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Conditions: Hemophilia
Interventions: rFVIII; Drug
Lead sponsor: Craig Seaman; Other
Eligibility: 18 Years and older · Male only
Enrollment: 30 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2015 – 2020
U.S. locations: 1
States / cities: Pittsburgh, Pennsylvania

Conditions: Healthy Volunteers, Anemia, Sickle Cell
Interventions: AG-946, AG-946 Matched Placebo; Drug
Lead sponsor: Agios Pharmaceuticals, Inc.; Industry
Eligibility: 18 Years to 70 Years
Enrollment: 122 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2020 – 2023
U.S. locations: 8
States / cities: La Jolla, California • Boston, Massachusetts • New York, New York + 4 more

Conditions: Vasculitis, Behcet's Disease, CNS Vasculitis, Cryoglobulinemic Vasculitis, Eosinophilic Granulomatosis With Polyangiitis (EGPA), Churg-Strauss Syndrome (CSS), Granulomatosis With Polyangiitis (GPA), Wegener's Granulomatosis, IgA Vasculitis, Henoch-Schoenlein Purpura (HSP), Microscopic Polyangiitis (MPA), Polyarteritis Nodosa (PAN), Takayasu Arteritis (TAK), Urticarial Vasculitis, Systemic Vasculitis
Interventions: Online questionnaires; Other
Lead sponsor: University of Pennsylvania; Other
Eligibility: 18 Years to 50 Years · Female only
Enrollment: 100 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2015 – 2027
U.S. locations: 1
States / cities: Tampa, Florida

Conditions: Sickle Cell Disease
Interventions: Dextromethorphan, Codeine, Morphine; Drug
Lead sponsor: Children's National Research Institute; Other
Eligibility: 7 Years to 18 Years
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2006 – 2012
U.S. locations: 1
States / cities: Washington D.C., District of Columbia

Conditions: Leukemia, Myelodysplastic Syndromes
Interventions: medical chart review, questionnaire administration, quality-of-life assessment; Other · Procedure
Lead sponsor: Fred Hutchinson Cancer Center; Other
Eligibility: 18 Years to 60 Years
Enrollment: 96 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2008 – 2011
U.S. locations: 2
States / cities: Portland, Oregon • Seattle, Washington

Conditions: Cord Blood Transplant, Hematologic Malignancy, Inherited Metabolic Disease
Interventions: TXA127; Drug
Lead sponsor: Tarix Pharmaceuticals; Industry
Eligibility: 6 Months to 20 Years
Healthy volunteers: Healthy volunteers not accepted
Timeline: Started 2012
U.S. locations: 1
States / cities: Durham, North Carolina

Conditions: Sickle Cell Anemia, Sickle Cell Disease, Chronic Hemolytic Disorders
Interventions: Sodium Nitrite Cream; Drug
Lead sponsor: National Heart, Lung, and Blood Institute (NHLBI); NIH
Eligibility: 18 Years to 99 Years
Enrollment: 28 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2011 – 2018
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Sickle Cell Disease
Interventions: Linkage Coordinator; Behavioral
Lead sponsor: RTI International; Other
Eligibility: 18 Years and older
Enrollment: 500 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2023 – 2027
U.S. locations: 9
States / cities: Birmingham, Alabama • Phoenix, Arizona • Oakland, California + 6 more

Conditions: Acute Myeloid Leukemia in Remission, de Novo Myelodysplastic Syndrome, Fanconi Anemia, Previously Treated Myelodysplastic Syndrome
Interventions: Allogeneic Bone Marrow Transplantation, Cyclophosphamide, Cyclosporine, Fludarabine Phosphate, Laboratory Biomarker Analysis, Mycophenolate Mofetil, Nonmyeloablative Allogeneic Hematopoietic Stem Cell Transplantation, Total-Body Irradiation; Procedure · Drug · Other + 1 more
Lead sponsor: Fred Hutchinson Cancer Center; Other
Eligibility: Not listed
Enrollment: 6 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2007 – 2013
U.S. locations: 4
States / cities: Oakland, California • Nashville, Tennessee • Seattle, Washington + 1 more

