hemophilia B

Summary

hemophilia B (MONDO:0010604) is a disease caused by F9 (GenCC Strong), with 3 cohort genes and 170 clinical trials. Top therapeutic interventions include coagulation factor ix recombinant human, eftrenonacog alfa, and etranacogene dezaparvovec.

At a glance

• Prevalence: 1-9 / 100 000 (Europe) [Orphanet-validated]
• Causal gene: F9 (GenCC Strong)
• Cohort genes: 3
• ClinVar variants: 635
• Phenotypes (HPO): 14
• Clinical trials: 170

Clinical features

Epidemiology

Prevalence records

101 prevalence record(s), Orphanet, top 20 (validated / broadest geography first):

Type	Class	Value	Geography	Validation
Prevalence at birth	1-9 / 100 000	1.665	Worldwide	Validated
Point prevalence	1-9 / 100 000	3	Europe	Validated
Point prevalence	1-9 / 100 000	1	Albania	Validated
Point prevalence	1-9 / 1 000 000	0.44	Algeria	Validated
Point prevalence	1-9 / 1 000 000	0.54	Argentina	Validated
Point prevalence	1-9 / 1 000 000	0.2	Armenia	Validated
Point prevalence	1-9 / 1 000 000	0.665	Austria	Validated
Point prevalence	1-9 / 1 000 000	0.16	Azerbaijan	Validated
Point prevalence	<1 / 1 000 000	0.015	Bangladesh	Validated
Point prevalence	1-9 / 1 000 000	0.84	Belarus	Validated
Point prevalence	1-9 / 100 000	1.32	Belgium	Validated
Point prevalence	1-9 / 100 000	1.155	Belize	Validated
Point prevalence	<1 / 1 000 000	0.045	Bolivia	Validated
Point prevalence	1-9 / 1 000 000	0.265	Bosnia and Herzegovina	Validated
Point prevalence	1-9 / 1 000 000	0.535	Brazil	Validated
Point prevalence	1-9 / 1 000 000	0.775	Bulgaria	Validated
Point prevalence	1-9 / 1 000 000	0.795	Chile	Validated
Point prevalence	1-9 / 1 000 000	0.405	Colombia	Validated
Point prevalence	1-9 / 1 000 000	0.645	Costa rica	Validated
Point prevalence	1-9 / 100 000	1.625	Croatia	Validated

Signs & symptoms

Clinical features (HPO)

14 HPO clinical features (Orphanet curated; top 14 by frequency):

HPO ID	Term	Frequency
HP:0000790	Hematuria	Very frequent (80-99%)
HP:0001058	Poor wound healing	Very frequent (80-99%)
HP:0002170	Intracranial hemorrhage	Very frequent (80-99%)
HP:0003010	Prolonged bleeding time	Very frequent (80-99%)
HP:0003645	Prolonged partial thromboplastin time	Very frequent (80-99%)
HP:0004406	Spontaneous, recurrent epistaxis	Very frequent (80-99%)
HP:0004846	Prolonged bleeding after surgery	Very frequent (80-99%)
HP:0005261	Joint hemorrhage	Very frequent (80-99%)
HP:0006298	Prolonged bleeding after dental extraction	Very frequent (80-99%)
HP:0011858	Reduced factor IX activity	Very frequent (80-99%)
HP:0012233	Intramuscular hematoma	Very frequent (80-99%)
HP:0012541	Cephalohematoma	Very frequent (80-99%)
HP:0040232	Delayed onset bleeding	Very frequent (80-99%)
HP:0400008	Menometrorrhagia	Very frequent (80-99%)

Identifiers

Disease identifiers

Field	Value
Canonical name	hemophilia B
Mondo ID	MONDO:0010604
MeSH	D002836
OMIM	306900
Orphanet	98879
DOID	DOID:12259
ICD-10-CM	D67
ICD-11	1901375668
NCIT	C26721
SNOMED CT	41788008
UMLS	C0008533
MedGen	945
GARD	0008732
MedDRA	10016077
NORD	1222
Is cancer (heuristic)	no

