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Conditions: Hemophilia B
Interventions: CSL222 (Adeno-associated viral vector serotype 5 [AAV5]-hFIXco-Padua); Genetic
Lead sponsor: CSL Behring; Industry
Eligibility: 138 Months to 206 Months · Male only
Enrollment: 20 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2025 – 2033
U.S. locations: 5
States / cities: Orange, California • Gainesville, Florida • Atlanta, Georgia + 2 more

Conditions: Cardiopulmonary Bypass
Interventions: Recombinant activated factor VII, Prothrombin Complex Concentrate; Drug
Lead sponsor: Emory University; Other
Eligibility: Up to 30 Days
Enrollment: 24 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2011 – 2012
U.S. locations: 1
States / cities: Atlanta, Georgia

Conditions: Hemophilia B
Interventions: FLT180a; Biological
Lead sponsor: University College, London; Other
Eligibility: 18 Years and older · Male only
Enrollment: 10 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2017 – 2020
U.S. locations: 1
States / cities: Memphis, Tennessee

Conditions: Complex Cardiovascular Surgery With Cardiopulmonary Bypass
Interventions: BE1116, FFP; Biological
Lead sponsor: CSL Behring; Industry
Eligibility: 18 Years and older
Enrollment: 200 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2025 – 2027
U.S. locations: 16
States / cities: Los Angeles, California • Atlanta, Georgia • Chicago, Illinois + 12 more

Conditions: Hemophilia B
Interventions: Gene Transfer, scAAV2/8-LP1-hFIXco; Genetic · Drug
Lead sponsor: St. Jude Children's Research Hospital; Other
Eligibility: 18 Years and older · Male only
Enrollment: 14 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2010 – 2026
U.S. locations: 3
States / cities: Stanford, California • Memphis, Tennessee • Temple, Texas

Conditions: Factor VIII Deficiency, Congenital
Interventions: Emicizumab; Drug
Lead sponsor: Indiana Hemophilia &Thrombosis Center, Inc.; Other
Eligibility: 5 Years to 45 Years · Male only
Enrollment: 20 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2027
U.S. locations: 1
States / cities: Indianapolis, Indiana

Conditions: Hemophilia A, Hemophilia B
Interventions: Testing of hepatic AAV Vector integration; Diagnostic Test
Lead sponsor: Pfizer; Industry
Eligibility: 18 Years and older · Male only
Enrollment: 173 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2040
U.S. locations: 15
States / cities: Rancho Cordova, California • Sacramento, California • San Francisco, California + 6 more

Conditions: Hemophilia B
Interventions: IB1001; Biological
Lead sponsor: Medexus Pharma, Inc.; Industry
Eligibility: Up to 12 Years
Enrollment: 9 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2010 – 2016
U.S. locations: 3
States / cities: Chicago, Illinois • Indianapolis, Indiana • Houston, Texas

Conditions: Hemophilia B
Interventions: DTX101; Genetic
Lead sponsor: Ultragenyx Pharmaceutical Inc; Industry
Eligibility: 18 Years and older · Male only
Enrollment: 6 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2015 – 2017
U.S. locations: 6
States / cities: Little Rock, Arkansas • Los Angeles, California • Gainesville, Florida + 3 more

Conditions: Hemophilia B
Interventions: REGV131, LNP1265; Drug
Lead sponsor: Regeneron Pharmaceuticals; Industry
Eligibility: 2 Years and older · Male only
Enrollment: 130 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2024 – 2047
U.S. locations: 16
States / cities: Phoenix, Arizona • Los Angeles, California • Sacramento, California + 11 more

Conditions: Hemophilia B
Interventions: rFIXFc, FIX; Drug
Lead sponsor: Bioverativ Therapeutics Inc.; Industry
Eligibility: Up to 11 Years · Male only
Enrollment: 30 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2012 – 2014
U.S. locations: 7
States / cities: Phoenix, Arizona • Sacramento, California • Atlanta, Georgia + 4 more

