Diseases / Hemophilia A
Hemophilia A
DiseaseHemophilia A is an inherited bleeding disorder caused by deficiency or dysfunction of clotting factor VIII, leading to impaired coagulation and prolonged bleeding. Clinical severity ranges from mild to severe and can include spontaneous joint and muscle hemorrhage in severe disease. It is the most common form of hemophilia.
- Trials
- 777
- Recruiting
- 70
- Completed
- 458
- With results
- 166
Last revised
Pipeline
Phase by status
| Phase | Recruiting | Not yet | By invitation | Active | Completed | Terminated | Withdrawn | Suspended | Unknown | Approved for marketing | Total |
|---|---|---|---|---|---|---|---|---|---|---|---|
| Early Phase 1 | 2 | — | — | — | 2 | — | 1 | — | 1 | — | 6 |
| Phase 1 | 4 | 2 | — | 1 | 58 | 5 | 2 | — | 3 | — | 75 |
| Phase 1/2 | 5 | — | — | 4 | 10 | 3 | — | — | 1 | — | 23 |
| Phase 2 | 1 | 1 | — | — | 23 | 7 | — | — | 2 | — | 34 |
| Phase 2/3 | 1 | 1 | — | 1 | 9 | 1 | — | — | 1 | — | 14 |
| Phase 3 | 11 | 2 | — | 14 | 87 | 13 | 4 | — | 7 | — | 138 |
| Phase 4 | 9 | — | — | 3 | 38 | 7 | 3 | — | 7 | — | 67 |
| Not applicable | 6 | 7 | 3 | 1 | 68 | 8 | 2 | — | 24 | — | 119 |
| Unknown | 31 | 17 | 7 | 12 | 163 | 11 | 6 | 1 | 52 | 1 | 301 |
Trials started per year
Conditions studied, by phase
| Condition | Early Phase 1 | Phase 1 | Phase 1/2 | Phase 2 | Phase 2/3 | Phase 3 | Phase 4 | Not applicable | Unknown | Total |
|---|---|---|---|---|---|---|---|---|---|---|
| Hemophilia A | 6 | 75 | 23 | 34 | 14 | 138 | 67 | 119 | 301 | 777 |
| Hemophilia B | 1 | 21 | 3 | 6 | 1 | 15 | 5 | 11 | 71 | 134 |
| Hematologic Diseases | — | — | 6 | — | 1 | 10 | 3 | — | 13 | 33 |
| Blood Coagulation Disorders | — | 2 | 6 | — | 1 | 12 | 3 | 1 | 7 | 32 |
| Joint Diseases | — | — | — | — | — | — | 1 | 13 | 16 | 30 |
| Coagulation Protein Disorders | — | 1 | 6 | — | 1 | 10 | 2 | — | 7 | 27 |
| Hemostatic Disorders | — | — | 3 | 1 | — | 6 | 3 | 1 | 12 | 26 |
| Hemorrhagic Disorders | — | — | 6 | — | 1 | 9 | — | — | 6 | 22 |
| Genetic Diseases, Inborn | — | — | 6 | — | — | 7 | — | — | 7 | 20 |
| Motor Activity | — | — | — | — | — | — | — | 9 | 9 | 18 |
Most active sponsors
| # | Sponsor | Trials | Phase 3 |
|---|---|---|---|
| 1 | Novo Nordisk A/S | 67 | 16 |
| 2 | Bayer | 56 | 8 |
| 3 | Baxalta now part of Shire | 40 | 19 |
| 4 | Pfizer | 33 | 9 |
| 5 | Investigación en Hemofilia y Fisioterapia | 19 | 0 |
| 6 | Hoffmann-La Roche | 16 | 10 |
| 7 | Octapharma | 15 | 8 |
| 8 | Swedish Orphan Biovitrum | 14 | 2 |
| 9 | CSL Behring | 13 | 7 |
| 10 | Wyeth is now a wholly owned subsidiary of Pfizer | 12 | 5 |
Most studied interventions
| # | Intervention | Trials |
|---|---|---|
| 1 | Factor VIII | 123 |
| 2 | F8 protein, human | 52 |
| 3 | emicizumab | 33 |
| 4 | BAY 14-2222 | 22 |
| 5 | factor VIII-Fc fusion protein | 19 |
| 6 | recombinant factor VIII N8 | 19 |
| 7 | BAX 855 | 18 |
| 8 | Injections | 16 |
| 9 | recombinant factor VIII SQ | 14 |
| 10 | Blood Specimen Collection | 11 |
Evidence
- Results reported
- 36.2%166 of 458 completed
- Published
- 13.8%63 of 458 completed
- Primary endpoint met
- 74.5%41 of 55 analyses
- Median months to report
- 11
Documents disclosed
Trial profile
- Median enrollment
- 42
- Median duration
- 24months
- Countries
- 89
- Study sites
- 5437
Who is enrolled
Median enrollment by phase
- Early Phase 1
- 5
- Phase 1
- 17
- Phase 1/2
- 15
- Phase 2
- 23
- Phase 2/3
- 40
- Phase 3
- 66
- Phase 4
- 30
- Not applicable
- 24
- Unknown
- 81
Where trials run
Busiest cities
| # | City | Country | Trials |
|---|---|---|---|
| 1 | London | United Kingdom | 104 |
| 2 | Madrid | Spain | 93 |
| 3 | Milan | Italy | 92 |
| 4 | Los Angeles | United States | 77 |
| 5 | Berlin | Germany | 69 |
| 6 | Bonn | Germany | 66 |
| 7 | Florence | Italy | 63 |
| 8 | Indianapolis | United States | 61 |
| 9 | Tianjin | China | 60 |
| 10 | Houston | United States | 58 |
Terminations
- Terminated
- 55
- Withdrawn
- 18
- Suspended
- 1
- Stopped for recruitment
- 1.4%11 of 777 trials
Stated reasons
Reasons are grouped from the sponsor's own wording with a rule-based classifier; the original text is shown below.
Recently stopped
| Trial | Status | Stated reason | Stopped |
|---|---|---|---|
| NCT06297486 Withdrawn | Withdrawn | Strategic Decision | 2035-09-04 |
| NCT02196207 Withdrawn | Withdrawn | The trial was revised to be two protocols, one Prevention, one Eradication Trials. | 2027-07-31 |
| NCT04690322 Withdrawn | Withdrawn | Due to COVID-19 pandemic, recruitment was temporarily put on hold. We are withdrawing this registration due to a recent amendment to the protocol that includes major changes to data elements. A new registration will be submitted for the new protocol. | 2027-07-31 |
| NCT06345833 Withdrawn | Withdrawn | Lack of Participants | 2026-07-01 |
| NCT06702579 Terminated | Terminated | The study was discontinued due to a lack of available personnel to complete the study | 2026-06-16 |
| NCT06273865 Suspended | Suspended | Performing interim analysis before continuing with next center | 2026-05-01 |
| NCT06634836 Withdrawn | Withdrawn | Business decision | 2025-12-31 |
| NCT06530030 Terminated | Terminated | Early discontinuation based on sponsor decision not driven by any safety concerns. | 2025-09-18 |
| NCT05695391 Terminated | Terminated | Feasibility considerations - extended timelines to complete recruitment | 2025-07-07 |
| NCT05789524 Terminated | Terminated | Study terminated due to business and strategic decision | 2025-02-28 |
Trial records
Individual study records live on ClinicalTrials.gov. A sample of the trials counted above: