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Showing 1–24 of 879 matching trials from the live ClinicalTrials.gov search.
Local D1 index available.
Completed Phase 1Phase 2 Interventional Results available

A Gene Transfer Study for Hemophilia A

NCT03003533
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
Accelerated Phase Myeloproliferative Neoplasm, Blast Phase Myeloproliferative Neoplasm, Essential Thrombocythemia, Myelodysplastic/Myeloproliferative Neoplasm, Myeloproliferative Neoplasm, Not Otherwise Specified, Polycythemia Vera, Primary Myelofibrosis, Secondary Myelofibrosis
Interventions
Biospecimen Collection, Bone Marrow Aspiration, Bone Marrow Biopsy, Decitabine and Cedazuridine, Iadademstat
Procedure · Drug
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
18 Years and older
Enrollment
62 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
31
States / cities
Irvine, California • Orange, California • Palo Alto, California + 27 more
Terminated Phase 1 Interventional Accepts healthy volunteers Results available

A First in HumanTrial to Evaluate The Safety, Tolerability, And Pharmacokinetics Of PF-06755347

NCT03275740
Conditions
Healthy, Primary Immune Thrombocytopenia, Polyradiculoneuropathy, Chronic Inflammatory Demyelinating
Interventions
PF-06755347 intravenous healthy participant, Placebo intravenous healthy participant, PF-06755347 subcutaneous healthy participant, Placebo subcutaneous healthy participant, PF-06755347 subcutaneous ITP
Drug
Lead sponsor
Pfizer
Industry
Eligibility
18 Years to 55 Years
Enrollment
58 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2017 – 2023
U.S. locations
1
States / cities
New Haven, Connecticut
Completed Phase 1 Interventional Accepts healthy volunteers

Dose Response to Recombinant Factor VIIa When Administered for Bleed

NCT00104455
Conditions
Congenital Bleeding Disorder, Healthy
Interventions
activated recombinant human factor VII
Drug
Lead sponsor
Novo Nordisk A/S
Industry
Eligibility
18 Years to 60 Years · Male only
Enrollment
64 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2004
U.S. locations
1
States / cities
Overland Park, Kansas
Conditions
Coagulation Disorder, Bleeding
Interventions
Coagulation Laboratory Testing
Diagnostic Test
Lead sponsor
Mayo Clinic
Other
Eligibility
18 Years and older
Enrollment
80 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018
U.S. locations
1
States / cities
Rochester, Minnesota
Conditions
Chronic HCV Infection
Interventions
LDV/SOF, SOF, RBV
Drug
Lead sponsor
Gilead Sciences
Industry
Eligibility
18 Years and older
Enrollment
122 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2015
U.S. locations
13
States / cities
Sacramento, California • San Diego, California • San Francisco, California + 9 more
Conditions
Hemophilia B
Interventions
HEMGENIX, Factor IX (FIX)
Genetic · Biological
Lead sponsor
CSL Behring
Industry
Eligibility
18 Years and older
Enrollment
500 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2043
U.S. locations
1
States / cities
Rochester, New York
Conditions
Snake Bite, Blood Coagulation Disorders
Interventions
Anavip, CroFab
Biological
Lead sponsor
Instituto Bioclon S.A. de C.V.
Industry
Eligibility
18 Years to 70 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2007
U.S. locations
1
States / cities
Tucson, Arizona
Conditions
Immune Thrombocytopenia (ITP)
Interventions
SAR445088 (BIVV020)
Drug
Lead sponsor
Bioverativ, a Sanofi company
Industry
Eligibility
18 Years and older
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2023
U.S. locations
2
States / cities
Washington D.C., District of Columbia • Tamarac, Florida
Conditions
Chronic Myeloproliferative Disorders, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic Syndromes, Precancerous Condition, Small Intestine Cancer, Unspecified Adult Solid Tumor, Protocol Specific
Interventions
pyroxamide
Drug
Lead sponsor
Memorial Sloan Kettering Cancer Center
Other
Eligibility
18 Years and older
Healthy volunteers
Healthy volunteers not accepted
Timeline
2002
U.S. locations
1
States / cities
New York, New York
Not listed Phase 1Phase 2 Interventional

