hemophilia B
Information
- Disease name
- hemophilia B
- Disease ID
- DOID:12259
- Description
- "A blood coagulation disease that has_material_basis_in Factor IX deficiency, which makes coagulation much more prolonged. The disease is inherited as an X-linked recessive trait." [url:http\://www.nlm.nih.gov/medlineplus/ency/article/000539.htm]
Disease area statistics
[No Data.]
Chromosome band
[No Data.]
Annotation
| Genes | Mutation | Description | Source | Links |
|---|
| NCT ID | Status | Phase | Summary | Start date | Completion date |
|---|---|---|---|---|---|
| NCT04628871 | Active, not recruiting | Long Term Follow-up (LTFU) of Subjects Who Received SB-318, SB-913, or SB-FIX | November 3, 2020 | January 1, 2030 | |
| NCT04135300 | Active, not recruiting | N/A | Gene Therapy for Chinese Hemophilia B | October 16, 2019 | December 2039 |
| NCT03861273 | Active, not recruiting | Phase 3 | 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 | July 29, 2019 | September 5, 2030 |
| NCT03938792 | Active, not recruiting | Phase 3 | Study of the Efficacy and Safety PF-06741086 in Adult and Teenage Participants With Severe Hemophilia A or Moderately Severe to Severe Hemophilia B | March 9, 2020 | June 16, 2025 |
| NCT03587116 | Active, not recruiting | Phase 3 | A 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%) | July 26, 2018 | July 26, 2024 |
| NCT04073498 | Active, not recruiting | Phase 1/Phase 2 | The Safety and Tolerability of SerpinPC in Healthy Men and in Men With Severe Blood Disorders (Haemophilia A and B) | August 14, 2019 | October 2024 |
| NCT03901755 | Active, not recruiting | An International Study to Evaluate the Real-world Effectiveness and Usage of Alprolix in Patients With Haemophilia B | September 12, 2019 | May 31, 2024 | |
| NCT05360706 | Active, not recruiting | Study of AAV5-hFIX in Severe or Moderately Severe Haemophilia B | March 18, 2021 | May 2026 | |
| NCT01687608 | Active, not recruiting | Phase 1/Phase 2 | Open-Label Single Ascending Dose of Adeno-associated Virus Serotype 8 Factor IX Gene Therapy in Adults With Hemophilia B | February 11, 2013 | January 17, 2030 |
| NCT03569891 | Active, not recruiting | Phase 3 | HOPE-B: Trial of AMT-061 in Severe or Moderately Severe Hemophilia B Patients | June 27, 2018 | March 2025 |
| NCT05203679 | Active, not recruiting | Phase 3 | Evaluation of the Safety and Efficacy of Hemophilia B Gene Therapy Drug | December 30, 2021 | June 30, 2028 |
| NCT03307980 | Active, not recruiting | Phase 2 | Long-term Safety and Efficacy Study and Dose-Escalation Substudy of PF 06838435 in Individuals With Hemophilia B | June 22, 2017 | May 29, 2029 |
| NCT00979238 | Active, not recruiting | Phase 1 | Dose-Escalation Study Of A Self Complementary Adeno-Associated Viral Vector For Gene Transfer in Hemophilia B | February 22, 2010 | December 31, 2032 |
| NCT00581438 | Completed | Study Evaluating Approach to Treatment of Haemophilia A and B in Spain | June 2007 | December 2007 | |
| NCT00714415 | Completed | Registry For Patients Treated With BeneFix In Usual Care Setting In Germany | January 2008 | October 2016 | |
| NCT00716716 | Completed | Phase 1 | Phase I/IIa Study of FIXFc in Hemophilia B Patients | April 2008 | October 2009 |
| NCT00749476 | Completed | Phase 4 | Study Evaluating BeneFIX in Patients With Haemophilia B, Previously Treated With Plasma Derived Factor IX | April 2008 | January 2009 |
| NCT00768287 | Completed | Phase 2/Phase 3 | Study of Recombinant Factor IX Product, IB1001, in Subjects With Hemophilia B | January 2009 | December 2016 |
| NCT00824798 | Completed | Gait Evaluation in Haemophiliac Patients | January 2009 | January 2011 | |
| NCT00835068 | Completed | Post Marketing Observational Study of Reformulated BeneFIX | January 2009 | October 2013 | |
| NCT00851721 | Completed | Phase 3 | 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 | March 31, 2009 | October 17, 2012 |
| NCT00866606 | Completed | Phase 3 | Study Evaluating On-Demand Treatment With BeneFIX In Chinese Subjects | February 2009 | December 2009 |
| NCT00927992 | Completed | Study Evaluating Liver Transplantation in Haemophilia Patients in Spain | July 2009 | June 2010 | |
| NCT00936312 | Completed | Females With Severe or Moderate Hemophilia A or B: an International Multi-center Study | March 2008 | August 2011 | |
| NCT00936845 | Completed | Females With Severe or Moderate Hemophilia A or B: A Multi-Center Study | April 2005 | January 2010 | |
| NCT01090206 | Completed | N/A | Correlation Between Vitamin D Status and Bone Mineral Density in Patients With Hemophilia | March 2010 | March 2015 |
| NCT01128881 | Completed | Phase 4 | IMMUNINE Pre-Treatment Study | May 31, 2010 | August 28, 2012 |
| NCT01154231 | Completed | BeneFIX Drug Use Results Survey [All-Case Surveillance] | February 2010 | February 27, 2017 | |
| NCT01174446 | Completed | Phase 3 | Pivotal Study (Pharmacokinetics, Efficacy, Safety) of BAX 326 (rFIX) in Hemophilia B Patients | July 29, 2010 | May 3, 2012 |
| NCT01217255 | Completed | Comparing the Burden of Illness of Hemophilia in the Developing and the Developed World | September 2010 | October 2013 | |
| NCT01233440 | Completed | Phase 1 | Safety and Pharmacokinetic Study of a Recombinant Coagulation Factor IX Albumin Fusion Protein in Subjects With Hemophilia B | October 2010 | July 2011 |
| NCT01286779 | Completed | Phase 3 | BAX 326 (rFIX) Continuation Study | April 12, 2011 | June 29, 2017 |
| NCT01335061 | Completed | Phase 3 | Study To Compare On-Demand Treatment To A Prophylaxis Regimen Of BeneFIX In Subjects With Moderately Severe to Severe Hemophilia B | September 2011 | April 2014 |
| NCT01361126 | Completed | Phase 1/Phase 2 | A Safety and Efficacy Study of a Recombinant Factor IX in Patients With Severe Hemophilia B | July 2011 | July 2012 |
| NCT00004801 | Completed | Phase 1/Phase 2 | Phase I/II Study of Monoclonal Factor IX Concentrate for Factor IX Deficiency | March 1992 | |
| NCT01488994 | Completed | Phase 2/Phase 3 | BAX 326 Pediatric Study | December 20, 2011 | May 14, 2013 |
| NCT01496274 | Completed | Phase 2/Phase 3 | A Safety and Efficacy Study of a Recombinant Fusion Protein Linking Coagulation Factor IX With Albumin (rIX-FP) in Patients With Hemophilia B | February 2012 | |
| NCT01507896 | Completed | Phase 3 | BAX 326 Surgery Study in Hemophilia B Patients | December 19, 2011 | May 15, 2014 |
| NCT01510418 | Completed | Socialization of Adult Men With Congenital Hemophilia A or B | May 2011 | December 2013 | |
| NCT01589848 | Completed | Study on Von Willebrand Disease and Hemophilia in Cuenca, Ecuador | March 2013 | June 2013 | |
| NCT01662531 | Completed | Phase 3 | A Safety, Efficacy and Pharmacokinetics Study of a Recombinant Fusion Protein Linking Coagulation Factor IX With Albumin (rIX-FP) in Children With Hemophilia B | January 2013 | |
| NCT01748201 | Completed | Phase 4 | Viscosupplementation in Patients With Hemophilic Arthropathy | November 2012 | March 2013 |
| NCT01757405 | Completed | Phase 3 | Recombinant Factor VIIa BI (rFVIIa BI) Treatment of Acute Bleeding Episodes Per an On-demand Regimen | February 20, 2013 | November 11, 2014 |
| NCT01921855 | Completed | Phase 1 | Study of FVIIa Variant BAY86-6150 (B0189) in Subjects With Moderate or Severe Hemophilia Types A or B With or Without Inhibitors | January 2009 | December 2009 |
