Biomarker & Intervention Discovery

Haemophilia A / B

This page lists 8 druggable gene targets, 56 approved or trial-tested drug candidates and 15 recent clinical trials for Haemophilia A / B, with each agent ranked by the strength of its evidence (clinical, mechanistic or correlative). Candidates include existing drugs that could be repurposed. Sources are linked so you can check every claim.

Spontaneous remissionNo spontaneous remission; lifelong clotting factor deficiency; joint damage progressive with each bleed without…
Best-intervention remissionEmicizumab prophylaxis (HAVEN studies): 87% reduction in bleeds vs on-demand FVIII; fitusiran (antithrombin RNAi):…
Gap sizeLarge — gene therapy offers functional cure but access is essentially the entire gap
Primary barrieraccess
Full remission profile →

Remission & chronicity

Spontaneous remission
No spontaneous remission; lifelong clotting factor deficiency; joint damage progressive with each bleed without treatment; with prophylaxis, normal lifespan achievable
Best-intervention remission
Emicizumab prophylaxis (HAVEN studies): 87% reduction in bleeds vs on-demand FVIII; fitusiran (antithrombin RNAi): monthly subcutaneous injection, 89% reduction in bleeds; gene therapy (valoctocogene roxaparvovec): 94% reduction in bleeds at 5 years (GENEr8-1)
Gap size
Large — gene therapy offers functional cure but access is essentially the entire gap
Primary barrier
access
Gene therapy for haemophilia A (valoctocogene roxaparvovec/Roctavian, FDA approved 2023) and B (fidanacogene elaparvovec/Beqvez) represent functional cures in eligible patients but cost $2.9-3.5M; IgG immune responses limit retreatment; durability questions persist at 5 years; most patients in LMICs remain on factor concentrates

Biomarker Targets & Therapeutic Agents

Gene targets queried against DGIdb, Open Targets, ChEMBL, PubMed, and Europe PMC. Agents ranked: Clinical > Mechanistic > Correlative.

F10 242F2 241F7 182F8 27F9 223SERPINC1 35TFPI 213VWF 21
Therapeutic Agent Effect Direction Evidence Tier Potency Source
FACTOR IX ? unclear Mechanistic
SSR-126517E ? unclear Mechanistic
COAGULATION FACTOR IX ? unclear Mechanistic
COAGULATION FACTOR X HUMAN ? unclear Mechanistic
LETAXABAN ? unclear Mechanistic
IDRAPARINUX ? unclear Mechanistic
COAGULATION FACTOR X, HUMAN ? unclear Mechanistic
NADROPARIN CALCIUM ? unclear Mechanistic
EDOXABAN ↓ inhibits Mechanistic
RIVAROXABAN ↓ inhibits Mechanistic
THROMBIN ? unclear Mechanistic
LY-517717 ? unclear Mechanistic
SR-123781A ? unclear Mechanistic
ERIBAXABAN ↓ inhibits Mechanistic
TRIMETHOPRIM/SULFADOXINE ? unclear Mechanistic
HEPARIN ? unclear Mechanistic
APIXABAN ↓ inhibits Mechanistic
MANGIFERIN HEPTASULFATE ? unclear Mechanistic
DX-9065A ↓ inhibits Mechanistic
COAGULATION FACTOR VIIA ? unclear Mechanistic
EDOXABAN TOSYLATE ↓ inhibits Mechanistic
TANOGITRAN ? unclear Mechanistic
2-O, 3-O DESULFATED HEPARIN ? unclear Mechanistic
BAY 3389934 ↓ inhibits Mechanistic
FACTOR VIII ? unclear Mechanistic
SEMULOPARIN ? unclear Mechanistic
IDRABIOTAPARINUX SODIUM ↓ inhibits Mechanistic
ANDEXANET ALFA ? unclear Mechanistic
BEMIPARIN ↓ inhibits Mechanistic
VITAMIN K3 ? unclear Mechanistic
DAREXABAN ↓ inhibits Mechanistic
ANTITHROMBIN III ? unclear Mechanistic
FONDAPARINUX ? unclear Mechanistic
PROTHROMBIN COMPLEX CONCENTRATE ? unclear Mechanistic
DANAPAROID ? unclear Mechanistic
GW-813893 ? unclear Mechanistic
IDRAPARINUX SODIUM ↓ inhibits Mechanistic
BETRIXABAN ↓ inhibits Mechanistic
NONACOG ALFA ? unclear Mechanistic
EP-217609 ? unclear Mechanistic

Showing top 40 of 242 agents ranked by evidence tier.

