Disease · MONDO_0010602

hemophilia A

6 sourcesdiseases-2026-09-18-0.1.0

Cite this record

ConductScience Library. hemophilia A. Diseases, record MONDO_0010602. Release diseases-2026-09-18-0.1.0, 18 Sept 2026. https://conductscience.org/library/diseases/MONDO_0010602

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Disease

Names and IDs as Open Targets gives them. Its IDs in MeSH, Orphanet, the Disease Ontology and the NCI Thesaurus are those Open Targets states for it.

Name: hemophilia A; Open Targets
Open Targets ID: MONDO_0010602; Open Targets
Other names: Hemophilia A (disorder), Subhemophilia, congenital factor VIII disorder, factor VIII deficiency, haemophilia a, X-linked recessive, haemophilia type A, hemophilia a, X-linked recessive, hemophilia type A, hereditary Factor VIII deficiency, hereditary Factor VIII deficiency disease; Open Targets
Disease Ontology: DOID:12134; Disease Ontology
MeSH: D006467; MeSH
NCI Thesaurus: C27146; NCI Thesaurus
Orphanet: ORPHA:98878; Orphanet
Highest stage of its drugs: Approval, for 18 of its 42 drugs, from ClinicalTrials.gov, DailyMed, EMA Human Drugs, FDA, PMDA and TTD; Open Targets

Drugs42

The drugs Open Targets' clinical reports study for the disease, the highest stage first, then the most trial reports. The stage is Open Targets', the highest any of its sources reports for the pair, and each row names the sources that report it.

Drug	ChEMBL ID	Type	Open Targets' stage	Stage reported by	Reports	Of them trials	Trials shown here	Source
OCTOCOG ALFA	CHEMBL4297873	Protein	Approval	EMA Human Drugs	107	102	28	Drugs and targets
MOROCTOCOG ALFA	CHEMBL2109137	Protein	Approval	EMA Human Drugs	88	87	13	Drugs and targets
ANTIHEMOPHILIC FACTOR, PEGYLATED (MW 20000) HUMAN SEQUENCE RECOMBINANT	CHEMBL4594584		Approval	EMA Human Drugs	74	72	9	Drugs and targets
LONOCTOCOG ALFA	CHEMBL4594603	Protein	Approval	EMA Human Drugs	66	65	2	Drugs and targets
EFMOROCTOCOG ALFA	CHEMBL4297945	Protein	Approval	EMA Human Drugs and TTD	59	57	7	Drugs and targets
TUROCTOCOG ALFA	CHEMBL2108286	Protein	Approval	EMA Human Drugs	34	32	14	Drugs and targets
EMICIZUMAB	CHEMBL3833393	Antibody	Approval	ClinicalTrials.gov, EMA Human Drugs, FDA and TTD	25	22	22	Drugs and targets
EPTACOG ALFA (ACTIVATED)	CHEMBL2108334	Protein	Approval	EMA Human Drugs	20	18	11	Drugs and targets
DAMOCTOCOG ALFA PEGOL	CHEMBL5315069	Protein	Approval	EMA Human Drugs and TTD	14	12	12	Drugs and targets
TUROCTOCOG ALFA PEGOL	CHEMBL5315071	Protein	Approval	EMA Human Drugs	14	12	12	Drugs and targets

1 to 10 of 42

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Targets93

The targets ChEMBL's mechanism records give for the disease's drugs, through Open Targets, the most drugs first. A mechanism record says what a drug acts on, not that the target causes the disease.

Target	Protein	Its drugs that act on it	Mechanisms	Highest stage of those drugs	Gene in Genes	Source
F8	Coagulation factor VIII	14	Coagulation factor VIII exogenous gene; Coagulation factor VIII exogenous protein	Approval	https://conductscience.org/library/genes/2157	Drugs and targets
F9	Coagulation factor IX	4	Coagulation factor IX and X other; Coagulation factor IX exogenous protein	Approval	https://conductscience.org/library/genes/2158	Drugs and targets
IFNAR1	Interferon alpha/beta receptor	3	Interferon alpha/beta receptor agonist	Phase 2	https://conductscience.org/library/genes/3454	Drugs and targets
IFNAR2	Interferon alpha/beta receptor	3	Interferon alpha/beta receptor agonist	Phase 2	https://conductscience.org/library/genes/3455	Drugs and targets
TFPI	Tissue factor pathway inhibitor	3	Tissue factor pathway inhibitor inhibitor	Approval	https://conductscience.org/library/genes/7035	Drugs and targets
F7	Coagulation factor VII	2	Coagulation factor VII exogenous protein	Approval	https://conductscience.org/library/genes/2155	Drugs and targets
AVPR1A	Vasopressin receptor	1	Vasopressin receptor agonist	Unknown	https://conductscience.org/library/genes/552	Drugs and targets
AVPR1B	Vasopressin receptor	1	Vasopressin receptor agonist	Unknown	https://conductscience.org/library/genes/553	Drugs and targets
AVPR2	Vasopressin receptor	1	Vasopressin receptor agonist	Unknown	https://conductscience.org/library/genes/554	Drugs and targets
F10	Coagulation factor IX and X	1	Coagulation factor IX and X other	Approval	https://conductscience.org/library/genes/2159	Drugs and targets

