A Study of TAK-771 in Japanese People With Primary Immunodeficiency Diseases (PID)

NCT05150340 | Completed Phase 3 Results

• 2 other identifiers: TAK-771-3004jRCT2031210457
• Study Type: interventional
• Target: 16
• Locations: 1 country
• Sites: 12

Brief Summary

The main aim of the study is to check how much TAK-771 stays in their blood over time, side effect from the study treatment or TAK-771, how much TAK-771 participants can receive without getting side effects from it, and if TAK-771 improves symptoms of primary immunodeficiency diseases (PID). This will help the study sponsor (Takeda) to work out the best dose to give people in the future. The participants will be treated with TAK-771 for totally 27 or 30 weeks. Treatment period is consist of two periods called Epoch 1 and Epoch 2. In Epoch 1, different groups of participants will receive lower to higher doses of TAK-771 for 3 to 6 weeks. The study doctors will check for side effects from each dose of TAK-771. In Epoch 2, participants will receive TAK-771 once a 3 or 4 weeks until the end of 24 weeks. There will be many clinic visits. The number of visits will depend on the infusion cycles of study drug (every 3, or 4 weeks).

Conditions

Primary Immunodeficiency Diseases (PID)

Outcome Measures

Primary Outcomes (1)

• Epoch 2: Serum Trough Levels of Total IgG Antibodies After Administration of TAK-771

  The data was reported at Week 7, 11, 15, 19, 23, 27, and 31 for 4-Week interval and at Week 4, 7, 10, 13, 16, 19, 22, 25 and 28 for 3-Week interval.

  Up to Week 31 for Participants with 4-Week Dosing Interval or Up to Week 28 for Participants with 3-Week Dosing Interval

Secondary Outcomes (44)

• Epoch 2: Maximum Concentration (Cmax) of Total Serum Levels of IgG and IgG Subclasses

  Pre-infusion at Week 27 for participants with 4-Week or Week 25 with 3-Week dosing interval and post infusion at multiple time points up to Week 31 for participants with 4-Week dosing interval and up to Week 28 with 3-Week dosing interval.

• Epoch 2: Time to Maximum Concentration (Tmax) of Total Serum Levels of IgG and IgG Subclasses (IgG1, IgG2, IgG3, and IgG4)

  Pre-infusion at Week 27 for participants with 4-Week or Week 25 with 3-Week dosing interval and post infusion at multiple time points up to Week 31 for participants with 4-Week dosing interval and up to Week 28 with 3-Week dosing interval.

• Epoch 2: Area Under the Curve (AUC) of Total Serum Levels of IgG and IgG Subclasses (IgG1, IgG2, IgG3, and IgG4)

  Pre-infusion at Week 27 for participants with 4-Week or Week 25 with 3-Week dosing interval and post infusion at multiple time points up to Week 31 for participants with 4-Week dosing interval and up to Week 28 with 3-Week dosing interval.

• Epoch 2: Half-life of Total Serum Levels of IgG and IgG Subclasses (IgG1, IgG2, IgG3, and IgG4)

  At multiple time points post-infusion from Week 7 for participants with 4-Week or Week 4 with 3-Week dosing interval up to Week 31 for participants with 4-Week dosing interval and up to Week 28 with 3-Week dosing interval

• Epoch 2: Apparent Total Clearance (CL/F) of Total Serum Levels of IgG and IgG Subclasses (IgG1, IgG2, IgG3, and IgG4)

  Pre-infusion at Week 27 for participants with 4-Week or Week 25 with 3-Week dosing interval and post infusion at multiple time points up to Week 31 for participants with 4-Week dosing interval and up to Week 28 with 3-Week dosing interval.

Study Arms (2)

Epoch 1: TAK-771 Ramp up Period

EXPERIMENTAL

TAK-771 included IGI 10% and Recombinant Human Hyaluronidase (rHuPH20). Participants received subcutaneous infusion of rHuPH20 solution at a dose of 80 U/g IgG first, followed by SC infusion of 10% IGI within 10 minutes of completion of the infusion of rHuPH20 solution. The dose of 10% IGI was increased from 1/3 of full dose to full dose in 3 weeks for participants who received TAK-771 once every 3 weeks, or from 1/4 of full dose to full dose in 6 weeks for participants who received TAK-771 once every 4 weeks.

Drug: TAK-771

Epoch 2: TAK-771 Full Dose Treatment Period

EXPERIMENTAL

TAK-771 included Immune Globulin Infusion (IGI) 10% and Recombinant Human Hyaluronidase (rHuPH20). Participants received subcutaneous infusion of rHuPH20 solution at a dose of 80 U/g IgG first, followed by SC infusion of 10% IGI within 10 minutes of completion of the infusion of rHuPH20 solution, every 3, or 4 weeks for up to Week 27 for participants with 4-Week dosing interval or Week 25 for participants with 3-Week dosing interval.