Therapeutic Agent Effect Direction Evidence Tier Potency Source
DESIRUDIN ↓ inhibits Mechanistic
CATECHIN ? unclear Mechanistic
BIVALIRUDIN ↓ inhibits Mechanistic
ICHORCUMAB ↓ inhibits Mechanistic
APRAMIDE A ? unclear Mechanistic
ANTITHROMBIN III ? unclear Mechanistic
XIMELAGATRAN ↓ inhibits Mechanistic
ASPIRIN TRELAMINE ? unclear Mechanistic
CHEMBL:CHEMBL586628 ? unclear Mechanistic
ATECEGATRAN METOXIL ↓ inhibits Mechanistic
WX-UK1 ↓ inhibits Mechanistic
ARGATROBAN ↓ inhibits Mechanistic
ODIPARCIL ? unclear Mechanistic
HIRUDIN ? unclear Mechanistic
DABIGATRAN ↓ inhibits Mechanistic
VITAMIN K3 ? unclear Mechanistic
ANISINDIONE ? unclear Mechanistic
ANTITHROMBIN ALFA ↓ inhibits Mechanistic
MELAGATRAN ↓ inhibits Mechanistic 4700.0 nM (IC50)
PYRIDOXAL PHOSPHATE ANHYDROUS ? unclear Mechanistic
AZD-8165 ? unclear Mechanistic
DPOC-4088 ? unclear Mechanistic
TAMOXIFEN ? unclear Mechanistic
DUP-714 ↓ inhibits Mechanistic
BAY 3389934 ↓ inhibits Mechanistic
PROTHROMBIN COMPLEX CONCENTRATE ? unclear Mechanistic
PEGMUSIRUDIN ? unclear Mechanistic
DABIGATRAN ETEXILATE MESYLATE ↓ inhibits Mechanistic
DEFIBROTIDE ↓ inhibits Mechanistic
PROTHROMBIN ? unclear Mechanistic
HEPARIN ? unclear Mechanistic
SR-123781A ? unclear Mechanistic
CYANIN ? unclear Mechanistic
BORTEZOMIB ? unclear Mechanistic 13000.0 nM (Ki)
AZ12971554 ↓ inhibits Mechanistic
ENOXAPARIN SODIUM ? unclear Mechanistic
AVATROMBOPAG ? unclear Mechanistic
SILIBININ ? unclear Mechanistic
QUERCETIN ? unclear Mechanistic
LUSUTROMBOPAG ? unclear Mechanistic

Showing top 40 of 241 agents ranked by evidence tier.

