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Conditions: Hemophilia A
Interventions: SPK-8011; Genetic
Lead sponsor: Spark Therapeutics, Inc.; Industry
Eligibility: 18 Years and older · Male only
Enrollment: 25 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2017 – 2023
U.S. locations: 11
States / cities: Sacramento, California • Gainesville, Florida • Boston, Massachusetts + 7 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
Interventions: Antihemophilic factor, recombinant, manufactured protein-free (rAHF-PFM). (Antihemophilic factor is also known as Factor VIII); Biological
Lead sponsor: Baxalta now part of Shire; Industry
Eligibility: 2 Years to 65 Years
Enrollment: 52 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2008 – 2009
U.S. locations: 11
States / cities: Washington D.C., District of Columbia • Atlanta, Georgia • Lexington, Kentucky + 8 more

Conditions: Hemophilia A With Inhibitor
Interventions: efanesoctacog alpha, Emicizumab; Drug
Lead sponsor: Versiti Blood Health; Other
Eligibility: 0 Years to 17 Years
Enrollment: 15 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2025 – 2032
U.S. locations: 1
States / cities: Milwaukee, Wisconsin

Conditions: Hemophilia A
Interventions: Not listed
Lead sponsor: Octapharma; Industry
Eligibility: Male only
Enrollment: 80 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2015 – 2020
U.S. locations: 5
States / cities: Gainesville, Florida • Miami, Florida • New Orleans, Louisiana + 2 more

Conditions: Hemophilia A
Interventions: Emicizumab, FVIII; Drug
Lead sponsor: Wayne State University; Other
Eligibility: 6 Years to 19 Years
Enrollment: 72 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2023 – 2029
U.S. locations: 10
States / cities: Little Rock, Arkansas • Palo Alto, California • San Diego, California + 7 more

Conditions: Cardiac Disease, Surgery, Bleeding
Interventions: FEIBA, FFP; Drug
Lead sponsor: Northwell Health; Other
Eligibility: 18 Years and older
Enrollment: 140 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2025 – 2029
U.S. locations: 1
States / cities: Manhasset, New York

Conditions: Congenital Bleeding Disorder, Haemophilia A
Interventions: turoctocog alfa; Drug
Lead sponsor: Novo Nordisk A/S; Industry
Eligibility: Up to 11 Years · Male only
Enrollment: 65 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2010 – 2011
U.S. locations: 18
States / cities: Phoenix, Arizona • Orange, California • Torrance, California + 15 more

Conditions: Hemophilia A and B, Liver Transplantation
Interventions: Not listed
Lead sponsor: American Thrombosis and Hemostasis Network; Network
Eligibility: 18 Years and older
Enrollment: 86 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2020 – 2022
U.S. locations: 15
States / cities: Los Angeles, California • San Francisco, California • Peoria, Illinois + 12 more

Conditions: Hemophilia A
Interventions: Moroctocog alfa (AF-CC); Biological
Lead sponsor: Pfizer; Industry
Eligibility: 6 Months to 15 Years · Male only
Enrollment: 66 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2007 – 2018
U.S. locations: 7
States / cities: Cincinnati, Ohio • Portland, Oregon • Dallas, Texas + 1 more

Conditions: Hemophilia B
Interventions: AAV5-hFIXco-Padua, Factor IX (FIX); Genetic · Biological
Lead sponsor: CSL Behring; Industry
Eligibility: 18 Years and older · Male only
Enrollment: 67 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2025
U.S. locations: 19
States / cities: Phoenix, Arizona • Little Rock, Arkansas • Los Angeles, California + 14 more

Conditions: Congenital Bleeding Disorder, Haemophilia A
Interventions: turoctocog alfa; Drug
Lead sponsor: Novo Nordisk A/S; Industry
Eligibility: 0 Years to 6 Years · Male only
Enrollment: 60 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2012 – 2018
U.S. locations: 25
States / cities: Phoenix, Arizona • Long Beach, California • Sacramento, California + 20 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 – 2033
U.S. locations: 4
States / cities: Sacramento, California • Washington D.C., District of Columbia • Indianapolis, Indiana + 1 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: Hemophilia A
Interventions: BAY94-9027; Biological
Lead sponsor: Bayer; Industry
Eligibility: Up to 12 Years · Male only
Enrollment: 73 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2013 – 2020
U.S. locations: 7
States / cities: Sacramento, California • Pensacola, Florida • Cincinnati, Ohio + 4 more

Conditions: Hemophilia A, Immune Tolerance
Interventions: FVIII, Emicizumab; Drug
Lead sponsor: Children's Hospital Los Angeles; Other
Eligibility: 0 Years to 12 Years · Male only
Enrollment: 52 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2020 – 2025
U.S. locations: 1
States / cities: Los Angeles, California

