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Hemophilia A trial readouts

registry pull 2026-08-19

Primary-completion dates that listed drug and biotech companies filed with ClinicalTrials.gov for studies in Hemophilia A. They are the sponsor's own estimated windows, revised as a study runs — not announcement dates. sources →

Source: ClinicalTrials.gov, retrieved 2026-08-19

Upcoming readout windows

next 180 days · by month

3 studies across 2 companies, in the next 180 days.

October 2026 1

TickerNameStudyPhaseStatusPrimary completionReadout inUpdated
NVONovo Nordisk A/SPatient Preferences in Treatment of Haemophilia A With and Without Inhibitors: a Discrete Choice Experiment in GermanyNCT07653139observationalChoice of treatment alternative in discrete choice tasksNot yet recruiting2026-10-26in 67 d2026-06-17

November 2026 1

TickerNameStudyPhaseStatusPrimary completionReadout inUpdated
SOBI.STSwedish Orphan BiovitrumA Study to Investigate the Course of Synovial Hypertrophy in Patients With Haemophilia A on Efanesoctocog Alfa ProphylaxisNCT06752850Haemophilia Early Arthropathy Detection with Ultrasound (HEAD-US) synovial hypertrophy domain score decrease.Phase 4Active, not recruiting2026-11-17in 89 d2026-04-30

February 2027 1

TickerNameStudyPhaseStatusPrimary completionReadout inUpdated
NVONovo Nordisk A/SA Research Study Looking at How Different Doses of Study Medicine (Inno8) Work in the Body of People With Haemophilia ANCT07220564Number of treatment emergent adverse eventsPhase 1Recruiting2027-02-05in 169 d2026-08-10

Primary completion is the date the sponsor filed with the registry — their own projection, revised whenever they revise it, unless the row is marked actual.

Every registered study in Hemophilia A

most recently updated first

154 studies match. Showing the first 100 of 154 studies — page through, or narrow the filters.