CEP-701 (Lestaurtinib) in Myelofibrosis

NCT00668421
Conditions
Myelofibrosis, Essential Thrombocythemia, Polycythemia Vera
Interventions
CEP-701 (Lestaurtinib)
Drug
Lead sponsor
Ronald Hoffman
Other
Eligibility
18 Years and older
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2015
U.S. locations
7
States / cities
Palo Alto, California • Washington D.C., District of Columbia • Baltimore, Maryland + 4 more
Conditions
Immune Thrombocytopenia (ITP)
Interventions
NVG-2089
Drug
Lead sponsor
Nuvig Therapeutics, Inc.
Industry
Eligibility
18 Years to 80 Years
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
1
States / cities
Greenville, North Carolina
Enrolling by invitation Not applicable Interventional Accepts healthy volunteers

Palliative Care Needs of Children With Rare Diseases and Their Families

NCT06938542
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
Coagulation Disorder
Interventions
Chamomile Tea, Chamomile Extract Capsule
Dietary Supplement
Lead sponsor
Stony Brook University
Other
Eligibility
18 Years to 75 Years
Enrollment
8 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2022
U.S. locations
1
States / cities
Stony Brook, New York
Conditions
Wiskott-Aldrich Syndrome
Interventions
OTL-103
Genetic
Lead sponsor
Fondazione Telethon
Other
Eligibility
Up to 65 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2027
U.S. locations
1
States / cities
Atlanta, Georgia
Conditions
Chronic Myeloproliferative Disorders, Leukemia, Lymphoma, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Neoplasms
Interventions
alemtuzumab, therapeutic allogeneic lymphocytes, fludarabine phosphate, melphalan, tacrolimus, thiotepa, peripheral blood stem cell transplantation
Biological · Drug · Procedure
Lead sponsor
Yale University
Other
Eligibility
18 Years to 55 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2008
U.S. locations
1
States / cities
New Haven, Connecticut
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
Myeloproliferative Diseases
Interventions
Pacritinib, Busulfan, Questionnaires, Phone Calls, Allogeneic Stem Cell Transplantation, Fludarabine
Drug · Behavioral · Procedure
Lead sponsor
M.D. Anderson Cancer Center
Other
Eligibility
18 Years to 70 Years
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2017
U.S. locations
1
States / cities
Houston, Texas
Conditions
Hemophilia B
Interventions
CSL222 (AAV5-hFIXco-Padua)
Genetic
Lead sponsor
CSL Behring
Industry
Eligibility
18 Years and older
Enrollment
35 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2032
U.S. locations
3
States / cities
San Diego, California • Ann Arbor, Michigan • Pittsburgh, Pennsylvania
Conditions
Accelerated Phase Chronic Myelogenous Leukemia, Acute Undifferentiated Leukemia, Adult Acute Lymphoblastic Leukemia in Remission, 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), Adult Grade III Lymphomatoid Granulomatosis, Adult Nasal Type Extranodal NK/T-cell Lymphoma, Aggressive NK-cell Leukemia, AIDS-related Diffuse Large Cell Lymphoma, AIDS-related Diffuse Mixed Cell Lymphoma, AIDS-related Diffuse Small Cleaved Cell Lymphoma, AIDS-related Immunoblastic Large Cell Lymphoma, AIDS-related Lymphoblastic Lymphoma, AIDS-related Peripheral/Systemic Lymphoma, AIDS-related Primary CNS Lymphoma, AIDS-related Small Noncleaved Cell Lymphoma, Anaplastic Large Cell Lymphoma, Angioimmunoblastic T-cell Lymphoma, Blastic Phase Chronic Myelogenous Leukemia, Childhood Acute Lymphoblastic Leukemia in Remission, Childhood Acute Myeloid Leukemia in Remission, Childhood Burkitt Lymphoma, Childhood Chronic Myelogenous Leukemia, Childhood Diffuse Large Cell Lymphoma, Childhood Grade III Lymphomatoid Granulomatosis, Childhood Immunoblastic Large Cell Lymphoma, Childhood