Clinical Trials

An Important Note:
Clinical trials listed for ITP are a starting point for discussion with a healthcare professional. Be sure you understand all the implications of the proposed treatment by reading the informed consent very carefully and speaking with your doctor before enrolling in a clinical trial.


Doctor with patientWhat are Clinical Trials?

Clinical Trials are controlled patient studies that must be performed before a new drug or treatment is approved by the Food and Drug Administration (FDA) in the United States and similar organizations in other countries. Clinical trials are also completed after a drug or treatment is approved to document the effectiveness of the treatment, or to gain approval to use the drug or therapy for a different disease or age group.

There are three main phases to clinical trials, completed in sequence. A Phase I study tests the safety and side effects of the drug. A Phase II study is initiated to determine the efficacy of the drug, to see if it works as anticipated. And, a Phase III study tests the efficacy of drug or treatment and documents the side effects in a large population of volunteers.


Finding a Clinical Trial

The best way to find the latest information on clinical trials is by visiting ClinicalTrials.gov, a database of privately and publicly funded clinical studies conducted around the world. Pharmaceutical companies and researchers update this site frequently to ensure it contains the most comprehensive and up-to-date clinical trial listings. The database features an easy-to-use search facility and detailed guidance for patients and industry.


Current ITP Clinical Trials


A Study of PRN1008 in Adult Patients With Immune Thrombocytopenic Purpura (ITP)

Principia Biopharma is conducting a study of their oral drug PRN1008 in patients with ITP who are refractory or relapsed with no available and approved therapeutic options, with a platelet count <30,000/μL on two counts no sooner than 7 days apart in the 15 days before treatment begins. Preclinical findings show that PRN1008 decreases the activity of B cells and other inflammatory-causing immune cells, and in a mouse model of ITP show that PRN1008 can reduce platelet loss. Results in a human Phase 2 trial in a different autoimmune disease called pemphigus show that PRN1008 rapidly reduces skin inflammation.

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Actively Recruiting for a Clinical Study for the Treatment of Autoimmune Hemolytic Anemia (AIHA)

Idiopathic thrombocytopenia purpura (ITP) is the destruction of platelets in the body. Autoimmune hemolytic anemia (AIHA) is a rare disease characterized by the body’s premature destruction of red blood cells (hemolysis). Both of these conditions are autoimmune disorders, which means that the body’s immune system malfunctions and attacks healthy blood cells. These two disorders can occur at the same time (destruction of both red blood cells and platelets) or one disorder develops after the other. Evans syndrome is the combination of AIHA with ITP. The cause of Evans syndrome is currently unknown.

A Phase 2 clinical study of an investigational drug is seeking AIHA patients who meet certain criteria.

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Open-Label, Dose Escalation Study of PRTX-100 in Adults With Persistent/Chronic Immune Thrombocytopenia

Protalex, Inc., a clinical-stage biopharmaceutical company, is now enrolling patients in a Phase I/II open-label, dose-escalating study of PRTX-100 in adults with persistent/chronic ITP. Pre-clinical data indicate that PRTX-100 may have the potential to treat ITP by reducing the immune-mediated destruction of the platelets.

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  Additional Resources

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