RareWays is a research directory. It helps you understand the science. It is not medical advice. Always discuss your care options with your healthcare team.
Coverage: 2020-01-01 onwards, with up to 300 results per query from PubMed and Europe PMC and separate limits on other sources. This is a selected index, not a complete literature search.
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Haemophilia: Research Summary
Printed from RareWays (rareways.org)
For general awareness only. Not medical advice. Discuss all care options with your healthcare team.
5 Most Recent Research Articles
- 1.
Cases of High-Titer Inhibitor Formation After AAV Gene Therapy for Hemophilia A.
Crary Shelley E et al., Blood (16 October 2026)
https://pubmed.ncbi.nlm.nih.gov/42886677/
- 2.
Feasibility and perceptions among hemophilia patients and doctors towards ultrasound for the assessment of subclinical abnormalities in hemophilic joints.
Ye Ming et al., Medicine (16 October 2026)
https://pubmed.ncbi.nlm.nih.gov/42888591/
- 3.
Effects of secondary factor VIII prophylaxis on joint and bone health in a hemophilia A mouse model.
Monica Lara et al., Haematologica (15 October 2026)
https://pubmed.ncbi.nlm.nih.gov/42876293/
- 4.
Socio‑Economic and Education Profile of Children With Haemophilia A Receiving Prophylaxis and Their Families: Results From a Brazilian Cohort.
Cardoso Patrícia Santos Resende et al., Haemophilia : the official journal of the World Federation of Hemophilia (14 October 2026)
https://pubmed.ncbi.nlm.nih.gov/42873602/
- 5.
Association of TNFAIP3 Polymorphisms With Bleeding Phenotypes and FVIII Inhibitor Development in Patients With Hemophilia A: A Case-Control Study.
Karimi Fatemeh et al., Haemophilia : the official journal of the World Federation of Hemophilia (14 October 2026)
https://pubmed.ncbi.nlm.nih.gov/42873604/
Clinical Trials : Currently Recruiting (Australia)
Ask your doctor whether you or your child may be eligible for any of these trials. Each has its ID and full eligibility criteria at the link shown.
- 1.
A Clinical Study to Evaluate the Effects of NXT007 Compared to Emicizumab Prophylaxis in People With Hemophilia A
NCT07416604 · Recruiting · Phase 3 · Hoffmann-La Roche
Ages 12 and over
https://clinicaltrials.gov/study/NCT07416604
- 2.
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
NCT07080905 · Recruiting · Phase 3 · CSL Behring
Ages 138 Months to 206 Months
https://clinicaltrials.gov/study/NCT07080905
- 3.
A Study to Investigate the Safety and Effectiveness of a Coagulation Factor IX Gene Insertion Therapy (REGV131-LNP1265) in Pediatric, Adolescent and Adult Participants With Hemophilia B
NCT06379789 · Recruiting · Phase 1 / Phase 2 · Regeneron Pharmaceuticals
Ages 2 and over
https://clinicaltrials.gov/study/NCT06379789
- 4.
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)
NCT06003387 · Recruiting · Phase 3 · CSL Behring
Ages 18 and over
https://clinicaltrials.gov/study/NCT06003387
- 5.
Safety and Effectiveness of Giroctocogene Fitelparvovec or Fidanacogene Elaparvovec in Patients With Hemophilia A or B Respectively
NCT05568719 · Recruiting · Phase 3 · Pfizer
Ages 18 and over
https://clinicaltrials.gov/study/NCT05568719
Source: RareWays research directory. Data from PubMed, Europe PMC, OpenAlex, ClinicalTrials.gov.
Always verify information with your healthcare team before making any decisions about your care.
Haemophilia
Haemophilia is a rare inherited bleeding disorder caused by deficiency of clotting factor VIII (Haemophilia A) or factor IX (Haemophilia B), leading to prolonged bleeding after injury or surgery. Extended half-life factor replacement and non-factor therapies such as emicizumab have transformed management. Gene therapy is now approved in several countries.
This overview is general information, not an individual medical assessment. Research is selected automatically and may include mismatches. How we select and explain research.
Most Recent Research
Gene therapy for hemophilia A (HA) aims to provide durable hemostatic control and reduce treatment burden, but AAV-based products to date have limitations: inconsistent peak expression, declining factor levels, and immunogenicity causing transaminitis and expression loss. ASC618 (ASC Therapeutics, Milpitas, CA, USA) was developed as a second-generation gene therapy product to overcome some of these limitations, using ET3, a bioengineered chimeric human/porcine B-domain-deleted FVIII transgene, paired with a minimal liver promoter and liver codon optimization. In a phase 1/2 trial (NCT04676048), two patients received ASC618. Despite initial factor expression, both developed high-titer FVIII inhibitors and are now maintained on emicizumab prophylaxis. Although inhibitor titers have declined, both remain high-titer nearly two years after a single infusion. The best explanation for inhibitor formation is that the novel FVIII with the human-porcine hybrid construct (ET3) contained epitopes for which these patients had not developed tolerance despite both having more than 1000 exposure days to exogenous FVIII in the past. Although this trial has been halted, these cases provide important lessons for the development of future hemophilia gene therapies. They highlight the need for careful immunogenicity assessment when using bioengineered transgenes, even in previously treated patients with extensive factor VIII exposure.
Common Questions
What is Haemophilia?
Haemophilia is a rare inherited bleeding disorder caused by deficiency of clotting factor VIII (Haemophilia A) or factor IX (Haemophilia B), leading to prolonged bleeding after injury or surgery. Extended half-life factor replacement and non-factor therapies such as emicizumab have transformed management. Gene therapy is now approved in several countries.
How many clinical trials are available for Haemophilia?
RareWays currently indexes 574 clinical trials for Haemophilia, of which 47 are actively recruiting. These counts cover indexed trials across countries, not just your country. An Australian site does not necessarily mean that site is recruiting. Check locations, eligibility and current availability with the study team.
Where does the research data for Haemophilia come from?
RareWays aggregates research from PubMed, Europe PMC, OpenAlex, and ClinicalTrials.gov. Data is refreshed automatically every day. All articles and trials link directly to their original sources.
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