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Conditions: Hemophilia A
Interventions: Colorado Adult Joint Assessment Evaluation; Other
Lead sponsor: Bayer; Industry
Eligibility: 18 Years to 50 Years · Male only
Enrollment: 30 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2015
U.S. locations: 4
States / cities: Orange, California • Aurora, Colorado • Indianapolis, Indiana + 1 more

Conditions: Factor VIII Deficiency
Interventions: efanesoctocog alfa (BIVV001); Biological
Lead sponsor: Bioverativ, a Sanofi company; Industry
Eligibility: 12 Years and older
Enrollment: 159 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019 – 2022
U.S. locations: 8
States / cities: Los Angeles, California • San Diego, California • Gainesville, Florida + 4 more

Conditions: Hemophilia A
Interventions: NXT007; Drug
Lead sponsor: Hoffmann-La Roche; Industry
Eligibility: 2 Years to 59 Years · Male only
Enrollment: 60 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2023 – 2030
U.S. locations: 4
States / cities: Sacramento, California • Washington D.C., District of Columbia • Indianapolis, Indiana + 1 more

Conditions: Trisomy 13 Syndrome, Arthrogryposis Congenita Multiplex With Intestinal Atresia, Asparagine Synthetase Deficiency, CHARGE Syndrome, Early Infantile Epileptic Encephalopathy, FOXG1 Syndrome, KBG Syndrome, Noonan Syndrome, Severe Hemophilia A, Short Bowel Syndrome, Beta-Propeller Protein-Associated Neurodegeneration, Brain Injury of Prematurity With Periventricular Leukomalacia, Chromosome 17p13.3 Microdeletion Syndrome, Chromosome 1q43-1q44 Deletion, Cockayne Syndrome, Congenital Diaphragmatic Hernia, End-Stage Renal Disease With Cloacal Anomaly, Mitochondrial Depletion Disorder, Severe Factor VII Deficiency
Interventions: Family Centered pediatric palliative care for family caregivers of children with rare diseases.; Behavioral
Lead sponsor: Children's National Research Institute; Other
Eligibility: 12 Months to 99 Years
Enrollment: 480 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2025 – 2029
U.S. locations: 1
States / cities: Washington D.C., District of Columbia

Conditions: Hemophilia A, Hemophilia B
Interventions: PF-06741086; Drug
Lead sponsor: Pfizer; Industry
Eligibility: 12 Years to 74 Years · Male only
Enrollment: 189 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2020 – 2025
U.S. locations: 4
States / cities: Tampa, Florida • Iowa City, Iowa • New Hyde Park, New York + 1 more

Conditions: Congenital Bleeding Disorder, Haemophilia A
Interventions: turoctocog alfa pegol; Drug
Lead sponsor: Novo Nordisk A/S; Industry
Eligibility: 12 Years and older · Male only
Enrollment: 21 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2016 – 2017
U.S. locations: 5
States / cities: Torrance, California • Iowa City, Iowa • Baltimore, Maryland + 2 more

Conditions: Hemophilia A
Interventions: rVIII-SingleChain, Octocog alfa; Biological
Lead sponsor: CSL Behring; Industry
Eligibility: 12 Years to 65 Years · Male only
Enrollment: 175 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2012 – 2014
U.S. locations: 9
States / cities: Sacramento, California • San Diego, California • Aurora, Colorado + 6 more

Conditions: Hemophilia B
Interventions: PF-06838435/ fidanacogene elaparvovec; Biological
Lead sponsor: Pfizer; Industry
Eligibility: 18 Years to 65 Years · Male only
Enrollment: 51 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019 – 2031
U.S. locations: 17
States / cities: San Francisco, California • Aurora, Colorado • Indianapolis, Indiana + 3 more

Conditions: Hemophilia A, Factor VIII
Interventions: Eloctate, Adynovate, Emicizumab; Drug
Lead sponsor: University of Texas Southwestern Medical Center; Other
Eligibility: Up to 17 Years
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2027
U.S. locations: 1
States / cities: Dallas, Texas

Conditions: Hemophilia A
Interventions: PF-07055480 (giroctocogene fitelparovec): Recombinant AAV2/6 Human Factor VIII Gene Therapy; Biological
Lead sponsor: Pfizer; Industry
Eligibility: 18 Years to 64 Years · Male only
Enrollment: 77 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2020 – 2026
U.S. locations: 19
States / cities: Mountain View, California • Oakland, California • Palo Alto, California + 5 more

Conditions: Hemophilia A
Interventions: SIG-001; Combination Product
Lead sponsor: Eli Lilly and Company; Industry
Eligibility: 18 Years and older · Male only
Enrollment: 3 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2020 – 2023
U.S. locations: 3
States / cities: Indianapolis, Indiana • Boston, Massachusetts • Seattle, Washington

Conditions: Severe Hemophilia A
Interventions: Eloctate Prophylaxis, Emicizumab Prophylaxis, Eloctate ITI plus Emicizumab, Eloctate ITI; Drug
Lead sponsor: Margaret Ragni; Other
Eligibility: 4 Months to 99 Years · Male only
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2020 – 2027
U.S. locations: 1
States / cities: Pittsburgh, Pennsylvania

Conditions: Hemophilia A
Interventions: BAY94-9027; Biological
Lead sponsor: Bayer; Industry
Eligibility: 12 Years to 65 Years · Male only
Enrollment: 145 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2012 – 2019
U.S. locations: 14
States / cities: Tucson, Arizona • Sacramento, California • San Diego, California + 11 more

Conditions: Treatment of Bleeding, Prophylaxis of Bleeding, Hemophilia A, Children
Interventions: Damoctocog alfa pegol (Jivi, BAY94-9027); Biological
Lead sponsor: Bayer; Industry
Eligibility: 7 Years to 11 Years · Male only
Enrollment: 36 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2025
U.S. locations: 1
States / cities: Orlando, Florida

Conditions: Hemophilia A
Interventions: Recombinant Protein-Free Factor VIII (rAHF-PFM); Drug
Lead sponsor: Baxalta now part of Shire; Industry
Eligibility: 18 Years to 70 Years
Enrollment: 85 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2006 – 2015
U.S. locations: 10
States / cities: Los Angeles, California • Washington D.C., District of Columbia • Chicago, Illinois + 7 more

Conditions: Haemostasis, Haemophilia A
Interventions: Concizumab, Turoctocog alfa; Drug
Lead sponsor: Novo Nordisk A/S; Industry
Eligibility: 18 Years and older · Male only
Enrollment: 36 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2017 – 2020
U.S. locations: 5
States / cities: Los Angeles, California • Indianapolis, Indiana • Oklahoma City, Oklahoma + 2 more

Conditions: Hemophilia A
Interventions: BAY2599023 (DTX201); Drug
Lead sponsor: Bayer; Industry
Eligibility: 18 Years and older · Male only
Enrollment: 11 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2026
U.S. locations: 3
States / cities: Little Rock, Arkansas • Ann Arbor, Michigan • Madison, Wisconsin

Conditions: Hemophilia A
Interventions: SB-525 (PF-07055480); Biological
Lead sponsor: Pfizer; Industry
Eligibility: 18 Years and older · Male only
Enrollment: 13 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2017 – 2024
U.S. locations: 22
States / cities: Little Rock, Arkansas • Duarte, California • Sacramento, California + 7 more

Conditions: Severe Hemophilia A
Interventions: Human cl rhFVIII; Biological
Lead sponsor: Octapharma; Industry
Eligibility: Male only
Enrollment: 110 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2013 – 2019
U.S. locations: 3
States / cities: Sacramento, California • St. Petersburg, Florida • Boston, Massachusetts

Conditions: Severe Hemophilia A
Interventions: Human-cl rhFVIII; Biological
Lead sponsor: Octapharma; Industry
Eligibility: 12 Years to 65 Years · Male only
Enrollment: 3 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2011 – 2012
U.S. locations: 1
States / cities: Washington D.C., District of Columbia

Conditions: Hemophilia A, Hemophilia B
Interventions: PF-06741086; Drug
Lead sponsor: Pfizer; Industry
Eligibility: 1 Year to 74 Years · Male only
Enrollment: 245 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2030
U.S. locations: 4
States / cities: Iowa City, Iowa • New Hyde Park, New York • Philadelphia, Pennsylvania + 1 more