1 to 10 of 93

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Trials173

Trials an Open Targets clinical report links to the disease, posted results first, then the largest. A trial is shown when the release's checks confirm its link to the disease and Drugs and targets shows its link to one of the disease's drugs. 76 more are not shown, since the checks do not confirm them.

Trial	Title	Phase	Participants	Results first posted	Conditions	Drugs	Linked by	Source
NCT00984126	Safety and Efficacy of Turoctocog Alfa (N8) in Prevention and On-demand Treatment of Bleeding Episodes in Subjects With Haemophilia A: An Extension to Trials NN7008-3543, NN7008-3545, NN7008-3600, NN7008-3893 and NN7008-4015	Phase 3	214	2017-07-27	Congenital Bleeding Disorder, Haemophilia A	TUROCTOCOG ALFA	Open Targets' reading, confirmed by NLM's indexing of its papers	ClinicalTrials.gov
NCT00884390	Study Evaluating Safety Of Patients Switching To ReFacto AF In Usual Care Settings	Phase 4	208	2014-09-01	Hemophilia A	MOROCTOCOG ALFA	Its record names the disease	ClinicalTrials.gov
NCT01480180	Evaluation of Safety and Efficacy, Including Pharmacokinetics, of NNC 0129-0000-1003 When Administered for Treatment and Prophylaxis of Bleeding in Subjects With Haemophilia A	Phase 3	186	2020-04-20	Congenital Bleeding Disorder, Haemophilia A	TUROCTOCOG ALFA PEGOL	Its record names the disease	ClinicalTrials.gov
NCT01486927	An Open-label Safety, Efficacy and Pharmacokinetic Study of a Recombinant FVIII Compared to Recombinant Human Antihemophilic FVIII in Patients With Severe Hemophilia A	Phase 2/3	175	2016-08-09	Hemophilia A	LONOCTOCOG ALFA, OCTOCOG ALFA	Its record names the disease	ClinicalTrials.gov
NCT01181128	Study to Evaluate the Safety, Pharmacokinetics and Efficacy of Recombinant Factor VIII Fc Fusion Protein (rFVIIIFc) in Previously Treated Subjects With Severe Hemophilia A	Phase 3	165	2014-08-29	Severe Hemophilia A	OCTOCOG ALFA	Its record names the disease	ClinicalTrials.gov
NCT03528551	A Research Study Looking at How a Factor VIII Medicine Called Turoctocog Alfa Pegol (N8-GP) Works in People With Haemophilia A	Phase 3	160	2021-11-26	Congenital Bleeding Disorder, Haemophilia A	TUROCTOCOG ALFA PEGOL	Its record names the disease	ClinicalTrials.gov
NCT04161495	A Phase 3 Open-label Interventional Study of Intravenous Recombinant Coagulation Factor VIII Fc-von Willebrand Factor-XTEN Fusion Protein, Efanesoctocog Alfa (BIVV001), in Patients With Severe Hemophilia A	Phase 3	159	2023-05-24	Factor VIII Deficiency	EFANESOCTOCOG ALFA	Its record names the disease	ClinicalTrials.gov
NCT00840086	Safety and Efficacy of Turoctocog Alfa in Haemophilia A Subjects	Phase 3	150	2014-09-04	Congenital Bleeding Disorder, Haemophilia A	TUROCTOCOG ALFA	Open Targets' reading, confirmed by NLM's indexing of its papers	ClinicalTrials.gov
NCT03370913	Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients (BMN 270-301)	Phase 3	144	2023-11-28	Hemophilia A	VALOCTOCOGENE ROXAPARVOVEC	Its record names the disease	ClinicalTrials.gov
NCT02585960	BAX 855 PK-guided Dosing	Phase 3	135	2019-08-26	Hemophilia A	ANTIHEMOPHILIC FACTOR, PEGYLATED (MW 20000) HUMAN SEQUENCE RECOMBINANT	Its record names the disease	ClinicalTrials.gov

1 to 10 of 173

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Comparisons in its trials51

Comparisons Trial comparisons calculated in the trials shown here, most participants first. A comparison in one of these trials does not always compare a treatment for the disease, so read the two groups.