TickerNameMarket capStudyPhaseStatusInterventionsConditionsEnrollmentStartPrimary completionReadout inUpdated
PFEPfizer$155.3BStudy to Evaluate the Efficacy and Safety of PF-07055480 / Giroctocogene Fitelparvovec Gene Therapy in Moderately Severe to Severe Hemophilia A AdultsNCT04370054results2025-08-17Total Annualized Bleeding Rate (ABR)Phase 3Active, not recruitingBiological: PF-07055480 (giroctocogene fitelparovec): Recombinant AAV2/6 Human Factor VIII Gene TherapyHemophilia A772020-08-182024-06-17actual2026-08-19
PFEPfizer$155.3BStudy of the Efficacy and Safety PF-06741086 in Adult and Teenage Participants With Severe Hemophilia A or Moderately Severe to Severe Hemophilia BNCT03938792results2026-08-14Model-Based Annualized Bleeding Rate (ABR) of Treated Bleeding Events: Inhibitor Cohort (Participants With Prior OD Therapy at OP)Phase 3CompletedDrug: PF-06741086Hemophilia A, Hemophilia B1882020-03-092025-04-29actual2026-08-14
PFEPfizer$155.3BA Clinical Trial of Study Medicine (Marstacimab) in Pediatric Patients With Hemophilia A or Hemophilia BNCT05611801Annualized bleeding rate (ABR) of treated bleeding eventsPhase 3RecruitingDrug: marstacimabHemophilia A, Hemophilia B1002022-12-092028-09-10in 752 d2026-08-10
NVONovo Nordisk A/SA Research Study Looking at How Different Doses of Study Medicine (Inno8) Work in the Body of People With Haemophilia ANCT07220564Number of treatment emergent adverse eventsPhase 1RecruitingDrug: NNC0442-0344 AHaemophilia A302025-10-232027-02-05in 169 d2026-08-10
BMRNBioMarin Pharmaceutical$13.0BSafety, Tolerability, and Efficacy Study of Valoctocogene Roxaparvovec in Hemophilia A With Active or Prior InhibitorsNCT04684940Number of participants with treatment-related adverse events, as assessed by Common Terminology Criteria for Adverse Events (CTCAE) v5.0 after administration of BMN 270.Phase 1/2CompletedBiological: Valoctocogene roxaparvovecHemophilia A With Inhibitor, Hemophilia A With Anti Factor VIII102020-12-102026-04-20actual2026-07-10
PFEPfizer$155.3BOpen-Label Extension Study of Marstacimab in Hemophilia Participants With or Without InhibitorsNCT05145127Number of subject reporting Adverse EventsPhase 3RecruitingDrug: PF-06741086Hemophilia A, Hemophilia B2452021-11-172030-07-31in 1,441 d2026-07-01
NVONovo Nordisk A/SA Research Study Looking at Long-term Treatment With Mim8 in People With Haemophilia ANCT05685238Arm 1 and 2: Number of treatment emergent adverse eventsPhase 3RecruitingDrug: Mim8Haemophilia A, Haemophilia A With Inhibitors4512023-02-132028-06-30in 680 d2026-06-30
SOBI.STSwedish Orphan BiovitrumLong-term Study Evaluating Joint Health in People With Haemophilia A Receiving Real-world Prophylactic Treatment With Efanesoctocog AlfaNCT06940830Annualized joint bleeding rate (AjBR) over the prospective observation period.Phase 4RecruitingDrug: Efanesoctocog alfaHemophilia A2502025-05-21Dec 2028≤ 864 d2026-06-30
SOBI.STSwedish Orphan BiovitrumStudy to Provide Continued Access to Treatment for Patients Completing a Previous Trial With Efanesoctocog AlfaNCT06716814Number of injections to treat a bleeding episodePhase 3RecruitingDrug: Efanesoctocog alfaHaemophilia A (Moderate or Severe)1042025-03-06Mar 2027≤ 223 d2026-06-26
SOBI.STSwedish Orphan BiovitrumPharmacokinetic Comparison of Efanesoctocog Alfa vs Other EHL-rFVIII Products in Participants With Severe Haemophilia ANCT06579144Half-life (t½) of efanesoctocog alfa, damactocog alfa pegol and turoctocog alfa pegol after a single i.v. injectionPhase 1CompletedDrug: Efanesoctocog alfaHemophilia A242025-02-242026-05-21actual2026-06-25
NVONovo Nordisk A/SStudy for Turoctocog Alfa Treatment Regimen in Iraqi Haemophilia A PatientsNCT06574984observationalAnnual bleeding Rate (ABRs) among patients treated with different regimen of turoctocog alfa after previous FVIII replacement therapyCompletedDrug: Turoctocog alfaCongenital Bleeding Disorder, Haemophilia A3292024-11-102025-11-04actual2026-06-17
NVONovo Nordisk A/SA Research Study Looking at How Food Intake Affects Inno8 in the Body of Healthy PeopleNCT07238816AUC0-∞: the area under the plasma Inno8 concentration-time curve from time 0 to infinity after a single oral dosePhase 1CompletedDrug: NNC0442-0344 AHaemophilia A802025-11-182026-05-08actual2026-06-17
NVONovo Nordisk A/SPatient Preferences in Treatment of Haemophilia A With and Without Inhibitors: a Discrete Choice Experiment in GermanyNCT07653139observationalChoice of treatment alternative in discrete choice tasksNot yet recruitingOther: No treatment givenHaemophilia A1502026-07-102026-10-26in 67 d2026-06-17
NVONovo Nordisk A/SPost-Marketing Surveillance (Use-results Surveillance) With Esperoct®NCT04334057observationalNumber of adverse reactions (ARs) reported during the observation periodCompletedDrug: Turoctocog alfa pegolHaemophilia A232021-03-312023-11-30actual2026-06-15
NVONovo Nordisk A/SPost-Marketing Surveillance (All Case Surveillance) on Treatment With Alhemo® in Patients With Haemophilia A or Haemophilia B With InhibitorsNCT06285071observationalNumber of adverse reaction (AR)Enrolling by invitationDrug: ConcizumabHaemophilia A, Haemophilia B232024-08-082030-04-30in 1,349 d2026-06-15