| NCT02035605 | Completed | Phase 1 | A Phase 1 Study of an Investigational Drug, ALN-AT3SC, in Healthy Volunteers and Hemophilia A or B Patients | January 20, 2014 | July 20, 2017 |
| NCT02053792 | Completed | Phase 3 | A Safety and Efficacy Extension Study of a Recombinant Fusion Protein Linking Coagulation Factor IX With Albumin (rIX-FP) in Patients With Hemophilia B | February 6, 2014 | June 2, 2021 |
| NCT02061072 | Completed | Web-based Application for the Population Pharmacokinetic Service - Phase 1 | January 2015 | June 2019 | |
| NCT02190149 | Completed | Study of Prophylaxis, ACtivity and Effectiveness (SPACE) in Hemophilia Patients Currently Treated With ADVATE or RIXUBIS | July 22, 2014 | March 29, 2016 | |
| NCT02199717 | Completed | An Institutional Pilot Study to Investigate Physical Activity Patterns in Boys With Hemophilia | September 2013 | September 2016 | |
| NCT02213250 | Completed | Phase 1 | An Open-Label, Single Dose Pharmacokinetic Study of Benefix (Recombinant Factor IX) in Male Chinese Subjects With Hemophilia B | March 2015 | April 2015 |
| NCT02234310 | Completed | Phase 3 | Study to Determine the Safety and Efficacy of rFIXFc in Previously Untreated Males With Severe Hemophilia B | November 13, 2014 | August 20, 2019 |
| NCT02336178 | Completed | Phase 4 | Safety and Efficacy of Benefix in Patients With Hemophilia B in Usual Care Settings in China | January 2015 | August 2016 |
| NCT02396342 | Completed | Phase 1/Phase 2 | Trial of AAV5-hFIX in Severe or Moderately Severe Hemophilia B | June 10, 2015 | April 15, 2021 |
| NCT02418793 | Completed | Phase 1 | A Study of a Long-Acting r-Factor 7a (Factor VIIa) in Adult Men With Hemophilia A or B | May 2015 | September 2018 |
| NCT02484092 | Completed | Phase 2 | A Gene Therapy Study for Hemophilia B | November 18, 2015 | April 8, 2019 |
| NCT02554773 | Completed | Phase 1/Phase 2 | An Open-label Extension Study of an Investigational Drug, Fitusiran, in Patients With Moderate or Severe Hemophilia A or B | September 18, 2015 | March 21, 2023 |
| NCT02571569 | Completed | Phase 1 | A Single Escalating Dose and Multiple Dose Study of BAY 1093884 in Subjects With Severe Hemophilia Types A or B, With or Without Inhibitors | October 28, 2015 | October 11, 2018 |
| NCT02634918 | Completed | Ultrasonography in Hemophilic Joint Disease and Serum Markers | January 2016 | December 2019 | |
| NCT02740413 | Completed | Treatment Patterns And Outcomes In Patients Treated With Benefix Or Refacto/Refacto AF - A Swedish Cohort Study | January 11, 2016 | December 31, 2017 | |
| NCT01440946 | Completed | Phase 3 | Study of Recombinant Coagulation Factor IX Fc Fusion Protein, BIIB029, in Previously Treated Pediatric Participants With Hemophilia B | June 2012 | November 2014 |
| NCT00037557 | Completed | Phase 3 | Study Evaluating rFIX; BeneFIX in Severe Hemophilia B | September 2002 | November 2007 |
| NCT00093171 | Completed | Phase 3 | Study Evaluating rFIX; BeneFIX® in Hemophilia B | May 2005 | |
| NCT00093210 | Completed | Phase 3 | Study Evaluating of Recombinant Human Factor IX (BeneFIX) and a New Formulation of BeneFIX (rFIX-R) in Moderate to Severe Hemophilia B | September 2005 | |
| NCT00139828 | Completed | Phase 4 | Post Marketing Study in Haemophilia B Patients Using Nonafact® (Human Coagulation Factor IX) | May 2003 | February 2007 |
| NCT00167973 | Completed | Prospective Registry of European Hemophilia B Patients Receiving BeneFIX® for Usual Use | January 2002 | June 2009 | |
| NCT00195221 | Completed | Study to Describe the Allergic Reactions to Factor IX in Patients With Hemophilia B | February 2005 | November 2007 | |
| NCT00212459 | Completed | N/A | The Effect of Patient Counseling on Adolescent Hemophilia Patient Compliance With Bleeding Logs | May 2005 | April 2008 |
