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Conditions: Hemophilia
Interventions: Fitusiran; Drug
Lead sponsor: Genzyme, a Sanofi Company; Industry
Eligibility: 1 Year to 11 Years · Male only
Enrollment: 32 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2020 – 2026
U.S. locations: 4
States / cities: Los Angeles, California • Hackensack, New Jersey • Cleveland, Ohio + 1 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

Conditions: Bleeding Disorder, Von Willebrand Disease (VWD)
Interventions: Not listed
Lead sponsor: Children's Hospital Medical Center, Cincinnati; Other
Eligibility: Not listed
Enrollment: 1,000 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2025 – 2027
U.S. locations: 1
States / cities: Cincinnati, Ohio

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: Adult Acute Myeloid Leukemia in Remission, Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities, Adult Acute Myeloid Leukemia With Del(5q), Adult Acute Myeloid Leukemia With Inv(16)(p13;q22), Adult Acute Myeloid Leukemia With t(15;17)(q22;q12), Adult Acute Myeloid Leukemia With t(16;16)(p13;q22), Adult Acute Myeloid Leukemia With t(8;21)(q22;q22), Childhood Acute Myeloid Leukemia in Remission, Childhood Myelodysplastic Syndromes, de Novo Myelodysplastic Syndromes, Essential Thrombocythemia, Myelodysplastic Syndrome With Isolated Del(5q), Polycythemia Vera, Previously Treated Myelodysplastic Syndromes, Primary Myelofibrosis, Recurrent Adult Acute Myeloid Leukemia, Recurrent Childhood Acute Myeloid Leukemia, Secondary Acute Myeloid Leukemia, Secondary Myelodysplastic Syndromes, Secondary Myelofibrosis, Untreated Adult Acute Myeloid Leukemia, Untreated Childhood Acute Myeloid Leukemia and Other Myeloid Malignancies
Interventions: cyclophosphamide, busulfan, tacrolimus, methotrexate, cytogenetic analysis, flow cytometry, pharmacological study, pharmacogenomic studies, peripheral blood stem cell transplantation, allogeneic hematopoietic stem cell transplantation; Drug · Genetic · Other + 1 more
Lead sponsor: Fred Hutchinson Cancer Center; Other
Eligibility: Up to 65 Years
Enrollment: 52 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2006 – 2013
U.S. locations: 1
States / cities: Seattle, Washington

Conditions: Focal Segmental Glomerulosclerosis, Minimal Change Disease, Immunoglobulin A Nephropathy, IgA Vasculitis, Alport Syndrome
Interventions: Sparsentan; Drug
Lead sponsor: Travere Therapeutics, Inc.; Industry
Eligibility: 1 Year to 17 Years
Enrollment: 67 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2027
U.S. locations: 23
States / cities: Los Angeles, California • Wilmington, Delaware • Miami, Florida + 17 more

Conditions: Chronic Myeloproliferative Disorders, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic Syndromes
Interventions: rabbit anti-thymocyte globulin (ATG), therapeutic allogeneic lymphocytes, busulfan, fludarabine phosphate, tacrolimus, allogeneic hematopoietic stem cell transplantation, peripheral blood stem cell transplantation, methotrexate; Biological · Drug · Procedure
Lead sponsor: UNC Lineberger Comprehensive Cancer Center; Other
Eligibility: 18 Years to 55 Years
Enrollment: 54 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2005 – 2015
U.S. locations: 1
States / cities: Chapel Hill, North Carolina

Conditions: Hemophilia A
Interventions: ReFacto; Drug
Lead sponsor: Wyeth is now a wholly owned subsidiary of Pfizer; Industry
Eligibility: 6 Years and older · Male only
Enrollment: 15 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2003 – 2007
U.S. locations: 4
States / cities: Los Angeles, California • New Brunswick, New Jersey • Cincinnati, Ohio + 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: 186 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2012 – 2018
U.S. locations: 31
States / cities: Phoenix, Arizona • Long Beach, California • Sacramento, California + 26 more