Conditions: Granuloma, Granulomatous Disease, Chronic, Leukocyte Disease, Genetic Disease, X-Linked, Genetic Disease, Inborn
Interventions: Not listed
Lead sponsor: National Institute of Allergy and Infectious Diseases (NIAID); NIH
Eligibility: 18 Years to 70 Years
Enrollment: 850 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: Started 1994
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Sickle Cell Disease, Chronic Pain
Interventions: Not listed
Lead sponsor: Duke University; Other
Eligibility: 12 Years to 100 Years
Enrollment: 21 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2012 – 2014
U.S. locations: 1
States / cities: Durham, North Carolina

Conditions: Erythroplakia, Fanconi Anemia, Lichen Planus, Oral Cavity and Lip Precancerous Condition, Oral Cavity Leukoplakia, Oral Neoplasm, Premalignant Lesion, Tobacco Use Disorder
Interventions: Cytology Specimen Collection Procedure, Diagnostic Microscopy, Diagnostic Procedure, Fluorescence Imaging, Fluorescence Spectroscopy; Other · Procedure
Lead sponsor: M.D. Anderson Cancer Center; Other
Eligibility: 18 Years and older
Enrollment: 338 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2007 – 2027
U.S. locations: 1
States / cities: Houston, Texas

Conditions: Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Diseases
Interventions: filgrastim, cyclophosphamide, fludarabine phosphate, mycophenolate mofetil, tacrolimus, allogeneic bone marrow transplantation, radiation therapy; Biological · Drug · Procedure + 1 more
Lead sponsor: Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins; Other
Eligibility: Up to 70 Years
Healthy volunteers: Healthy volunteers not accepted
Timeline: Started 1999
U.S. locations: 1
States / cities: Baltimore, Maryland

Conditions: Hemoglobin SC Disease
Interventions: Hydroxyurea; Drug
Lead sponsor: Baylor College of Medicine; Other
Eligibility: 18 Years to 69 Years
Enrollment: 1 participant
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2015 – 2017
U.S. locations: 2
States / cities: Houston, Texas

Conditions: Hemophilia A
Interventions: Damoctocog alfa pegol (Jivi, BAY94-9027); Drug
Lead sponsor: Bayer; Industry
Eligibility: Not listed
Enrollment: 10 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2024
U.S. locations: 2
States / cities: Orange, California • New Orleans, Louisiana

Conditions: Pediatric Cancer, Sickle Cell Disease, Quality of Life, Depressive Symptoms, Coping Skills
Interventions: FOCUS; Behavioral
Lead sponsor: Georgia State University; Other
Eligibility: 12 Years to 18 Years
Enrollment: 60 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2020 – 2021
U.S. locations: 2
States / cities: Atlanta, Georgia

Conditions: Hereditary Hemorrhagic Telangiectasia (HHT)
Interventions: VAD044 Part I, VAD044 Part II; Drug
Lead sponsor: Vaderis Therapeutics AG; Industry
Eligibility: 18 Years and older
Enrollment: 75 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2027
U.S. locations: 1
States / cities: Boston, Massachusetts

Conditions: Sickle Cell Disease
Interventions: nula-cel Drug Product; Genetic
Lead sponsor: Kamau Therapeutics; Industry
Eligibility: 12 Years to 50 Years
Enrollment: 15 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2028
U.S. locations: 6
States / cities: Los Angeles, California • Palo Alto, California • St Louis, Missouri + 2 more

Conditions: Intracranial Hemorrhage, Traumatic, Intracranial Hemorrhage, Spontaneous
Interventions: Four Factor Prothrombin Complex Concentrate, Fresh Frozen Plasma; Drug · Biological
Lead sponsor: University of Utah; Other
Eligibility: 18 Years and older
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2015 – 2016
U.S. locations: 1
States / cities: Salt Lake City, Utah

Conditions: Sickle Cell Disease, Sickle Cell Trait
Interventions: CHOICES, eBook; Other
Lead sponsor: University of Florida; Other
Eligibility: 18 Years to 45 Years
Enrollment: 506 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2027
U.S. locations: 1
States / cities: Gainesville, Florida