| NCT00244114 | Completed | Study Evaluating Allergic Reactions To Benefix In Hemophilia B Patients | February 2006 | December 2009 | |
| NCT00364182 | Completed | Phase 3 | Study Comparing On-Demand Treatment With Two Prophylaxis Regimens Of BeneFIX In Patients With Severe Hemophilia B | May 2007 | October 2010 |
| NCT00484185 | Completed | Post Marketing Surveillance To Observe Safety and Efficacy Of BeneFIX In Patients With Hemophilia B | August 2007 | June 2012 | |
| NCT00515710 | Completed | LTFU for Gene Transfer Subjects With Hemophilia B | August 2007 | December 2017 | |
| NCT00581126 | Completed | Phase 4 | Study Evaluating BENEFIX in Previously Treated Patients With Hemophilia B | December 2001 | July 2004 |
| NCT02922231 | Completed | Post-Marketing Surveillance (Usage Results Study) of RIXUBIS in Adult and Pediatric Patients With Haemophilia B in South Korea | January 6, 2017 | April 4, 2019 | |
| NCT02937831 | Completed | RIXUBIS Drug Use-Result Survey (Japan) | November 16, 2016 | May 11, 2022 | |
| NCT02971969 | Completed | Long-Term Safety, Tolerability, and Efficacy of DTX101 (AAVrh10FIX) in Adults With Moderate/Severe to Severe Hemophilia B | January 2017 | November 6, 2021 | |
| NCT03054389 | Completed | Non-Interventional Study to Capture the Patient Experience on Gene Therapy for Hemophilia Within the AskBio009-101 Study Through Patient and Investigator Interviews | March 31, 2017 | June 6, 2017 | |
| NCT03091751 | Completed | Phase 2 | Efficacy and Safety of AlphaNine Versus BeneFIX in Patients With Severe Hereditary Haemophilia B | August 2005 | October 2009 |
| NCT03185897 | Completed | Global Epidemiologic Study of Preexisting Immunity to AAV in Adults With Severe Hemophilia | June 14, 2017 | March 17, 2021 | |
| NCT03186677 | Completed | Phase 1 | Dose-escalation Study to Investigate the Safety, PK, and PD of ISU304/CB2679d in Hemophilia B Patients | June 3, 2017 | February 22, 2019 |
| NCT03358836 | Completed | Joint Health Study | November 15, 2017 | August 17, 2020 | |
| NCT03417102 | Completed | Phase 3 | A Study of Fitusiran (ALN-AT3SC) in Severe Hemophilia A and B Patients With Inhibitors | February 14, 2018 | June 23, 2021 |
| NCT03417245 | Completed | Phase 3 | A Study of Fitusiran (ALN-AT3SC) in Severe Hemophilia A and B Patients Without Inhibitors | March 1, 2018 | July 14, 2021 |
| NCT03489291 | Completed | Phase 2 | Dose Confirmation Trial of AAV5-hFIXco-Padua | July 24, 2018 | September 21, 2023 |
| NCT03507582 | Completed | N/A | Virtual Reality for Hemophilia | April 2016 | December 28, 2016 |
| NCT03565237 | Completed | Phase 4 | RIXUBIS PMS India (RIXUBIS PMS) | December 7, 2018 | August 11, 2021 |
| NCT03818529 | Completed | ATHN 8: Previously Untreated Patients (PUPs) Matter Study | October 3, 2018 | December 31, 2022 | |
| NCT03855280 | Completed | Phase 3 | Evaluation of a Recombinant Factor IX Product, APVO101, in Previously-Treated Pediatric Patients With Hemophilia B | January 16, 2020 | July 4, 2022 |
| NCT03995784 | Completed | Phase 2 | Study of Next-Generation Recombinant Factor IX Variant in Adult Subjects With Hemophilia B | June 18, 2019 | April 30, 2020 |
| NCT04072237 | Completed | Phase 1 | Study of Coagulation Faction VIIa Variant Marzeptacog Alfa (Activated) in Adult Subjects With Hemophilia | September 24, 2019 | June 17, 2020 |
| NCT04272554 | Completed | AAV Gene Therapy Screening/Observational Protocol (ECLIPSE) | February 14, 2020 | October 10, 2022 | |
| NCT04286412 | Completed | Phase 4 | Nonacog Alfa Prophylaxis And Treatment Of Bleeding Episodes In Previously Treated Patients With Hemophilia B | February 10, 2020 | September 24, 2020 |