Conditions: Hemophilia A
Interventions: Fitusiran (SAR439774), Clotting factor concentrates (CFC) or bypassing agents (BPA), Antithrombin concentrate (ATIIIC), Emicizumab; Drug · Biological
Lead sponsor: Sanofi; Industry
Eligibility: 12 Years and older · Male only
Enrollment: 20 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2024 – 2030
U.S. locations: 1
States / cities: Los Angeles, California

Conditions: Hemophilia A
Interventions: Antihemophilic factor, recombinant, manufactured protein-free; Biological
Lead sponsor: Baxalta now part of Shire; Industry
Eligibility: 12 Years to 65 Years
Enrollment: 38 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2006 – 2007
U.S. locations: 8
States / cities: Little Rock, Arkansas • Los Angeles, California • Peoria, Illinois + 5 more

Conditions: Hemophilia A, Hemophilia B
Interventions: Not listed
Lead sponsor: Weill Medical College of Cornell University; Other
Eligibility: 1 Month and older · Female only
Enrollment: 22 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2005 – 2010
U.S. locations: 18
States / cities: Duarte, California • Aurora, Colorado • Washington D.C., District of Columbia + 15 more

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Colorado Adult Joint Assessment Scale (CAJAS) Validation

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

A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of NXT007 in Persons With Severe or Moderate Hemophilia A

Palliative Care Needs of Children With Rare Diseases and Their Families

Study of the Efficacy and Safety PF-06741086 in Adult and Teenage Participants With Severe Hemophilia A or Moderately Severe to Severe Hemophilia B

A 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 A

An Open-label Safety, Efficacy and Pharmacokinetic Study of a Recombinant FVIII Compared to Recombinant Human Antihemophilic FVIII in Patients With Severe Hemophilia A

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

POCUS: Hemostatic Potential and Joint Health in Patients With Severe Hemophilia A on Novel Replacement Therapies

Study to Evaluate the Efficacy and Safety of PF-07055480 / Giroctocogene Fitelparvovec Gene Therapy in Moderately Severe to Severe Hemophilia A Adults

Safety & Efficacy of Encapsulated Allogeneic FVIII Cell Therapy in Haemophilia A

Hemophilia Inhibitor Clinical Trials (INHIBIT) Platform

A Trial Investigating Safety and Efficacy of Treatment With BAY94-9027 in Severe Hemophilia A

A Study to Learn How Safe the Study Treatment BAY94-9027 is and How it Affects the Body in Previously Treated Children Aged 7 to Less Than 12 Years With Severe Hemophilia A, a Genetic Bleeding Disorder That is Caused by the Lack of a Protein Called Clotting Factor 8 (FVIII) in the Blood

Phase 3/4 Study of a Recombinant Protein-Free Factor VIII (rAHF-PFM): Comparison of Continuous Infusion Versus Intermittent Bolus Infusion in Hemophilia A Subjects Undergoing Major Orthopedic Surgery

A Trial Evaluating Efficacy and Safety of Prophylactic Administration of Concizumab in Patients With Severe Haemophilia A Without Inhibitors

Study to Test the Safety and How Well Patients With Severe Hemophilia A Respond to Treatment With BAY 2599023 (DTX 201), a Drug Therapy That Delivers a Healthy Version of the Defective Factor VIII Gene Into the Nucleus of Liver Cells Using an Altered, Non-infectious Virus (AAV) as a "Shuttle"

A Study of Recombinant AAV2/6 Human Factor 8 Gene Therapy SB-525 (PF-07055480) in Subjects With Severe Hemophilia A

Human Cell Line-derived Recombinant Factor VIII (Human-cl-rhFVIII) in Previously Untreated Patients

Study to Investigate the Long-term Efficacy and Safety of Human-cl rhFVIII in Previously Treated Patients (PTPs)

Open-Label Extension Study of Marstacimab in Hemophilia Participants With or Without Inhibitors

A Study to Test a Medicine (Fitusiran) for Preventing Bleeds in People With Severe Hemophilia Who Previously Received Preventive Treatment With Emicizumab

Dose-Response Study of Recombinant Factor VIII Manufactured Protein-Free (rAHF-PFM) in Patients With Hemophilia A

Females With Severe or Moderate Hemophilia A or B: A Multi-Center Study