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: 14
States / cities: Rancho Cordova, California • Sacramento, California • San Francisco, California + 5 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 A
Interventions: PEGylated Recombinant factor VIII (rFVIII); Biological
Lead sponsor: Baxalta now part of Shire; Industry
Eligibility: 12 Years to 65 Years · Male only
Enrollment: 30 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2013 – 2016
U.S. locations: 8
States / cities: Gainesville, Florida • Peoria, Illinois • Louisville, Kentucky + 5 more

Conditions: Hemophilia A
Interventions: BAX 888; Drug
Lead sponsor: Baxalta now part of Shire; Industry
Eligibility: 18 Years to 75 Years · Male only
Enrollment: 4 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2024
U.S. locations: 8
States / cities: Phoenix, Arizona • Los Angeles, California • Sacramento, California + 5 more

Conditions: Severe Combined Immunodeficiency (SCID), Immunodeficiency With Predominant T-cell Defect, Unspecified, Severe Chronic Neutropenia, Chronic Granulomatous Disease (CGD), Hyper IgE Syndromes, Hyper IgM Deficiencies, Wiskott-Aldrich Syndrome, Mendelian Susceptibility to Mycobacterial Disease, Common Variable Immune Deficiency (CVID)
Interventions: CD3/CD19 negative allogeneic hematopoietic stem cells; Biological
Lead sponsor: Paul Szabolcs; Other
Eligibility: 5 Years to 45 Years
Enrollment: 16 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2013 – 2027
U.S. locations: 1
States / cities: Pittsburgh, Pennsylvania

Conditions: Haemostasis, Congenital Bleeding Disorder, Haemophilia B
Interventions: No treatment given; Other
Lead sponsor: Novo Nordisk A/S; Industry
Eligibility: 18 Years and older
Enrollment: 449 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2015
U.S. locations: 1
States / cities: Plainsboro, New Jersey

Conditions: Primary Immunodeficiency Diseases (PID), Immune Thrombocytopenic Purpura (ITP), Kawasaki Syndrome
Interventions: Immune Globulin Intravenous (Human), 10%; Biological
Lead sponsor: Baxalta now part of Shire; Industry
Eligibility: 24 Months and older
Enrollment: 61 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2002 – 2003
U.S. locations: 11
States / cities: Los Angeles, California • Stanford, California • Englewood, Colorado + 8 more

Conditions: Haemophilia A, Haemophilia B, Obesity
Interventions: No treatment given; Other
Lead sponsor: Novo Nordisk A/S; Industry
Eligibility: 18 Years and older
Enrollment: 317 participants
Timeline: 2017 – 2018
U.S. locations: 1
States / cities: Plainsboro, New Jersey

Conditions: Blood Coagulation Disorders, Inherited, Thrombotic Disorder
Interventions: Not listed
Lead sponsor: The University of Texas Health Science Center, Houston; Other
Eligibility: 1 Day to 98 Years
Enrollment: 1,000 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2002 – 2028
U.S. locations: 2
States / cities: Houston, Texas

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: Chronic Myeloproliferative Disorders, Leukemia, Lymphoma, Lymphoproliferative Disorder, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Neoplasms, Pain, Precancerous Condition, Psychosocial Effects of Cancer and Its Treatment
Interventions: music therapy; Procedure
Lead sponsor: Wake Forest University Health Sciences; Other
Eligibility: 18 Years to 120 Years
Enrollment: 65 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2006 – 2010
U.S. locations: 1
States / cities: Winston-Salem, North Carolina

Conditions: Hemophilia A, Hemophilia B
Interventions: Virtual Reality Distraction, Standard of Care Distraction; Other
Lead sponsor: Nationwide Children's Hospital; Other
Eligibility: 6 Years to 19 Years
Enrollment: 25 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2016
U.S. locations: 1
States / cities: Columbus, Ohio

