Clinical trial NCT06076642: 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
Overall status: RECRUITING
Phase: PHASE3
Conditions: Primary Immunodeficiency Diseases (PID)
Lead sponsor: Takeda
Sponsor class: INDUSTRY
Collaborators: name: Takeda Development Center Americas, Inc.
name: Baxalta Innovations GmbH, now part of Takeda
First posted: 2023-10-11
Enrollment: 39
Sex: ALL
Minimum age (years): 2
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.

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).
* 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.

Primary outcomes:
measure: Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
timeFrame: Up to Week 121
measure: Number of Participants With Infusion Withdrawals, Interruptions, and Infusion Rate Reductions due to TAK-881-related TEAEs
timeFrame: Up to Week 121
Secondary outcomes:
measure: Annualized Rate of all Infections
timeFrame: Up to Week 121
measure: Annualized Rate of Acute Serious Bacterial Infections (ASBIs)
timeFrame: Up to Week 121
measure: Annualized Rate of Episodes of Fever
timeFrame: Up to Week 121
measure: Time to First ASBI
timeFrame: Up to Week 121
measure: Duration of Infections
timeFrame: Up to Week 121
measure: Number of Participants With Positive Binding Antibodies and Neutralizing Antibodies to rHuPH20
timeFrame: Up to Week 121
measure: Trough Level of Total IgG
timeFrame: Up to Week 121
measure: Doses of TAK-881
timeFrame: Up to Week 121
measure: Treatment Interval of TAK-881
timeFrame: Up to Week 121
measure: Number of Infusions Per Month With Investigational Medical Device Using TAK-881
timeFrame: Up to Week 121
measure: Number of Infusions Sites (Needle Sticks) per Month With Investigational Medical Device Using TAK-881
timeFrame: Up to Week 121
measure: Number of Infusions Sites (Needle Sticks) per infusion With Investigational Medical Device Using TAK-881
timeFrame: Up to Week 121
measure: Duration of Infusions (minutes) With Investigational Medical Device Using TAK-881
timeFrame: Up to Week 121
measure: Monthly Infusion Time (minutes/month) With Investigational Medical Device Using TAK-881
timeFrame: Up to Week 121
measure: Maximum Tolerated Infusion Rate per Site (milliliter/hour/site) With Investigational Medical Device Using TAK-881
timeFrame: Up to Week 121
measure: Infusion Volume per Site (milliliter/site) With Investigational Medical Device Using TAK-881
timeFrame: Up to Week 121
measure: Number of Participants who have Infusions of TAK-881 at Physical Location
timeFrame: Up to Week 121
measure: Number of Participants, Caregiver, or Healthcare Professional (HCP) who have Administration of TAK-881
timeFrame: Up to Week 121
measure: Treatment Satisfaction Questionnaire for Medication (TSQM-9) Scores in Participants Aged >=16 Years
timeFrame: From Week 13 up to Week 121
measure: Health-Related Quality of Life (HRQoL): EuroQol 5 Dimension 5 Level Questionnaire (EQ-5D-5L) Score in Participants Aged >=12 Years
timeFrame: From Baseline up to Week 121
measure: Treatment Preference Measured by a Disease-specific Questionnaire for Participants and the Investigational Medical Device Using TAK-881 in Participants Aged >=12 Years
timeFrame: At Weeks 25 and 73
measure: 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
timeFrame: Up to Week 121
measure: Number of Days on Antibiotics
timeFrame: Up to Week 121
measure: Number of Hospitalizations With Indications (Infection or other Illnesses)
timeFrame: Up to Week 121
measure: Number of Days of Hospitalization
timeFrame: Up to Week 121
measure: Number of Acute Physician Visits Due to Infection or Other Illnesses
timeFrame: Up to Week 121
measure: Infusion Preparation Time For TAK-881 by HCP
timeFrame: Up to Week 121
measure: Infusion Preparation Time For TAK-881 by Participants/Caregiver
timeFrame: Up to Week 121
measure: Number of Participants With Adverse Events (AEs) Related to the Investigational Medical Device
timeFrame: Up to Week 121
Other outcomes:
Locations:
city: Macon
country: United States
facility: Central Georgia Infectious Disease Consultants
state: Georgia
city: Normal
country: United States
facility: Sneeze, Wheeze, & Itch Associates, LLC
state: Illinois
city: Bronx
country: United States
facility: Montefiore Medical Center
state: New York
city: Durham
country: United States
facility: Duke Asthma, Allergy and Airway Center
state: North Carolina
city: Dallas
country: United States
facility: Allergy Partners of North Texas
state: Texas
Study officials:
affiliation: Takeda
name: Study Director
role: STUDY_DIRECTOR
References: