A transforming therapeutic landscape worth monitoring daily
Hemophilia is one of the most commercially active rare disease therapeutic areas, undergoing a fundamental transformation. The approval of gene therapies for hemophilia A (fitusiran, valoctocogene roxaparvovec) and hemophilia B (etranacogene dezaparvovec) has validated the approach commercially, while next-generation programs seek to improve durability and expand eligible patient populations. Simultaneously, non-factor replacement therapies, led by emicizumab, continue expanding their role in the treatment paradigm.
Key signals in the hemophilia pipeline in 2026:
- Next-generation AAV gene therapy programs for hemophilia A (Factor VIII deficiency) with improved durability and seroprevalence coverage
- Hemophilia B (Factor IX deficiency) gene therapy programs and long-term follow-up studies for approved products
- Non-factor replacement therapies: bispecific antibodies, fitusiran (antithrombin targeting), and rebalancing agents
- Inhibitor management programs, patients who develop inhibitors to factor replacement represent a particularly challenging and commercially important subgroup
- Gene editing approaches (CRISPR, base editing) for hemophilia as curative strategies
- Pediatric programs: eligibility expansion for gene therapy and non-factor approaches to younger patients
- Real-world evidence and long-term safety studies following gene therapy approvals
Get daily hemophilia trial digest
Set your profile once. Receive a filtered daily digest of new and updated hemophilia trials. Free plan, no credit card required.
Get the Free DigestA primary completion date pushed out, enrollment cut, or a primary endpoint changed in the last 90 days. Active trials only.
| Trial | Warning signs | Status |
|---|---|---|
| Lentiviral FVIII Gene Therapy NCT03217032 | Primary completion moved at least 12 months later | Not Yet Recruiting |
11 more on the Trials at Risk page. See which trials →
Phase 2 and Phase 3 trials with a primary completion date in the next 180 days, as ClinicalTrials.gov estimates it today. Active trials only.
| Trial | Scheduled | Phase |
|---|---|---|
| Efficacy and Safety of KN057 Prophylaxis in Patients With Haemophilia A or B… NCT06569108 | Oct 15, 2026 · Estimated | Phase 3 |
12 more on the Completions page. See the calendar →
What DataLookout monitors for hemophilia
Configure your profile with condition and mechanism keywords matched to your specific intelligence priorities:
- Condition keywords: "hemophilia A", "hemophilia B", "hemophilia", "Factor VIII deficiency", "Factor IX deficiency", "von Willebrand disease"
- Mechanism keywords: "gene therapy", "AAV", "Factor VIII", "Factor IX", "emicizumab", "fitusiran", "non-factor", "bispecific antibody"
- Subtype specific: "hemophilia with inhibitors", "severe hemophilia", "moderate hemophilia"
- Phase filter: Phase 2/3 for commercial and BD intelligence, or all phases for comprehensive pipeline coverage
- Sponsor filter: Industry-sponsored for competitive tracking, NIH/academic for emerging science
What DataLookout adds to ClinicalTrials.gov
ClinicalTrials.gov RSS feeds tell you that a study record was updated. DataLookout compares each record against the previous day across 13 tracked fields and tells you which one changed, with the old and new values, for every Hemophilia trial on your watchlist. Each change is marked Stopped, Notable or Minor, so a trial being terminated does not read the same as a few sites being added.
Who uses hemophilia trial monitoring
Rare disease pharma BD and strategy teams
Companies active in hemophilia, spanning gene therapy, protein engineering, and novel mechanisms, track new trial registrations to identify partnership opportunities and anticipate competitive dynamics. The gene therapy landscape in hemophilia is evolving rapidly, with durability, immune response management, and patient selection all active areas of clinical investigation.
Hematology biotech analysts and investors
Investors covering rare hematology use hemophilia trial registrations as leading indicators of pipeline development. Gene therapy program phase transitions and non-factor therapy expansion into new patient populations represent significant investment signals that daily trial monitoring enables.