Also known as: Christmas disease · congenital factor IX deficiency · congenital factor IX disorder · factor IX deficiency · haemophilia B Leyden · haemophilia B(M) · haemophilia b, X-linked recessive · haemophilia type B · hem B · HEMB · hemophilia B · hemophilia b, X-linked recessive · hemophilia type B · hereditary Factor IX deficiency · hereditary Factor IX deficiency disease

Data availability: 635 ClinVar variants · 71 ClinGen variant curations · 2 GenCC gene-disease records · 6 cell lines.

Disease family

An umbrella term covering 4 Mondo subtypes.

Classification path: disease › human disease › disease by body system or component › hematologic disorder › hemorrhagic disease › hemophilia B

Related subtypes (27): inherited bleeding disorder, platelet-type, factor VII deficiency, factor X deficiency, purpura, vascular hemostatic disease, congenital factor V deficiency, congenital high-molecular-weight kininogen deficiency, congenital factor XII deficiency, alpha-2-plasmin inhibitor deficiency, hemophilia A, East Texas bleeding disorder, congenital factor XI deficiency, inherited prekallikrein deficiency, congenital plasminogen activator inhibitor type 1 deficiency, thrombomodulin-related bleeding disorder, congenital vitamin K-dependent coagulation factors deficiency, hemorrhagic disease due to alpha-1-antitrypsin Pittsburgh mutation, multiple sclerosis-ichthyosis-factor VIII deficiency syndrome, congenital factor XIII deficiency, congenital fibrinogen deficiency, combined deficiency of factor V and factor VIII, acquired hemophilia, fetal and neonatal alloimmune thrombocytopenia, hereditary von Willebrand disease, acquired von willebrand syndrome, prothrombin deficiency, hemophilia B leyden

Subtypes (4): severe hemophilia B, moderately severe hemophilia B, mild hemophilia B, symptomatic form of hemophilia B in female carriers

Genetics & variants

GWAS landscape

No GWAS associations recorded — common-variant (GWAS) studies don’t cover this disease (typical for Mendelian / rare diseases). See the curated gene cohort and Mendelian overlap below.

Variant details and genetic-evidence tiers

ClinVar germline variants

600 retrieved; paginated sample, class counts are floors:

186 pathogenic, 173 likely benign, 88 likely pathogenic, 67 uncertain significance, 36 benign, 29 pathogenic/likely pathogenic, 14 conflicting classifications of pathogenicity, 6 benign/likely benign, 1 benign; association

