Genetic and immune clues in pediatric paroxysmal nocturnal hemoglobinuria (PNH)
Official title: Project 3: Genetic and immunological signatures of disease presentation and progression in pediatric paroxysmal nocturnal hemoglobinuria
Funded at Boston Children's Hospital · NIH-11453343 · Data updated 2026-11-12
A multi-institution study in children with PNH looks for genetic and immune markers that explain how the disease presents and progresses.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Boston Children's Hospital NIH-funded |
| Lab location | 1 site (Boston, United States) |
| Project start | August 21, 2026 |
| Project end | June 30, 2031 |
| Funding year | 2026 |
| Award (this year) | $164,160 |
| NIH project number | 1U54HD123051-01 |
| Project ID | NIH-11453343 on NIH RePORTER |
Who could benefit from this research
✓Could help
Children and adolescents diagnosed with PNH, especially those with bone marrow failure features, who are treated or evaluated at participating centers.
✕Less likely to help
Only non-PNH patients or those not eligible under the study’s data-sharing criteria would likely not benefit.
What this research studies
This project focuses on pediatric PNH, a rare blood disorder, to understand why symptoms vary and how the disease changes over time. It includes collaboration across centers to collect consistent data and to study underlying biology and possible biomarkers. The goal is to tailor treatment decisions for children, potentially guiding when to use immunosuppression or transplant and which regimens to consider. By studying how the developing immune system in children affects PNH, researchers hope to improve care and outcomes for young patients.
Why it matters
Potential benefit: Findings could lead to personalized treatment plans and better outcomes for children with PNH.
How similar studies have gone: Prior studies have shown that multi-institution collaborations can identify biomarkers and inform treatment in rare diseases, though pediatric PNH remains under-studied.
Where this research is happening
-
Boston Children's Hospital
Boston, United States Active
Researchers
- Principal investigator: Ware, Russell E — Boston Children's Hospital
- Study coordinator: Ware, Russell E
Related research
Other NIH-funded projects on the same topics.
-
Oral HS-10542 for paroxysmal nocturnal hemoglobinuria
This study will test whether the oral drug HS-10542 can reduce hemolysis and raise hemoglobin in adults with PNH, including those not fully helped by C5 inhibitor therapy.
-
Danicopan added to ravulizumab or eculizumab for PNH in Korea
This will see if adding Danicopan to ravulizumab or eculizumab is safe and helps people with PNH who are treated in routine care in Korea.
-
Iptacopan use in Korean patients with PNH or C3G
This post-marketing surveillance study tests how well iptacopan works and how safe it is for Korean adults with PNH or C3G in real-world care.
-
Pozelimab + cemdisiran for adults with PNH whose current treatment isn't working
This trial tests whether combining pozelimab and cemdisiran can better reduce hemolysis in adults with PNH whose C5 inhibitor treatment isn’t controlling their disease.
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Evaluating MY008211A for treating anemia in PNH patients on anti-C5 therapy
This study is testing a new treatment called MY008211A to see if it can help people with PNH who still have anemia even while on anti-C5 therapy.
-
Iptacopan treatment for adults with PNH in China
This study will try iptacopan (LNP023) in adults with PNH in China to see if it improves clinical and laboratory outcomes and changes healthcare use.
About this research
- This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
- Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
- For full project details, budget, and progress reports, visit the official NIH RePORTER page.