Drug: TAK-771

Interventions

TAK-771 DRUG

Intervention description; Immune Globulin Infusion (IGI) 10% and Recombinant Human Hyaluronidase (rHuPH20)

Also known as: Immune Globulin Infusion 10% (Human) with Recombinant Human Hyaluronidase

Epoch 1: TAK-771 Ramp up Period; Epoch 2: TAK-771 Full Dose Treatment Period

Eligibility Criteria

• Age: 2 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Be a Japanese person.
• Participant must have a documented diagnosis of a form of primary humoral immunodeficiency involving antibody formation and requiring gammaglobulin replacement, as defined according to the International Union of Immunological Societies(IUIS) Committee 2017. The diagnosis must be confirmed by the Medical Director prior to TAK-771 treatment.
• Participant has been receiving a stable clinical dose of intravenous immunoglobulin (IVIG) or conventional subcutaneous immunoglobulin (cSCIG), which is equivalent to approximately 200 to 600 mg/kg body weight per 3 to 4 week period for IVIG and approximately 50 to 200 mg/kg body weight per week for cSCIG based on the description in the package insert, consistently over a period of at least 3 months prior to screening, or Participant has been receiving of TAK-664 with fixed dose and dosing frequency at least 3 months prior to enrollment. That is, participant is about to complete Study TAK-664-3001 or participating in Study TAK-664-3002.
• Participant who has been receiving IVIG or cSCIG had all serum trough levels of total immunoglobulin G (IgG) \>=5 g/L within 1 month prior to the screening/enrollment.
• Serum trough levels at screening/enrollment meet one of the following:
• IVIG-treated or cSCIG-treated participants Participant who had serum trough levels of IgG \>=5 g/L at the last 2 points in screening procedure before the first administration of TAK-771.
• TAK-664-treated participants Participant who had serum trough levels of IgG \>=5 g/L at the last 2 points in TAK-664 studies before the first administration of TAK-771.
• Participant is willing and able to comply with use of digital tools and applications.

You may not qualify if:

• Participant has a known history of or is positive at screening/enrollment for one or more of the following: hepatitis B surface antigen (HBsAg), polymerase chain reaction (PCR) for hepatitis C virus (HCV), PCR for human immunodeficiency virus (HIV) Type 1/2 For participants who are switching from TAK-664 studies, the eligibility will be reconfirmed after result of the specialty test conducted at Week 1 become available.
• Abnormal laboratory values at screening/enrollment meeting any one of the following criteria (abnormal tests may be repeated once to determine if they are persistent):
• Persistent alanine aminotransferase (ALT) and aspartate aminotransferase (AST) \>2.5 times the upper limit of normal (ULN) for the testing laboratory
• Persistent severe neutropenia (defined as an absolute neutrophil count \[ANC\] =\<500/mm\^3)
• Participant has presence of renal function impairment defined by eGFR \<60 mL/min/1.73m\^2.
• Participant has been diagnosed with, or had a malignancy (other than adequately treated basal cell or squamous cell carcinoma of the skin or carcinoma in situ of the cervix) unless the disease-free period prior to screening exceeds 5 years.
• Participant is receiving anti-coagulation therapy or has a history of thrombotic episodes (including deep vein thrombosis, myocardial infarction, cerebrovascular accident, pulmonary embolism) within 12 months prior to screening/enrollment or a history of thrombophilia.
• Participant has abnormal protein loss (protein losing enteropathy, nephrotic syndrome)
• Participant has anemia that would preclude phlebotomy for laboratory studies according to standard practice at the site.
• Participant has an ongoing history of hypersensitivity or persistent reactions (urticaria, breathing difficulty, severe hypotension, or anaphylaxis) following IVIG, subcutaneous immunoglobulin (SCIG), and/or Immune Serum Globulin infusions
• Participant has immunoglobulin A (IgA) deficiency (serum IgA less than 0.07g/L) and history of hypersensitivity, or history of confirmed anti-IgA antibodies, or both.
• Participant is on preventative (prophylactic) systemic antibacterial antibiotics at doses sufficient to treat or prevent bacterial infections, and cannot stop these antibiotics at the time of screening/enrollment.
• Participant has active infection and is receiving antibiotic therapy for the treatment of infection at the time of screening/enrollment or had a serious bacterial infection within the 3 months prior to screening/enrollment
• Participant has a bleeding disorder, or a platelet count less than 20,000/microL, or in the opinion of the investigator, would be at significant risk of increased bleeding or bruising as a result of subcutaneous (SC) therapy.
• Participant has total protein \>9 g/dL or myeloma, or macroglobulinemia (IgM) or paraproteinemia.

Sponsors & Collaborators

• Takeda: lead

Study Sites (12)

Nagoya University Hospital

Nagoya, Aichi-ken, Japan

Location

Hospital of University of Occupational and Environmental Health

Kitakyushu, Fukuoka, Japan

Location

Kurume University Hospital

Kurume, Fukuoka, Japan

Location

Kanagawa Children's Medical Center

Yokohama, Kanagawa, Japan

Location

Shinshu University Hospital

Matsumoto, Nagano, Japan

Location

Tokyo Medical Dental University Hospital

Bunkyo-ku, Tokyo, Japan

Location

National Center for Child Health and development

Setagaya-ku, Tokyo, Japan

Location

Kyushu University Hospital

Fukuoka, Japan

Location

Gifu University Hospital

Gifu, Japan

Location

Hiroshima University Hospital

Hiroshima, Japan

Location

Saitama Prefectual Children's Medical Center

Saitama, Japan

Location

Shizuoka Childrens Hospital

Shizuoka, Japan

Location

Related Links

• To obtain more information on the study, click here/on this link.

MeSH Terms

Conditions

Primary Immunodeficiency Diseases

Condition Hierarchy (Ancestors)

Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Immunologic Deficiency Syndromes; Immune System Diseases

Results Point of Contact

• Title: Study Director
• Organization: Takeda

TrialDisclosures@takeda.com

+1-877-825-3327

Study Officials

• Study Director

  Takeda

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 24, 2021
• First Posted: December 9, 2021
• Study Start: January 24, 2022
• Primary Completion: August 28, 2023
• Study Completion: August 28, 2023
• Last Updated: November 25, 2024
• Results First Posted: November 25, 2024

Record last verified: 2024-09

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF, CSR
• 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.

Locations

JP (12)