Independent directory Public ClinicalTrials.gov records United States
Clear filters

These results were retrieved from the live ClinicalTrials.gov registry. Filters and sort order apply to this result list.

ClinicalTrials.gov public records Last synced Oct 5, 2026, 5:50 PM EDT

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

Showing 73–96 of 331 matching trials from the live ClinicalTrials.gov search.
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
Terminated Phase 2 Interventional Results available

Study of Ataluren (PTC124) in Hemophilia A and B

NCT00947193
Conditions
Hemophilia A, Hemophilia B
Interventions
Ataluren
Drug
Lead sponsor
PTC Therapeutics
Industry
Eligibility
18 Years and older · Male only
Enrollment
13 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2011
U.S. locations
7
States / cities
Peoria, Illinois • Indianapolis, Indiana • Worcester, Massachusetts + 4 more
Conditions
Hemophilia A
Interventions
Advate (Low Dose), Advate (High Dose), BIVV001 (Low Dose), BIVV001 (High Dose)
Biological
Lead sponsor
Bioverativ, a Sanofi company
Industry
Eligibility
18 Years to 65 Years · Male only
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2018
U.S. locations
7
States / cities
Los Angeles, California • Indianapolis, Indiana • Iowa City, Iowa + 4 more
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
Hemophilia B
Interventions
Non-Interventional
Other
Lead sponsor
Regeneron Pharmaceuticals
Industry
Eligibility
16 Years and older · Male only
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
9
States / cities
Aurora, Colorado • New Haven, Connecticut • Washington D.C., District of Columbia + 6 more
Conditions
Hemophilia A
Interventions
BAY94-9027
Biological
Lead sponsor
Bayer
Industry
Eligibility
18 Years and older
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017
U.S. locations
1
States / cities
Cleveland, Ohio
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
Completed No phase listed Observational Accepts healthy volunteers

Biomarkers Serum Collection Methodology Pilot Study

NCT01318707
Conditions
Hemophilia A
Interventions
Not listed
Lead sponsor
Georgetown University
Other
Eligibility
18 Years to 75 Years
Enrollment
20 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2010 – 2011
U.S. locations
1
States / cities
Washington D.C., District of Columbia
Conditions
Hemophilia A
Interventions
ADYNOVI/ADYNOVATE
Biological
Lead sponsor
Baxalta now part of Shire
Industry
Eligibility
Not listed
Enrollment
207 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2030
U.S. locations
1
States / cities
Gainesville, Florida
Conditions
Hemophilia, Hemophilia A, Hemophilia B, Hemophilia A With Inhibitor, Hemophilia B With Inhibitor, Haemophilia B Without Inhibitor, Haemophilia A Without Inhibitor, Haemophilia
Interventions
Not listed
Lead sponsor
American Thrombosis and Hemostasis Network
Network
Eligibility
0 Years to 13 Years
Enrollment
237 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2022
U.S. locations
27
States / cities
Phoenix, Arizona • Madera, California • San Diego, California + 24 more
Conditions
Congenital Bleeding Disorder, Haemophilia B
Interventions
nonacog beta pegol
Drug
Lead sponsor
Novo Nordisk A/S
Industry
Eligibility
0 Years to 6 Years · Male only
Enrollment
54 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2022
U.S. locations
23
States / cities
Long Beach, California • Los Angeles, California • Gainesville, Florida + 20 more
Conditions
Hemophilia A With Inhibitors
Interventions
recombinant activated factor VII
Drug
Lead sponsor
Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico
Other
Eligibility
Up to 8 Years · Male only
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2014
U.S. locations
3
States / cities
Los Angeles, California • Atlanta, Georgia • Kansas City, Missouri
Conditions
Liver Cirrhosis, Hemophilia, Hemophilia A, Hemophilia B, Liver Transplant Disorder, Liver Failure, Coagulation Factor Deficiency
Interventions
Not listed
Lead sponsor
Stanford University
Other
Eligibility
18 Years and older
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2024
U.S. locations
1
States / cities
Palo Alto, California
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
Case scenarios - questionnaire, Case scenarios - Focus groups, Use scenarios - Usability test, Use scenarios - interview
Other
Lead sponsor
Enzyre B.V.
Industry
Eligibility
12 Years and older
Enrollment
74 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024
U.S. locations
1
States / cities
Indianapolis, Indiana
Conditions
Hemophilia A
Interventions
rFVIII (BAY81-8973) on demand, rFVIII (BAY81-8973) prophylaxis low-dose, rFVIII (BAY81-8973) prophylaxis high-dose
Biological
Lead sponsor
Bayer
Industry
Eligibility
12 Years to 65 Years · Male only
Enrollment
80 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2012
U.S. locations
2
States / cities
Dayton, Ohio • Houston, Texas
Conditions
Hemophilia A, Hemophilia B
Interventions
PF-06741086
Drug
Lead sponsor
Pfizer
Industry
Eligibility
1 Year to 74 Years · Male only
Enrollment
245 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2030
U.S. locations
4
States / cities
Iowa City, Iowa • New Hyde Park, New York • Philadelphia, Pennsylvania + 1 more
Conditions
Hemophilia A
Interventions
Desmopressin
Drug
Lead sponsor
Emory University
Other
Eligibility
18 Years to 60 Years · Female only
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2018
U.S. locations
2
States / cities
Atlanta, Georgia
Conditions
Hemophilia A
Interventions
Available Recombinant, and Human Factor VIII products incl. Kogenate FS (Recombinant Factor VIII, BAY14-2222)
Drug
Lead sponsor
Bayer
Industry
Eligibility
16 Years and older · Male only
Enrollment
272 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2018
U.S. locations
7
States / cities
Phoenix, Arizona • Aurora, Colorado • Washington D.C., District of Columbia + 4 more
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
Completed Phase 3 Interventional Results available

BAX 855 Pediatric Study

NCT02210091
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
Up to 11 Years
Enrollment
75 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2015
U.S. locations
12
States / cities
Phoenix, Arizona • Denver, Colorado • Gainesville, Florida + 9 more
Conditions
Hemophilia
Interventions
Coagulation Factor VIIa (Recombinant)
Biological
Lead sponsor
Laboratoire français de Fractionnement et de Biotechnologies
Industry
Eligibility
12 Years to 65 Years · Male only
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2025
U.S. locations
5
States / cities
Los Angeles, California • Atlanta, Georgia • New Orleans, Louisiana + 2 more
Conditions
Hemophilia A
Interventions
Factor IX
Drug
Lead sponsor
Tulane University
Other
Eligibility
12 Years to 120 Years · Male only
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2024
U.S. locations
2
States / cities
Metairie, Louisiana • New Orleans, Louisiana
Conditions
HIV Infections, Hemophilia A
Interventions
Ibuprofen, Zidovudine
Drug
Lead sponsor
University of Pittsburgh
Other
Eligibility
12 Years and older
Healthy volunteers
Healthy volunteers not accepted
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania