- 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
Search Results
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Search by query text, NCT ID, condition, intervention, sponsor, city, state, recruitment status, phase, study type, healthy volunteer eligibility, sex, or age. Results are retrieved from ClinicalTrials.gov and synchronized into the directory. Search pages remain noindex by default.
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Showing 1–24
of 52
matching trials from the live ClinicalTrials.gov search.
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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
- THC, Cannabis, Cannabis Smoking, Cannabis Use, Unspecified, Cigarette Smoking, Tobacco Use, Nicotine Dependence, Nicotine Withdrawal, Cardiovascular Risk Factor
- Interventions
- Smoked Cannabis, Vaped Cannabis, Tobacco Cigarette
- Other
- Lead sponsor
- University of California, San Francisco
- Other
- Eligibility
- 21 Years and older
- Enrollment
- 14 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2020 – 2024
- U.S. locations
- 2
- States / cities
- San Francisco, California
- Conditions
- Factor 10 Deficiency
- Interventions
- Coagadex
- Drug
- Lead sponsor
- Bio Products Laboratory
- Other
- Eligibility
- 12 Years and older
- Enrollment
- 3 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2021
- U.S. locations
- 3
- States / cities
- Gainesville, Florida • New Orleans, Louisiana • Kalamazoo, Michigan
- 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
- Fibrinogen Deficiency
- Interventions
- Human Fibrinogen Concentrate
- Biological
- Lead sponsor
- CSL Behring
- Industry
- Eligibility
- 6 Years and older
- Enrollment
- 15 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2007 – 2008
- U.S. locations
- 6
- States / cities
- Aurora, Colorado • St. Petersburg, Florida • Chicago, Illinois + 3 more
- Conditions
- Adverse Drug Events, Adverse Drug Reactions, Drug Interaction Potentiation, Drug Metabolism, Poor, CYP2D6-RELATED, Drug Metabolism, Poor, CYP2C19-RELATED, Cytochrome P450 Enzyme Deficiency, Cytochrome P450 CYP2D6 Enzyme Deficiency, Cytochrome P450 CYP2C9 Enzyme Deficiency, Cytochrome P450 CYP2C19 Enzyme Deficiency, Cytochrome P450 CYP3A Enzyme Deficiency, Poor Metabolizer Due to Cytochrome P450 CYP2C9 Variant, Poor Metabolizer Due to Cytochrome P450 CYP2C19 Variant, Poor Metabolizer Due to Cytochrome P450 CYP2D6 Variant
- Interventions
- Not listed
- Lead sponsor
- Genelex Corporation
- Industry
- Eligibility
- 65 Years and older
- Enrollment
- 800 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2015
- U.S. locations
- 7
- States / cities
- Garden Grove, California • Denver, Colorado • Lexington, Kentucky + 3 more
- Conditions
- Hemophilia A
- Interventions
- Antihemophilic factor, recombinant, manufactured protein-free
- Drug
- Lead sponsor
- Baxalta now part of Shire
- Industry
- Eligibility
- Up to 6 Years
- Enrollment
- 50 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2002 – 2005
- U.S. locations
- 10
- States / cities
- Los Angeles, California • Atlanta, Georgia • Chicago, Illinois + 7 more
- Conditions
- Hemophilia A, Hemophilia B, Vitamin D Deficiency
- Interventions
- Vitamin D and calcium
- Dietary Supplement
- Lead sponsor
- Virginia Commonwealth University
- Other
- Eligibility
- 2 Years to 21 Years · Male only
- Enrollment
- 20 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2010 – 2015
- U.S. locations
- 1
- States / cities
- Richmond, Virginia
- Conditions
- Factor X Deficiency
- Interventions
- FACTOR X
- Biological
- Lead sponsor
- Bio Products Laboratory
- Other
- Eligibility
- 12 Years and older
- Enrollment
- 4 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2011 – 2014
- U.S. locations
- 1
- States / cities
- Houston, Texas
- Conditions
- Wound; Rupture, Surgery, Cesarean Section, Postpartum Hemorrhage, Surgery
- Interventions
- Hemoleven
- Biological
- Lead sponsor
- Laboratoire français de Fractionnement et de Biotechnologies
- Industry
- Eligibility
- 6 Months and older
- U.S. locations
- 1
- States / cities
- Nashville, Tennessee
- Conditions
- Congenital Fibrinogen Deficiency, Afibrinogenemia
- Interventions
- Octafibrin, Haemocomplettan® P or RiaSTAPTM
- Biological
- Lead sponsor
- Octapharma
- Industry
- Eligibility
- 12 Years and older
- Enrollment
- 22 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2013 – 2015
- U.S. locations
- 2
- States / cities
- Aurora, Colorado • New Hyde Park, New York
- Conditions
- HIV Infections
- Interventions
- Zidovudine
- Drug
- Lead sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
- NIH
- Eligibility
- 12 Years and older
- Enrollment
- 538 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- Ends 1990