Myelodysplastic Syndromes, Childhood Nasal Type Extranodal NK/T-cell Lymphoma, Chronic Eosinophilic Leukemia, Chronic Myelomonocytic Leukemia, Chronic Neutrophilic Leukemia, Chronic Phase Chronic Myelogenous Leukemia, Contiguous Stage II Adult Burkitt Lymphoma, Contiguous Stage II Adult Diffuse Large Cell Lymphoma, Contiguous Stage II Adult Diffuse Mixed Cell Lymphoma, Contiguous Stage II Adult Diffuse Small Cleaved Cell Lymphoma, Contiguous Stage II Adult Immunoblastic Large Cell Lymphoma, Contiguous Stage II Adult Lymphoblastic Lymphoma, Contiguous Stage II Grade 1 Follicular Lymphoma, Contiguous Stage II Grade 2 Follicular Lymphoma, Contiguous Stage II Grade 3 Follicular Lymphoma, Contiguous Stage II Mantle Cell Lymphoma, Contiguous Stage II Marginal Zone Lymphoma, Contiguous Stage II Small Lymphocytic Lymphoma, Cutaneous B-cell Non-Hodgkin Lymphoma, Essential Thrombocythemia, Extramedullary Plasmacytoma, Extranodal Marginal Zone B-cell Lymphoma of Mucosa-associated Lymphoid Tissue, Hepatosplenic T-cell Lymphoma, HIV Infection, HIV-associated Hodgkin Lymphoma, Intraocular Lymphoma, Isolated Plasmacytoma of Bone, Juvenile Myelomonocytic Leukemia, Mast Cell Leukemia, Meningeal Chronic Myelogenous Leukemia, Myelodysplastic/Myeloproliferative Neoplasm, Unclassifiable, Myeloid/NK-cell Acute Leukemia, Nodal Marginal Zone B-cell Lymphoma, Noncontiguous Stage II Adult Burkitt Lymphoma, Noncontiguous Stage II Adult Diffuse Large Cell Lymphoma, Noncontiguous Stage II Adult Diffuse Mixed Cell Lymphoma, Noncontiguous Stage II Adult Diffuse Small Cleaved Cell Lymphoma, Noncontiguous Stage II Adult Immunoblastic Large Cell Lymphoma, Noncontiguous Stage II Adult Lymphoblastic Lymphoma, Noncontiguous Stage II Grade 1 Follicular Lymphoma, Noncontiguous Stage II Grade 2 Follicular Lymphoma, Noncontiguous Stage II Grade 3 Follicular Lymphoma, Noncontiguous Stage II Mantle Cell Lymphoma, Noncontiguous Stage II Marginal Zone Lymphoma, Noncontiguous Stage II Small Lymphocytic Lymphoma, Noncutaneous Extranodal Lymphoma, Peripheral T-cell Lymphoma, Polycythemia Vera, Post-transplant Lymphoproliferative Disorder, Previously Treated Myelodysplastic Syndromes, Primary Central Nervous System Lymphoma, Primary Myelofibrosis, Primary Systemic Amyloidosis, Progressive Hairy Cell Leukemia, Initial Treatment, Prolymphocytic Leukemia, Secondary Acute Myeloid Leukemia, Secondary Myelodysplastic Syndromes, Small Intestine Lymphoma, Splenic Marginal Zone Lymphoma, Stage 0 Chronic Lymphocytic Leukemia, Stage I Adult Burkitt Lymphoma, Stage I Adult Diffuse Large Cell Lymphoma, Stage I Adult Diffuse Mixed Cell Lymphoma, Stage I Adult Diffuse Small Cleaved Cell Lymphoma, Stage I Adult Hodgkin Lymphoma, Stage I Adult Immunoblastic Large Cell Lymphoma, Stage I Adult Lymphoblastic Lymphoma, Stage I Adult T-cell Leukemia/Lymphoma, Stage I Childhood Anaplastic Large Cell Lymphoma, Stage I Childhood Hodgkin Lymphoma, Stage I Childhood Large Cell Lymphoma, Stage I Childhood Lymphoblastic Lymphoma, Stage I Childhood Small Noncleaved Cell Lymphoma, Stage I Chronic Lymphocytic Leukemia, Stage I Cutaneous T-cell Non-Hodgkin Lymphoma, Stage I Grade 1 Follicular Lymphoma, Stage I Grade 2 Follicular Lymphoma, Stage I Grade 3 Follicular Lymphoma, Stage I Mantle Cell Lymphoma, Stage I Marginal Zone Lymphoma, Stage I Multiple Myeloma, Stage I Small Lymphocytic Lymphoma, Stage IA Mycosis Fungoides/Sezary Syndrome, Stage IB Mycosis Fungoides/Sezary Syndrome, Stage II Adult Hodgkin Lymphoma, Stage II Adult T-cell Leukemia/Lymphoma, Stage II Childhood Anaplastic Large Cell Lymphoma, Stage II Childhood Hodgkin Lymphoma, Stage II Childhood Large Cell Lymphoma, Stage II Childhood Lymphoblastic Lymphoma, Stage II Childhood Small