ClinVar	Variant (HGVS)	Gene	Classification	Review
665638	NC_000023.10:g.(?138612860)(139587225_?)del	ATP11C	Pathogenic	criteria provided, single submitter
10098	NM_000132.4(F8):c.6683G>A (p.Arg2228Gln)	F8	Pathogenic	criteria provided, multiple submitters, no conflicts
10111	NM_000132.4(F8):c.1172G>A (p.Arg391His)	F8	Pathogenic	criteria provided, multiple submitters, no conflicts
10126	NM_000132.4(F8):c.6977G>A (p.Arg2326Gln)	F8	Pathogenic/Likely pathogenic	criteria provided, multiple submitters, no conflicts
10177	NM_000132.4(F8):c.541G>A (p.Val181Met)	F8	Pathogenic	criteria provided, multiple submitters, no conflicts
10196	NM_000132.4(F8):c.935T>C (p.Phe312Ser)	F8	Pathogenic/Likely pathogenic	criteria provided, multiple submitters, no conflicts
10217	NM_000132.4(F8):c.1492G>A (p.Gly498Arg)	F8	Pathogenic	criteria provided, multiple submitters, no conflicts
10222	NM_000132.4(F8):c.1636C>T (p.Arg546Trp)	F8	Pathogenic/Likely pathogenic	criteria provided, multiple submitters, no conflicts
10232	NM_000132.4(F8):c.1804C>T (p.Arg602Ter)	F8	Pathogenic	reviewed by expert panel
10236	NM_000132.4(F8):c.1834C>T (p.Arg612Cys)	F8	Pathogenic	reviewed by expert panel
10245	NM_000132.4(F8):c.2149C>T (p.Arg717Trp)	F8	Pathogenic/Likely pathogenic	criteria provided, multiple submitters, no conflicts
10246	NM_000132.4(F8):c.2167G>A (p.Ala723Thr)	F8	Pathogenic	criteria provided, multiple submitters, no conflicts
10253	NM_000132.4(F8):c.3637del (p.Ile1213fs)	F8	Pathogenic	reviewed by expert panel
10319	NM_000132.4(F8):c.6533G>A (p.Arg2178His)	F8	Pathogenic/Likely pathogenic	criteria provided, multiple submitters, no conflicts
10327	NM_000132.4(F8):c.6744G>T (p.Trp2248Cys)	F8	Pathogenic	reviewed by expert panel
10332	NM_000132.4(F8):c.6956C>T (p.Pro2319Leu)	F8	Pathogenic	criteria provided, multiple submitters, no conflicts
439683	NM_000132.4(F8):c.6089G>A (p.Ser2030Asn)	F8	Pathogenic/Likely pathogenic	criteria provided, multiple submitters, no conflicts
618098	NM_000132.4(F8):c.5954G>A (p.Arg1985Gln)	F8	Pathogenic/Likely pathogenic	criteria provided, multiple submitters, no conflicts
618104	NM_000132.4(F8):c.1094A>G (p.Tyr365Cys)	F8	Pathogenic	reviewed by expert panel
626933	NM_000132.4(F8):c.6547A>G (p.Met2183Val)	F8	Pathogenic/Likely pathogenic	criteria provided, multiple submitters, no conflicts
626935	NM_000132.4(F8):c.6686T>C (p.Leu2229Pro)	F8	Pathogenic/Likely pathogenic	criteria provided, multiple submitters, no conflicts
626992	NM_000132.4(F8):c.1364T>G (p.Phe455Cys)	F8	Pathogenic	criteria provided, single submitter
627033	NM_000132.4(F8):c.2043G>C (p.Met681Ile)	F8	Pathogenic	criteria provided, single submitter
627109	NM_000132.4(F8):c.6434T>G (p.Phe2145Cys)	F8	Pathogenic	criteria provided, single submitter
627121	NM_000132.4(F8):c.6920A>C (p.Asp2307Ala)	F8	Pathogenic/Likely pathogenic	criteria provided, multiple submitters, no conflicts
627165	NM_000132.4(F8):c.979C>G (p.Leu327Val)	F8	Pathogenic	reviewed by expert panel
627191	NM_000132.4(F8):c.1020A>C (p.Glu340Asp)	F8	Pathogenic	criteria provided, single submitter
627254	NM_000132.4(F8):c.1621A>T (p.Thr541Ser)	F8	Pathogenic/Likely pathogenic	criteria provided, multiple submitters, no conflicts
627259	NM_000132.4(F8):c.1804C>G (p.Arg602Gly)	F8	Pathogenic/Likely pathogenic	criteria provided, multiple submitters, no conflicts
627340	NM_000132.4(F8):c.5918A>T (p.His1973Leu)	F8	Pathogenic	criteria provided, single submitter

Genes & proteins

Mendelian disease overlap and somatic drivers

GenCC: 7 · Orphanet: 8 · OMIM-shared: 0 · Dual-evidence (GWAS+Mendelian): 0

GenCC gene–disease validity (cohort genes)

the Disease column is the GenCC-asserted condition — a cohort gene’s strongest validity may be for a related predisposition syndrome.