NVONovo Nordisk A/SResearch Study to Look at How Well the Drug Concizumab Works in Your Body if You Have Haemophilia With InhibitorsNCT04083781results2025-04-27Rate of Treated Spontaneous and Traumatic Bleeding EpisodesPhase 3Active, not recruitingDrug: ConcizumabHaemophilia A With Inhibitors, Haemophilia B With Inhibitors1342019-10-212021-12-27actual2026-06-12
SNYSanofiA Study to Test a Medicine (Fitusiran) for Preventing Bleeds in People With Severe Hemophilia Who Previously Received Preventive Treatment With EmicizumabNCT06145373Number of participants with Adverse events (AEs) during the fitusiran treatmentPhase 4RecruitingDrug: Fitusiran (SAR439774) · Biological: Clotting factor concentrates (CFC) or bypassing agents (BPA), Antithrombin concentrate (ATIIIC), EmicizumabHemophilia A202024-03-012028-05-28in 647 d2026-06-02
PFEPfizer$155.3BSafety and Effectiveness of Giroctocogene Fitelparvovec or Fidanacogene Elaparvovec in Patients With Hemophilia A or B RespectivelyNCT05568719Incidence of thromboembolic eventsPhase 3RecruitingDiagnostic test: Testing of hepatic AAV Vector integrationHemophilia A, Hemophilia B1732022-12-282040-02-25in 4,937 d2026-05-27
SOBI.STSwedish Orphan BiovitrumA Study to Investigate the Course of Synovial Hypertrophy in Patients With Haemophilia A on Efanesoctocog Alfa ProphylaxisNCT06752850Haemophilia Early Arthropathy Detection with Ultrasound (HEAD-US) synovial hypertrophy domain score decrease.Phase 4Active, not recruitingDrug: Efanesoctocog alfaHaemophilia A (Moderate or Severe), Hemophilia, Classic, Haemophilia, Hemophilia, Classic Hemophilia, Synovial Hypertrophy372024-12-192026-11-17in 89 d2026-04-30
NVONovo Nordisk A/SA Study Looking at How Different Doses of Study Medicine (Inno8) Works in the Body of Healthy MenNCT06649630SAD: Number of treatment emergent adverse eventsPhase 1CompletedDrug: NNC0442-0344 A, PlaceboHaemophilia A952024-09-232026-03-26actual2026-04-01
PFEPfizer$155.3BA Study to Learn About the Study Medicine -Hympavzi in Congenital Hemophilia Patients Without Inhibitors in Japan.NCT07161687observationalNumber of the participants with adverse drug reactionsActive, not recruitingDrug: MarstacimabHemophilia A or B502025-10-302030-04-11in 1,330 d2026-03-30
SOBI.STSwedish Orphan BiovitrumA 48-Month Study to Evaluate Long-Term Effectiveness of Elocta on Joint HealthNCT04293523observationalJoint health: Target joint developmentActive, not recruitingDrug: ELOCTAHemophilia A4272020-03-30Jul 20262026-03-19
BMRNBioMarin Pharmaceutical$13.0BPhase 3 Study for Efficacy and Safety Outcomes Data in Japanese Patients With Severe Hemophilia ANCT06224907Change in the human coagulation factor VIII (hFVIII) activity, as measured by chromogenic substrate assay, during Weeks 49 to 52 post BMN 270 infusion from Baseline.Phase 3Active, not recruitingBiological: Valoctocogene roxaparvovecHemophilia A62023-12-252025-04-16actual2026-03-19
SNYSanofiGoal Attainment and Physical Activity in People With Hemophilia ANCT06530030observationalProportion of total goals achievedTerminatedsponsor's stated reason: Early discontinuation based on sponsor decision not driven by any safety concerns.Drug: efanesoctocog alfaHemophilia A32024-11-262025-09-18actual2026-01-20
BMRNBioMarin Pharmaceutical$13.0BA Long-Term Follow-Up Study in Severe Hemophilia A Subjects Who Received BMN 270 in a Prior BioMarin Clinical Trial (270-401)NCT05768386observationalEvaluate the long-term safety of BMN 270Enrolling by invitationHemophilia A1722023-01-01Jan 2040≤ 4,912 d2026-01-12
NVONovo Nordisk A/SEfficacy and Safety of Turoctocog Alfa Pegol (N8-GP) for Prophylaxis and Treatment of Bleeding Episodes in Previously Treated Chinese Patients With Haemophilia A (pathfinder10)NCT05082116results2024-01-18Number of Bleeding Episodes Per Year (Annualised Bleeding Rate)Phase 3CompletedDrug: turoctocog alfa pegol (N8-GP)Haemophilia A362021-09-272022-12-28actual2026-01-06
NVONovo Nordisk A/SSafety and Efficacy of Turoctocog Alfa Pegol (N8-GP) in Previously Untreated Patients With Haemophilia ANCT02137850results2024-10-31Number of Participants With Inhibitory Antibodies Against Coagulation Factor VIII (FVIII)Phase 3CompletedDrug: turoctocog alfa pegolCongenital Bleeding Disorder, Haemophilia A1242014-06-262023-06-07actual2025-12-23
NVONovo Nordisk A/SA Study Following Males With Haemophilia A on Prophylaxis With Esperoct®NCT04574076observationalNumber of Adverse Events (AEs) reported during the study periodEnrolling by invitationDrug: Turoctocog alfa pegol (N8-GP)Haemophilia A602020-10-232027-06-03in 287 d2025-12-22
NVONovo Nordisk A/SA Research Study Investigating Mim8 in People With Haemophilia ANCT04204408Part 1: Number of treatment emergent adverse eventsPhase 2CompletedDrug: NNC0365-3769 (Mim8), Placebo (Mim8)Healthy Volunteers, Haemophilia A With or Without Inhibitors2752020-01-102023-10-06actual2025-12-19
NVONovo Nordisk A/SA Research Study Investigating Mim8 in Adults and Adolescents With Haemophilia A With or Without InhibitorsNCT05053139Number of treated bleedsPhase 3CompletedDrug: NNC0365-3769 (Mim8)Haemophilia A, Haemophilia A With Inhibitors2812021-12-022024-03-11actual2025-12-08
NVONovo Nordisk A/SA Research Study Looking at How Safe it is to Switch From Emicizumab to Mim8 in People With Haemophilia A (FRONTIER 5)NCT05878938Number of treatment-emergent adverse eventsPhase 3CompletedDrug: NNC0365-3769 (Mim8) PPXHaemophilia A, Haemophilia A With Inhibitors612023-06-262024-07-18actual2025-12-08