Therapeutic Agent Effect Direction Evidence Tier Potency Source
VITAMIN K3 ? unclear Mechanistic
PCI-27483 ↓ inhibits Mechanistic
COAGULATION FACTOR VII HUMAN ? unclear Mechanistic
COAGULATION FACTOR VIIA (RECOMBINANT)-JNCW ? unclear Mechanistic
COMPOUND 11 [PMID: 16413183] ↓ inhibits Mechanistic
ANPOCOGIN ? unclear Mechanistic
PROTHROMBIN COMPLEX CONCENTRATE ? unclear Mechanistic
EPTACOG ALFA PEGOL (ACTIVATED) ? unclear Mechanistic
COAGULATION FACTOR VIIA ? unclear Mechanistic
COMPOUND 1 [PMID: 16650987] ↓ inhibits Mechanistic
EPTACOG BETA (ACTIVATED) ? unclear Mechanistic
EPTACOG ALFA (ACTIVATED) ? unclear Mechanistic
VATREPTACOG ALFA (ACTIVATED) ? unclear Mechanistic
ANTITHROMBIN III ? unclear Mechanistic
MARZEPTACOG ALFA (ACTIVATED) ? unclear Mechanistic
CHEMBL2373350 ↓ inhibits Mechanistic 2500.0 nM (Ki)
CHEMBL2373559 ↓ inhibits Mechanistic 2200.0 nM (Ki)
CHEMBL2373397 ↓ inhibits Mechanistic 1900.0 nM (Ki)
CHEMBL2373341 ↓ inhibits Mechanistic 7700.0 nM (Ki)
CHEMBL2373349 ↓ inhibits Mechanistic 7160.0 nM (Ki)
CHEMBL2375178 ↓ inhibits Mechanistic 4200.0 nM (Ki)
CHEMBL2373347 ↓ inhibits Mechanistic 2600.0 nM (Ki)
CHEMBL2373555 ↓ inhibits Mechanistic 14000.0 nM (Ki)
CHEMBL2373348 ↓ inhibits Mechanistic 1400.0 nM (Ki)
CHEMBL2373401 ↓ inhibits Mechanistic 3940.0 nM (Ki)
CHEMBL2373475 ↓ inhibits Mechanistic 2100.0 nM (Ki)
CHEMBL2373402 ↓ inhibits Mechanistic 2400.0 nM (Ki)
CHEMBL2373345 ↓ inhibits Mechanistic 2700.0 nM (Ki)
CHEMBL2373344 ↓ inhibits Mechanistic 1500.0 nM (Ki)
CHEMBL2373405 ↓ inhibits Mechanistic 3000.0 nM (Ki)
CHEMBL130464 ↓ inhibits Mechanistic 570.0 nM (IC50)
CHEMBL132395 ↓ inhibits Mechanistic 3180.0 nM (IC50)
CHEMBL128019 ↓ inhibits Mechanistic 13700.0 nM (IC50)
CHEMBL339956 ↓ inhibits Mechanistic 3300.0 nM (IC50)
CHEMBL131192 ↓ inhibits Mechanistic 1290.0 nM (IC50)
CHEMBL429170 ↓ inhibits Mechanistic 200.0 nM (IC50)
CHEMBL1790570 ↓ inhibits Mechanistic 300.0 nM (IC50)
CHEMBL132057 ↓ inhibits Mechanistic 900.0 nM (IC50)
CHEMBL427751 ↓ inhibits Mechanistic 1450.0 nM (IC50)
CHEMBL77142 ↓ inhibits Mechanistic 340.0 nM (IC50)

Showing top 40 of 182 agents ranked by evidence tier.