Hemophilia treatment centers and medical affairs teams
Hemophilia treatment centers (HTCs) and medical affairs professionals at hemophilia companies track new trial registrations to understand the competitive landscape, identify opportunities for site participation in upcoming trials, and stay current on emerging evidence in gene therapy long-term outcomes.
National Hemophilia Foundation and patient advocacy
Patient advocacy organizations monitor the clinical trial pipeline to connect patients, particularly those with inhibitors or those who are not candidates for current gene therapy programs due to pre-existing AAV antibodies, with appropriate clinical study opportunities.
Frequently asked questions
How current is the hemophilia trial data?
Our pipeline fetches from ClinicalTrials.gov every morning. Studies posted or updated in the preceding 24 hours appear in that day's digest. ClinicalTrials.gov is updated as sponsors register new trials or submit protocol amendments, typically within 24–48 hours.
Can I track hemophilia A and hemophilia B programs separately?
Yes. On Pro ($49/month, or $490/year), you can save up to 10 searches, for example, one focused on hemophilia A gene therapy and another monitoring hemophilia B programs and non-factor therapies, each delivering a focused daily digest.
Does DataLookout cover gene therapy long-term follow-up studies?
Yes. Long-term follow-up (LTFU) studies required after gene therapy approval are registered on ClinicalTrials.gov and will appear in your digest when matching your profile keywords.
Does DataLookout cover von Willebrand disease trials?
Yes, adding "von Willebrand disease" or "VWD" to your profile keywords will surface relevant clinical trials, which are often conducted alongside or in comparison to hemophilia programs.
177 active trials, 86 recruiting.
Industry sponsors run 87 of these trials. The rest are run by universities, hospitals, government bodies and other non-commercial sponsors. The table below lists the 5 largest of 29.
| Top industry sponsor | Active trials |
|---|---|
| Novo Nordisk | 14 |
| Pfizer | 8 |
| Roche / Genentech | 7 |
| Bayer | 6 |
| CSL Behring | 6 |
| Trial | Status | Latest tracked change | Seen |
|---|---|---|---|
| Adverse Event Data Collection From the EUHASS Registry on Turoctocog Alfa Pegol NCT04682145 | Enrolling by Invitation | Completion pushed from Feb 1, 2026 to Dec 1, 2026 | 2026-10-09 |
| Determinants and Consequences of the Transition to Adulthood for Adolescents… NCT07418827 | Recruiting | Recruitment opened | 2026-10-09 |
| Patient Preferences in Treatment of Haemophilia A With and Without Inhibitors… NCT07653139 | Enrolling by Invitation | Completion pushed from Oct 26, 2026 to Dec 26, 2026 | 2026-10-07 |
| A Clinical Study to Evaluate the Effects of NXT007 Compared to Emicizumab… NCT07416604 | Recruiting | Trial sites expanded: 42 → 59 locations | 2026-10-07 |
| A Prospective Assessment of Bone Health in Patients With Severe Hemophilia A on… NCT07582276 | Recruiting | Completion pushed from Aug 1, 2032 to Sep 2032 | 2026-10-06 |
Each trial page shows every change DataLookout has recorded for that trial.
DataLookout checks ClinicalTrials.gov every day and records what changed on each trial: status, enrollment, phase, primary endpoint, completion dates, sites, sponsor, and the stated reason a trial stopped. Add a sponsor or a disease to your watchlist, or save any search, and the changes arrive in a daily or weekly email digest.
Free, $0 forever: 1 sponsor + 1 disease watchlist, 1 saved search in your email digest, track up to 5 individual trials, daily change detection, Trials at Risk (top 2 trials), the weekly report (top 3 trials). No credit card.
Pro, $49/month or $490/year: 10 sponsor + 10 disease watchlists, 10 saved searches in your email digest, track up to 25 individual trials, full Trials at Risk list, Completions calendar, the full weekly report, landscape reports, CSV export.