NVONovo Nordisk A/SA Research Study Looking at Mim8 in Children With Haemophilia A With or Without InhibitorsNCT05306418Number of treatment emergent adverse eventsPhase 3CompletedDrug: Mim8Haemophilia A With or Without Inhibitors702022-04-042024-11-13actual2025-11-13
PFEPfizer$155.3BA Study to Evaluate Prospective Efficacy and Safety Data of Current FIX Prophylaxis Replacement Therapy in Adult Hemophilia B Subjects (FIX:C≤2%) or Current FVIII Prophylaxis Replacement Therapy in Adult Hemophilia A Subjects (FVIII:C≤1%)NCT03587116results2025-11-06Annualized Bleeding Rate (ABR) for Treated Bleeds and All Bleeds During Prospective Data Collection Period in Hemophilia B Participants: Efficacy Analysis SetPhase 3CompletedDrug: Standard of Care FIX Replacement therapy, Standard of Care FVIII Replacement therapyHemophilia B, Hemophilia A2122018-07-262024-12-13actual2025-11-06
NVONovo Nordisk A/SA Study to Compare the Blood Levels of Mim8 in Healthy Men When Administered With the DV3407-C1 Pen Injector or a Syringe and CartridgeNCT05681845AUC0-112 days, SD: area under the Mim8 plasma concentration-time curve from time 0 until 112 days after a single dosePhase 1CompletedDrug: NNC0365-3769 (Mim8)Haemophilia A2052023-01-022023-10-13actual2025-10-14
BMRNBioMarin Pharmaceutical$13.0BStudy to Evaluate the Efficacy and Safety of Valoctocogene Roxaparvovec, With Prophylactic Steroids in Hemophilia ANCT04323098results2024-04-04Change From Baseline in FVIII Activity as Measured by Chromogenic Substrate Assay at Week 52.Phase 3CompletedBiological: valoctocogene roxaparvovecHemophilia A222020-12-082023-01-27actual2025-10-08
SNYSanofiA Study to Evaluate Impact of Efanesoctocog Alfa on Long-term Joint Health in Participants With Hemophilia ANCT05911763observationalChange from baseline in Annualized joint bleeding rate (AjBR) for treated bleedsActive, not recruitingDrug: Efanesoctocog Alfa BIVV001Hemophilia A2002023-06-302028-06-06in 656 d2025-10-02
SNYSanofiA Study to Evaluate Impact of Efanesoctocog Alfa on Long-term Joint Health in Participants With Hemophilia A in TaiwanNCT06684314observationalChange from baseline in total scores of the Hemophilia Joint Health Score (HJHS) for all jointsRecruitingDrug: Efanesoctocog alfaHemophilia A1002024-11-112031-08-28in 1,834 d2025-10-02
PFEPfizer$155.3BA Study of Recombinant AAV2/6 Human Factor 8 Gene Therapy SB-525 (PF-07055480) in Subjects With Severe Hemophilia ANCT03061201results2025-09-30Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)Phase 2CompletedBiological: SB-525 (PF-07055480)Hemophilia A132017-06-212024-07-16actual2025-09-30
NVONovo Nordisk A/SAdverse Event Data Collection From the EUHASS Registry on Turoctocog Alfa PegolNCT04682145observationalAdverse events (AEs) reported to the registry with suspected relation to turoctocog alfa pegol, Adverse Drug Reactions (ADRs), in patients with haemophilia A for renal, hepatic and neurological events.Enrolling by invitationDrug: Turoctocog alfa pegolHaemophilia A502020-12-092026-02-012025-09-25
SNYSanofiPharmacokinetic Assessment of Standard Half-Life (SHL) FVIII, Extended Half-Life (EHL) FVIII, and Efanesoctocog Alfa (BIVV001) in Severe Hemophilia ANCT05042440Half-life of BIVV001Phase 1CompletedDrug: Efanesoctocog alfa, Octocog alfa, Rurioctocog alfa pegolHemophilia A132021-08-112021-11-24actual2025-09-18
PFEPfizer$155.3BPatient Interview Study to Explore the Impact of Gene Therapy in Hemophilia A & BNCT06634836observationalFrequency of self-reported symptoms pre and post hemophilia gene therapyWithdrawnsponsor's stated reason: Business decisionHemophilia A, Hemophilia B02025-03-172025-12-312025-08-29
BMRNBioMarin Pharmaceutical$13.0BGene Therapy Study in Severe Hemophilia A Patients With Antibodies Against AAV5NCT03520712results2025-08-22Number of Participants With Treatment Emergent Adverse EventsPhase 1/2Terminatedsponsor's stated reason: Early rollover into long-term extension studyBiological: Valoctocogene RoxaparvovecHemophilia A, Gene Therapy, Clotting Disorders, Blood Disorder32018-04-242024-08-07actual2025-08-22
PFEPfizer$155.3BTreatment Patterns And Outcomes In Patients Treated With Benefix Or Refacto/Refacto AF - A Swedish Cohort StudyNCT02740413results2019-05-03observationalAge of Participants at Disease DiagnosisCompletedHemophilia A, Hemophilia B862016-01-112017-12-31actual2025-07-16
PFEPfizer$155.3BStudy Evaluating On-Demand Treatment Of Xyntha In Chinese SubjectsNCT00868530results2011-02-08Investigator Hemostatic Efficacy Assessment 8 Hours Post InfusionPhase 3CompletedBiological: XynthaHemophilia A53Sep 2008Dec 2009actual2025-06-12
NVONovo Nordisk A/SAn Observational Research Study of the Health of Joints in People With Haemophilia Taking the Medicine EsperoctNCT05621746observationalChange in haemophilia joint health score (HJHS)RecruitingDrug: EsperoctHaemophilia A1002022-11-232026-06-302025-06-02
BMRNBioMarin Pharmaceutical$13.0BSingle-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients (BMN 270-301)NCT03370913results2023-11-28Change From Baseline in Annualized Number of Bleeding Episodes Irrespective of Exogenous FVIII Replacement Treatment [Annualized Bleeding Rate (ABR) for All Bleeds] in EEP.Phase 3CompletedBiological: valoctocogene roxaparvovecHemophilia A1442017-12-192020-11-16actual2025-03-25
