventions
BIOLOGICALTAK-881
Participants will receive SC infusion of TAK-881.
The single-use only SC needle set will be used to administer TAK-881 to the target depth below the skin surface. One needle set (single or bifurcated) will be used per infusion.
Sponsors
Takeda Development Center Americas, Inc.
Baxalta Innovations GmbH, now part of Takeda
Takeda
Study design
Allocation
NON_RANDOMIZED
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
2 Years to No maximum
Healthy volunteers
No
Inclusion criteria
Participants who meet ALL of the following criteria are eligible for this study: * Participant must have completed Study TAK-881-3001 (NCT05755035). * Participant/Participant's parent(s)/legal guardian(s) is/are willing and able to comply with the requirements of the protocol. * Participant/Participant's parent(s)/legal guardian(s) has/have provided informed consent/assent (if applicable), including providing consent for use of the investigational medical device, 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. * Women of childbearing potential who meet any one of the following criteria:- 1. Participant has a positive pregnancy test. 2. Participant does not agree to employ a highly effective form of contraception for the duration of the study. * If female, participant is pregnant or lactating at the time of screening or intends to become pregnant or begin lactating during the study.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Number of Participants With Treatment-Emergent Adverse Events (TEAEs) | Up to Week 121 |
| Number of Participants With Infusion Withdrawals, Interruptions, and Infusion Rate Reductions due to TAK-881-related TEAEs | Up to Week 121 |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants who have Infusions of TAK-881 at Physical Location | Up to Week 121 | Physical location includes site/infusion center or home. |
| Treatment Interval of TAK-881 | Up to Week 121 | — |
| Number of Infusions Per Month With Investigational Medical Device Using TAK-881 | Up to Week 121 | — |
| Number of Infusions Sites (Needle Sticks) per Month With Investigational Medical Device Using TAK-881 | Up to Week 121 | — |
| Number of Infusions Sites (Needle Sticks) per infusion With Investigational Medical Device Using TAK-881 | Up to Week 121 | — |
| Duration of Infusions (minutes) With Investigational Medical Device Using TAK-881 | Up to Week 121 | — |
| Monthly Infusion Time (minutes/month) With Investigational Medical Device Using TAK-881 | Up to Week 121 | — |
| Maximum Tolerated Infusion Rate per Site (milliliter/hour/site) With Investigational Medical Device Using TAK-881 | Up to Week 121 | — |
| Annualized Rate of all Infections | Up to Week 121 | — |
| Annualized Rate of Acute Serious Bacterial Infections (ASBIs) | Up to Week 121 | — |
| Annualized Rate of Episodes of Fever | Up to Week 121 | — |
| Time to First ASBI | Up to Week 121 | — |
| Duration of Infections | Up to Week 121 | — |
| Infusion Volume per Site (milliliter/site) With Investigational Medical Device Using TAK-881 | Up to Week 121 | — |
| Trough Level of Total IgG | Up to Week 121 | — |
| Doses of TAK-881 | Up to Week 121 | — |
| Treatment Satisfaction Questionnaire for Medication (TSQM-9) Scores in Participants Aged >=16 Years | From Week 13 up to Week 121 | 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 | From Baseline up to Week 121 | 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 for Participants and the Investigational Medical Device Using TAK-881 in Participants Aged >=12 Years | At Weeks 25 and 73 | 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 not Able to go to School, Work, Daycare, or to Perform Normal Daily Activities Due to Infections and/or their Treatment or Other Illnesses | Up to Week 121 | — |
| Number of Days on Antibiotics | Up to Week 121 | — |
| Number of Hospitalizations With Indications (Infection or other Illnesses) | Up to Week 121 | — |
| Number of Days of Hospitalization | Up to Week 121 | — |
| Number of Acute Physician Visits Due to Infection or Other Illnesses | Up to Week 121 | — |
| Infusion Preparation Time For TAK-881 by HCP | Up to Week 121 | — |
| Infusion Preparation Time For TAK-881 by Participants/Caregiver | Up to Week 121 | — |
| Number of Participants With Adverse Events (AEs) Related to the Investigational Medical Device | Up to Week 121 | — |
| Number of Participants With Positive Binding Antibodies and Neutralizing Antibodies to rHuPH20 | Up to Week 121 | — |
| Number of Participants, Caregiver, or Healthcare Professional (HCP) who have Administration of TAK-881 | Up to Week 121 | — |
Countries
United States
Contacts
Primary ContactTakeda Contact
Outcome results
None listed