Enrollment Opens for Phase 3 Study of Intravenous Immunoglobulin in Primary Immunodeficiency Patients

According to a publication from BioSpace, New Jersey-based Kedrion Biopharma recently announced the enrollment of the first participant in their CARES10 study. CARES10 is a phase 3 study of a 10% concentration immunoglobulin administered intravenously for the treatment of patients with immunodeficiency diseases.

About Immunodeficiency Diseases

Primary immune deficiency diseases (PIDDs) are highly diverse — there are over 200 individually recognized conditions, each with a unique cause and set of symptoms. They are, as the name implies however, universally similar in that they are characterized by an absent or severely malfunctioning immune system. Without adequate care, the result is frequent and severe infection.

Perhaps the most famous immunodeficiency disorder is HIV. However, HIV and other immunodeficiency disorders are different from PIDDs in that PIDDs are, by definition, present from birth — the result of congenital genetic mutations that are inherited or, sometimes, completely idiopathic.

Because of the incredible diversity of the conditions, prognoses vary significantly. Some PIDDs are more frequently fatal than others, but cases vary on an individual basis.

An Experimental Immunoglobulin Infusion

In all likelihood, there are immunoglobulins in your bloodstream right now — and a lot of them. They’re a group of a number of blood proteins, comprising about 20% of the weight of all proteins found in the blood.

More synecdochically, they’re called “antibodies.” They’re produced by your blood plasma, and play an important part of the immune response, binding to pathogens and aiding in their destruction. Immunodeficient individuals often have few functioning immunoglobulins.

Externally prepared solutions of immunoglobulins for blood transfusion were first used to treat PIDDs as early as 1952. Patients who require immunoglobulin infusions likely need it regularly and frequently — sometimes taking as long as a few hours every couple of days.

In a new study, Kedrion Biopharma intends to study the effects of a “10% intravenous immunoglobulin” in up to 50 patients with PIDDs. As many as 20 have already been enrolled, with the remaining 30 spaces to be filled between now and January of next year. Participants will then receive the open-label treatment for as long as a year and a half, while the effects are monitored.

The study’s planned end date is mid-2021. Pending success in the phase 3 trial, Kendrion plans to apply for a biologics license application from the FDA — which would allow the administration of Kendrion’s IV solution country-wide.

PIDDs are rare but often serious conditions. What do you think of this exciting development? Share your thoughts with Patient Worthy!

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