Noncleaved Cell Lymphoma, Stage II Chronic Lymphocytic Leukemia, Stage II Cutaneous T-cell Non-Hodgkin Lymphoma, Stage II Multiple Myeloma, Stage IIA Mycosis Fungoides/Sezary Syndrome, Stage IIB Mycosis Fungoides/Sezary Syndrome, Stage III Adult Burkitt Lymphoma, Stage III Adult Diffuse Large Cell Lymphoma, Stage III Adult Diffuse Mixed Cell Lymphoma, Stage III Adult Diffuse Small Cleaved Cell Lymphoma, Stage III Adult Hodgkin Lymphoma, Stage III Adult Immunoblastic Large Cell Lymphoma, Stage III Adult Lymphoblastic Lymphoma, Stage III Adult T-cell Leukemia/Lymphoma, Stage III Childhood Anaplastic Large Cell Lymphoma, Stage III Childhood Hodgkin Lymphoma, Stage III Childhood Large Cell Lymphoma, Stage III Childhood Lymphoblastic Lymphoma, Stage III Childhood Small Noncleaved Cell Lymphoma, Stage III Chronic Lymphocytic Leukemia, Stage III Cutaneous T-cell Non-Hodgkin Lymphoma, Stage III Grade 1 Follicular Lymphoma, Stage III Grade 2 Follicular Lymphoma, Stage III Grade 3 Follicular Lymphoma, Stage III Mantle Cell Lymphoma, Stage III Marginal Zone Lymphoma, Stage III Multiple Myeloma, Stage III Small Lymphocytic Lymphoma, Stage IIIA Mycosis Fungoides/Sezary Syndrome, Stage IIIB Mycosis Fungoides/Sezary Syndrome, Stage IV Adult Burkitt Lymphoma, Stage IV Adult Diffuse Large Cell Lymphoma, Stage IV Adult Diffuse Mixed Cell Lymphoma, Stage IV Adult Diffuse Small Cleaved Cell Lymphoma, Stage IV Adult Hodgkin Lymphoma, Stage IV Adult Immunoblastic Large Cell Lymphoma, Stage IV Adult Lymphoblastic Lymphoma, Stage IV Adult T-cell Leukemia/Lymphoma, Stage IV Childhood Anaplastic Large Cell Lymphoma, Stage IV Childhood Hodgkin Lymphoma, Stage IV Childhood Large Cell Lymphoma, Stage IV Childhood Lymphoblastic Lymphoma, Stage IV Childhood Small Noncleaved Cell Lymphoma, Stage IV Chronic Lymphocytic Leukemia, Stage IV Cutaneous T-cell Non-Hodgkin Lymphoma, Stage IV Grade 1 Follicular Lymphoma, Stage IV Grade 2 Follicular Lymphoma, Stage IV Grade 3 Follicular Lymphoma, Stage IV Mantle Cell Lymphoma, Stage IV Marginal Zone Lymphoma, Stage IV Small Lymphocytic Lymphoma, Stage IVA Mycosis Fungoides/Sezary Syndrome, Stage IVB Mycosis Fungoides/Sezary Syndrome, T-cell Large Granular Lymphocyte Leukemia, Testicular Lymphoma, Unspecified Adult Solid Tumor, Protocol Specific, Unspecified Childhood Solid Tumor, Protocol Specific, Waldenström Macroglobulinemia
Interventions
fludarabine phosphate, total-body irradiation, peripheral blood stem cell transplantation, cyclosporine, mycophenolate mofetil, laboratory biomarker analysis
Drug · Radiation · Procedure + 1 more
Lead sponsor
Fred Hutchinson Cancer Center
Other
Eligibility
Up to 75 Years
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1999
U.S. locations
1
States / cities
Seattle, Washington
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
Acute Myeloid Leukemia, Essential Thrombocythemia, Myelodysplastic Syndrome, Myelodysplastic/Myeloproliferative Neoplasm, Myeloproliferative Neoplasm, Myeloproliferative Neoplasm, Not Otherwise Specified, Polycythemia Vera, Primary Myelofibrosis, Secondary Myelofibrosis
Interventions
Decitabine, Ruxolitinib, Fedratinib, Questionnaire Administration, Pacritinib, Biospecimen Collection
Drug · Other · Procedure
Lead sponsor
University of Washington
Other
Eligibility
18 Years and older
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2026
U.S. locations
1
States / cities
Seattle, Washington
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
Glanzmann Thrombasthenia
Interventions
Not listed
Lead sponsor
Rockefeller University
Other
Eligibility
Not listed
Enrollment
60 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2005 – 2030
U.S. locations
1
States / cities
New York, New York