SOBI.STSwedish Orphan BiovitrumAn 18-month Low-interventional Study to Assess Joint Health in Haemophilia A and B Patients on Prophylaxis With Efmoroctocog Alfa or Eftrenonacog AlfaNCT05856266results2025-01-16Change From Baseline in Total HEAD-US Score up to Month 18 (End of Study - EOS)Phase 4Terminatedsponsor's stated reason: The Sponsor has decided to stop the phase 4 study Sobi.HAEM89-007 (JOIN-us) due to a realignment of its development activities. The clinical trial hasn't been terminated due to a change in the benefit-risk balance.Procedure: Haemophilia Early Arthropathy Detection with Ultrasound (HEAD-US) · Other: Haemophilia Joint Health Score (HJHS)Hemophilia A, Hemophilia B102023-08-242023-12-06actual2025-01-16
SOBI.STSwedish Orphan BiovitrumProspective, Non-interventional Study to Evaluate the Effectiveness of Elocta Compared to Conventional Factor ProductsNCT02976753observationalAnnualised bleeding rate (ABR)CompletedDrug: efmoroctocog alfa, Factor VIIIHemophilia A361Dec 20162021-11-03actual2024-10-02
SOBI.STSwedish Orphan BiovitrumA Study to Evaluate the Real-world Usage and Effectiveness of Elocta and Alprolix in Patients With Haemophilia A or BNCT03055611observationalAnnualised bleeding rate (ABR)CompletedDrug: ELOCTA, ALPROLIXHaemophilia A, Haemophilia B2012017-05-092022-04-19actual2024-10-02
SOBI.STSwedish Orphan BiovitrumStudy of rFVIIIFc for Immune Tolerance Induction (ITI) in Haemophilia A Patients With Inhibitors Who Have Failed Previous ITI TherapiesNCT03103542results2021-11-30ITI SuccessPhase 4CompletedBiological: Recombinant coagulation factor (rFVIIIFc)Hemophilia A162017-08-292019-09-04actual2024-09-19
SOBI.STSwedish Orphan BiovitrumrFVIIIFc (Elocta®) ITI Chart Review in Patients With Haemophilia ANCT03951103observationalITI with rFVIIIFc: Main doseCompletedOther: rFVIIIFcHemophilia A With Inhibitor442018-11-072022-09-30actual2024-09-19
SOBI.STSwedish Orphan BiovitrumImpact on French Physician's Haemophilia Treatment Management Decision Based on Systematic Joint ExaminationNCT04133883Changes in haemophilia management based on systematic joint examinations of ankles, knees and elbows with HJHS and HEAD-USNot applicableCompletedProcedure: Ultrasound, Haemophilia Joint Health ScoreHemophilia A882020-01-132022-07-13actual2024-09-19
SOBI.STSwedish Orphan BiovitrumPsychometric Validation of the Hemophilia Functional Ability Scoring Tool (Hemo-FAST)NCT04731701observationalPsychometric validation of the Hemophilia Functional Ability Scoring Tool (Hemo-FAST).CompletedDiagnostic test: Hemophilia Joint Health Score (HJHS)Hemophilia A, Hemophilia B1802021-05-052022-07-19actual2024-09-19
LLYEli Lilly and Company$1.15TSafety & Efficacy of Encapsulated Allogeneic FVIII Cell Therapy in Haemophilia ANCT04541628results2024-09-04Number of Participants With Treatment Emergent Adverse Events (TEAEs)Phase 1/2Terminatedsponsor's stated reason: SIG-001 programme terminatedCombination product: SIG-001Hemophilia A32020-09-282022-10-28actual2024-09-04
BMRNBioMarin Pharmaceutical$13.0BA Prospective Study Evaluating Seroprevalence and Seroconversion of Antibodies Against Adeno-associated Virus (AAV)NCT05580692To qunatify Seroprevalence of antibodies to selected AAV serotypesNot applicableTerminatedsponsor's stated reason: Sponsor decision to terminate; sufficient sample size for statistical analysis has been achieved.Procedure: Biospecimen CollectionHemophilia A3212023-03-142024-07-31actual2024-08-19
BMRNBioMarin Pharmaceutical$13.0BA Study to Evaluate Seroprevalence and Seroconversion of Antibodies to Adeno-Associated Virus (AAV) in Patients With Hemophilia ANCT04560933observationalAssess the Seroprevalence of antibodies to AAV vectors in patients with hemophilia ACompletedProcedure: Blood sample collectionHemophilia A1862020-08-252023-09-20actual2024-04-24
SOBI.STSwedish Orphan BiovitrumManagement of Health-Related QoL Impairment, Including Pain, Depression and Anxiety, in People With Haemophilia A and BNCT03276130observationalUse of medication for pain, depression and anxiety (yes/no?)CompletedHaemophilia A, Haemophilia B2,3002018-10-302020-01-30actual2024-03-06
NVONovo Nordisk A/SSafety and Efficacy of Activated Recombinant Human Factor VII in Haemophilia Patients With Inhibitors During and After Major SurgeryNCT01561391Presence and maintenance of haemostasisPhase 4CompletedDrug: activated recombinant human factor VII, activated recombinant human factor VII, factor VIII · Other: factor IXCongenital Bleeding Disorder, Haemophilia A With Inhibitors, Haemophilia B With Inhibitors36Apr 1998May 2004actual2023-12-29
NVONovo Nordisk A/SA Long-term Assessment of Physical Activity, Range of Motion, and Functional Status Following Elective Orthopedic Surgery in Hemophilia Patients With InhibitorsNCT01830712observationalLong-term changes in general mobility/ambulation/activityCompletedDrug: eptacog alfa (activated)Congenital Bleeding Disorder, Haemophilia A With Inhibitors, Haemophilia B With Inhibitors15Apr 2011Aug 2011actual2023-12-29
NVONovo Nordisk A/STuroctocog Alfa in Haemophilic Italian Patients: Protection and Engagement in Recreational ActivitiesNCT04584892observationalIndividual prophylaxis (PPX) regimen with turoctocog alfa: Every second day, or three times a week (3TW), or twice a week (2TW)CompletedDrug: Turoctocog alfaHaemophilia A182021-04-122023-03-27actual2023-11-28