| NCT04731701 | Completed | Psychometric Validation of the Hemophilia Functional Ability Scoring Tool (Hemo-FAST) | May 5, 2021 | July 19, 2022 | |
| NCT05606640 | Completed | Gaining Insight Into the Complexity of Pain in Patients With Haemophilia | February 13, 2020 | October 13, 2023 | |
| NCT05804734 | Completed | Vitamin K Antagonist Versus Direct Oral Anticoagulant Treatments in Hemophilia | June 1, 2021 | December 31, 2021 | |
| NCT05962398 | Enrolling by invitation | Long-term Follow-up Study of Male Adults With Hemophilia B Previously Treated With Etranacogene Dezaparvovec (CSL222) | August 30, 2023 | March 2035 | |
| NCT03655223 | Enrolling by invitation | Early Check: Expanded Screening in Newborns | October 15, 2018 | December 31, 2025 | |
| NCT03533504 | Enrolling by invitation | Web Accessible Population Pharmacokinetics Service - Hemophilia: Sources of Variability | May 9, 2018 | June 30, 2024 | |
| NCT05981313 | Not yet recruiting | Viscoelastic Properties of Lower Extremity Muscles in Patients With Hemophilia | August 15, 2023 | December 30, 2023 | |
| NCT05889754 | Not yet recruiting | Reliability and Validity of the Turkish Version of the PedHAL | May 30, 2023 | December 30, 2023 | |
| NCT05441553 | Not yet recruiting | Phase 1/Phase 2 | A Study to Evaluate the Safety and Efficacy of VGB-R04 in Adult Hemophilia B Patients | July 2022 | January 2025 |
| NCT05442528 | Not yet recruiting | Lead-in Study of VGB-R04 Gene Therapy for Hemophilia B-- An Observational Survey Analysis Study | August 2022 | December 2024 | |
| NCT06399289 | Not yet recruiting | Phase 3 | Recombinant Fusion Protein Linking Coagulation Factor IX With Albumin (rIX-FP) in Chinese Subjects With Hemophilia B Previously Treated With FIX Therapy | June 2024 | December 2026 |
| NCT05641610 | Not yet recruiting | Phase 1/Phase 2 | A Study to Evaluate the Safety and Efficacy of ZS801 in Adult Hemophilia B Patients | March 2023 | December 2028 |
| NCT06147414 | Not yet recruiting | Development of Non-Invasive Prenatal Diagnosis for Single Gene Disorders | April 2024 | December 2026 | |
| NCT06379789 | Not yet recruiting | Phase 1/Phase 2 | A Trial to Learn if REGV131-LNP1265 is Safe and Works to Help the Body Make Clotting Factor in Pediatric, Adolescent and Adult Patients With Hemophilia B | July 25, 2024 | July 3, 2032 |
| NCT05980377 | Not yet recruiting | Patterns of Hemophilia Care in Assiut Children Patients | September 1, 2023 | August 1, 2024 | |
| NCT05630651 | Not yet recruiting | N/A | The Efficacy and Safety of ZS801 in Chinese Hemophilia B Patients. | December 2022 | December 2028 |
| NCT06014320 | Not yet recruiting | Alterations in Coagulation Factor Levels in Patients With End Stage Liver Disease | October 1, 2023 | October 1, 2024 | |
| NCT05932914 | Not yet recruiting | Liver Biopsy Following Gene Therapy For Hemophilia | July 2024 | May 2025 | |
| NCT05044845 | Recruiting | Needs Assessment of Knowledge, Beliefs, and Attitudes of Patients With Hemophilia B About Gene Therapy | January 18, 2022 | January 2025 | |
| NCT05145127 | Recruiting | Phase 3 | Open-Label Extension Study of Marstacimab in Hemophilia Participants With or Without Inhibitors | November 17, 2021 | July 31, 2030 |
| NCT05152732 | Recruiting | Early Phase 1 | Safety and Tolerability of VGB-R04 in Patients With Haemophilia B | December 28, 2021 | December 2025 |
| NCT06003387 | Recruiting | Phase 3 | 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) | January 30, 2024 | October 2028 |
| NCT05487976 | Recruiting | Phase 3 | Clinical Study of Recombinant Human Activated Coagulation Factor VII for Injection in Patients With Hemophilia With Inhibitor | April 28, 2022 | April 2023 |
