A Study About the Long-Term Safety of TAK-881 in People With Primary Immunodeficiency Diseases

Long-term Safety and Tolerability of TAK-881 in Subjects With Primary Immunodeficiency Diseases (PIDD)

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.

Study Overview

• Status: Not yet recruiting
• Conditions: Primary Immunodeficiency Diseases (PID)
• Intervention / Treatment: Biological: TAK-881

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.

• Study Type: Interventional
• Enrollment (Estimated): 39
• Phase: Phase 3

Contacts and Locations

• Study Contact: Name: Takeda Contact; Phone Number: +1-877-825-3327; Email: medinfoUS@takeda.com

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Non-Randomized
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm

Intervention / Treatment

Experimental: TAK-881; 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.

Biological: TAK-881; Participants will receive SC infusion of TAK-881.; Other Names: Immune Globulin Subcutaneous (Human), 20% Solution with Recombinant Human Hyaluronidase (rHuPH20).

What is the study measuring?

Primary Outcome Measures

Outcome 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 Outcome Measures

Outcome Measure	Measure Description	Time Frame
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
Number of Participants With Positive Binding Antibodies and Neutralizing Antibodies to rHuPH20		Up to Week 121
Trough Level of Total IgG		Up to Week 121
Doses of TAK-881		Up to Week 121
Treatment Interval of TAK-881		Up to Week 121
Number of Infusions Per Month With TAK-881		Up to Week 121
Number of Infusions Sites (Needle Sticks) per Month With TAK-881		Up to Week 121
Number of Infusions Sites (Needle Sticks) per infusion With TAK-881		Up to Week 121
Duration of Infusions (minutes) With TAK-881		Up to Week 121
Monthly Infusion Time (minutes/month) With TAK-881		Up to Week 121
Maximum Infusion Rate per Site (milliliter/hour/site) With TAK-881		Up to Week 121
Infusion Volume per Site (milliliter/site) With TAK-881		Up to Week 121
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.	From Week 13 up to Week 121
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.	From Baseline up to Week 121
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.	At Weeks 25 and 73
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)		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 (Office and Emergency Room) Due to Infection or Other Illnesses		Up to Week 121
Infusion Preparation Time For TAK-881 by Nurse/Pharmacist in Participant Aged >=16 Years		Up to Week 121

Collaborators and Investigators

• Sponsor: Takeda
• Collaborators: Takeda Development Center Americas, Inc.
• Investigators: Study Director: Study Director, Takeda

Study record dates

Study Major Dates

• Study Start (Estimated): December 6, 2024
• Primary Completion (Estimated): May 7, 2027
• Study Completion (Estimated): May 7, 2027

Study Registration Dates

• First Submitted: October 4, 2023
• First Submitted That Met QC Criteria: October 4, 2023
• First Posted (Actual): October 11, 2023

Study Record Updates

• Last Update Posted (Actual): October 11, 2023
• Last Update Submitted That Met QC Criteria: October 4, 2023
• Last Verified: October 1, 2023

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Immune System Diseases; Genetic Diseases, Inborn; Immunologic Deficiency Syndromes; Primary Immunodeficiency Diseases; Physiological Effects of Drugs; Immunologic Factors; Immunoglobulins; Immunoglobulins, Intravenous; gamma-Globulins; Rho(D) Immune Globulin
• Other Study ID Numbers: TAK-881-3002; 2023-505946-24 (Other Identifier: EU CTIS)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD 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 Supporting Information Type: STUDY_PROTOCOL; SAP; ICF; CSR

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No