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All 29 Paroxysmal Nocturnal Hemoglobinuria trials and their US sites
Enter your ZIP code above to sort them by how far their nearest open site is from you.
- Study to Assess the Pharmacokinetics, Safety, and Tolerability of Iptacopan in Pediatric PNH PatientsMost sites in Georgia, New Jersey and PennsylvaniaRecruitingPhase 3Ages 2-18Novartis Pharmaceuticals4US sites
4 states - A Study of Pegcetacoplan in Pediatric Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)Only in GeorgiaRecruitingPhase 2Ages 12-17Apellis Pharmaceuticals1US site
- Global PNH Patient RegistryOnly in MarylandRecruitingAplastic Anemia and MDS International Foundation1US site
- Haplo-identical Transplantation for Severe Aplastic Anemia, Hypo-plastic MDS and PNH Using Peripheral Blood Stem Cells and Post-transplant Cyclophosphamide for GVHD ProphylaxisOnly in MarylandRecruitingPhase 2Ages 4-75National Heart, Lung, and Blood Institute (NHLBI)1US site
- Home Reported Outcomes in PNHOnly in New JerseyRecruitingAges 18-99Novartis Pharmaceuticals1US site
- MT2023-20: Hematopoietic Cell Transplant With Reduced Intensity Conditioning and Post-transplant Cyclophosphamide for Severe Aplastic Anemia and Other Forms of Acquired Bone Marrow Failure.Only in MinnesotaRecruitingPhase 2Ages 0-75Masonic Cancer Center, University of Minnesota1US site
- Study of Ultomiris® (Ravulizumab) Safety in PregnancyOnly in MassachusettsRecruitingAlexion Pharmaceuticals1US site
- The International PNH Interest Group PNH RegistryOnly in FloridaRecruitingInternational PNH Interest Group1US site
- A First-in-Human Safety and Efficacy Study of ALN-CFB, a Small Interfering RNA (siRNA) Targeting Complement Factor B, in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria With Persistent Anemia on a C5 InhibitorRecruitingPhase 1/2Ages 18+Regeneron PharmaceuticalsNo US site yet
- A Multi-center, Ambispective Cohort Study to Evaluate the Impact of Iptacopan for Adult Patients With PNH in ChinaRecruitingAges 18-80Novartis PharmaceuticalsNo US site yet
- A Phase I Clinical Trial to Evaluate CMS-D017 Following Single and Multiple Doses in Healthy ParticipantsRecruitingPhase 1Ages 18-55Shenzhen Kangzhe Biotechnology Co.No US site yet
- A Phase Ib, Multicenter, Open-Label Study of Multiple-Dose EA5 in Adults With Paroxysmal Nocturnal Hemoglobinuria (PNH)RecruitingPhase 1Ages 18+Shanghai Lanyi Therapeutics Co.No US site yet
- A Phase II Study Evaluating the Efficacy and Safety of XH-S003 Capsules in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)RecruitingPhase 2Ages 18+S-INFINITY Pharmaceuticals Co.No US site yet
- A Study in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) to Evaluate How Safe Long-term Treatment With Pozelimab + Cemdisiran Combination Therapy is and How Well it WorksRecruitingPhase 3Ages 18+Regeneron PharmaceuticalsNo US site yet
- A Study of Iptacopan in Korean Patients With Paroxysmal Nocturnal Hemoglobinuria or C3 GlomerulopathyRecruitingAges 18-99Novartis PharmaceuticalsNo US site yet
- A Study to Evaluate How Pozelimab + Cemdisiran Combination Therapy Works in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Whose Current Treatment is Not Working EfficientlyRecruitingPhase 3Ages 18+Regeneron PharmaceuticalsNo US site yet
- Danicopan PMS in KoreaRecruitingAstraZenecaNo US site yet
- Enhancing the Clinical Management of CMF Through a National Pediatric and Adult Network Based on Biological and Genetic DataRecruitingAges 28 Days-65Istituto Giannina GasliniNo US site yet
- Investigation of Pharmacokinetics,Safety,and Pharmacodynamics of HSK39297 in Subjects With Hepatic ImpairmentRecruitingPhase 1Ages 18-70Haisco Pharmaceutical Group Co.No US site yet
- Long-term Safety and Tolerability of HSK39297 in Patients With Paroxysmal Nocturnal HemoglobinuriaRecruitingPhase 2Ages 18+Haisco Pharmaceutical Group Co.No US site yet
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Who runs the Paroxysmal Nocturnal Hemoglobinuria trials in the US, and what stage they are in
Of the 8 Paroxysmal Nocturnal Hemoglobinuria trials with a US site, 1 is in Phase 3, the last stage before a company can ask the FDA for approval. Earlier Phase 1 or 2 tests of safety and dosing make up 3 of them. The remaining 4 have no drug phase, such as studies that follow people over time or test a device or a way of giving care. Companies run 4 of them, and universities, hospitals, foundations and government groups run 4. Novartis Pharmaceuticals runs the most, with 2. The most recently listed one first appeared on ClinicalTrials.gov on April 18, 2025.