Therapeutic Agent Effect Direction Evidence Tier Potency Source
SORBITOL ? unclear Mechanistic
DAMOCTOCOG ALFA PEGOL ? unclear Mechanistic
OMFILOCTOCOG ALFA ? unclear Mechanistic
EFANESOCTOCOG ALFA ? unclear Mechanistic
RECOMBINANT C5A ? unclear Mechanistic
TB-402 ? unclear Mechanistic
FACTOR VIII ? unclear Mechanistic
LONOCTOCOG ALFA ? unclear Mechanistic
MOROCTOCOG ALFA ? unclear Mechanistic
SIMOCTOCOG ALFA ? unclear Mechanistic
SUSOCTOCOG ALFA ? unclear Mechanistic
TUROCTOCOG ALFA PEGOL ? unclear Mechanistic
RANITIDINE HYDROCHLORIDE ? unclear Mechanistic
ANTIHEMOPHILIC FACTOR, PEGYLATED (MW 20000) HUMAN SEQUENCE RECOMBINANT ? unclear Mechanistic
EFMOROCTOCOG ALFA ? unclear Mechanistic
ANTIHEMOPHILIC FACTOR (RECOMBINANT) PEGYLATED-EXEI ? unclear Mechanistic
OCTOCOG ALFA ? unclear Mechanistic
THROMBIN ? unclear Mechanistic
GIROCTOCOGENE FITELPARVOVEC ? unclear Mechanistic
TUROCTOCOG ALFA ? unclear Mechanistic
DROTRECOGIN ALFA ↓ inhibits Mechanistic
RECOMBINANT FACTOR VIIA ? unclear Mechanistic
DESMOPRESSIN ? unclear Mechanistic
DROTRECOGIN ALFA (ACTIVATED) ? unclear Mechanistic
CHEMBL1614804 ↓ inhibits Mechanistic 7800.0 nM (IC50)
CHEMBL261865 ↓ inhibits Mechanistic 8900.0 nM (IC50)
HUMAN COAGULATION FACTOR VIII ? unclear Clinical
Therapeutic Agent Effect Direction Evidence Tier Potency Source
HYDROGEN PEROXIDE ? unclear Mechanistic
ALBUTREPENONACOG ALFA ? unclear Mechanistic
EMICIZUMAB ? unclear Mechanistic
COAGULATION FACTOR IX (RECOMBINANT), GLYCOPEGYLATED ? unclear Mechanistic
TTP889 ? unclear Mechanistic
VITAMIN K3 ? unclear Mechanistic
DEXAMETHASONE ? unclear Mechanistic
COAGULATION FACTOR IX RECOMBINANT HUMAN ? unclear Mechanistic
SOMATROPIN (RECOMBINANT DNA ORIGIN) ? unclear Mechanistic
NONACOG BETA PEGOL ? unclear Mechanistic
AZATHIOPRINE ? unclear Mechanistic
TRENONACOG ALFA ? unclear Mechanistic
NONACOG ALFA ? unclear Mechanistic
LEVOTHYROXINE SODIUM ? unclear Mechanistic
ANTITHROMBIN III ? unclear Mechanistic
PROTHROMBIN COMPLEX CONCENTRATE ? unclear Mechanistic
VALOCTOCOGENE ROXAPARVOVEC ? unclear Mechanistic
ETRANACOGENE DEZAPARVOVEC ? unclear Mechanistic
COMPOUND 57 [PMID: 20121197] ↓ inhibits Mechanistic
PEGNIVACOGIN ↓ inhibits Mechanistic
FACTOR VIII ? unclear Mechanistic
EFTRENONACOG ALFA ? unclear Mechanistic
FIDANACOGENE ELAPARVOVEC ? unclear Mechanistic
HYDROCORTISONE BUTYRATE ? unclear Mechanistic
COUMARIN ? unclear Mechanistic
COAGULATION FACTOR IX HUMAN ? unclear Mechanistic
DEXTRAN SULFATE SODIUM ? unclear Mechanistic
CHEMBL559015 ↓ inhibits Mechanistic 2200.0 nM (Ki)
CHEMBL554926 ↓ inhibits Mechanistic 3500.0 nM (Ki)
CHEMBL176140 ↓ inhibits Mechanistic 2200.0 nM (Ki)
RAZAXABAN HYDROCHLORIDE ↓ inhibits Mechanistic 9000.0 nM (Ki)
CHEMBL185330 ↓ inhibits Mechanistic 4900.0 nM (Ki)
CHEMBL184896 ↓ inhibits Mechanistic 1250.0 nM (Ki)
CHEMBL363310 ↓ inhibits Mechanistic 390.0 nM (Ki)
CHEMBL363110 ↓ inhibits Mechanistic 930.0 nM (Ki)
CHEMBL367575 ↓ inhibits Mechanistic 250.0 nM (Ki)
CHEMBL182736 ↓ inhibits Mechanistic 780.0 nM (Ki)
CHEMBL180207 ↓ inhibits Mechanistic 1730.0 nM (Ki)
CHEMBL362648 ↓ inhibits Mechanistic 960.0 nM (Ki)
CHEMBL182939 ↓ inhibits Mechanistic 580.0 nM (Ki)

Showing top 40 of 223 agents ranked by evidence tier.

