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Showing 1–24 of 109 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
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
Congenital Bleeding Disorder, Haemophilia A
Interventions
turoctocog alfa
Drug
Lead sponsor
Novo Nordisk A/S
Industry
Eligibility
Up to 11 Years · Male only
Enrollment
65 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2011
U.S. locations
18
States / cities
Phoenix, Arizona • Orange, California • Torrance, California + 15 more
Conditions
Congenital Bleeding Disorder, Haemophilia A
Interventions
turoctocog alfa
Drug
Lead sponsor
Novo Nordisk A/S
Industry
Eligibility
0 Years to 6 Years · Male only
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2018
U.S. locations
25
States / cities
Phoenix, Arizona • Long Beach, California • Sacramento, California + 20 more
Conditions
Hemophilia A
Interventions
BAY94-9027
Biological
Lead sponsor
Bayer
Industry
Eligibility
Up to 12 Years · Male only
Enrollment
73 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2020
U.S. locations
7
States / cities
Sacramento, California • Pensacola, Florida • Cincinnati, Ohio + 4 more
Completed Phase 3 Interventional Results available

BAY81-8973 Pediatric Safety and Efficacy Trial

NCT01311648
Conditions
Haemophilia A
Interventions
Recombinant Factor VIII (Kovaltry, BAY81-8973)
Biological
Lead sponsor
Bayer
Industry
Eligibility
0 Years to 12 Years · Male only
Enrollment
94 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2020
U.S. locations
4
States / cities
New Orleans, Louisiana • Cincinnati, Ohio • Cleveland, Ohio + 1 more
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
Acquired Hemophilia A
Interventions
OBIZUR
Biological
Lead sponsor
Baxalta now part of Shire
Industry
Eligibility
18 Years and older
Enrollment
53 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2019
U.S. locations
16
States / cities
Aurora, Colorado • Gainesville, Florida • Atlanta, Georgia + 13 more
Conditions
Hemophilia
Interventions
Coagulation Factor VIIa (Recombinant)
Biological
Lead sponsor
Laboratoire français de Fractionnement et de Biotechnologies
Industry
Eligibility
6 Months to 75 Years · Male only
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2017
U.S. locations
1
States / cities
Dallas, Texas
Conditions
Hemophilia A
Interventions
OBI-1
Biological
Lead sponsor
Baxalta now part of Shire
Industry
Eligibility
6 Years and older
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2013
U.S. locations
1
States / cities
Indianapolis, Indiana
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
Active, not recruiting Phase 1Phase 2 Interventional

Gene Therapy for Haemophilia A.

NCT03001830
Conditions
Hemophilia A
Interventions
AAV2/8-HLP-FVIII-V3
Biological
Lead sponsor
University College, London
Other
Eligibility
18 Years and older · Male only
Enrollment
14 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2029
U.S. locations
3
States / cities
Boise, Idaho • Lexington, Kentucky • Memphis, Tennessee
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
Haemophilia A, Haemophilia A With Inhibitors, Haemophilia B, Haemophilia B With Inhibitors
Interventions
No treatment given
Other
Lead sponsor
Novo Nordisk A/S
Industry
Eligibility
12 Years and older · Male only
Enrollment
231 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2021
U.S. locations
29
States / cities
Los Angeles, California • Sacramento, California • Aurora, Colorado + 24 more
Conditions
Haemostasis, Haemophilia A
Interventions
Concizumab, Turoctocog alfa
Drug
Lead sponsor
Novo Nordisk A/S
Industry
Eligibility
18 Years and older · Male only
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2020
U.S. locations
5
States / cities
Los Angeles, California • Indianapolis, Indiana • Oklahoma City, Oklahoma + 2 more
Conditions
Congenital Bleeding Disorder, Haemophilia A With Inhibitors, Haemophilia B With Inhibitors
Interventions
activated recombinant human factor VII, Feiba VH
Drug
Lead sponsor
Novo Nordisk A/S
Industry
Eligibility
Male only
Enrollment
52 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2009
U.S. locations
1
States / cities
Plainsboro, New Jersey
Conditions
Congenital Bleeding Disorder, Haemophilia A With Inhibitors, Haemophilia B With Inhibitors
Interventions
eptacog alfa (activated)
Drug
Lead sponsor
Novo Nordisk A/S
Industry
Eligibility
5 Years and older
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011
U.S. locations
1
States / cities
Princeton, New Jersey
Terminated Phase 2 Interventional Results available

Alphanate in Immune Tolerance Induction Therapy

NCT03095287
Conditions
Hemophilia A, Congenital
Interventions
Alphanate
Biological
Lead sponsor
Grifols Therapeutics LLC
Industry
Eligibility
Up to 12 Years · Male only
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2020
U.S. locations
8
States / cities
Atlanta, Georgia • Lexington, Kentucky • Minneapolis, Minnesota + 5 more
Conditions
Congenital Hemophilia A
Interventions
rVIII-SingleChain
Biological
Lead sponsor
CSL Behring
Industry
Eligibility
Up to 11 Years · Male only
Enrollment
84 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2015
U.S. locations
2
States / cities
Aurora, Colorado • Chicago, Illinois
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
Haemophilia A, Haemophilia A With Inhibitors
Interventions
Mim8
Drug
Lead sponsor
Novo Nordisk A/S
Industry
Eligibility
Not listed
Enrollment
451 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2028
U.S. locations
15
States / cities
Los Angeles, California • Aurora, Colorado • Miami, Florida + 11 more
Conditions
Haemophilia A, Haemophilia B, Obesity
Interventions
No treatment given
Other
Lead sponsor
Novo Nordisk A/S
Industry
Eligibility
18 Years and older
Enrollment
317 participants
Timeline
2017 – 2018
U.S. locations
1
States / cities
Plainsboro, New Jersey