| NCT05568459 | Recruiting | A Trial That Evaluates Disease Characteristics in Hemophilia B Adult Male Participants Receiving Prophylaxis With Standard of Care Factor IX Protein (FIX) Replacement Therapy | January 17, 2024 | April 2, 2026 | |
| NCT05568719 | Recruiting | Phase 3 | Safety and Effectiveness of Giroctocogene Fitelparvovec or Fidanacogene Elaparvovec in Patients With Hemophilia A or B Respectively | December 28, 2022 | April 8, 2038 |
| NCT05605678 | Recruiting | Hemophilia Non-Interventional Study Prior to SerpinPC Intervention (PRESent-5) | December 9, 2022 | November 2024 | |
| NCT05611801 | Recruiting | Phase 3 | A Clinical Trial of Study Medicine (Marstacimab) in Pediatric Patients With Hemophilia A or Hemophilia B | December 9, 2022 | September 10, 2028 |
| NCT02912143 | Recruiting | German Pediatric Hemophilia Research Database | January 1, 2017 | December 31, 2028 | |
| NCT05687474 | Recruiting | Baby Detect : Genomic Newborn Screening | September 1, 2022 | August 31, 2025 | |
| NCT05709288 | Recruiting | Phase 1 | Gene Therapy for Hemophilia B Patients Aged 12-18 Years Old | March 23, 2023 | November 2035 |
| NCT03327779 | Recruiting | World Bleeding Disorders Registry | January 26, 2018 | January 2028 | |
| NCT05789524 | Recruiting | Phase 2 | The Efficacy and Safety of SerpinPC in Participants With Severe Hemophilia A or Moderately Severe to Severe Hemophilia B | July 6, 2023 | June 5, 2026 |
| NCT05789537 | Recruiting | Phase 2 | A Study of SerpinPC in Participants With Hemophilia B (HemB) With Inhibitors | July 28, 2023 | October 28, 2025 |
| NCT06025552 | Recruiting | Phase 1 | Study of TU7710 in Warfarin Anti-coagulated Healthy Male Subjects | August 2, 2023 | February 2024 |
| NCT05879549 | Recruiting | N/A | Comparison of Efficiency of Closed Kinetic Chain Exercises Versus Proprioceptive Exercises Patient With Hemophilia | November 15, 2023 | May 1, 2025 |
| NCT06120582 | Recruiting | Phase 1/Phase 2 | Study of the Safety, Pharmacodynamics and Efficacy of ANB-002 in Patients With Hemophilia B (SAFRAN) | May 2, 2023 | November 2029 |
| NCT06008938 | Recruiting | An Observational Cohort Study to Characterize the Effectiveness and Safety of HEMGENIX® in Patients With Hemophilia B | June 15, 2023 | August 2043 | |
| NCT06158334 | Recruiting | Study of Surgical Practices in Patients With Haemophilia A or B Treated With an Extended Half-life Recombinant Factor VIII-Fc or IX-Fc (ELOCTA®, ALPROLIX®) | June 1, 2022 | December 1, 2024 | |
| NCT03615053 | Recruiting | N/A | Personalized Medicine for Canadians With Hemophilia | July 24, 2019 | September 30, 2023 |
| NCT04645199 | Recruiting | National Longitudinal Cohort of Hematological Diseases | December 1, 2020 | December 1, 2030 | |
| NCT06349473 | Recruiting | Phase 1 | A Study of Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of SR604 in Two Participants Groups (Part A: Healthy Participants, and Part B: Participants With Hemophilia A or Hemophilia B) | April 15, 2024 | March 30, 2026 |
| NCT02618915 | Terminated | Phase 1/Phase 2 | Safety and Dose Finding Study of DTX101 (AAVrh10FIX) in Adults With Moderate/Severe to Severe Hemophilia B | December 16, 2015 | October 18, 2017 |
| NCT03641703 | Terminated | Phase 1/Phase 2 | A Long-Term Follow-Up Study of Haemophilia B Patients Who Have Undergone Gene Therapy | July 10, 2018 | May 24, 2023 |
| NCT02392156 | Terminated | Fc factOrs and Real-World hemophiliA Patient-ReporteD Outcomes | July 2015 | December 2015 | |
| NCT01620801 | Terminated | Phase 1 | Hemophilia B Gene Therapy With AAV8 Vector | October 2012 | March 2016 |
| NCT01460147 | Terminated | N/A | Osteoporosis and MRI Study in Hemophilia | October 30, 2011 | April 13, 2012 |
