A Study of TAK-664 in People With Primary Immunodeficiency Diseases Who Have Not Yet Been Treated With Immunoglobulins

ConditionPrimary Immunodeficiency Diseases (PID)

Trial statusNot yet recruiting

Trial phasePhase 4

Trial typeInterventional

Biological sexAll

Age6+

SponsorTakeda

About this trial

Primary immunodeficiency disease (or PIDD) is a group of conditions in which the immune system does not work properly. Some people with PIDD do not make enough antibodies. Antibodies are proteins that help to either protect the body from infections or fight infections. The main type of antibodies that helps protect the body from infection is IgG. People who do not make enough IgG often need medical treatment that gives the body those antibodies. This treatment is called IgG replacement therapy. It can be given through a vein (intravenous or IV) or under the skin (subcutaneous SC). TAK-664 is approved worldwide for SC IG replacement therapy.

The study wants to learn more about TAK-664 given to people with PIDD who have not yet been treated with IG (called 'treatment-naïve').

The main aim of the study is to check if giving TAK-664 daily for 5 days, and then once more 3 days later (Day 8), can raise IgG to the target level and keep IgG there.

Another aim of the study is to learn if TAK-664 given once a week can keep the IgG at the target levels during the study. The study also wants to find out how much the IgG levels raise and learn how many infections occur and how they are treated.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

The participant or the participant's legally authorized representative is willing and able to understand and fully comply with trial procedures and requirements, in the opinion of the investigator.

The participant or the participant's legally authorized representative has provided informed consent or assent, if applicable (that is, in writing, documented via a signed and dated informed consent form [ICF]), and any required privacy authorization before the initiation of any trial procedures.

The participant is at least 6 years of age at the time of signing the ICF or assent, if applicable.

The participant has a documented diagnosis of a form of primary humoral immunodeficiency involving a defect in antibody formation and requiring IG replacement, as defined according to the International Union of Immunological Societies (IUIS) Committee (Human Inborn Errors of Immunity: 2024 update on the phenotypic classification from the IUIS Expert Committee).

Disqualifiers

The participant has significant proteinuria (greater than or equal to [>=] 3 and/or known urinary protein loss greater than [>]1 gram per 24 [g/24] hours or nephrotic syndrome), has acute renal failure, is on dialysis, and/or has severe renal impairment on screening laboratory testing (blood urea nitrogen [BUN] or creatinine >2.5 × upper limit of the normal range [ULN]).

The participant has immunoglobulin A (IgA) deficiency (IgA less than [<] 0.07 grams per liter [g/L]) associated with known anti-IgA antibodies and a history of hypersensitivity.

The participant has a condition(s) that could alter protein catabolism and/or IgG use (for example, protein-losing enteropathies or nephrotic syndrome).

The participant has a known history of a positive result or is positive at screening for one or more of the following: hepatitis B virus surface antigen (HBsAg), polymerase chain reaction (PCR) for hepatitis C virus (HCV), or PCR for human immunodeficiency virus (HIV) Type 1 and Type 2. Note: Cured participants with a history of hepatitis C infection who have a negative PCR test at screening are eligible.

Trial design

Design model

Single group

Treatments tested in this trial

TAK-664
Biological/Vaccine
Participants will receive TAK-664 infusion.

Treatment groups

9 Participants

are divided into 1 treatment group

Group A: TAK-664, 150 mg/kgExperimental treatment 1 intervention