Therapeutic Agent Effect Direction Evidence Tier Potency Source
PROGESTERONE ? unclear Mechanistic
FONDAPARINUX ↑ activates Mechanistic
PARNAPARIN SODIUM ↑ activates Mechanistic
HEPARIN CALCIUM ? unclear Mechanistic
LUSUTROMBOPAG ? unclear Mechanistic
SULODEXIDE ? unclear Mechanistic
ESTRADIOL VALERATE ? unclear Mechanistic
AVATROMBOPAG ? unclear Mechanistic
DOCIPARSTAT SODIUM ? unclear Mechanistic
DELPARANTAG ? unclear Mechanistic
DANAPAROID ↑ activates Mechanistic
GEMCITABINE ? unclear Mechanistic
2-O, 3-O DESULFATED HEPARIN ? unclear Mechanistic
ENOXAPARIN SODIUM ↑ activates Mechanistic
HEPARIN ↑ activates Mechanistic
BEMIPARIN SODIUM ↑ activates Mechanistic
SYNTHETIC CONJUGATED ESTROGENS, A ? unclear Mechanistic
SEMULOPARIN SODIUM ↓ inhibits Mechanistic
UNFRACTIONATED HEPARIN ? unclear Mechanistic
TINZAPARIN SODIUM ↑ activates Mechanistic
HEPARIN SODIUM ↑ activates Mechanistic
CARBOPLATIN ? unclear Mechanistic
NADROPARIN CALCIUM ↑ activates Mechanistic
ELTROMBOPAG ? unclear Mechanistic
ANTITHROMBIN ALFA ? unclear Mechanistic
FONDAPARINUX SODIUM ? unclear Mechanistic
DANAPAROID SODIUM ? unclear Mechanistic
CHEMBL609990 ↓ inhibits Mechanistic 58.0 nM (Kd)
CHEMBL1909451 ↓ inhibits Mechanistic 7.0 nM (Kd)
CHEMBL2303814 ↓ inhibits Mechanistic 2.8 nM (Kd)
CHEMBL2303815 ↓ inhibits Mechanistic 1.9 nM (Kd)
CHEMBL51173 ↓ inhibits Mechanistic 570.0 nM (IC50)
IDRAPARINUX SODIUM ↓ inhibits Mechanistic 1.0 nM (Kd)
DALTEPARIN SODIUM ? unclear Clinical
FITUSIRAN ? unclear Clinical
Therapeutic Agent Effect Direction Evidence Tier Potency Source
CONCIZUMAB ↓ inhibits Mechanistic
BAY-1093884 ↓ inhibits Mechanistic
ARC-19499 ? unclear Mechanistic
TIFACOGIN ? unclear Mechanistic
HEPARIN ? unclear Mechanistic
ATORVASTATIN CALCIUM TRIHYDRATE ? unclear Mechanistic
FENOFIBRATE MICRONIZED ? unclear Mechanistic
DACTINOMYCIN ? unclear Mechanistic
ANDEXANET ALFA ↓ inhibits Mechanistic
FULVESTRANT ? unclear Mechanistic
MARSTACIMAB ↓ inhibits Mechanistic
LOVASTATIN ? unclear Mechanistic
TINZAPARIN SODIUM ? unclear Mechanistic
CHONDROITIN SULFATES ? unclear Mechanistic
AZITHROMYCIN ? unclear Mechanistic
CREMOPHOR ? unclear Mechanistic
DEFIBROTIDE ? unclear Mechanistic
MUPARFOSTAT ? unclear Mechanistic
CHEMBL4448151 ↑ activates Mechanistic 190.0 nM (EC50)
CHEMBL4474279 ↑ activates Mechanistic 6400.0 nM (EC50)
CHEMBL4451495 ↑ activates Mechanistic 550.0 nM (EC50)
CHEMBL4436964 ↑ activates Mechanistic 300.0 nM (EC50)
CHEMBL4452958 ↑ activates Mechanistic 1020.0 nM (EC50)
CHEMBL4461957 ↑ activates Mechanistic 630.0 nM (EC50)
CHEMBL4471001 ↑ activates Mechanistic 610.0 nM (EC50)
CHEMBL4434798 ↑ activates Mechanistic 1060.0 nM (EC50)
CHEMBL4442960 ↑ activates Mechanistic 270.0 nM (EC50)
CHEMBL4445250 ↑ activates Mechanistic 150.0 nM (EC50)
CHEMBL4528590 ↑ activates Mechanistic 160.0 nM (EC50)
CHEMBL4530936 ↑ activates Mechanistic 370.0 nM (EC50)
CHEMBL4434931 ↑ activates Mechanistic 950.0 nM (EC50)
CHEMBL4437116 ↓ inhibits Mechanistic 5000.0 nM (Ki)
CHEMBL4466576 ↓ inhibits Mechanistic 5000.0 nM (Ki)
CHEMBL4457048 ↓ inhibits Mechanistic 10000.0 nM (IC50)
CHEMBL4466979 ↓ inhibits Mechanistic 10000.0 nM (IC50)
CHEMBL4461745 ↓ inhibits Mechanistic 5000.0 nM (IC50)
CHEMBL4456032 ↓ inhibits Mechanistic 5000.0 nM (IC50)
CHEMBL4453063 ↓ inhibits Mechanistic 10000.0 nM (IC50)
CHEMBL4463120 ↓ inhibits Mechanistic 8481.0 nM (IC50)
CHEMBL4474899 ↓ inhibits Mechanistic 12912.0 nM (IC50)