- U.S. locations
- 23
- States / cities
- Los Angeles, California • San Diego, California • Stanford, California + 16 more
- Conditions
- Hemophilia A, Hemophilia A, Severe, Factor VIII (FVIII), FVIII Deficiency, Carrier of Hemophilia A, Inhibitors, Pregnancy, Maternal Blood Loss, Pregnancy Complications, Bleeding Disorder, Hemorrhage, Postpartum, Alloimmunization, X-Linked
- Interventions
- Not listed
- Lead sponsor
- University of Washington
- Other
- Eligibility
- Not listed
- Enrollment
- 500 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2024 – 2029
- U.S. locations
- 1
- States / cities
- Seattle, Washington
- Conditions
- Hematologic Disorder, Bleeding Disorder, Connective Tissue Disorder, Hemophilia, Thrombosis, Von Willebrand Diseases, Thrombophilia, Rare Bleeding Disorder, Platelet Disorder, Factor IX Deficiency, Factor VIII Deficiency, Thalassemia, Sickle Cell Disease
- Interventions
- Not listed
- Lead sponsor
- American Thrombosis and Hemostasis Network
- Network
- Eligibility
- Not listed
- Enrollment
- 3,000 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2020 – 2035
- U.S. locations
- 71
- States / cities
- Phoenix, Arizona • Little Rock, Arkansas • Los Angeles, California + 59 more
- Conditions
- Hemophilia A, Hemophilia B
- Interventions
- ADVATE (Antihemophilic Factor [Recombinant]), RIXUBIS (Coagulation Factor IX [Recombinant])
- Biological
- Lead sponsor
- Baxalta now part of Shire
- Industry
- Eligibility
- 13 Years to 65 Years
- Enrollment
- 64 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2016
- U.S. locations
- 17
- States / cities
- Los Angeles, California • San Diego, California • Aurora, Colorado + 14 more
- Conditions
- Hemophilia B
- Interventions
- Adeno-Associated Viral with Human Factor IX
- Genetic
- Lead sponsor
- Avigen
- Industry
- Eligibility
- 18 Years and older · Male only
- Enrollment
- 15 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- Started 2004
- U.S. locations
- 3
- States / cities
- Palo Alto, California • Philadelphia, Pennsylvania • Pittsburgh, Pennsylvania
- Conditions
- Acquired Hemophilia A
- Interventions
- OBI-1
- Biological
- Lead sponsor
- Baxalta now part of Shire
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 29 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2010 – 2013
- U.S. locations
- 8
- States / cities
- Indianapolis, Indiana • New Orleans, Louisiana • Bethesda, Maryland + 4 more
- Conditions
- Factor VIII Deficiency
- Interventions
- efanesoctocog alfa (BIVV001)
- Biological
- Lead sponsor
- Bioverativ, a Sanofi company
- Industry
- Eligibility
- 12 Years and older
- Enrollment
- 159 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2022
- U.S. locations
- 8
- States / cities
- Los Angeles, California • San Diego, California • Gainesville, Florida + 4 more
- Conditions
- Hemophilia A
- Interventions
- Recombinant Antihemophilic Factor Manufactured and Formulated without Added Human or Animal Proteins (rAHF-PFM)
- Biological
- Lead sponsor
- Baxalta now part of Shire
- Industry
- Eligibility
- Up to 6 Years
- Enrollment
- 66 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2004 – 2009
- U.S. locations
- 17
- States / cities
- Phoenix, Arizona • Little Rock, Arkansas • Los Angeles, California + 14 more
- Conditions
- Hemophilia, AIDS
- Interventions
- Not listed
- Lead sponsor
- National Cancer Institute (NCI)
- NIH
- Eligibility
- 4 Years to 100 Years
- Enrollment
- 333 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2002 – 2020
- U.S. locations
- 15
- States / cities
- La Jolla, California • Los Angeles, California • Indianapolis, Indiana + 11 more
- Conditions
- Factor IX Deficiency
- Interventions
- Standard care with blood and urine sample collection
- Other
- Lead sponsor
- Skane University Hospital
- Other
- Eligibility
- Not listed
- Enrollment
- 550 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015 – 2021
- U.S. locations
- 1
- States / cities
- Indianapolis, Indiana
- Conditions
- Complex Cardiovascular Surgery With Cardiopulmonary Bypass
- Interventions
- BE1116, FFP
- Biological
- Lead sponsor
- CSL Behring
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 200 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2026
- U.S. locations
- 10
- States / cities
- Los Angeles, California • Chicago, Illinois • Baltimore, Maryland + 7 more
- Conditions
- Liver Decompensation, Hepatocellular Carcinoma, Non-Hodgkin Lymphoma
- Interventions
- Not listed
- Lead sponsor
- National Cancer Institute (NCI)
- NIH
- Eligibility
- 13 Years and older
- Enrollment
- 2,565 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2001 – 2013
- U.S. locations
- 39
- States / cities
- Tucson, Arizona • Davis, California • Orange, California + 31 more