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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
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: 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: 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
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: 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: Hemophilia A
Interventions: rFVIIIFc; Biological
Lead sponsor: Bioverativ, a Sanofi company; Industry
Eligibility: Up to 5 Years · Male only
Enrollment: 108 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2015 – 2019
U.S. locations: 20
States / cities: Little Rock, Arkansas • Duarte, California • Sacramento, California + 16 more

Conditions: Hemophilia A
Interventions: ReFacto AF; Biological
Lead sponsor: Wyeth is now a wholly owned subsidiary of Pfizer; Industry
Eligibility: 12 Years and older · Male only
Enrollment: 30 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2006 – 2008
U.S. locations: 8
States / cities: La Jolla, California • Detroit, Michigan • Chapel Hill, North Carolina + 4 more

Conditions: Hemophilia A, Factor VIII (FVIII)
Interventions: Half-life factor VIII based replacement therapy, Non-FVIII based replacement therapy; Drug
Lead sponsor: University of Texas Southwestern Medical Center; Other
Eligibility: 6 Months and older
Enrollment: 60 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2026 – 2033
U.S. locations: 1
States / cities: Dallas, Texas

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: 14
States / cities: Los Angeles, California • Sacramento, California • San Francisco, California + 9 more

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: Hemophilia B, Allergic Reactions
Interventions: Not listed
Lead sponsor: Wyeth is now a wholly owned subsidiary of Pfizer; Industry
Eligibility: Not listed
Enrollment: 166 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2005 – 2007
U.S. locations: 15
States / cities: Phoenix, Arizona • Orange, California • Atlanta, Georgia + 12 more

Conditions: Hemophilia B, Hemophilia A
Interventions: Standard of Care FIX Replacement therapy, Standard of Care FVIII Replacement therapy; Drug
Lead sponsor: Pfizer; Industry
Eligibility: 18 Years to 64 Years · Male only
Enrollment: 212 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2024
U.S. locations: 13
States / cities: Palo Alto, California • San Francisco, California • Stanford, California + 7 more

Conditions: Hemophilia A
Interventions: Kogenate (BAY 14-2222); Drug
Lead sponsor: Bayer; Industry
Eligibility: 12 Years to 60 Years · Male only
Enrollment: 1 participant
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2006
U.S. locations: 2
States / cities: Detroit, Michigan • Las Vegas, Nevada

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 B
Interventions: SB-FIX; Biological
Lead sponsor: Sangamo Therapeutics; Industry
Eligibility: 18 Years and older · Male only
Enrollment: 1 participant
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2016 – 2021
U.S. locations: 1
States / cities: Washington D.C., District of Columbia

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 A
Interventions: efanesoctocog alfa (BIVV001); Drug
Lead sponsor: Bioverativ, a Sanofi company; Industry
Eligibility: Up to 11 Years · Male only
Enrollment: 74 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2023
U.S. locations: 10
States / cities: Los Angeles, California • Gainesville, Florida • Atlanta, Georgia + 7 more

Conditions: Hemophilia A, Hemophilia B
Interventions: fitusiran, Bypassing agents; Drug
Lead sponsor: Genzyme, a Sanofi Company; Industry
Eligibility: 12 Years and older · Male only
Enrollment: 60 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2021
U.S. locations: 12
States / cities: Phoenix, Arizona • Los Angeles, California • Orange, California + 9 more

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Palliative Care Needs of Children With Rare Diseases and Their Families

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

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

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

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

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

BAY81-8973 Pediatric Safety and Efficacy Trial

An Open Label Study to Determine the Safety and Efficacy of Replacement Factor VIII Protein (Known as rFVIIIFc) in Previously Untreated Males With Severe Hemophilia A

Study Evaluating B-Domain Deleted Recombinant Factor VIII (BDDrFVIII, ReFacto AF) in Patients With Hemophilia A Undergoing Elective Major Surgery

TG-INSIGHT With Joint POCUS, Hemostatic Potential in Patients With Severe Hemophilia A on Novel Replacement and Substitution FVIII Therapies

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

Colorado Adult Joint Assessment Scale (CAJAS) Validation

Study to Describe the Allergic Reactions to Factor IX in Patients With Hemophilia B

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%)

Assessment of the Risk of Inhibitor Formation in Previously Treated 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

Ascending Dose Study of Genome Editing by Zinc Finger Nuclease Therapeutic SB-FIX in Subjects With Severe Hemophilia B

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

Safety, Efficacy and PK of BIVV001 in Pediatric Patients With Hemophilia A

A Study of Fitusiran (ALN-AT3SC) in Severe Hemophilia A and B Patients With Inhibitors