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Severe hemophilia B is a form of hemophilia B characterized by a large deficiency of factor IX leading to frequent spontaneous hemorrhage and abnormal bleeding as a result of minor injuries, or following surgery or tooth extraction.
Biomarker and diagnostic research for severe hemophilia B has been reported in the published literature.
Estimated prevalence: 1-9 in 1,000,000 (Rare).
6 clinical trials registered, 4 recruiting. Interventions under study include drug therapy, gene therapy, and biologic therapy. Pipeline includes 5 PHASE3, 1 PHASE1. Research is primarily industry-sponsored.
NCT ID | Title | Phase | Sponsor | Status |
|---|---|---|---|---|
[NCT03861273](https://clinicaltrials.gov/study/NCT03861273) |
Data assembled from 4 of 12 sources · Last updated Sep 19, 2026, 6:54 PM UTC
Program availability and eligibility requirements are set by each foundation. Contact them directly to learn more about your options.
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European rare disease database
Genetic and Rare Diseases Info Center
A Study to Evaluate the Efficacy and Safety of Factor IX Gene Therapy With PF-06838435 in Adult Males With Moderately Severe to Severe Hemophilia B
PHASE3 |
Pfizer |
ACTIVE_NOT_RECRUITING |
[NCT07080905](https://clinicaltrials.gov/study/NCT07080905) | Phase 3, Open-label, Single-dose Study of CSL222 in Adolescent Male Subjects (≥ 12 to < 18 Years of Age) With Severe or Moderately Severe Hemophilia B | PHASE3 | CSL Behring | RECRUITING |
[NCT05145127](https://clinicaltrials.gov/study/NCT05145127) | Open-Label Extension Study of Marstacimab in Hemophilia Participants With or Without Inhibitors | PHASE3 | Pfizer | RECRUITING |
[NCT06003387](https://clinicaltrials.gov/study/NCT06003387) | Efficacy and Safety of CSL222 (Etranacogene Dezaparvovec) Gene Therapy in Adults With Hemophilia B With Pretreatment Adeno-associated Virus Serotype 5 (AAV5) Neutralizing Antibodies (Nabs) | PHASE3 | CSL Behring | RECRUITING |
[NCT05611801](https://clinicaltrials.gov/study/NCT05611801) | A Clinical Trial of Study Medicine (Marstacimab) in Pediatric Patients With Hemophilia A or Hemophilia B | PHASE3 | Pfizer | RECRUITING |
80 publications have been identified in PubMed for severe hemophilia B. Research spans Epidemiology / Natural History (31%), Clinical Trial Publication (16%), and Case Report / Case Series (15%).
Research Type | Count | % of Total |
|---|---|---|
Disease patterns and progression | 25 | 31% |
Clinical study results | 13 | 16% |
Patient case studies | 12 | 15% |
Research summaries | 11 | 14% |
Laboratory research | 9 | 11% |
New treatment approaches | 5 | 6% |
Other research | 4 | 5% |
Testing and diagnosis research | 1 | 1% |
Polack B (2026). [PMID: 41870437](https://pubmed.ncbi.nlm.nih.gov/41870437/). *J Comp Eff Res*. [Epidemiology / Natural History]
Mahlangu J (2026). [PMID: 42107711](https://pubmed.ncbi.nlm.nih.gov/42107711/). *J Thromb Haemost*. [Clinical Trial Publication]
Basta M (2026). [PMID: 32491315](https://pubmed.ncbi.nlm.nih.gov/32491315/). *Unknown Journal*. [Basic Science / Preclinical]
Contente S (2026). [PMID: 41644240](https://pubmed.ncbi.nlm.nih.gov/41644240/). *J Thromb Haemost*. [Epidemiology / Natural History]
Prameyllawati DM (2026). [PMID: 40887565](https://pubmed.ncbi.nlm.nih.gov/40887565/). *Appl Health Econ Health Policy*. [Review / Meta-Analysis]
Abu-Hanna J (2026). [PMID: 41607355](https://pubmed.ncbi.nlm.nih.gov/41607355/). *Transfusion*. [Basic Science / Preclinical]
Takenaka R (2026). [PMID: 41407872](https://pubmed.ncbi.nlm.nih.gov/41407872/). *Skeletal Radiol*. [Review / Meta-Analysis]
Lyu TW (2026). [PMID: 41549881](https://pubmed.ncbi.nlm.nih.gov/41549881/). *Haemophilia*. [Review / Meta-Analysis]
Safdari SM (2026). [PMID: 41742268](https://pubmed.ncbi.nlm.nih.gov/41742268/). *Thromb J*. [Review / Meta-Analysis]
Olivieri M (2026). [PMID: 42158717](https://pubmed.ncbi.nlm.nih.gov/42158717/). *Transfus Med Hemother*. [Review / Meta-Analysis]