Conditions: Congenital Bleeding Disorder, Haemophilia B
Interventions: N9-GP, ALPROLIX®; Drug
Lead sponsor: Novo Nordisk A/S; Industry
Eligibility: 18 Years to 70 Years · Male only
Enrollment: 15 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2017
U.S. locations: 7
States / cities: Phoenix, Arizona • Chicago, Illinois • Peoria, Illinois + 4 more

Conditions: Healthy Participants, Hemophilia A, Hemophilia B, Factor VII Deficiency
Interventions: SR604, Placebo; Drug
Lead sponsor: Equilibra Bioscience LLC; Industry
Eligibility: 18 Years to 60 Years
Enrollment: 31 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2024 – 2026
U.S. locations: 9
States / cities: Glendale, California • Los Angeles, California • Chicago, Illinois + 6 more

Conditions: Hemophilia
Interventions: Fitusiran, Clotting factor concentrates (CFC) or bypassing agents (BPA), Antithrombin concentrate (ATIIIC); Drug
Lead sponsor: Sanofi; Industry
Eligibility: 12 Years and older · Male only
Enrollment: 91 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2023 – 2029
U.S. locations: 8
States / cities: Orange, California • Minneapolis, Minnesota • Hackensack, New Jersey + 5 more

Conditions: Hemophilia A, Hemophilia B
Interventions: Ataluren; Drug
Lead sponsor: PTC Therapeutics; Industry
Eligibility: 18 Years and older · Male only
Enrollment: 13 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2009 – 2011
U.S. locations: 7
States / cities: Peoria, Illinois • Indianapolis, Indiana • Worcester, Massachusetts + 4 more

Conditions: Hemophilia B
Interventions: CSL222 (AAV5-hFIXco-Padua); Genetic
Lead sponsor: CSL Behring; Industry
Eligibility: 18 Years and older
Enrollment: 35 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2024 – 2032
U.S. locations: 4
States / cities: San Diego, California • Ann Arbor, Michigan • Pittsburgh, Pennsylvania + 1 more

Conditions: Reversal of Coagulopathy
Interventions: Beriplex® P/N (Kcentra), Fresh frozen plasma; Biological
Lead sponsor: CSL Behring; Industry
Eligibility: 18 Years and older
Enrollment: 176 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2009 – 2013
U.S. locations: 16
States / cities: Newark, Delaware • Lexington, Kentucky • Boston, Massachusetts + 13 more

Conditions: Heparin-induced Thrombocytopenia
Interventions: Danaparoid Sodium, Argatroban; Drug
Lead sponsor: Aspen Global Incorporated; Industry
Eligibility: 2 Weeks to 100 Years
Enrollment: 7 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019 – 2022
U.S. locations: 5
States / cities: Gainesville, Florida • Minneapolis, Minnesota • Durham, North Carolina + 2 more

Conditions: Severe Hemophilia B
Interventions: rFIXFc; Biological
Lead sponsor: Bioverativ Therapeutics Inc.; Industry
Eligibility: Male only
Enrollment: 120 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2011 – 2017
U.S. locations: 10
States / cities: Phoenix, Arizona • Sacramento, California • Aurora, Colorado + 7 more

Conditions: Hemophilia B
Interventions: Not listed
Lead sponsor: Ultragenyx Pharmaceutical Inc; Industry
Eligibility: 18 Years and older · Male only
Enrollment: 6 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2017 – 2021
U.S. locations: 3
States / cities: Little Rock, Arkansas • Gainesville, Florida • Ann Arbor, Michigan

Conditions: Trauma Injury
Interventions: Prothrombin Complex Concentrate, Human, Placebo; Drug
Lead sponsor: Oregon Health and Science University; Other
Eligibility: 18 Years and older
Enrollment: 166 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2025
U.S. locations: 1
States / cities: Portland, Oregon

