Recruiting Studies
Advanced SearchData: ClinicalTrials.gov · Source of record for eligibility and locations
Top Cities for Paroxysmal Nocturnal Hemoglobinuria Clinical Trials
Paroxysmal Nocturnal Hemoglobinuria clinical trials are recruiting across 10 cities. Here are the cities with the most active studies:
Paroxysmal Nocturnal Hemoglobinuria Trial Phases
7 research organizations are sponsoring paroxysmal nocturnal hemoglobinuria trials.
About Paroxysmal Nocturnal Hemoglobinuria
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, acquired blood disease where the immune system destroys red blood cells, causing dark-colored urine, fatigue, and blood clots. It is caused by a genetic mutation in blood stem cells. Treatment includes complement inhibitors like eculizumab.
Clinical trials are advancing new treatments for paroxysmal nocturnal hemoglobinuria. Currently, 8 studies are recruiting a combined 3,137 participants across the United States. Research is being conducted by 7 organizations including International PNH Interest Group, Aplastic Anemia and MDS International Foundation, Children's Hospital Medical Center, Cincinnati and 4 others.
2026 Paroxysmal Nocturnal Hemoglobinuria Research Landscape
As of July 2026, the paroxysmal nocturnal hemoglobinuria clinical trial landscape includes 8 actively recruiting studies across 10 cities in the United States. These studies are collectively seeking 3,137 participants, with an average enrollment target of 392 per study.
The research spans multiple phases of development: 2 studies are in PHASE1, 2 studies are in PHASE2, 1 study is in PHASE3. The presence of Phase 3 trials indicates that some paroxysmal nocturnal hemoglobinuria treatments are in late-stage testing and may be approaching regulatory review.
Research is being led by 7 different organizations, including International PNH Interest Group, Aplastic Anemia and MDS International Foundation, Children's Hospital Medical Center, Cincinnati, Novartis Pharmaceuticals, CRISPR Therapeutics AG, and 2 others.
Geographically, paroxysmal nocturnal hemoglobinuria trials are most concentrated in Bethesda, Maryland (2 trials); Altamonte Springs, Florida (1 trial); Cincinnati, Ohio (1 trial); East Hanover, New Jersey (1 trial); Rochester, Minnesota (1 trial) and 5 other cities.
Featured Paroxysmal Nocturnal Hemoglobinuria Studies
Highlighted recruiting studies for paroxysmal nocturnal hemoglobinuria, selected by enrollment size and research scope.
The International PNH Interest Group PNH Registry
The aim of this International PNH Interest Group (IPIG) registry is to develop an international database to prospectively collect data on patients with PNH covering clinical outcomes, patient reported outcomes (PROs), and health-resource utilization (HRU) on all enrolled patients, as well as long term safety data.
Global PNH Patient Registry
The primary aim of the Global Paroxysmal Nocturnal Hemoglobinuria (PNH) Patient Registry is to conduct a prospectively-planned and efficient natural history study that will result in a more comprehensive understanding of the disease and its course and pace over time. Other registry objectives include the following: * Provide a convenient online platform for participants (or caregivers) to self-re...
Congenital Hemolytic and Dyserythropoietic Anemias
The main reason for this research study is to further understand how some red blood cells are formed incorrectly or they have an abnormal metabolism in a way that they break easier in the circulation or during their passage through the spleen. Participants and/or family members diagnosed with non-immune hemolytic anemia due to a genetic disorder, such as, hemoglobin disorder, erythrocyte membrane...
Frequently Asked Questions About Paroxysmal Nocturnal Hemoglobinuria Clinical Trials
Are there paroxysmal nocturnal hemoglobinuria clinical trials near me?
Yes, there are 8 paroxysmal nocturnal hemoglobinuria clinical trials currently recruiting across 10+ cities in the United States, including Bethesda, Maryland; Altamonte Springs, Florida; Cincinnati, Ohio. Browse the studies above to find one at a location convenient for you.
How do I join a paroxysmal nocturnal hemoglobinuria clinical trial?
To join a paroxysmal nocturnal hemoglobinuria clinical trial: 1) Browse the available studies on this page, 2) Click on a study that interests you, 3) Check the study locations to find a site near you, 4) Review the eligibility criteria, and 5) Contact the study site or complete the eligibility form. The process is free and you can withdraw at any time.
Are paroxysmal nocturnal hemoglobinuria clinical trials free?
Yes, participation in paroxysmal nocturnal hemoglobinuria clinical trials is free. Study-related treatments, medical tests, and doctor visits are provided at no cost to participants. Many studies also offer compensation for your time and travel expenses.
What types of paroxysmal nocturnal hemoglobinuria treatments are being studied?
Current paroxysmal nocturnal hemoglobinuria clinical trials are testing a range of approaches across PHASE1 (2 studies), PHASE2 (2 studies), PHASE3 (1 study). These include new drugs, combination therapies, medical devices, and other interventions sponsored by 7 research organizations.
Is it safe to participate in paroxysmal nocturnal hemoglobinuria clinical trials?
Clinical trials are carefully regulated by the FDA and institutional review boards (IRBs). All trials must follow strict safety protocols, and participants receive close medical monitoring throughout the study. You can withdraw from a trial at any time without penalty.
Data updated July 16, 2026 from ClinicalTrials.gov
About This Data
Data: ClinicalTrials.gov · Source of record for eligibility and locations
Clinical trial information on this page is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health (NIH) and National Library of Medicine (NLM). Study data is refreshed every hour to ensure accuracy.
Medical Disclaimer: The information provided on this page is for informational purposes only and does not constitute medical advice. Always consult with a qualified healthcare provider before making decisions about clinical trial participation or changes to your treatment plan.
Page reviewed by the HelloStudys Research Team · Last updated July 16, 2026 · Data from ClinicalTrials.gov