Comparison	Group 1, with the outcome	Group 2, with the outcome	Participants	Trial	Source
Previously Treated Patients (PTPs) vs Previously Untreated Patients (PUPs): Number of Participants Who Discontinued the Use of Study Drug	10 of 123	5 of 12	135	NCT03169972	Trial comparisons
Previously Treated Patients (PTPs) vs Previously Untreated Patients (PUPs): deaths from any cause	0 of 123	0 of 12	135	NCT03169972	Trial comparisons
Previously Treated Patients (PTPs) vs Previously Untreated Patients (PUPs): serious adverse events	2 of 123	1 of 12	135	NCT03169972	Trial comparisons
Part A: Main Study vs Part B: ITI Portion: Number of Participants With At Least One Clinically Significant Changes in Vital Signs	0 of 120	0 of 7	127	NCT02615691	Trial comparisons
Part A: Main Study vs Part B: ITI Portion: Number of Participants With At Least One Clinically Significant Changes in Clinical Laboratory Parameters	53 of 120	1 of 7	127	NCT02615691	Trial comparisons
Pre-prophylaxis vs Prophylaxis: Number of Participants With Confirmed High Titre Inhibitors (Defined as Inhibitor Titre Above 5 Bethesda Units (BU)	3 of 55	8 of 68	123	NCT02137850	Trial comparisons
Pre-prophylaxis vs Prophylaxis: Number of Participants With Inhibitory Antibodies Against Coagulation Factor VIII (FVIII)	11 of 55	10 of 68	123	NCT02137850	Trial comparisons
Factor On-demand vs Fitusiran 80 mg Prophylaxis: deaths from any cause	0 of 40	0 of 79	119	NCT03417245	Trial comparisons
Factor On-demand vs Fitusiran 80 mg Prophylaxis: serious adverse events	5 of 40	5 of 79	119	NCT03417245	Trial comparisons
BAX 855 Safety Analysis Set vs ADVATE Received Before BAX 855: serious adverse events	3 of 66	0 of 31	97	NCT02210091	Trial comparisons

1 to 10 of 51

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Papers from its trials134

The papers the trials shown here list on ClinicalTrials.gov as their results (RESULT) or as drawn from them (DERIVED), results first, newest first.

PMID	Listed as	Trials listing it	Source
38853005	RESULT	1	PubMed
37538505	RESULT	1	PubMed
37309365	RESULT	1	PubMed
36626898	RESULT	1	PubMed
36192847	RESULT	1	PubMed
32940955	RESULT	1	PubMed
32544297	RESULT	1	PubMed
32293786	RESULT	2	PubMed
32197139	RESULT	1	PubMed
31697801	RESULT	3	PubMed

1 to 10 of 134

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Data dates

The date of each source this release read.

Open Targets: 2026-09-18; Open Targets
Drug links and their checks, from Drugs and targets: 2026-09-18; Drugs and targets
ClinicalTrials.gov, from Trials: 2026-11-12; Trials
Comparisons, from Trial comparisons: 2026-03-23; Trial comparisons
PubMed, for the checks: 2026-11-06; PubMed
MeSH, for the checks: 2026-11-10; MeSH

Sources 6

ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
ChEMBL, through Open TargetsCC BY-SA 3.0
ClinicalTrials.govNo license stated
NLM's MeSHNLM's terms
Open TargetsCC0 1.0
PubMedNLM's terms

Each source's terms

ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
ChEMBL, through Open TargetsCC BY-SA 3.0, for the parts from ChEMBL. Read on 8 Nov 2026 at https://platform-docs.opentargets.org/licence
ClinicalTrials.govnone stated
NLM's MeSHNLM's
Open TargetsCC0 1.0. Open Targets asks that users cite its latest paper: Buniello et al. 2025, Open Targets Platform: facilitating therapeutic hypotheses building in drug discovery, Nucleic Acids Research 53(D1):D1467, PMID 39657122. Read on 8 Nov 2026 at https://platform-docs.opentargets.org/licence
PubMedNLM's terms

ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.

For software

Read this disease from software.

The record and each of its lists are JSON, twenty rows a page. No key.

GET https://conductscience.org/api/diseases/v1/MONDO_0010602

GET https://conductscience.org/api/diseases/v1/MONDO_0010602/drugs?page=2

API reference For AI assistants