BMRNBioMarin Pharmaceutical$13.0BSingle-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients at a Dose of 4E13 vg/kgNCT03392974results2021-10-08Change of the Median Factor VIII (FVIII) ActivityPhase 3CompletedBiological: Valoctocogene RoxaparvovecHemophilia A12018-03-142019-05-22actual2023-10-03
NVONovo Nordisk A/SA Research Study Looking at How a Factor VIII Medicine Called Turoctocog Alfa Pegol (N8-GP) Works in People With Haemophilia ANCT03528551results2021-11-26Number of Adverse Events ReportedPhase 3CompletedDrug: Turoctocog alfa pegol, Turoctocog alfa pegol, Turoctocog alfa pegolCongenital Bleeding Disorder, Haemophilia A1602018-04-302020-12-03actual2022-12-22
PFEWyeth is now a wholly owned subsidiary of Pfizer$155.3BStudy Evaluating The Efficacy And Safety Of Xyntha In Children Less Than 6 Years Of AgeNCT00759655results2011-01-04Percentage of Participants With Factor VIII (FVIII) Inhibitor DevelopmentPhase 3Terminatedsponsor's stated reason: See termination reason in detailed description.Biological: Moroctocog alfaHemophilia A1Jun 2009Dec 2009actual2022-06-15
NVONovo Nordisk A/SA Study Following People With Haemophilia A and B, With or Without Inhibitors, When on Usual Treatment (Explorer™6)NCT03741881observationalThe number of treated bleeding episodesCompletedOther: No treatment givenHaemophilia A, Haemophilia A With Inhibitors, Haemophilia B, Haemophilia B With Inhibitors2312018-12-182021-10-25actual2021-11-23
NVONovo Nordisk A/SA Trial Evaluating Efficacy and Safety of Prophylactic Administration of Concizumab in Patients With Severe Haemophilia A Without InhibitorsNCT03196297results2021-05-10The Number of Bleeding Episodes During at Least 24 Weeks From Treatment OnsetPhase 2CompletedDrug: Concizumab, Turoctocog alfaHaemostasis, Haemophilia A362017-08-162018-06-22actual2021-11-16
NVONovo Nordisk A/STo Investigate Safety and Efficacy of NovoEight® (rFVIII) During Long-term Treatment of Haemophilia A in JapanNCT02207218observationalFrequency of Adverse Events (AEs)CompletedDrug: turoctocog alfaCongenital Bleeding Disorder, Haemophilia A402014-08-042020-11-10actual2021-11-15
NVONovo Nordisk A/SStudy Investigating Novoeight®/NovoEight® (Turoctocog Alfa) in Mexican Haemophilia A PatientsNCT03179748observationalFrequency of adverse reactionsCompletedDrug: turoctocog alfaCongenital Bleeding Disorder, Haemophilia A102018-06-202021-06-16actual2021-11-15
NVONovo Nordisk A/SA Trial Evaluating the Efficacy and Safety of Prophylactic Administration of Concizumab in Haemophilia A and B Patients With InhibitorsNCT03196284results2021-10-22The Number of Bleeding EpisodesPhase 2CompletedDrug: Concizumab, Eptacog alfaCongenital Bleeding Disorder, Haemophilia A With Inhibitors, Haemophilia B With Inhibitors262017-08-102018-09-19actual2021-10-22
PFEPfizer$155.3BPF-06741086 Long-term Treatment in Severe HemophiliaNCT03363321results2021-07-27Number of Participants With Treatment-Emergent Adverse Events (TEAEs), TEAEs by Severity, and Serious Adverse Events (SAEs) (All Causality and Treatment-Related)Phase 2CompletedBiological: PF-06741086Hemophilia A or B202018-05-302020-08-05actual2021-07-27
OPKOPKO Health, Inc.$1.0BA Study of a Long-Acting r-Factor 7a (Factor VIIa) in Adult Men With Hemophilia A or BNCT02418793Composite safety and tolerability parameters as measured by adverse events, electrocardiograms (ECG), laboratory results, vital signs and injection site reactionsPhase 1CompletedDrug: MOD-5014Hemophilia A, Hemophilia B27May 2015Sep 2018actual2021-05-20
PFEPfizer$155.3BMoroctocog Alfa (AF-CC) for Prophylaxis and Treatment of Bleeding Episodes in Previously Treated Hemophilia A PatientsNCT04396639results2021-05-03Percentage of Participants Who Developed Factor VIII (FVIII) InhibitorsPhase 4CompletedBiological: Moroctocog-alfa (AF-CC)Hemophilia A502020-01-252020-09-24actual2021-05-03
SNYSanofiA Phase 1 Study of an Investigational Drug, ALN-AT3SC, in Healthy Volunteers and Hemophilia A or B PatientsNCT02035605The safety of ALN-AT3SC evaluated by the proportion of subjects experiencing adverse events (AEs), serious adverse events (SAEs), dose-limiting toxicities (DLTs), and AEs leading to study drug discontinuation.Phase 1CompletedDrug: ALN-AT3SC, Sterile Normal Saline (0.9% NaCl)Hemophilia A, Hemophilia B512014-01-202017-07-20actual2020-12-19
NVONovo Nordisk A/SSafety and Efficacy of Turoctocog Alfa in Prevention and Treatment of Bleeds in Previously Untreated Children With Haemophilia ANCT01493778results2018-10-30Incidence Rate of Factor VIII Inhibitors (Above or Equal to 0.6 BU (Bethesda Units)/mL) for the Main Phase of the TrialPhase 3CompletedDrug: turoctocog alfaCongenital Bleeding Disorder, Haemophilia A602012-09-172017-08-16actual2020-11-30
NVONovo Nordisk A/SEvaluation of Safety and Efficacy, Including Pharmacokinetics, of NNC 0129-0000-1003 When Administered for Treatment and Prophylaxis of Bleeding in Subjects With Haemophilia ANCT01480180results2020-04-20The Incidence Rate of FVIII-inhibitors ≥0.6 BU: After Approximately 19 MonthsPhase 3CompletedDrug: turoctocog alfa pegolCongenital Bleeding Disorder, Haemophilia A1862012-01-302018-12-10actual2020-11-23
NVONovo Nordisk A/SA Multinational, Open-Label, Non-Controlled Trial on Safety, Efficacy and Pharmacokinetics of NNC 0129-0000-1003 in Previously Treated Paediatric Patients With Severe Haemophilia ANCT01731600results2019-10-30Number of Participants With Inhibitory Antibodies Against Coagulation Factor VIII (FVIII) ≥0.6 Bethesda UnitsPhase 3CompletedDrug: turoctocog alfa pegolCongenital Bleeding Disorder, Haemophilia A682013-02-202014-09-15actual2020-11-23
