What is the mechanism of action of this treatment?

The most common treatment for Primary Immune Deficiency (PID) is immunoglobulin replacement therapy, which includes subcutaneous human immunoglobulin (SCIG). SCIG provides necessary antibodies that PID patients lack, thereby enhancing their immune system's ability to fight infections. This therapy is crucial for PID patients as it helps prevent recurrent infections, reduces the severity of infections, and improves overall quality of life. By maintaining adequate antibody levels, SCIG minimizes the risk of complications associated with infections, which is vital for the health and well-being of individuals with PID.

What side effects are associated with this type of intervention?

Common side effects of subcutaneous human immunoglobulin (SCIG) treatments, such as those being studied in the trial for Newnorm, include local reactions at the injection site (e.g., redness, swelling, itching, and pain). Systemic side effects may include headaches, fatigue, fever, nausea, and muscle pain. These side effects are generally mild to moderate and often resolve on their own. Serious side effects are rare but can include severe allergic reactions and thrombotic events.

What prior FDA approvals exist?

FDA-approved treatments for Primary Immune Deficiency (PID) often involve immunoglobulin replacement therapy, which provides essential antibodies to prevent infections. Similar to the subcutaneous human immunoglobulin (Newnorm) being studied, other approved treatments include intravenous immunoglobulin (IVIG) and subcutaneous immunoglobulin (SCIG) products. These therapies, such as Hizentra, Gammagard, and Gamunex-C, are designed to boost the immune system by supplying the necessary antibodies that patients with PID lack, thereby reducing the frequency and severity of infections.

What other types of research exist?

Promising novel alternatives to Subcutaneous Human Immunoglobulin (Newnorm) for primary immunodeficiency patients include monoclonal antibodies such as omalizumab, which has shown efficacy in increasing the threshold for food allergens and may have broader immunomodulatory effects. Additionally, RNA interference therapies like vutrisiran, which have been effective in other conditions like amyloidosis, could potentially be repurposed for primary immunodeficiency. These alternatives are still under investigation, and their use should be discussed with a healthcare provider.

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Immunoglobulin

Newnorm for Primary Immunodeficiency

Phase 3

Recruiting

Research Sponsored by Octapharma

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Trough level of IgG ≥5 g/L at screening and documentation of an IgG trough level of ≥5 g/L at least once within the previous 12 weeks.

Documented and confirmed diagnosis of PID as defined by European Society of Immunodeficiencies (ESID) and the Pan American Group for Immune Deficiency (PAGID) and requiring immunoglobulin replacement therapy due to hypogammaglobulinaemia or agammaglobulinaemia. The exact type of PID must be recorded.

Must not have

Current medical condition or history of condition known to cause secondary immune deficiency, for example, chronic lymphocytic leukaemia, lymphoma, multiple myeloma, or chronic or recurrent neutropenia (absolute neutrophil count <1000/μL).

Severe liver dysfunction (alanine aminotransferase [ALT] >3 times the upper limit of normal for the expected normal range for the testing laboratory) at screening.

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 52 weeks

Awards & highlights

Pivotal Trial

No Placebo-Only Group

Summary

This trial is testing a new antibody treatment called Newnorm, which is given through an injection under the skin. It aims to help people with primary immunodeficiency diseases, whose immune systems are weak. The treatment provides extra antibodies to help their bodies fight infections.

Who is the study for?

This trial is for people aged 2 to 75 with Primary Immunodeficiency Diseases (PID) who need immunoglobulin therapy. They must have been on stable treatment doses for at least 12 weeks and agree to protocol requirements, including blood sampling. Excluded are those with severe allergies to IgA, obesity (BMI >40), liver dysfunction, uncontrolled diabetes or hypertension, certain heart conditions, recent blood product exposure other than IgG for PID, or active infections.

What is being tested?

The study is testing 'Newnorm,' a subcutaneous human immunoglobulin treatment for patients with PID. It's an open-label Phase 3 trial assessing how the body processes the drug and its effectiveness and safety across multiple centers.

What are the potential side effects?

While specific side effects of Newnorm aren't listed here, typical reactions to immunoglobulin treatments can include injection site discomfort, headache, fatigue, nausea or vomiting. Severe allergic reactions are rare but possible.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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My IgG levels have been above 5 g/L recently and at screening.

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I have a confirmed PID diagnosis and need immunoglobulin therapy.

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I've been on a stable dose of IVIG, SCIG, or fSCIG for 12 weeks.

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I am between 2 and 75 years old.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I have a condition like leukemia or lymphoma that weakens my immune system.

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My liver is not working well (ALT levels are very high).

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I have a specific type of immune deficiency.

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I have had a blood clot or related condition in the past year or twice in my life.

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I have kidney problems or a high risk of kidney failure.

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My diabetes is not under control (HbA1c > 7%).

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My blood pressure is not controlled and is above the recommended level for my age.

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I haven't had any live vaccines in the last month or plan to during the study.

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I haven't needed IV antibiotics for infections or had a serious bacterial infection in the last 3 months.

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I am on blood thinners that make certain injections risky.

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I have a condition causing significant protein loss in my urine.

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I am a man and agree to use birth control during the study.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 52 weeks

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~52 weeks

This trial's timeline: 3 weeks for screening, Varies for treatment, and 52 weeks for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Average total IgG concentration

Rate of Serious Bacterial Infections

Secondary study objectives

Child Health Questionnaire-Parent Form

Days lost from work, school, kindergarten, or day care due to infection

Episodes of fever

+7 more

Awards & Highlights

Pivotal Trial

The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups

Experimental Treatment

Group I: NewnormExperimental Treatment1 Intervention

Newnorm is a 20% human normal immunoglobulin for SC infusion

0 / 16Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

The most common treatment for Primary Immune Deficiency (PID) is immunoglobulin replacement therapy, which includes subcutaneous human immunoglobulin (SCIG). SCIG provides necessary antibodies that PID patients lack, thereby enhancing their immune system's ability to fight infections. This therapy is crucial for PID patients as it helps prevent recurrent infections, reduces the severity of infections, and improves overall quality of life. By maintaining adequate antibody levels, SCIG minimizes the risk of complications associated with infections, which is vital for the health and well-being of individuals with PID.