Gene	Classification	Inheritance	Disease	Records
F9	Strong	X-linked	hemophilia B	7

Orphanet rare-disease linkage (cohort genes)

Gene	Orphanet ID	Rare disease
F9	Orphanet:169793	Severe hemophilia B
F9	Orphanet:169796	Moderate hemophilia B
F9	Orphanet:169799	Mild hemophilia B
F9	Orphanet:177929	Bleeding disorder in hemophilia B carriers
F8	Orphanet:169802	Severe hemophilia A
F8	Orphanet:169805	Moderate hemophilia A
F8	Orphanet:169808	Mild hemophilia A
F8	Orphanet:177926	Bleeding disorder in hemophilia A carriers

Cohort genes → proteins

3 cohort genes, 3 distinct canonical proteins.

Evidence partition

Subset	Genes
multi_evidence	3

Cohort genes (full)

Symbol	HGNC	Ensembl	UniProt	Name	Evidence
F9	HGNC:3551	ENSG00000101981	P00740	Coagulation factor IX	gencc,clinvar
ATP11C	HGNC:13554	ENSG00000101974	Q8NB49	Phospholipid-transporting ATPase IG	clinvar
F8	HGNC:3546	ENSG00000185010	P00451	Coagulation factor VIII	clinvar

Cohort function summary

Lead sentence per gene, UniProt-curated.

Symbol	Protein name	Function (lead sentence)
F9	Coagulation factor IX	Factor IX is a vitamin K-dependent plasma protein that participates in the intrinsic pathway of blood coagulation by converting factor X to its active form in the presence of Ca(2+) ions, phospholipids, and factor VIIIa.
ATP11C	Phospholipid-transporting ATPase IG	Catalytic component of a P4-ATPase flippase complex which catalyzes the hydrolysis of ATP coupled to the transport of aminophospholipids, phosphatidylserines (PS) and phosphatidylethanolamines (PE), from the outer to the inner leaflet of t…
F8	Coagulation factor VIII	Factor VIII, along with calcium and phospholipid, acts as a cofactor for F9/factor IXa when it converts F10/factor X to the activated form, factor Xa.

Protein-family classification

Druggable: 1 · Difficult: 1 · Unknown: 1 · Druggable fraction: 0.33

Family distribution

Cohort families vs a genome-wide background (hypergeometric, BH-FDR; fold = observed/expected). Counts kept; sorted by enrichment, so the catch-all Other/Unknown bucket no longer leads.

Family	Genes	Fold	FDR
Protease	1	12.2×	0.239
Transcription factor	1	2.8×	0.482
Other/Unknown	1	0.6×	0.914

Per-gene assignment

Symbol	Family	Druggable?	EC	InterPro (top 3)
F9	Protease	yes	3.4.21.22	EGF-type_Asp/Asn_hydroxyl_site, GLA_domain, EGF
ATP11C	Transcription factor	no	7.6.2.1	P_typ_ATPase, P-type_ATPase_IV, ATPase_P-typ_transduc_dom_A_sf
F8	Other/Unknown	no		FA58C, Cupredoxin, Galactose-bd-like_sf

Expression context

Cohort genes with no expression data: 0.

3 cohort genes are a single-cell marker in ≥1 SCXA experiment.

Breadth distribution (Bgee present_calls)

Bucket	Genes
narrow (1-5 tissues)	0
moderate (6-20)	0
broad (>20)	3
unknown	0

Top tissues across cohort

Tissue	Cohort genes
liver	1
right lobe of liver	1
secondary oocyte	1
calcaneal tendon	1
male germ line stem cell (sensu Vertebrata) in testis	1
seminal vesicle	1
heart right ventricle	1
left ventricle myocardium	1
myocardium	1

Per-gene tissue summary (top 30)

Symbol	Bgee breadth	FANTOM5 breadth	SCXA	Top tissues
F9	45	tissue_specific	marker	right lobe of liver, liver, secondary oocyte
ATP11C	250	ubiquitous	marker	seminal vesicle, calcaneal tendon, male germ line stem cell (sensu Vertebrata) in testis
F8	266	broad	marker	left ventricle myocardium, heart right ventricle, myocardium

Protein interactions among cohort

Intra-cohort edges: 2.

