- 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
Search Results
Search by objective public record fields.
Search by query text, NCT ID, condition, intervention, sponsor, city, state, recruitment status, phase, study type, healthy volunteer eligibility, sex, or age. Results come from ClinicalTrials.gov. When the live registry is unavailable, a clearly labeled stored copy may be shown.
These results were retrieved from the live ClinicalTrials.gov registry. Filters and sort order apply to this result list.
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.
- 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
- 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
- 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