| NCT04394286 | Terminated | Phase 1/Phase 2 | A Phase 1/2 Study of SHP648, an Adeno-Associated Viral Vector for Gene Transfer in Hemophilia B Subjects | May 13, 2020 | May 3, 2021 |
| NCT01271868 | Terminated | Phase 3 | Study of Recombinant Factor IX Product, IB1001, in Previously Treated Pediatric Subjects With Hemophilia B | November 2010 | December 2016 |
| NCT05164471 | Terminated | Phase 1/Phase 2 | Phase 1/2 Dose Confirmation Study of FLT180a in Hemophilia B | December 6, 2021 | May 31, 2023 |
| NCT00947193 | Terminated | Phase 2 | Study of Ataluren (PTC124) in Hemophilia A and B | October 14, 2009 | August 30, 2011 |
| NCT00076557 | Terminated | Phase 1/Phase 2 | Safety of a New Type of Treatment Called Gene Transfer for the Treatment of Severe Hemophilia B | January 2004 | |
| NCT02807753 | Terminated | The Hemophilia Ultrasound Project | September 16, 2016 | December 7, 2020 | |
| NCT05856266 | Terminated | Phase 4 | An 18-month Low-interventional Study to Assess Joint Health in Haemophilia A and B Patients on Prophylaxis With Efmoroctocog Alfa or Eftrenonacog Alfa | August 24, 2023 | December 6, 2023 |
| NCT03248141 | Terminated | Understanding Hemophilia A and B Drug Dosage Administration Patterns | September 1, 2017 | March 14, 2018 | |
| NCT02695160 | Terminated | Phase 1 | Ascending Dose Study of Genome Editing by Zinc Finger Nuclease Therapeutic SB-FIX in Subjects With Severe Hemophilia B | November 15, 2016 | April 19, 2021 |
| NCT03369444 | Terminated | Phase 1/Phase 2 | A Factor IX Gene Therapy Study (FIX-GT) | December 5, 2017 | October 20, 2020 |
| NCT02402829 | Terminated | A Study Comparing Factor Level and Inhibitor Titer Testing Results Drawn From Central Venous Lines and Venipuncture | November 2014 | November 28, 2018 | |
| NCT05439642 | Unknown status | N/A | Validation and Reliability of the CHO-KLAT in Turkish | June 1, 2022 | December 1, 2022 |
| NCT05187936 | Unknown status | Pre-clinical Models for Mesenchymal Stem Cell Therapy in Hemophilic Arthropathy | October 1, 2021 | December 31, 2022 | |
| NCT02938156 | Unknown status | UK - EHL Outcomes Registry | December 17, 2016 | October 2019 | |
| NCT02061033 | Unknown status | Global Hemostatic Methods in Hemophilia and Von Willebrand's Disease | March 2013 | December 2018 | |
| NCT03961243 | Unknown status | Phase 1 | Lentiviral FIX Gene Therapy | June 1, 2020 | June 1, 2022 |
| NCT03946384 | Unknown status | Mutation p.Ile112Thr : Discrepancy Between Factor IX Level and Bleeding Phenotype | June 2019 | October 2019 | |
| NCT04541875 | Withdrawn | Medication Adherence and Non-adherence in Adults With Rare Disease | January 2021 | September 2022 | |
| NCT02048111 | Withdrawn | Phase 3 | Study of Recombinant Factor IX Product, IB1001, in Previously Treated Subjects With Hemophilia B | March 2014 | November 2015 |
- Disase is a (Disease Ontology)
- DOID:1247
- Cross Reference ID (Disease Ontology)
- GARD:8732
- Cross Reference ID (Disease Ontology)
- ICD10CM:D67
- Cross Reference ID (Disease Ontology)
- ICD9CM:286.1
- Cross Reference ID (Disease Ontology)
- MESH:D002836
- Cross Reference ID (Disease Ontology)
- MIM:306900
- Cross Reference ID (Disease Ontology)
- NCI:C26721
- Cross Reference ID (Disease Ontology)
- SNOMEDCT_US_2023_03_01:41788008
- Cross Reference ID (Disease Ontology)
- UMLS_CUI:C0008533
- Exact Synonym (Disease Ontology)
- Congenital factor IX deficiency
- Exact Synonym (Disease Ontology)
- Congenital factor IX disorder
- Exact Synonym (Disease Ontology)
- deficiency, functional factor IX
- Exact Synonym (Disease Ontology)
- factor IX deficiency
- OrphaNumber from OrphaNet (Orphanet)
- 98879
- MeSH unique ID (MeSH (Medical Subject Headings))
- D002836