Hub genes (top 10 by interactor count)

Symbol	Interactor count
F8	1,900
ATP11C	1,740
F9	1,451

Intra-cohort edges

A	B	Sources
ATP11C	F9	string_interaction
F8	F9	intact, string_interaction

Structural data

PDB: 3 · AlphaFold-only: 0 · No structure: 0

Cohort genes with PDB structures (top 30)

Symbol	UniProt	PDB entries
F9	P00740	56
F8	P00451	25
ATP11C	Q8NB49	13

Function

Pathway analysis

Distinct Reactome pathways touched by cohort: 27. Enrichment computed across 3 evidence-associated genes (3 with Reactome annotation).

Pathways by enrichment

Over-representation of cohort genes vs the genome-wide background (hypergeometric test, Benjamini-Hochberg FDR; fold = observed/expected over 3 annotated cohort genes). Counts and members are kept as ground-truth; sorted by enrichment.

Pathway	Cohort genes	Fold	FDR	Sample cohort genes
Defective factor IX causes thrombophilia	2	2537.8×	1e-06	F9, F8
Defective cofactor function of FVIIIa variant	2	2537.8×	1e-06	F9, F8
Defective F9 variant does not activate FX	2	2537.8×	1e-06	F9, F8
Amplification and propagation of coagulation cascade	2	423.0×	5e-05	F9, F8
Initiation of coagulation cascade	2	317.2×	7e-05	F9, F8
Regulation of clotting cascade	2	155.4×	2e-04	F9, F8
Defective F8 accelerates dissociation of the A2 domain	1	3806.7×	7e-04	F8
Defective F8 binding to the cell membrane	1	3806.7×	7e-04	F8
Defective F8 secretion	1	3806.7×	7e-04	F8
Defective F9 secretion	1	3806.7×	7e-04	F9
Defective F8 binding to von Willebrand factor	1	1903.3×	0.001	F8
Defective gamma-carboxylation of F9	1	1903.3×	0.001	F9
Defective F8 cleavage by thrombin	1	1268.9×	0.002	F8
Defective F8 sulfation at Y1699	1	1268.9×	0.002	F8
Defective F9 activation	1	634.4×	0.003	F9
Transport of gamma-carboxylated protein precursors from the endoplasmic reticulum to the Golgi apparatus	1	423.0×	0.004	F9
Gamma-carboxylation of protein precursors	1	380.7×	0.004	F9
Removal of aminoterminal propeptides from gamma-carboxylated proteins	1	380.7×	0.004	F9
FXIIa, PKa-dependent activation of coagulation pathway	1	380.7×	0.004	F9
Protein hydroxylation	1	181.3×	0.007	F9
Gamma carboxylation, hypusinylation, hydroxylation, and arylsulfatase activation	1	141.0×	0.009	F8
Cargo concentration in the ER	1	112.0×	0.011	F8
Ion transport by P-type ATPases	1	69.2×	0.017	ATP11C
COPII-mediated vesicle transport	1	54.4×	0.021	F8
Ion channel transport	1	32.0×	0.033	ATP11C
Platelet degranulation	1	29.3×	0.035	F8
Transport of small molecules	1	8.4×	0.115	ATP11C

GO biological processes by enrichment

Over-representation of cohort genes vs the genome-wide background (hypergeometric test, Benjamini-Hochberg FDR; fold = observed/expected over 3 annotated cohort genes). Counts and members are kept as ground-truth; sorted by enrichment.

