Primary Immunodeficiency Clinical Trials 2023

What Are Primary Immunodeficiency Clinical Trials?

A person with primary immunodeficiency (PI) has a malfunctioning immune system, indicating that those with PI are more vulnerable to infections and getting quite ill from them. The severity of the more than 400 different varieties of PI determines how quickly they are discovered. Primary immunodeficiencies are illnesses in which a component of the immune system is absent or malfunctions improperly. The etiology of the immune deficiency must not be secondary for it to qualify as a primary immunodeficiency (PID). Although milder versions may not be discovered until maturity, most primary immunodeficiencies are hereditary diseases often identified in infants under the age of one.

Why Is Primary Immunodeficiency Being Studied Through Clinical Trials?

Primary Immunodeficiency Clinical Trials are necessary to assess the efficacy and safety of novel drugs and medical devices and to give the Food and Drug Administration (FDA) sufficient data to approve or disapprove these goods. To create safe and effective medications with minimal side effects and the greatest therapeutic value, human testing, commonly referred to as a clinical trial or protocol, is required.

To assess if novel medicines are safe enough to try on people, they are typically initially examined in lab animals. It is critical that participants in clinical trials represent a range of ages and genders to ensure that the new product under test will be effective for various populations.

What Are the Types of Treatments Available for Primary Immunodeficiency?

Primary immunodeficiencies are treated differently depending on the kind of problem. Somatic therapy is still in its infancy for predominantly hereditary disorders. In order to be passive and palliative, most therapy focuses on controlling infections and strengthening the immune system.

Prophylactic antibiotics or antivirals are frequently indicated, and reducing exposure to infections may also be advised. From the use of gammaretroviral vectors in the 1990s to more specialized self-inactivating vector platforms around 2006, gene therapy has advanced.

What Are Some Recent Breakthrough Clinical Trials for Primary Immunodeficiency?

2016: Adoptive immunotherapy for primary immunodeficiency disorders with virus-specific T lymphocytes - PID patients who had received VST treatment according to earlier or current procedures were examined together. Clinical data were assessed, including transplantation specifics, viral infections, and usage of immunosuppressive and antiviral medication.  The researchers compared information on VST production, infusions, and negative effects.

2020: Biomarkers and Precision Therapy for Primary Immunodeficiencies - The goal is to use iPSCs from patients with AT or other kinds of AGS to examine a panel of immunomodulators’ cytotoxicity. Sendai virus was used to reprogram the cells of AT and AGS patients as well as the BJ normal human fibroblast line as a control, yielding iPSCs. After exposure for 72 hours, the cytotoxic impacts of 2 medications suggested for the treatment of AT and AGS, respectively, were examined using the 3-(4,5-dimethylthiazol-2-yl)-2,5-diphenyltetrazolium bromide assay.

Who Are Some of the Key Research Institutions Conducting Primary Immunodeficiency Clinical Trial Research?

St. Jude Children’s Research Hospital - The discovery of gene treatments and better methods for bone marrow transplantation (BMT) are the two primary focuses of St. Jude's research. Treatment options for SCID and other primary immunodeficiency illnesses include bone marrow transplants and other hematopoietic stem cell transplants (HCT). Through the restoration of the body's immune system, a successful transplant may be able to cure a patient.

Immune Deficiency Foundation - The Immune Deficiency Foundation fosters a community strengthened by research and education to enhance the treatment and diagnosis of those with primary immunodeficiency. The National Institutes of Health estimate that there are 500,000 people in the United States who have primary immunodeficiency. Numerous people go undetected each year.