Paroxysmal Nocturnal Hemoglobinuria (PNH) Clinical Trials

Paroxysmal Nocturnal Hemoglobinuria results from acquired somatic mutations in the PIGA gene, which encodes a protein essential for biosynthesis of glycosylphosphatidylinositol (GPI) anchors. These anchors normally attach proteins that protect blood cells from complement-mediated destruction. Loss of GPI anchors leaves red blood cells, white blood cells, and platelets vulnerable to complement-mediated lysis.

The most common symptoms of Paroxysmal Nocturnal Hemoglobinuria include Hemoglobinuria (dark red or brown urine, especially after sleep), Fatigue and weakness from hemolytic anemia, Shortness of breath and chest discomfort, Back pain, abdominal pain, or leg pain (from thrombosis), Headaches and neurologic symptoms, among others. Approximately 1-2 cases per million people; estimated 2,000-5,000 patients in the United States. Symptoms can range from mild to severe and may vary between individuals.

Trial Friend lists all active clinical trials for Paroxysmal Nocturnal Hemoglobinuria from ClinicalTrials.gov, with eligibility criteria translated into plain English. Patients can filter by trial phase and recruitment status, search by location, and use the Match Me tool to score trials against their profile. Treatment for Paroxysmal Nocturnal Hemoglobinuria has been transformed by complement inhibitors, with multiple therapies now FDA-approved and newer agents continuing to expand available options. Research is examining how to best combine complement inhibitors, determine optimal timing for treatment initiation, and achieve complete complement inhibition that maximizes symptom control.

Yes. As of September 2026, Trial Friend lists 3 FDA-approved drugs used to treat Paroxysmal Nocturnal Hemoglobinuria: Fabhalta (iptacopan), first approved in 2023; Piasky (crovalimab), first approved in 2024; and Voydeya (danicopan), first approved in 2024.

Each has its own page with what the label covers, side effects and how it is given, and the Treatments section of this page adds any further approved drugs found in FDA labeling data.

Paroxysmal Nocturnal Hemoglobinuria arises from acquired (somatic) genetic changes in the affected cells, commonly involving PIGA, rather than from an inherited variant. Almost all cases are an acquired PIGA variant in a hematopoietic stem cell; diagnosis is by flow cytometry for missing GPI-anchored proteins, not inherited DNA testing. Approximately 1-2 cases per million people; estimated 2,000-5,000 patients in the United States.

For Paroxysmal Nocturnal Hemoglobinuria the genetic testing that matters is usually of the tumor or affected blood cells rather than inherited DNA, looking for PIGA. Almost all cases are an acquired PIGA variant in a hematopoietic stem cell; diagnosis is by flow cytometry for missing GPI-anchored proteins, not inherited DNA testing.

Those results guide treatment choice and are often the entry requirement for targeted-therapy trials, so ask whether the biopsy was sent for molecular testing and request the report. The Genetic Testing section of this page lists laboratories in the NIH Genetic Testing Registry.

Eligibility for Paroxysmal Nocturnal Hemoglobinuria clinical trials depends on factors like age, diagnosis, treatment history, and overall health. Each trial sets its own inclusion and exclusion criteria. Trial Friend translates these requirements from medical language into plain English so patients can review them more easily. The Match Me tool can score your profile against active trials to help identify studies that may be a fit.

Participating in a Paroxysmal Nocturnal Hemoglobinuria clinical trial typically involves scheduled visits, medical tests, and close monitoring by a research team. Depending on the study phase, you may receive an investigational treatment, a placebo, or the current standard of care. Trials may last from a few weeks to several years. Study participation is voluntary, and you can withdraw at any time.