Conditions: Hemophilia A
Interventions: Antihemophilic factor, recombinant, manufactured protein-free; Drug
Lead sponsor: Baxalta now part of Shire; Industry
Eligibility: 7 Years to 65 Years
Enrollment: 82 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2006 – 2010
U.S. locations: 9
States / cities: Los Angeles, California • Chicago, Illinois • Indianapolis, Indiana + 6 more

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: Hemophilia A
Interventions: Recomb. Factor VIII (Kogenate FS Liposome, BAY79-4980), Recombinant Factor VIII (Kogenate FS, BAY14-2222); Biological
Lead sponsor: Bayer; Industry
Eligibility: 12 Years to 60 Years · Male only
Enrollment: 27 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2005 – 2006
U.S. locations: 2
States / cities: Davis, California • Orange, California

Conditions: Hemophilia A
Interventions: HEMLIBRA; Drug
Lead sponsor: Montefiore Medical Center; Other
Eligibility: Up to 36 Months
Enrollment: 71 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2024 – 2025
U.S. locations: 10
States / cities: Hartford, Connecticut • New Haven, Connecticut • Lebanon, New Hampshire + 7 more

Conditions: Hemophilia A, Hemophilia B
Interventions: BAY86-6150, eptacog alfa [activated]; Drug
Lead sponsor: Bayer; Industry
Eligibility: 12 Years to 62 Years · Male only
Enrollment: 10 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2012 – 2014
U.S. locations: 2
States / cities: Sacramento, California • Chattanooga, Tennessee

Conditions: Haemophilia A
Interventions: Recombinant Factor VIII (Kovaltry, BAY81-8973); Biological
Lead sponsor: Bayer; Industry
Eligibility: 0 Years to 12 Years · Male only
Enrollment: 94 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2011 – 2020
U.S. locations: 4
States / cities: New Orleans, Louisiana • Cincinnati, Ohio • Cleveland, Ohio + 1 more

Conditions: Mild Hereditary Factor VIII Deficiency Disease Without Inhibitor, Moderate Hereditary Factor VIII Deficiency Disease Without Inhibitor, Hemophilia A
Interventions: Emicizumab; Drug
Lead sponsor: Hoffmann-La Roche; Industry
Eligibility: Not listed
Enrollment: 73 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2020 – 2025
U.S. locations: 5
States / cities: Los Angeles, California • Atlanta, Georgia • Indianapolis, Indiana + 2 more

Conditions: Hemophilia A
Interventions: Moroctocog alfa(AF-CC), Blood draw for laboratory testing; Biological · Procedure
Lead sponsor: Pfizer; Industry
Eligibility: 12 Years and older · Male only
Enrollment: 12 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2009 – 2011
U.S. locations: 5
States / cities: Los Angeles, California • Washington D.C., District of Columbia • Detroit, Michigan + 2 more

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A Gene Transfer Study for Hemophilia A

Palliative Care Needs of Children With Rare Diseases and Their Families

Pharmacokinetic Study of ADVATE Reconstituted in 2 mL Sterile Water for Injection

Understanding Treatment Outcomes and Immunologic Mechanisms in Altuviiio Immune Tolerance Induction

Non-interventional Post-authorisation Study to Document the Immunogenicity, Safety, and Efficacy of NUWIQ

Prevention of Bleeding in Patients With Moderate and Severe Hemophilia A Playing Sports: A Comparison Between Factor VIII and Emicizumab Prophylaxis

Factor VIII Inhibitor Bypass Activity (FEIBA) Versus Fresh Frozen Plasma As First Line Therapy For Bleeding After Cardiac Surgery

Safety and Efficacy of Turoctocog Alfa in Previously Treated Male Children With Haemophilia A

ATHN 11: Liver Transplantation Outcomes Study

Study Evaluating Prophylaxis Treatment & Characterizing Efficacy, Safety, & PK Of B-Domain Deleted Recombinant FVIII

HOPE-B: Trial of AMT-061 in Severe or Moderately Severe Hemophilia B Patients

Safety and Efficacy of Turoctocog Alfa in Prevention and Treatment of Bleeds in Previously Untreated Children With Haemophilia A

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

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

Safety and Efficacy of BAY94-9027 in Previously Treated Male Children With Haemophilia A

Preventing Inhibitor Recurrence Indefinitely

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

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

Pharmacokinetics and Safety of a Single Intravenous Infusion of BAY 79-4980

Real World Use of Emicizumab in Infants and Children Ages 0-3 Years With Hemophilia A

A Phase 2/ 3 Trial to Evaluate the Efficacy and Safety of BAY86-6150

BAY81-8973 Pediatric Safety and Efficacy Trial

A Study to Evaluate the Safety, Efficacy, Pharmacokinetics and Pharmacodynamics of Emicizumab in Participants With Mild or Moderate Hemophilia A Without FVIII Inhibitors

Study Evaluating The Safety Of Xyntha In Usual Care Settings