Showing top 40 of 213 agents ranked by evidence tier.

Therapeutic Agent Effect Direction Evidence Tier Potency Source
EGAPTIVON PEGOL ↓ inhibits Mechanistic
PHENYLEPHRINE HYDROCHLORIDE ? unclear Mechanistic
DA-697B ? unclear Mechanistic
HEPARIN ? unclear Mechanistic
THALIDOMIDE ? unclear Mechanistic
PENTOXIFYLLINE ? unclear Mechanistic
WARFARIN ? unclear Mechanistic
VINCRISTINE ? unclear Mechanistic
VONICOG ALFA ? unclear Mechanistic
CAPLACIZUMAB ↓ inhibits Mechanistic
STREPTOZOCIN ? unclear Mechanistic
O-CHLOROACETYLCARBAMOYLFUMAGILLOL ? unclear Mechanistic
RIBAVIRIN ? unclear Mechanistic
VON WILLEBRAND FACTOR HUMAN ? unclear Mechanistic
METHYLENE BLUE ANHYDROUS ? unclear Mechanistic
TYROSINE KINASE INHIBITOR ? unclear Mechanistic
PREDNISONE ? unclear Mechanistic
MITOMYCIN ANTINEOPLASTIC ANTIBIOTIC ? unclear Mechanistic
VITAMIN B6 ? unclear Mechanistic
ACETYLCYSTEINE ? unclear Mechanistic
FACTOR VIII ? unclear Mechanistic

Clinical Trials

Drug, biologic and supplement trials (treatment or prevention) from ClinicalTrials.gov whose main condition is Haemophilia A / B: recruiting, active or completed, most recently updated first. Data retrieved 2026-10-28.

NCT05145127 Phase: 3 Started: 2021-11-17

Study B7841007 is an open-label extension study to assess the long-term safety, tolerability, and efficacy of prophylaxis treatment with marstacimab in participants who did not require "Early Termination" from the Phase 3 Study B7841005 and from the Phase 3 Study B7841008. Study B7841005: approximat...

NCT05611801 Phase: 3 Started: 2022-12-09

The purpose of this clinical trial is to learn about the safety and effects of the study medicine (called marstacimab) for the potential treatment of hemophilia in pediatric patients. This study will enroll pediatric participants from ages 1 to 17 years in a sequential manner. The study opened the e...

NCT03588299 Phase: 1/2 Started: 2018-11-07

In this study researchers want to gather more information about safety and effectiveness of BAY 2599023 (DTX201), a drug therapy that delivers the human factor VIII gene into the human body by use of a viral vector to treat the disease. By replacing the defective gene with a healthy copy the human b...

NCT05135559 Phase: 3 Started: 2022-03-24

This study will test how well a new medicine called concizumab works for participants who have haemophilia A or B with or without inhibitors. The purpose is to show that concizumab can prevent bleeds and is safe to use. Participants will have to inject the study medicine every day under the skin wit...

NCT04082429 Phase: 3 Started: 2019-11-13

This study will test how well a new medicine called concizumab works in the body of people with haemophilia A or B without inhibitors. The purpose is to show that concizumab can prevent bleeds in the body and is safe to use. Participants who usually only take medicine to treat bleeds (on-demand) wil...

NCT04083781 Phase: 3 Started: 2019-10-21

This study will test how well a new medicine called concizumab works in the body of people with haemophilia A or B with inhibitors. The purpose is to show that concizumab can prevent bleeds in the body and is safe to use. Participants who usually only take medicine to treat bleeds (on-demand) will b...

