Ongoing Clinical Trials for Primary Immunodeficiency Syndrome

Currently, there are 4 ongoing clinical trials investigating treatments for Primary Immunodeficiency Syndrome. These studies are evaluating various immunoglobulin therapies designed to help boost the immune system and prevent infections in patients whose bodies cannot produce sufficient antibodies on their own. The trials are being conducted across multiple European countries and focus on assessing the safety, effectiveness, and optimal dosing of different immunoglobulin products.

Clinical trial locations

• Czechia
  • Study on Long-term Safety of TAK-881 (Human Normal Immunoglobulin, Hyaluronidase) for Patients with Primary Immunodeficiency Diseases
  • Study on the Effects of TAK-881 and Hyaluronidase in Adults with Primary Immunodeficiency Diseases
• Denmark
  • Study on Long-term Safety of TAK-881 (Human Normal Immunoglobulin, Hyaluronidase) for Patients with Primary Immunodeficiency Diseases
  • Study on the Effects of TAK-881 and Hyaluronidase in Adults with Primary Immunodeficiency Diseases
• Germany
  • Study on Long-term Safety of TAK-881 (Human Normal Immunoglobulin, Hyaluronidase) for Patients with Primary Immunodeficiency Diseases
  • Study on the Effects of Subcutaneous Human Immunoglobulin (Newnorm) for Patients with Primary Immunodeficiency Diseases
  • Study on the Effects of TAK-881 and Hyaluronidase in Adults with Primary Immunodeficiency Diseases
• Greece
  • Study on Long-term Safety of TAK-881 (Human Normal Immunoglobulin, Hyaluronidase) for Patients with Primary Immunodeficiency Diseases
  • Study on the Effects of TAK-881 and Hyaluronidase in Adults with Primary Immunodeficiency Diseases
• Hungary
  • Study on the Effects of Subcutaneous Human Immunoglobulin (Newnorm) for Patients with Primary Immunodeficiency Diseases
• Italy
  • Study on the Effects of Subcutaneous Human Immunoglobulin (Newnorm) for Patients with Primary Immunodeficiency Diseases
• Netherlands
  • Study on Long-term Safety of TAK-881 (Human Normal Immunoglobulin, Hyaluronidase) for Patients with Primary Immunodeficiency Diseases
  • Study on the Effect of Human Normal Immunoglobulin on Lung Disease in Patients with Primary Antibody Deficiency
  • Study on the Effects of TAK-881 and Hyaluronidase in Adults with Primary Immunodeficiency Diseases
• Poland
  • Study on Long-term Safety of TAK-881 (Human Normal Immunoglobulin, Hyaluronidase) for Patients with Primary Immunodeficiency Diseases
  • Study on the Effects of Subcutaneous Human Immunoglobulin (Newnorm) for Patients with Primary Immunodeficiency Diseases
  • Study on the Effects of TAK-881 and Hyaluronidase in Adults with Primary Immunodeficiency Diseases
• Slovakia
  • Study on Long-term Safety of TAK-881 (Human Normal Immunoglobulin, Hyaluronidase) for Patients with Primary Immunodeficiency Diseases
  • Study on the Effects of Subcutaneous Human Immunoglobulin (Newnorm) for Patients with Primary Immunodeficiency Diseases
  • Study on the Effects of TAK-881 and Hyaluronidase in Adults with Primary Immunodeficiency Diseases
• Spain
  • Study on Long-term Safety of TAK-881 (Human Normal Immunoglobulin, Hyaluronidase) for Patients with Primary Immunodeficiency Diseases
  • Study on the Effects of TAK-881 and Hyaluronidase in Adults with Primary Immunodeficiency Diseases

Study on Long-term Safety of TAK-881 (Human Normal Immunoglobulin, Hyaluronidase) for Patients with Primary Immunodeficiency Diseases

This study is evaluating the long-term safety and tolerability of TAK-881, a treatment that combines human normal immunoglobulin with hyaluronidase. The medication is delivered under the skin through subcutaneous infusion.

Inclusion criteria: Participants must have completed a previous study called TAK-881-3001. Women who can become pregnant need to have a negative pregnancy test at the start and must use highly effective birth control throughout the study. Participants or their legal representatives must be willing and able to follow all study requirements.

Exclusion criteria: Individuals without a diagnosis of Primary Immunodeficiency Diseases cannot participate. Participants must fall within specified age ranges. Vulnerable populations, such as children or people with certain disabilities requiring special protection, may not be eligible.

Focus of the trial: The study monitors participants for any side effects or reactions to the treatment over an extended period, expected to continue until February 2029. Researchers will track infection rates, antibody development against the treatment, and how the body processes the medication. They will also assess quality of life, including satisfaction with treatment, days missed from work or daily activities, use of antibiotics, hospitalizations, and doctor visits.

Investigational drug: TAK-881 is an immunomodulatory agent designed to enhance or support immune system function in people with these immune disorders.

Study on the Effect of Human Normal Immunoglobulin on Lung Disease in Patients with Primary Antibody Deficiency

This trial focuses specifically on preventing the progression of lung disease in people with Primary Antibody Deficiency, which includes conditions like Common Variable Immunodeficiency, IgA deficiency, and Agammaglobulinemia. The study uses multiple immunoglobulin products including Nanogam, HyQvia, Privigen, KIOVIG, Gammanorm, CUTAQUIG, GAMMAGARD S/D, Hizentra, Octagam, and Cuvitru.