PFEPfizer$155.3BPatient-reported Outcomes in Subjects Treated With ReFacto AF Routine ProphylaxisNCT02718677observationalThe health-related quality of life in subjects treated with ReFacto AF routine prophylaxisWithdrawnsponsor's stated reason: Company decision to not conduct studyDrug: Refacto AFHemophilia A02019-09-012021-05-012020-11-16
NVONovo Nordisk A/SEvaluating the Haemostatic Effect of NNC 0129-0000-1003 During Surgical Procedures in Subjects With Haemophilia A.NCT01489111results2020-01-21Haemostatic Effect During Surgery Evaluated by the Four-point Scale, Assessed by the Investigator/Surgeon at the Day of Surgery - Four-point Response Scale: Excellent, Good, Moderate or NonePhase 3CompletedDrug: turoctocog alfa pegolCongenital Bleeding Disorder, Haemophilia A362012-08-032018-12-10actual2020-08-10
NVONovo Nordisk A/SEfficacy and Safety of Turoctocog Alfa for Prophylaxis and Treatment of Bleeding Episodes in Previously Treated Chinese Patients With Haemophilia ANCT02938585results2019-08-26Haemostatic Effect of Turoctocog Alfa (Treatment of Bleeds): 6 MonthsPhase 3CompletedDrug: turoctocog alfa, turoctocog alfaCongenital Bleeding Disorder, Haemophilia A682016-12-122018-03-16actual2020-07-27
NVONovo Nordisk A/SEvaluation of Safety Following Immune Tolerance Induction Treatment With Turoctocog Alfa in Patients With Haemophilia A Following Inhibitor Development in NN7170-4213 TrialNCT03588741results2020-07-07Number of Adverse EventsPhase 3Terminatedsponsor's stated reason: The trial was terminated as the participant withdrew from the trial.Drug: Turoctocog alfaHaemophilia A12018-06-122019-06-19actual2020-07-07
PTCTPTC Therapeutics$6.1BStudy of Ataluren (PTC124) in Hemophilia A and BNCT00947193results2020-06-16Number of Participants With a Plasma FVIII/FIX Activity Response at Day 14Phase 2Terminatedsponsor's stated reason: Study was terminated early due to Sponsor decision and not reflective of adverse safety findings.Drug: AtalurenHemophilia A, Hemophilia B132009-10-142011-08-30actual2020-06-16
NVONovo Nordisk A/SSafety and Efficacy of Turoctocog Alfa During Long-Term Treatment of Severe and Moderately Severe Haemophilia ANCT02035384observationalIncidence rate of FVIII inhibitors (at least 0.6 Bethesda Units (BU) for central laboratory analyses, or above the specific local laboratory reference range) represented as the percentage of patients developing inhibitorsCompletedDrug: turoctocog alfaCongenital Bleeding Disorder, Haemophilia A692014-06-052020-01-15actual2020-04-27
NVONovo Nordisk A/SResearch Study to Look at Side Effects During Regular Injection With Factor VIII Medicine Named Turoctocog Alfa for a 8 Weeks PeriodNCT03449342results2020-03-18Occurrence of Confirmed FVIII Inhibitor Development (≥ 0.6 BU)Phase 4CompletedDrug: turoctocog alfaCongenital Bleeding Disorder, Haemophilia A602018-03-012019-03-25actual2020-04-17
NVONovo Nordisk A/SSafety, Tolerability, and Pharmacokinetics Study of Turoctocog Alfa Pegol Injected Under the Skin in Patients With Haemophilia ANCT02994407Number of adverse eventsPhase 1CompletedDrug: turoctocog alfa pegolCongenital Bleeding Disorder, Haemophilia A502017-01-302018-10-15actual2020-02-05
PFEPfizer$155.3BPF-06741086 Multiple Dose Study in Severe HemophiliaNCT02974855results2019-12-04Number of Participants With Treatment Emergent Adverse Events (TEAEs)Phase 2CompletedBiological: PF-06741086, PF-06741086, PF-06741086, PF-06741086Hemophilia A or B272017-03-082018-12-03actual2019-12-04
NVONovo Nordisk A/SA Study of the Impact of Hemophilia and Its Treatment on Brain Development, Thinking and Behaviour in Children With HemophiliaNCT03660774observationalCognition composite score according to the BAYLEY-III instrumentCompletedOther: No treatment givenHaemophilia A, Haemophilia B5632018-09-272019-10-11actual2019-11-22
PFEPfizer$155.3BStudy of Safety And Efficacy Of ReFacto AF In Previously Untreated Hemophilia A Patients In The Usual Care SettingNCT00950170results2019-07-19Percentage of Participants Who Developed Clinically Significant Factor VIII (FVIII) Inhibitors During the Course of the StudyPhase 4CompletedProcedure: Laboratory TestsHemophilia A232010-02-102016-11-24actual2019-07-19
PFEPfizer$155.3BUnderstanding Hemophilia A and B Drug Dosage Administration PatternsNCT03248141results2019-05-20observationalResource Utilization PatternTerminatedsponsor's stated reason: Study recruitment was stopped due to difficulty in enrolling the targeted number of patients on March 21, 2018. See details in Description section.Drug: Hemophilia B standard half-life, Hemophilia B extended half-life, Hemophilia A standard half-life, Hemophilia A extended half-lifeHemophilia A, Hemophilia B112017-09-012018-03-14actual2019-05-20
NVONovo Nordisk A/SSafety of NNC 0172-0000-2021 in Healthy Male Subjects and Subjects With Haemophilia A or BNCT01228669Number of adverse Events (AEs), including Serious Adverse Events (SAEs)Phase 1CompletedDrug: NNC 0172-0000-2021, NNC 0172-0000-2021, placeboCongenital Bleeding Disorder, Haemophilia A, Haemophilia B, Healthy522010-10-252012-09-10actual2019-05-15