NCT04563520 Phase: 3 Started: 2026-09

The purpose of the aPCC-emicizumab safety study is to investigate the hemostatic efficacy as measured by thrombin generation, of a low personalized dose of aPCC (FEIBA) in children and adults with hemophilia A and inhibitors on emicizumab prophylaxis.

NCT03938792 Phase: 3 Started: 2020-03-09

Treatment with PF-06741086 is anticipated to demonstrate a clinically relevant advantage and/or a major contribution to patient care in comparison to current methods of treatment for hemophilia A or B because it works differently than factor replacement products and will work in the presence of inhi...

NCT07644832 Phase: 1/2 Started: 2024-05-31

The purpose of this study is to evaluate the safety, tolerability, immunogenicity , PK, and PD of a single dose of SR604 in participants with Hemophilia A or Hemophilia B, with or without inhibitors (Part A)and to evaluate the safety, PK, PD, and efficacy of multiple doses of SR604 in participants w...

NCT07545395 Phase: 3 Started: 2026-03-13

The purposes of this open-label, multicenter III clinical trial are to evaluate the safety and efficacy of long-term preventive treatment with KN057 in Haemophilia A or B patients with or without inhibitors, and to assess the pharmacokinetic characteristics of the new and old processes KN057. The pa...

NCT04647227 Phase: 4 Started: 2021-06-28

Phase IV multi-center, US-centric, open-label, safety study enrolling participants with Hemophilia A or B with inhibitors, 12 years of age and older, who are either on long term prophylactic treatment (e.g., emicizumab) at risk of experiencing a breakthrough bleeding event (BE), or who are not on pr...

NCT06312475 Phase: 3 Started: 2024-01-09

The purpose of this study is to show that KN057 can prevent bleeds in patients with haemophilia A or B with inhibitors and is safe to use. Successfully screened participants will be randomly assigned to KN057 Prophylaxis (Arm 1) versus No Prophylaxis (Arm 2) at a ratio of 2:1. Participants in KN057 ...

NCT06569108 Phase: 3 Started: 2024-04-01

The purpose of this study is to show that KN057 can prevent bleeds in patients with haemophilia A or B without inhibitors and is safe to use. Participants receiving on-demand treatment prior to screening will be randomly assigned to Experimental group or Control group at a ratio of 2:1 in Part A. Pa...

NCT03370172 Phase: 1/2 Started: 2018-02-27

The main aim of this study is to check if there are side effects from BAX 888 and to determine the dose of BAX 888 for treating severe hemophilia A in male adults. Participants will receive one infusion with BAX 888 at the hemophilia treatment center. During the study, participants will visit their ...

NCT04073498 Phase: 1/2 Started: 2019-08-14

The purpose of this study is to investigate the safety and activity in the body of a new drug called SerpinPC. The study will be split into 7 parts: Part 1a will be conducted in healthy male volunteers in the UK (up to 15) and Parts 1b, 2, 3, 4, 5 and 6 will be conducted in haemophilia A \& B patien...


Agents Found By Disease-Level Search

Searched directly from “Haemophilia A / B” via ClinicalTrials.gov interventions and Open Targets' disease→drug data — independent of the 8-gene target panel above. This surfaces agents whose mechanism doesn't route through a curated gene (combination therapies, standard-of-care drugs, targets outside the panel).

56 agents shown (approved or with trial evidence) — 50 not found by the gene-target search above.

STSP-0601 for InjectionNOT in gene-target list

· 8 trials · source: ClinicalTrials.gov
NCT05619926 NCT04747964 NCT04747964

Emicizumab

Approved · 6 trials · source: ClinicalTrials.gov, Open Targets
NCT04023019 NCT04567511 NCT07416604

BAY Factor VII (BAY86-6150)NOT in gene-target list

· 4 trials · source: ClinicalTrials.gov
NCT01921855 NCT01921855 NCT01921855

Turoctocog alfa pegol

Approved · 3 trials · source: ClinicalTrials.gov, Open Targets
NCT03528551 NCT03528551 NCT03528551

