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

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Takeda Development Center Americas Inc., Baxalta Innovations GmbH

Participant type

Pediatric, Patients

Age range

0-17 years, 18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Immune System Diseases [C20]

Trial duration

15 May 2025 → ongoing

Decision date (initial)

2024-03-13

Transition trial

No

Low-intervention

No

Rare-disease indication

No

Vulnerable population

Yes

Funding sources

Takeda Development Center Americas, Inc

External identifiers

EU CT number

2023-505946-24-00

ClinicalTrials.gov

NCT06076642

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Efficacy, Others, Pharmacokinetic, Safety

To evaluate the long-term safety and tolerability of TAK-881 in all subjects

Secondary objectives 10

1. To assess infections in all subjects.
2. To evaluate long-term immunogenicity of TAK-881 in all subjects.
3. To assess IgG trough levels in all subjects.
4. To assess the treatment regimen with TAK-881 in all subjects.
5. To evaluate infusion parameters with TAK-881 in all subjects.
6. To assess subject satisfaction with TAK-881 treatment in subjects aged ≥16 years.
7. To assess subject HRQoL while on TAK-881 treatment in subjects aged ≥12 years.
8. To assess treatment preferences for TAK-881 in subjects aged ≥12 years.
9. To assess HRU for subjects using TAK-881.
10. To assess infusion preparation time for TAK-881 by nurse/pharmacist/subject/caregiver recordings.

Conditions and MedDRA coding

Primary Immunodeficiency Diseases

Regulatory references

Scientific advice from competent authorities

European Medicines Agency

Plan to share 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.

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 3

1. Has completed Study TAK-881-3001.
2. Subject or, in the case of minors, legally designated representative(s) is/are willing and able to comply with the requirements of the protocol.
3. The subject or, in the case of minors, legally designated representative(s) has/have provided informed consent/assent (if applicable), including providing consent for use of the investigational medicinal device (in US sites only), prior to the initiation of any study procedures.

Exclusion criteria 7

1. Subject has a serious medical condition such that the subject’s safety or medical care would be impacted by participation in this long-term follow-up study.
2. New medical condition that developed during participation in Study TAK-881-3001 that, in the judgment of the investigator, could increase risk to the subject or interfere with the evaluation of TAK-881 and/or conduct of the study.
3. Subject was enrolled in another clinical study involving an IP or investigational device within 30 days prior to screening (with the exception of Study TAK-881-3001).
4. Subject is scheduled to participate in another clinical study involving an IP or investigational device during the course of this study.
5. Subject is a family member or employee of the investigator or the investigator’s site staff.
6. Subjects with potential to become pregnant who meet any one of the following criteria: a. Subject has a positive pregnancy test. b. Subject does not agree to employ a highly effective form of contraception for the duration of the study.
7. Subject is pregnant or lactating at the time of screening or intends to become pregnant or begin lactating during the study.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 2

1. Occurrence of TEAEs.
2. Occurrence of infusion withdrawals, interruptions, and infusion rate reductions due to TAK-881-related TEAEs.

Secondary endpoints 10

1. Efficacy • Annualized rate of all infections. • Annualized rate of ASBIs. • Annualized rate of episodes of fever. • Time to first ASBI. • Duration of infections.
2. Immunogenicity • Occurrence of positive binding (defined as titer ≥1:160) and neutralizing antibodies to rHuPH20.
3. Pharmacokinetics • Trough level of total IgG.
4. Treatment regimen • Dose of TAK-881. • Treatment interval of TAK-881.
5. Infusion parameters: • Number of infusions per month. • Number of infusion sites (needle sticks) per month. • Number of infusion sites (needle sticks) per infusion. • Duration of infusions (minutes). • Monthly infusion time (minutes/month). • Maximum tolerated infusion rate per site (mL/hour/site). • Infusion volume per site (mL/site). • Physical location of infusion (site/infusion center or home). • Subject, caregiver, or healthcare professional (HCP) administration of IP.
6. Subject satisfaction • Subject satisfaction evaluated by subject-reported scores on the Treatment Satisfaction Questionnaire for Medication (TSQM) - 9 items (TSQM-9).
7. Health-related quality of life • Subject HRQoL evaluated via EQ-5D-5L.
8. Treatment preferences • Treatment preferences measured by a disease-specific questionnaire.
9. Healthcare resource utilization • 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. • Days on antibiotics. • Number of hospitalizations, indication for the hospitalization (infection or other illnesses) and days hospitalized. • Number of acute physician visits due to infection or other illnesses.
10. Infusion preparation • HCP-measured time of infusion preparation per guidelines in the Pharmacy Manual/Site Infusion Manual. • Subject/caregiver measured time of infusion preparation per guidelines in the Subject Infusion Manual.

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Takeda Development Center Americas Inc.

Sponsor organisation

Takeda Development Center Americas Inc.

Address

500 Kendall Street

City

Cambridge

Postcode

02142-1108

Country

United States

Scientific contact point

Organisation

Takeda Development Center Americas Inc.

Contact name

Andras Nagy

Public contact point

Organisation

Takeda Development Center Americas Inc.

Contact name

Takeda

Third parties 11

Baxalta Innovations GmbH

Sponsor organisation

Baxalta Innovations GmbH

Address

Industriestrasse 67, Donaustadt Donaustadt

City

Vienna

Postcode

1221

Country

Austria

Sponsor responsibilities

Article 77 compliance

Takeda Development Center Americas Inc.

Contact point sponsor

Takeda Development Center Americas Inc.

Article 77 implementation

Takeda Development Center Americas Inc.

Locations

8 EU/EEA countries · 19 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 146 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

22 submissions — initial application plus substantial / non-substantial modifications