Conditions: Congenital Bleeding Disorder, Haemophilia B
Interventions: nonacog beta pegol; Drug
Lead sponsor: Novo Nordisk A/S; Industry
Eligibility: 13 Years to 70 Years · Male only
Enrollment: 13 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2012 – 2013
U.S. locations: 7
States / cities: Los Angeles, California • San Francisco, California • Washington D.C., District of Columbia + 4 more

Conditions: Subjective Cognitive Complaints, Ehlers-Danlos Syndrome, Cognitive Dysfunction
Interventions: COG+OT Telerehabilitation; Behavioral
Lead sponsor: Medical University of South Carolina; Other
Eligibility: 18 Years to 99 Years
Enrollment: 16 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2026 – 2027
U.S. locations: 2
States / cities: Charleston, South Carolina

Conditions: Hemophilia A
Interventions: Antihemophilic Factor (Recombinant) - Plasma/Albumin Free Method, PEGylated Recombinant Factor VIII; Biological
Lead sponsor: Baxalta now part of Shire; Industry
Eligibility: 12 Years to 65 Years · Male only
Enrollment: 159 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2013 – 2014
U.S. locations: 17
States / cities: Aurora, Colorado • Gainesville, Florida • Atlanta, Georgia + 14 more

Conditions: Chronic Granulomatous Disease
Interventions: Specimen Donation; Diagnostic Test
Lead sponsor: Sanguine Biosciences; Industry
Eligibility: 18 Years to 85 Years
Enrollment: 6 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2023 – 2024
U.S. locations: 1
States / cities: Woburn, Massachusetts

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Weight-based Dosing in Hemophilia A

A Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of AG-946 in Healthy Volunteers and in Participants With Sickle Cell Disease

Vasculitis Pregnancy Registry

Assessment of Opioid Analgesia in Sickle Cell

Early Discharge and Outpatient Care After Chemotherapy in Patients With Myelodysplastic Syndrome or Acute Myeloid Leukemia

Evaluation of the Safety and Efficacy of TXA127 (Angiotensin 1-7) to Enhance Engraftment in Pediatric Patients Undergoing Single or Double Umbilical Cord Blood Transplantation

Topical Sodium Nitrite for Chronic Leg Ulcers in Adult Patients With Blood Disorders

Recruitment and Engagement in Care to Impact Practice Enhancement (RECIPE) for Sickle Cell Disease

Fludarabine Phosphate, Cyclophosphamide, and Total-Body Irradiation Followed by Donor Bone Marrow Transplant, Mycophenolate Mofetil, and Cyclosporine in Treating Patients With Fanconi Anemia

Recruitment and Apheresis Collection of Peripheral Blood Hematopoietic Stem Cells, Mononuclear Cells and Granulocytes

Use of a Mobile-based App for SCD Patients

Widefield Fluorescence and Reflectance Imaging Systems and Oral Tissue Samples in Monitoring Participants at Risk for Developing Oral Cancer

Cyclophosphamide Plus Bone Marrow Transplantation in Treating Patients With Hematologic Cancer

Treatment of Adult Patients With Hemoglobin SC Disease (SCYTHE)

Study to Learn More About the Physical Activity Level of Patients Suffering From Hemophilia A Treated With Damoctocog Alfa Pegol (LIFE ACTIVE Study)

FOCUS for Pediatric Sickle Cell Disease and Cancer

Assess Safety and Efficacy of VAD044 in HHT Patients

Gene Correction in Autologous CD34+ Hematopoietic Stem Cells (HbS to HbA) to Treat Severe Sickle Cell Disease

FFP Versus PCC in Intracranial Hemorrhage

CHOICES3: Sickle Cell Disease Parenting CHOICES

Efficacy and Safety of NNC-0156-0000-0009 During Surgical Procedures in Subjects With Haemophilia B

COG+OT Telerehabilitation for Adults With Subjective Cognitive Complaints

Study Investigating a PEGylated Recombinant Factor VIII (BAX 855) for Hemophilia A (PROLONG-ATE Study)

Whole Blood Biospecimen Collection for Subjects With Chronic Granulomatous Disease (CGD)