GO term	Cohort genes	Fold	FDR	Sample cohort genes
blood coagulation	2	115.8×	7e-04	F9, F8
blood coagulation, intrinsic pathway	1	702.2×	0.005	F8
zymogen activation	1	224.7×	0.008	F9
phospholipid translocation	1	208.1×	0.008	ATP11C
acute-phase response	1	140.4×	0.010	F8
monoatomic ion transmembrane transport	1	69.3×	0.017	ATP11C
proteolysis	1	11.4×	0.085	F9

Therapeutics

Drugs indicated for this disease

10 approved, 4 in late-stage (phase 3) trials. Disease-direct ChEMBL indications, not inferred from the associated-gene cohort below.

Drug	Development status
Albutrepenonacog Alfa	Approved (phase 4)
Coagulation Factor Ix Human	Approved (phase 4)
Coagulation Factor Ix Recombinant Human	Approved (phase 4)
Eftrenonacog Alfa	Approved (phase 4)
Eptacog Alfa (Activated)	Approved (phase 4)
Eptacog Beta (Activated)	Approved (phase 4)
Etranacogene Dezaparvovec	Approved (phase 4)
Fidanacogene Elaparvovec	Approved (phase 4)
Marstacimab	Approved (phase 4)
Nonacog Beta Pegol	Approved (phase 4)
Anti-Inhibitor Coagulant Complex	Phase 3 (in late-stage trials)
Concizumab	Phase 3 (in late-stage trials)
Fitusiran	Phase 3 (in late-stage trials)
Trenonacog Alfa	Phase 3 (in late-stage trials)

Earlier-phase candidates (phase 2, investigational — efficacy not yet established): Ataluren, Vatreptacog Alfa (Activated).

Drug target analysis

Approved (phase 4): 0 · Phase ≥3: 0 · Phased (≥1): 1 · Undrugged: 2

Druggability breadth: 2 of 3 evidence-associated genes (67%) have a ChEMBL target (buckets above are over the deeply-mined display cohort).

Top cohort targets by molecule count

Symbol	Molecules	Max phase
F9	2	2
ATP11C	0	0
F8	0	0

Drugs targeting cohort genes (top 30)

Molecule	Max phase	Targets in cohort
LETAXABAN	2	F9
RAZAXABAN	2	F9

Bioactivity and enzyme data

Enzyme cohort genes (≥1 EC): 2.

Cohort genes with ChEMBL bioactivity (full, sorted by assay count)

Symbol	Assays	Type breakdown
F9	108	Binding:107, ADMET:1
F8	8	Binding:8

Cohort enzymes (BRENDA EC)

Symbol	EC numbers	Names
F9	3.4.21.22	coagulation factor IXa
ATP11C	7.6.2.1	P-type phospholipid transporter

Cohort genes with high screening signal

≥100 ChEMBL assays — a studied-ness signal; see Therapeutics for approved-drug status.

Symbol	ChEMBL assays
F9	108

Pharmacogenomics

Cohort genes with a PharmGKB record: 3; with CPIC/DPWG dosing guidelines: 0.

No cohort gene has a CPIC/DPWG genotype-guided dosing guideline (PharmGKB).

Chemical tractability of cohort targets

2 approved/phased compounds have measured bioactivity against a cohort gene (and aren’t yet in disease-level trials). This is a research / tractability signal, NOT a therapeutic recommendation — a bioactivity row often reflects off-target or screening binding (e.g. promiscuous kinase inhibitors against a cohort kinase), implying no disease mechanism.

Compound	Max phase	Cohort target (bioactivity)
LETAXABAN	2	F9
RAZAXABAN	2	F9

Druggability pyramid

Cohort genes binned by druggability tier (high → low):

Tier	Definition	Genes	Symbols
A	Approved (phase 4 drug)	0
B	Phased (≥1) drug, not yet approved	1	F9
C	Druggable family + PDB, no drug	0
D	Druggable family + AlphaFold only, no drug	0
E	Difficult family or no structure, no drug	2	ATP11C, F8

Undrugged target profiles

2 cohort genes are undrugged. Ranked by ‘starting-point quality’ (assay depth + drugged-partner adjacency).