Paroxysmal Nocturnal Hemoglobinuria trials in the US, state by state
Of the 8 Paroxysmal Nocturnal Hemoglobinuria trials with a US site, 7 accept children under 18. Their open sites are in 8 states, most often Georgia, Maryland and Florida. A trial counts once in each state where it has an open site, and some trials accept both children and adults.
US hospitals running the most Paroxysmal Nocturnal Hemoglobinuria trials
Centers that run trials for a rare condition often see more people with it than most clinics do, so this list can also help you find doctors who know Paroxysmal Nocturnal Hemoglobinuria.
- Aplastic Anemia and MDS International FoundationBethesda, Maryland1 trial
- Cancer Institute of New JerseyNew Brunswick, New Jersey1 trial
- Children's Hospital of AtlantaAtlanta, Georgia1 trial
- Childrens Healthcare of AtlantaAtlanta, Georgia1 trial
- Childrens Hospital of PhiladelphiaPhiladelphia, Pennsylvania1 trial
- International PNH Interest GroupAltamonte Springs, Florida1 trial
- National Institutes of Health Clinical CenterBethesda, Maryland1 trial
- St Jude Childrens Research HospitalMemphis, Tennessee1 trial
- University of Minnesota Masonic Cancer CenterMinneapolis, Minnesota1 trial
Help getting to a Paroxysmal Nocturnal Hemoglobinuria trial far from home
Ask the study team whether the trial pays back travel, parking and hotel costs, because many sponsors do. The FDA's guidance on paying research volunteers says paying back these costs does not count as pressuring someone to join.
These nonprofits also help patients reach care, and each one sets its own rules.
- Mercy Medical Angels
- Pays for gas cards, ground rides and airline tickets anywhere in the US, and it lists clinical trials as a reason to travel. Household income at or below 300% of the federal poverty guidelines, and usually a trip of 50 miles or more each way.
- Air Charity Network
- Volunteer pilots in all 50 states fly patients for free in small private planes, and members also fly people taking part in clinical trials. You must be able to walk and sit upright for the flight, show financial need and have a backup plan if weather cancels it.
- Ronald McDonald House
- Gives families a room near the hospital while a child gets care, through 394 House programs around the world. Families of a sick child, with rules set by each local chapter.
- Healthcare Hospitality Network
- Keeps a map of hospitality houses near hospitals, and most member houses charge little or nothing to patients and families. Each house sets its own rules, so check with the house near your study site.
Program details were checked on each program's website on October 24, 2026.
Questions about finding a Paroxysmal Nocturnal Hemoglobinuria trial near you
Are there Paroxysmal Nocturnal Hemoglobinuria clinical trials near me?
As of November 13, 2026, 29 Paroxysmal Nocturnal Hemoglobinuria clinical trials are recruiting now and 14 are not yet recruiting. In the US, 8 of them have at least 1 open site, across 8 states. Enter your ZIP code on this page to rank every one of them by the distance to its closest open site, with the sites shown on a map.
Which states have Paroxysmal Nocturnal Hemoglobinuria clinical trial sites?
Recruiting Paroxysmal Nocturnal Hemoglobinuria trials list open sites in Florida, Georgia, Maryland, Massachusetts, Minnesota, New Jersey, Pennsylvania and Tennessee.
Are there Paroxysmal Nocturnal Hemoglobinuria trials for children near me?
Of the 8 Paroxysmal Nocturnal Hemoglobinuria trials with a US site, 7 accept children under 18. Their open sites are in 8 states, most often Georgia, Maryland and Florida. Each trial sets its own age limits, so check the ages listed on a trial before you contact it.
Who pays for travel to a Paroxysmal Nocturnal Hemoglobinuria clinical trial?
Many trial sponsors pay back travel, parking and hotel costs, so ask the study team before you rule out a far-away site. The FDA's guidance says paying back these costs does not count as pressuring someone to join. Charities such as Mercy Medical Angels and Air Charity Network can also help with rides and flights.
What if there is no Paroxysmal Nocturnal Hemoglobinuria trial near me?
Some trials send staff to your home or use a local lab, so fewer visits happen at the study site. You can also sign up on this page to get an email when a Paroxysmal Nocturnal Hemoglobinuria trial opens a new site or changes status.
How is the distance to a trial site measured?
Distances are straight-line miles from the center of your ZIP code to each study site's listed location, not driving distance. Your browser looks up the ZIP code's coordinates with a free map service, Zippopotam.us, and the ZIP code is never sent to or stored by Trial Friend.
FDA-approved for Paroxysmal Nocturnal Hemoglobinuria: Fabhalta (iptacopan), Piasky (crovalimab), Voydeya (danicopan), Empaveli (pegcetacoplan), Soliris (eculizumab), Ultomiris (ravulizumab-cwvz).
Clinical trials near you for related conditions
Source: ClinicalTrials.gov, trials with status Recruiting or Not yet recruiting. Refreshed every 6 hours; counts as of November 13, 2026. Sites that ClinicalTrials.gov lists as closed or withdrawn are left out. Children means the trial's age group includes people under 18.