Inclusion criteria: Participants must be between 8 and 60 years old with a confirmed diagnosis of Primary Antibody Deficiency or Common Variable Immunodeficiency Disorder. They must need immunoglobulin replacement therapy or already be receiving it, with current dosing between 0.25 and 0.6 grams per kilogram every 3 to 4 weeks. Participants must be receiving ongoing care at one of the participating centers and provide written informed consent.

Exclusion criteria: Individuals without primary antibody deficiency, those with other types of immune system problems not specified in the study, those outside the specified age range, and those considered part of vulnerable populations are excluded.

Focus of the trial: The study aims to determine whether higher doses of immunoglobulin can better protect against lung disease progression. Participants receive immunoglobulin through either intravenous or subcutaneous routes. CT scans are performed at the beginning and end of the study to measure changes in airway disease and lung damage. Participants maintain a daily diary to record respiratory symptoms and regular assessments track respiratory infections, days missed from school or work, and quality of life.

Investigational drug: Various immunoglobulin therapy products are being tested to evaluate whether higher dosing is more effective and cost-efficient in managing the condition and protecting lung health.

Study on the Effects of Subcutaneous Human Immunoglobulin (Newnorm) for Patients with Primary Immunodeficiency Diseases

This trial evaluates Newnorm, a form of human normal immunoglobulin given through subcutaneous administration once weekly. The study assesses how well this weekly treatment maintains protective levels of immunoglobulin G in the blood.

Inclusion criteria: Participants must be between 2 and 75 years old with a confirmed diagnosis of Primary Immunodeficiency Disease requiring immunoglobulin replacement therapy due to low antibody levels. They must have been receiving regular, stable treatment for at least 12 weeks before the study starts, with doses between 200 and 800 mg/kg/month. A trough level of IgG of at least 5 g/L is required at study start. Written informed consent is required from adults or from parents/legal guardians for children, along with willingness to follow all study procedures including blood sampling.

Exclusion criteria: Patients with known allergies to immunoglobulin products, history of severe blood clots, IgA deficiency with antibodies against IgA, severe allergic reactions to blood products, current use of other investigational drugs, chronic kidney disease, liver disease, heart disease, or stroke are excluded. Pregnant or breastfeeding women cannot participate.

Focus of the trial: The 52-week study monitors the effectiveness of Newnorm in preventing serious bacterial infections. Regular blood samples measure IgG levels to ensure they remain stable. Researchers track infection rates, antibiotic use, hospitalizations, and quality of life assessments. The dosage is adjusted for patients previously treated with intravenous immunoglobulin products.

Investigational drug: Newnorm is designed to provide necessary antibodies through weekly subcutaneous injections to help fight infections in patients with compromised immune systems.

Study on the Effects of TAK-881 and Hyaluronidase in Adults with Primary Immunodeficiency Diseases

This comparative trial evaluates TAK-881 against HyQvia, both containing human normal immunoglobulin and administered subcutaneously. The study assesses how the body processes these treatments in individuals aged 16 years and older.

Inclusion criteria: Patients must have documented Primary Immunodeficiency Disease affecting antibody production and requiring IgG replacement therapy. For one part of the study, participants must be between 2 and less than 16 years old; for another part, 16 years or older. Participants must have been receiving stable immunoglobulin therapy for at least 12 weeks before the study, with dose variations not exceeding 25% from the average. Required IgG levels in the blood must be maintained. Females who can become pregnant must have negative pregnancy tests and use highly effective birth control. Written consent and willingness to follow all study procedures, including blood sampling, are required.

Exclusion criteria: Individuals without Primary Immunodeficiency Diseases cannot participate. Participants must be at least 16 years old for the adult portion of the study. Certain vulnerable populations may be excluded for safety reasons.

Focus of the trial: The study, running for up to 27 weeks and expected to conclude by September 2026, compares how the body absorbs and processes TAK-881 versus HyQvia. Participants receive both treatments at different times. Regular monitoring assesses safety, side effects, infection frequency, and impact on daily life. Blood sampling evaluates pharmacokinetics and tracks annualized infection rates and health resource utilization.

Investigational drugs: TAK-881 is being studied to understand how it compares to HyQvia, an already-used treatment for the condition. Both are administered under the skin and provide antibodies the body needs to fight infections.

Summary

These four clinical trials represent ongoing efforts to improve treatment options for people living with Primary Immunodeficiency Syndrome across Europe. The studies are concentrated primarily in Central and Western European countries, with Germany, Poland, Slovakia, and the Netherlands participating in multiple trials. Spain, Greece, Denmark, and Czechia are also contributing to this research effort.

A notable focus across these studies is the investigation of subcutaneous immunoglobulin administration methods, which allow patients to receive treatment under the skin rather than through intravenous infusion. This approach may offer greater convenience and improved quality of life for patients requiring regular treatment.

TAK-881, combined with hyaluronidase, is being evaluated in two separate studies examining both its long-term safety profile and how it compares to existing treatments. The Newnorm product is being assessed specifically for weekly dosing schedules. Additionally, one unique study focuses specifically on lung health in patients with antibody deficiencies, recognizing that respiratory complications are a significant concern for this population.

These trials collectively aim to determine optimal dosing strategies, improve treatment convenience, enhance safety profiles, and ultimately provide better outcomes for individuals whose immune systems cannot produce sufficient antibodies to protect against infections.