Conditions: Hemophilia A, Bleeding Disorder
Interventions: Not listed
Lead sponsor: Oregon Health and Science University; Other
Eligibility: 10 Years to 65 Years · Female only
Enrollment: 256 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2022
U.S. locations: 1
States / cities: Portland, Oregon

Conditions: Immunologic Deficiency Syndromes, Chediak-Higashi Syndrome, Common Variable Immunodeficiency, Graft Versus Host Disease, X-Linked Lymphoproliferative Syndrome, Familial Erythrophagocytic Lymphohistiocytosis, Hemophagocytic Lymphohistiocytosis, X-linked Agammaglobulinemia, Wiskott-Aldrich Syndrome, Chronic Granulomatous Disease, X-linked Hyper IgM Syndrome, Severe Combined Immunodeficiency, Leukocyte Adhesion Deficiency Syndrome, Virus-Associated Hemophagocytic Syndrome
Interventions: anti-thymocyte globulin, busulfan, cyclophosphamide, cyclosporine, etoposide, methotrexate, methylprednisolone, prednisone, Allogeneic Bone Marrow Transplantation; Drug · Procedure
Lead sponsor: Fairview University Medical Center; Other
Eligibility: Up to 35 Years
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2000 – 2002
U.S. locations: 1
States / cities: Minneapolis, Minnesota

Conditions: Severe Hemophilia A, Moderate Hemophilia A
Interventions: Emicizumab; Drug
Lead sponsor: Hoffmann-La Roche; Industry
Eligibility: 13 Years to 69 Years
Enrollment: 136 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2027
U.S. locations: 3
States / cities: Los Angeles, California • Miami, Florida • Oklahoma City, Oklahoma

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Fitusiran Prophylaxis in Male Pediatric Subjects Aged 1 to Less Than 12 Years With Hemophilia A or B

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

The Bleeding Assessment Tool (BAT)

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

Cyclophosphamide and Busulfan Followed by Donor Stem Cell Transplant in Treating Patients With Myelofibrosis, Acute Myeloid Leukemia, or Myelodysplastic Syndrome

Study of Sparsentan Treatment in Pediatrics With Proteinuric Glomerular Diseases

Allogeneic Hematopoietic Cell Transplantation for Patients With Busulfex-based Regimen

Study Evaluating ReFacto® in Hemophilia A Undergoing Major Surgery

Evaluation of Safety and Efficacy, Including Pharmacokinetics, of NNC 0129-0000-1003 When Administered for Treatment and Prophylaxis of Bleeding in Subjects With Haemophilia A

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

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

Phase 3 Efficacy and Safety Study of BAX 855 in Severe Hemophilia A Patients Undergoing Surgical Procedures

A Study of BAX 888 in Male Adults With Severe Hemophilia A

Sequential Cadaveric Lung and Bone Marrow Transplant for Immune Deficiency Diseases

Bridging Hemophilia B Experiences, Results and Opportunities Into Solutions (B-HERO-S)

Safety and Efficacy Study of a 10% Intravenous Immune Globulin Solution in Subjects With Primary Immunodeficiency Disorders

Awareness, Care, and Treatment In Obesity Management of Patients With Hemophilia (ACTION) to Inform Hemophilia Obesity Patient Empowerment (HOPE)

Evaluation of Hemostasis in Bleeding and Thrombotic Disorders

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

Music in Reducing Anxiety and Pain in Adult Patients Undergoing Bone Marrow Biopsy for Hematologic Cancers or Other Diseases

Virtual Reality for Hemophilia

Characterizing the Impact and Treatment of Reproductive Tract Bleeding on Women and Post-menarchal Girls With Bleeding Disorders

Allogeneic Bone Marrow Transplantation in Patients With Primary Immunodeficiencies

A Study to Evaluate Overall Health, Physical Activity, and Joint Outcomes in Participants With Severe or Moderate Hemophilia A Without Factor VIII Inhibitors on Emicizumab Prophylaxis