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

Primary Immunodeficiency Diseases (PID) Clinical Trial

Official title:

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

Clinical Trial Details — Status: Not yet recruiting

Administrative data

• NCT number: NCT06076642
• Other study ID #: TAK-881-3002
• Secondary ID: 2023-505946-24
• Status: Not yet recruiting
• Phase: Phase 3
• Start date: December 6, 2024
• Est. completion date: May 7, 2027

Study information

• Verified date: October 2023
• Source: Takeda
• Contact: Takeda Contact
• Phone: +1-877-825-3327
• Email: medinfoUS[@]takeda.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Not yet recruiting
• Enrollment: 39
• Est. completion date: May 7, 2027
• Est. primary completion date: May 7, 2027
• Accepts healthy volunteers: No
• Gender: All
• Age group: 2 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Immunologic Deficiency Syndromes
• Primary Immunodeficiency Diseases
• Primary Immunodeficiency Diseases (PID)

Intervention

Biological:
• TAK-881
Participants will receive SC infusion of TAK-881.

Locations

Country	Name	City	State
n/a

Sponsors (2)

Lead Sponsor	Collaborator
Takeda	Takeda Development Center Americas, Inc.

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Number of Participants With Treatment-Emergent Adverse Events (TEAEs)		Up to Week 121
Primary	Number of Participants With Infusion Withdrawals, Interruptions, and Infusion Rate Reductions due to TAK-881-related TEAEs		Up to Week 121
Secondary	Annualized Rate of all Infections		Up to Week 121
Secondary	Annualized Rate of Acute Serious Bacterial Infections (ASBIs)		Up to Week 121
Secondary	Annualized Rate of Episodes of Fever		Up to Week 121
Secondary	Time to First ASBI		Up to Week 121
Secondary	Duration of Infections		Up to Week 121
Secondary	Number of Participants With Positive Binding Antibodies and Neutralizing Antibodies to rHuPH20		Up to Week 121
Secondary	Trough Level of Total IgG		Up to Week 121
Secondary	Doses of TAK-881		Up to Week 121
Secondary	Treatment Interval of TAK-881		Up to Week 121
Secondary	Number of Infusions Per Month With TAK-881		Up to Week 121
Secondary	Number of Infusions Sites (Needle Sticks) per Month With TAK-881		Up to Week 121
Secondary	Number of Infusions Sites (Needle Sticks) per infusion With TAK-881		Up to Week 121
Secondary	Duration of Infusions (minutes) With TAK-881		Up to Week 121
Secondary	Monthly Infusion Time (minutes/month) With TAK-881		Up to Week 121
Secondary	Maximum Infusion Rate per Site (milliliter/hour/site) With TAK-881		Up to Week 121
Secondary	Infusion Volume per Site (milliliter/site) With TAK-881		Up to Week 121
Secondary	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
Secondary	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
Secondary	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
Secondary	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
Secondary	Number of Days on Antibiotics		Up to Week 121
Secondary	Number of Hospitalizations With Indications (Infection or other Illnesses)		Up to Week 121
Secondary	Number of Days of Hospitalization		Up to Week 121
Secondary	Number of Acute Physician Visits (Office and Emergency Room) Due to Infection or Other Illnesses		Up to Week 121
Secondary	Infusion Preparation Time For TAK-881 by Nurse/Pharmacist in Participant Aged >=16 Years		Up to Week 121