NXT007NOT in gene-target list

· 3 trials · source: ClinicalTrials.gov
NCT07416604 NCT07416526 NCT05987449

activated recombinant human factor VIINOT in gene-target list

· 2 trials · source: ClinicalTrials.gov
NCT00571584 NCT00710619

efanesoctocog alfa (BIVV001)NOT in gene-target list

· 2 trials · source: ClinicalTrials.gov
NCT04161495 NCT04759131

Recombinant Protein-Free Factor VIII (rAHF-PFM)NOT in gene-target list

· 2 trials · source: ClinicalTrials.gov
NCT00357656 NCT00357656

ribavirin

· 2 trials · source: ClinicalTrials.gov
NCT00475072 NCT00055341

FRSW117NOT in gene-target list

· 2 trials · source: ClinicalTrials.gov
NCT04864743 NCT05265286

Human cl rhFVIIINOT in gene-target list

· 2 trials · source: ClinicalTrials.gov
NCT01712438 NCT00989196

Damoctocog alfa pegol (Jivi, BAY94-9027)NOT in gene-target list

· 2 trials · source: ClinicalTrials.gov
NCT05643560 NCT03932201

BAY1093884

Phase 2 · 2 trials · source: ClinicalTrials.gov, Open Targets
NCT02571569 NCT02571569

rFVIIIFcNOT in gene-target list

· 2 trials · source: ClinicalTrials.gov
NCT02502149 NCT02083965

Recombinant Factor VIIa BI (rFVIIa BI)NOT in gene-target list

· 2 trials · source: ClinicalTrials.gov
NCT01757405 NCT01757405

dirloctocogene samoparvovec (SPK-8011)NOT in gene-target list

· 2 trials · source: ClinicalTrials.gov
NCT03432520 NCT03003533

Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated)NOT in gene-target list

· 2 trials · source: ClinicalTrials.gov
NCT00851721 NCT00851721

NuwiqNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT04023019

OctanateNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT04023019

WilateNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT04023019

Recombinant factor VIIa (rFVIIa)NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT04023019

Activated prothrombin complex concentrate (aPCC)NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT04023019

Feiba VHNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT00710619

Recombinant Factor VIII (Kogenate FS, BAY14-2222)NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT01810666

rIX-FPNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT01662531

FEIBA and NovoSevenNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT00166309

peginterferon alfa-2a [Pegasys]NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT00475072

MOD-5014NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT02418793

rFVIII-FS (Kogenate FS, BAY14-2222) 70 IU/kg, dosing once per weekNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT00632814

rFVIII-FS (Kogenate FS, BAY14-2222), 70 IU/kg twice per week (30 IU/kg + 40 IU/kg)NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT00632814

rFVIII-FS (Kogenate FS, BAY14-2222) 75 IU/kg, dosing three times per week (3 x 25 IU/kg)NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT00632814

fidanacogene elaparvovec (SPK-9001)NOT in gene-target list

Approved · 1 trial · source: ClinicalTrials.gov
NCT02484092

Prophylaxis with EmicizumabNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT05279924

ADVATENOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT04864743

Human Coagulation Factor VIII

Phase 3 · 1 trial · source: ClinicalTrials.gov, Open Targets
NCT07416526

ASC618NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT04676048

Power Doppler SonographyNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT00212485

Oktokog alphaNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT03915080

Octocog alfa (recombinant human coagulation factor VIII)NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT02170402

Fibrogammin PNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT00640289

SPK-8016NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT03432520

Alphanate SD/HTNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT00323856

EsperoctNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT05621746

PF-06838435/ fidanacogene elaparvovecNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT03861273

SR604NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06349473

Vitamin D and calciumNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT01090206

SOFNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT01260350

RBVNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT01260350

PEGNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT01260350

LDVNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT01260350

radalbuvir (GS-9669)NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT01260350

LDV/SOFNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT01260350

custom-molded insoles and custom-molded shoesNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT00638001

TAK-672NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT04580407

NovoSevenNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT00375323

eptacog alfa (activated)

Approved · 1 trial · source: ClinicalTrials.gov, Open Targets
NCT01579968

Generated by the Open Source Medicine Foundation Biomarker Pipeline — 2026-10-08. Data sources: DGIdb, Open Targets, ChEMBL, PubMed, ClinicalTrials.gov. Not medical advice.