Symbol	ChEMBL assays	Drugged partners (top 3)
ATP11C	0	F9
F8	8	F9

Clinical trials & evidence

Clinical trials

Clinical trials: 170.

Phase distribution (across all retrieved trials)

Phase	Trials
Not specified	82
PHASE3	33
PHASE1/PHASE2	17
PHASE1	16
PHASE4	9
PHASE2	8
PHASE2/PHASE3	4
EARLY_PHASE1	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT00139828	PHASE4	COMPLETED	Post Marketing Study in Haemophilia B Patients Using Nonafact® (Human Coagulation Factor IX)
NCT00581126	PHASE4	COMPLETED	Study Evaluating BENEFIX in Previously Treated Patients With Hemophilia B
NCT00749476	PHASE4	COMPLETED	Study Evaluating BeneFIX in Patients With Haemophilia B, Previously Treated With Plasma Derived Factor IX
NCT01128881	PHASE4	COMPLETED	IMMUNINE Pre-Treatment Study
NCT01748201	PHASE4	COMPLETED	Viscosupplementation in Patients With Hemophilic Arthropathy
NCT02336178	PHASE4	COMPLETED	Safety and Efficacy of Benefix in Patients With Hemophilia B in Usual Care Settings in China
NCT03565237	PHASE4	COMPLETED	RIXUBIS PMS India (RIXUBIS PMS)
NCT04286412	PHASE4	COMPLETED	Nonacog Alfa Prophylaxis And Treatment Of Bleeding Episodes In Previously Treated Patients With Hemophilia B
NCT05856266	PHASE4	TERMINATED	An 18-month Low-interventional Study to Assess Joint Health in Haemophilia A and B Patients on Prophylaxis With Efmoroctocog Alfa or Eftrenonacog Alfa
NCT03861273	PHASE3	ACTIVE_NOT_RECRUITING	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
NCT05145127	PHASE3	RECRUITING	Open-Label Extension Study of Marstacimab in Hemophilia Participants With or Without Inhibitors
NCT05203679	PHASE2/PHASE3	ACTIVE_NOT_RECRUITING	Evaluation of the Safety and Efficacy of Hemophilia B Gene Therapy Drug
NCT05568719	PHASE3	RECRUITING	Safety and Effectiveness of Giroctocogene Fitelparvovec or Fidanacogene Elaparvovec in Patients With Hemophilia A or B Respectively
NCT05611801	PHASE3	RECRUITING	A Clinical Trial of Study Medicine (Marstacimab) in Pediatric Patients With Hemophilia A or Hemophilia B
NCT06003387	PHASE3	RECRUITING	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)
NCT06399289	PHASE3	ACTIVE_NOT_RECRUITING	Recombinant Fusion Protein Linking Coagulation Factor IX With Albumin (rIX-FP) in Chinese Subjects With Hemophilia B Previously Treated With FIX Therapy
NCT06700096	PHASE3	RECRUITING	An Open-Label, Comparative Study of the Efficacy, Safety and Pharmacodynamics of Single Dose of ANB-002 in Patients With Hemophilia B
NCT07080905	PHASE3	RECRUITING	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
NCT00037557	PHASE3	COMPLETED	Study Evaluating rFIX; BeneFIX in Severe Hemophilia B
NCT00093171	PHASE3	COMPLETED	Study Evaluating rFIX; BeneFIX® in Hemophilia B
NCT00093210	PHASE3	COMPLETED	Study Evaluating of Recombinant Human Factor IX (BeneFIX) and a New Formulation of BeneFIX (rFIX-R) in Moderate to Severe Hemophilia B
