Independent directory Public ClinicalTrials.gov records United States
Clear filters
ClinicalTrials.gov public records Last synced Jul 21, 2026, 10:22 PM EDT

Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.

Showing 1–24 of 52 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
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
Completed Not applicable Interventional Accepts healthy volunteers

THC Crossover Study

NCT04429568
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
Completed Phase 2 Interventional Results available

Human Fibrinogen - Pharmacokinetics

NCT00496262
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
Completed No phase listed Observational

YouScript IMPACT Registry

NCT02191358
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
Recruiting No phase listed Observational Accepts healthy volunteers

Hemophilia A Research Program

NCT07414511
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