NVONovo Nordisk A/SA Multi-centre, Open Labelled, Multiple Dosing Trial Investigating Safety, Pharmacokinetics and Pharmacodynamics of NNC 0172-2021 Administered Subcutaneously to Healthy Male Subjects and Haemophilia SubjectsNCT01631942Number of adverse events (AEs)Phase 1Terminatedsponsor's stated reason: See detailed descriptionDrug: NNC172-2021Congenital Bleeding Disorder, Haemophilia A, Healthy42012-06-262012-09-04actual2019-05-15
NVONovo Nordisk A/SEvaluating the Pharmacokinetics of NovoEight® (Turoctocog Alfa) in Relation to BMI in Subjects With Haemophilia ANCT02941354Plasma FVIII activity at 30 minutes(C30min)Phase 1CompletedDrug: turoctocog alfaCongenital Bleeding Disorder, Haemophilia A352016-10-102017-06-20actual2019-01-30
PFEPfizer$155.3BStudy Evaluating Prophylaxis Treatment & Characterizing Efficacy, Safety, & PK Of B-Domain Deleted Recombinant FVIIINCT00543439results2019-01-11Mean Annualized Bleed Rate (ABR) by Treatment: On Demand CohortPhase 3CompletedBiological: Moroctocog alfa (AF-CC), Moroctocog alfa (AF-CC)Hemophilia A66Dec 2007Apr 2018actual2019-01-11
NVONovo Nordisk A/SPharmacogenetic Testing of Saliva Samples From Patients With Five or More Exposure Days to rFVIIa Analogue in the Adept™2 TrialNCT02541942results2019-01-04Determination of HLA TypePhase 2CompletedOther: No treatment givenCongenital Bleeding Disorder, Haemophilia A With Inhibitors, Haemophilia B With Inhibitors192015-04-032016-04-15actual2019-01-04
PFEWyeth is now a wholly owned subsidiary of Pfizer$155.3BStudy Comparing Blood Levels of ReFacto and Advante in Hemophilia ANCT00168051Pharmacokinetic measurement of area under the plasma Factor VIII activity in plasma versus time curve for 48 hours.Phase 4WithdrawnDrug: ReFacto, AdvanteHemophilia A0Apr 2005Nov 2005actual2018-09-25
PFEPfizer$155.3BEvaluation Of Acceptability And Satisfaction Of Hemophilia Patients Treated With FusENGONCT01959919results2018-09-25observationalEase of Using Clotting Factor Treatment ScoreCompletedDevice: Refacto FusENGOHemophilia A862014-01-272016-12-12actual2018-09-25
NVONovo Nordisk A/SSafety and Efficacy of 3 Different Doses of Long Acting Factor VII in Haemophilia A or B Patients With InhibitorsNCT00951405ThrombogenecityPhase 2CompletedDrug: activated recombinant human factor VII, long acting, activated recombinant human factor VII, long acting, activated recombinant human factor VII, long actingCongenital Bleeding Disorder, Haemophilia A With Inhibitors, Haemophilia B With Inhibitors232009-09-012011-03-29actual2018-09-05
PFEPfizer$155.3BStudy Evaluating Pharmacovigilance Of Refacto AFNCT00895037results2018-07-17observationalNumber of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)CompletedDrug: ReFacto AF (Moroctocog alfa)Hemophilia A1012009-07-172016-10-19actual2018-07-17
NVONovo Nordisk A/SA Multi-centre, Comparative, Double Blind, Randomised Cross-over Trial Investigating Single Dose Pharmacokinetics and Safety of Turoctocog Alfa Pegol From the Pivotal Process and Turoctocog Alfa Pegol From the Commercial Process in Patients With Severe Haemophilia ANCT02920398Area under the FVIII activity-time curvePhase 1CompletedDrug: turoctocog alfa pegolCongenital Bleeding Disorder, Haemophilia A212016-10-042017-04-07actual2017-12-15
NVONovo Nordisk A/SSafety 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 NN7008-4015NCT00984126results2017-07-27Frequency of Development of FVIII Inhibitors (Greater Than or Equal to 0.6 Bethesda Units (BU)/mL)Phase 3CompletedDrug: turoctocog alfa, turoctocog alfaCongenital Bleeding Disorder, Haemophilia A2142009-10-262016-06-28actual2017-07-27
NVONovo Nordisk A/STrial Investigating Safety, Pharmacokinetics and Pharmacodynamics of Concizumab Administered Subcutaneously to Haemophilia A SubjectsNCT02490787Number of adverse events (AEs)Phase 1CompletedDrug: Concizumab, placeboCongenital Bleeding Disorder, Haemophilia A242015-09-102016-10-14actual2017-07-21
NVONovo Nordisk A/SEfficacy and Safety of NNC 0078-0000-0007 in Patients With Congenital Haemophilia and InhibitorsNCT01392547results2013-12-06Effective Bleeding Control Defined as no Additional Haemostatic Medication (Other Than Trial Product) GivenPhase 3CompletedDrug: vatreptacog alfa (activated), eptacog alfa (activated)Congenital Bleeding Disorder, Haemophilia A With Inhibitors, Haemophilia B With Inhibitors72Jul 2011Aug 2012actual2017-05-15

Primary completion is the date the sponsor filed with the registry — their own projection, revised whenever they revise it, unless the row is marked actual.

How Hemophilia A is matched — 7 exact registry spellings

A study is on this page when its ClinicalTrials.gov record names one of these conditions exactly (letter case aside). Nothing is matched by substring or stem, so a longer condition that merely contains one of these words is a different page. The list was curated by hand and last measured 2026-08-19; a spelling filed by only one study was not curated, so the counts here are a floor rather than the registry's own total for the disease.

  • Haemophilia A
  • Hemophilia A
  • Haemophilia A With Inhibitors
  • Hemophilia A or B
  • Haemophilia A (Moderate or Severe)
  • Haemophilia A With or Without Inhibitors
  • Hemophilia A With Inhibitor

Permalink · All conditions · Catalyst calendar · Every registered study · Search the whole pipeline for “Hemophilia A” (a text search, wider than this page)