NCT00364182	PHASE3	COMPLETED	Study Comparing On-Demand Treatment With Two Prophylaxis Regimens Of BeneFIX In Patients With Severe Hemophilia B
NCT00768287	PHASE2/PHASE3	COMPLETED	Study of Recombinant Factor IX Product, IB1001, in Subjects With Hemophilia B
NCT00851721	PHASE3	COMPLETED	Efficacy and Safety Study of Prophylactic Versus On-Demand Treatment With Feiba NF in Subjects With Hemophilia A or B and a High Titer Inhibitor
NCT00866606	PHASE3	COMPLETED	Study Evaluating On-Demand Treatment With BeneFIX In Chinese Subjects
NCT01174446	PHASE3	COMPLETED	Pivotal Study (Pharmacokinetics, Efficacy, Safety) of BAX 326 (rFIX) in Hemophilia B Patients
NCT01271868	PHASE3	TERMINATED	Study of Recombinant Factor IX Product, IB1001, in Previously Treated Pediatric Subjects With Hemophilia B
NCT01286779	PHASE3	COMPLETED	BAX 326 (rFIX) Continuation Study
NCT01335061	PHASE3	COMPLETED	Study To Compare On-Demand Treatment To A Prophylaxis Regimen Of BeneFIX In Subjects With Moderately Severe to Severe Hemophilia B
NCT01440946	PHASE3	COMPLETED	Study of Recombinant Coagulation Factor IX Fc Fusion Protein, BIIB029, in Previously Treated Pediatric Participants With Hemophilia B
NCT01488994	PHASE2/PHASE3	COMPLETED	BAX 326 Pediatric Study
NCT01496274	PHASE2/PHASE3	COMPLETED	A Safety and Efficacy Study of a Recombinant Fusion Protein Linking Coagulation Factor IX With Albumin (rIX-FP) in Patients With Hemophilia B
NCT01507896	PHASE3	COMPLETED	BAX 326 Surgery Study in Hemophilia B Patients
NCT01662531	PHASE3	COMPLETED	A Safety, Efficacy and Pharmacokinetics Study of a Recombinant Fusion Protein Linking Coagulation Factor IX With Albumin (rIX-FP) in Children With Hemophilia B
NCT01757405	PHASE3	COMPLETED	Recombinant Factor VIIa BI (rFVIIa BI) Treatment of Acute Bleeding Episodes Per an On-demand Regimen
NCT02048111	PHASE3	WITHDRAWN	Study of Recombinant Factor IX Product, IB1001, in Previously Treated Subjects With Hemophilia B
NCT02053792	PHASE3	COMPLETED	A Safety and Efficacy Extension Study of a Recombinant Fusion Protein Linking Coagulation Factor IX With Albumin (rIX-FP) in Patients With Hemophilia B
NCT02234310	PHASE3	COMPLETED	Study to Determine the Safety and Efficacy of rFIXFc in Previously Untreated Males With Severe Hemophilia B
NCT03417102	PHASE3	COMPLETED	A Study of Fitusiran (ALN-AT3SC) in Severe Hemophilia A and B Patients With Inhibitors
NCT03417245	PHASE3	COMPLETED	A Study of Fitusiran (ALN-AT3SC) in Severe Hemophilia A and B Patients Without Inhibitors

Drugs tested across these trials (top 30)

Molecule	Max phase	Trials referencing
COAGULATION FACTOR IX RECOMBINANT HUMAN	4	14
EFTRENONACOG ALFA	4	5
ETRANACOGENE DEZAPARVOVEC	4	4
LEVACETYLLEUCINE	4	3
MARSTACIMAB	4	3
FIDANACOGENE ELAPARVOVEC	4	2
ATALUREN	4	1
COAGULATION FACTOR IX HUMAN	4	1
SODIUM CHLORIDE	4	1
FITUSIRAN	3	4
TRENONACOG ALFA	3	3
BAY-1093884	2	1
VERBRINACOGENE SETPARVOVEC	1	3