Conditions: Hemophilia B, Mucopolysaccharidosis I, Mucopolysaccharidosis II
Interventions: SB-318, SB-913, SB-FIX; Biological
Lead sponsor: Sangamo Therapeutics; Industry
Eligibility: 18 Years and older
Enrollment: 13 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2020 – 2026
U.S. locations: 5
States / cities: Oakland, California • Chicago, Illinois • New York, New York + 2 more

Conditions: Hemophilia A, Hemophilia B
Interventions: Liver Biopsy; Procedure
Lead sponsor: St. Jude Children's Research Hospital; Other
Eligibility: 18 Years to 80 Years · Male only
Enrollment: 8 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2026 – 2030
U.S. locations: 1
States / cities: Memphis, Tennessee

Conditions: Hemophilia A, Hemophilia B
Interventions: rFVIIIFc, rFIXFc, non-Fc FVIII replacement products, non-Fc FIX replacement products; Biological · Drug
Lead sponsor: Bioverativ Therapeutics Inc.; Industry
Eligibility: Male only
Enrollment: 3 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2015
U.S. locations: 2
States / cities: Los Angeles, California • East Lansing, Michigan

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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

Thrombin Generation in Neonatal Plasma After Cardiopulmonary Bypass

A Factor IX Gene Therapy Study (FIX-GT)

Efficacy and Safety of 4F-PCC (4-Factor Prothrombin Complex Concentrate) in Adult Patients Undergoing Complex Cardiovascular Surgery With Cardiopulmonary Bypass (CPB)

Dose-Escalation Study Of A Self Complementary Adeno-Associated Viral Vector For Gene Transfer in Hemophilia B

Hemlibra in Mild Hemophilia A

Safety and Effectiveness of Giroctocogene Fitelparvovec or Fidanacogene Elaparvovec in Patients With Hemophilia A or B Respectively

Study of Recombinant Factor IX Product, IB1001, in Previously Treated Pediatric Subjects With Hemophilia B

Safety and Dose Finding Study of DTX101 (AAVrh10FIX) in Adults With Moderate/Severe to Severe Hemophilia B

A Study to Investigate the Safety and Effectiveness of a Coagulation Factor IX Gene Insertion Therapy (REGV131-LNP1265) in Pediatric, Adolescent and Adult Participants With Hemophilia B

Study of Recombinant Coagulation Factor IX Fc Fusion Protein, BIIB029, in Previously Treated Pediatric Participants With Hemophilia B

A Trial Comparing Nonacog Beta Pegol (N9-GP) and ALPROLIX® in Patients With Haemophilia B

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 or Factor VII Deficiency)

A Study to Test a Medicine (Fitusiran) Injected Under the Skin for Preventing Bleeding Episodes in Male Adolescent or Adult Participants With Severe Hemophilia

Study of Ataluren (PTC124) in Hemophilia A and B

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)

An Open-label, Randomized, Multicenter Phase IIIb Study to Assess the Efficacy, Safety and Tolerance of BERIPLEX® P/N (Kcentra) Compared With Plasma for Rapid Reversal of Coagulopathy Induced by Vitamin K Antagonists in Subjects Requiring an Urgent Surgical Procedure

Open-Label, Randomised, Active Controlled, Multi-Centre Phase 3 Study Safety and Efficacy of Danaparoid vs Argatroban

Long-Term Safety and Efficacy of rFIXFc in the Prevention and Treatment of Bleeding Episodes in Previously Treated Participants With Hemophilia B

Long-Term Safety, Tolerability, and Efficacy of DTX101 (AAVrh10FIX) in Adults With Moderate/Severe to Severe Hemophilia B

Prehospital Kcentra for Hemorrhagic Shock

Long Term Follow-up (LTFU) of Subjects Who Received SB-318, SB-913, or SB-FIX

Liver Biopsy Following Gene Therapy For Hemophilia

Fc factOrs and Real-World hemophiliA Patient-ReporteD Outcomes