Clinical trials: Hemophilia, Classic
Congenital Adrenal Hyperplasia, Familial Hyperinsulinemic Hypoglycemia 1
Baby Detect : Genomic Newborn Screening
Newborn screening (NBS) is a global initiative of systematic testing at birth to identify babies with pre-defined severe but treatable conditions.
- Conditions
- Congenital Adrenal Hyperplasia, Familial Hyperinsulinemic Hypoglycemia 1, Phosphoglucomutase 1 Deficiency, Maturity Onset Diabetes of the Young
- Trial name
- BabyDetect (NCT05687474)
- Where
- Belgium
- Volunteers
- 6,824 people, healthy volunteers welcome
- Sponsor
- Centre Hospitalier Universitaire de Liege
Haemophilia A
A Research Study Looking at How Food Intake Affects Inno8 in the Body of Healthy People
The purpose of this study is to test a new medicine called Inno8. The study will test how eating and drinking before and after taking Inno8 affects how well it is absorbed in the stomach. The study consists of four arms.
- Trial name
- NCT07238816
- Testing
- NNC0442-0344 A
- Where
- United States
- Volunteers
- 80 people, healthy volunteers welcome
- Sponsor
- Novo Nordisk A/S
Haemophilia
Functional and Proprioceptive Status in Patients With Hemophilic Ankle Arthropathy
Introduction: Hemophilic ankle arthropathy is manifested by functional degenerative alterations (muscle strength deficit, mobility and proprioception), intra-articular and chronic pain. Design: Case-control study.
- Trial name
- NCT05425888
- Testing
- Hemophilia group
- Where
- Spain
- Volunteers
- 32 people, healthy volunteers welcome
- Sponsor
- Investigación en Hemofilia y Fisioterapia
Raloxifene, Postmenopausal Women
Raloxifene on Coagulation System in the Postmenopausal Women
Objective: The aim of this study is to assess the effects of raloxifene HCl on coagulation parameters in healthy postmenopausal women.
- Trial name
- NCT04454775
- Testing
- coagulation system parameters; factor 8, von Willebrand factor, protein C, protein S, antithrombin III and fibrinogen were studied.
- Volunteers
- 80 people, healthy volunteers welcome
- Sponsor
- Karadeniz Technical University
Hemophilia a, Hemophilia B
Study of TU7710 in Warfarin Anti-coagulated Healthy Male Subjects
This is a Phase 1a, double-blind, randomized, placebo- controlled, SAD study to assess safety, tolerability, PK, and PD of TU7710 in warfarin treated healthy male participants.
- Conditions
- Hemophilia a, Hemophilia B, Blood Coagulation Disorders
- Trial name
- NCT06025552
- Testing
- TU7710, Normal saline
- Where
- South Korea
- Volunteers
- 40 people, healthy volunteers welcome
- Sponsor
- TiumBio Co., Ltd.
Hemophilia A, Healthy Adult Participants
A First-in Human Study of ARD001 in Healthy Volunteers and People With Hemophilia A
This study is evaluating ARD001, an investigational medicine being developed for the treatment of hemophilia A. In Phase 1, healthy adult participants will receive a single dose of ARD001 or placebo to assess its safety and…
- Trial name
- NCT07787091
- Testing
- ARD001
- Where
- Singapore
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- Arditus Pte Ltd
Hemophilia A
Version Testing of EnzySystem Version A for Hemophilia A
Background of the study: To measure blood clotting, blood is taken from a vein. This blood is processed in the laboratory and then tested.
- Trial name
- NCT06273865
- Testing
- Several assays
- Where
- 2 sites in Netherlands
- Volunteers
- 40 people, healthy volunteers welcome
- Sponsor
- Radboud University Medical Center
Hemophilia A
Impact of Emicizumab on Activated Clotting Time Using the i-STAT Alinity Analyzer
Emicizumab (Hemlibra®) is a subcutaneous hemostatic treatment approved in 2019 for the prophylaxis in severe hemophilia A. Emicizumab is a bispecific monoclonal antibody mimicking factor VIIIa that provides a constant level of…
- Trial name
- EMISTAT (NCT05856903)
- Testing
- ACT
- Where
- France
- Volunteers
- 15 people, healthy volunteers welcome
- Sponsor
- University Hospital, Grenoble
Haemophilia A
A Research Study Looking at How Different Doses of Study Medicine (Inno8) Work in the Body of People With Haemophilia A
This study will test how different doses of study medicine (Inno8) work in the bodies of people with haemophilia A. The purpose of the study is to see if Inno8 is safe to use for people with haemophilia A.
- Trial name
- VOYAGER2 (NCT07220564)
- Testing
- NNC0442-0344 A
- Where
- 33 sites in United States, Austria and 10 more
- Volunteers
- 30 people, healthy volunteers welcome
- Sponsor
- Novo Nordisk A/S
Spinal Muscular Atrophy, Fragile X Syndrome
Early Check: Expanded Screening in Newborns
Early Check provides voluntary screening of newborns for a selected panel of conditions. The study has three main objectives: 1) develop and implement an approach to identify affected infants, 2) address the impact on infants and…
- Conditions
- Spinal Muscular Atrophy, Fragile X Syndrome, Fragile X - Premutation, Duchenne Muscular Dystrophy
- Trial name
- NCT03655223
- Testing
- Confirmatory Testing
- Where
- United States
- Volunteers
- 30,000 people, healthy volunteers welcome
- Sponsor
- RTI International
Coronavirus Infection (COVID-19), Pulmonary Arterial Hypertension
Pharmacokinetics, Pharmacodynamics, and Safety Profile of Understudied Drugs Administered to Children Per Standard of Care (POPS)
The study investigators are interested in learning more about how drugs, that are given to children by their health care provider, act in the bodies of children and young adults in hopes to find the most safe and effective dose…
- Conditions
- Coronavirus Infection (COVID-19), Pulmonary Arterial Hypertension, Urinary Tract Infections in Children, Hypertension
- Trial name
- POPS or POP02 (NCT04278404)
- Testing
- The POP02 study is collecting bodily fluid samples (i.e., whole blood, effluent samples) of children prescribed the following drugs of interest per standard of care:
- Where
- 52 sites in United States, Canada
- Volunteers
- 5,000 people, healthy volunteers welcome
- Sponsor
- Duke University
Congenital Bleeding Disorder, Haemophilia A
Investigating the Bioequivalence of Eptacog Alfa A 6 mg and NovoSeven® in Healthy Male Subjects
This trial is conducted in Europe. The aim of the trial is to investigate the bioequivalence of eptacog alfa A 6 mg and NovoSeven® in healthy male subjects.
- Conditions
- Congenital Bleeding Disorder, Haemophilia A, Haemophilia A With Inhibitors, Haemophilia B
- Trial name
- NCT02084810
- Testing
- activated recombinant human factor VII, eptacog alfa (activated)
- Volunteers
- healthy volunteers welcome
- Sponsor
- Novo Nordisk A/S
Healthy Volunteers, Hemophilia A
A Study to Investigate the Pharmacokinetics, Safety, and Tolerability of Emicizumab in Healthy Chinese Volunteers
This single-center, open-label study will evaluate the pharmacokinetics, safety, and tolerability of emicizumab following a single subcutaneous (SC) administration to healthy Chinese subjects.
- Trial name
- NCT03380780
- Testing
- Emicizumab
- Where
- China
- Volunteers
- 16 people, healthy volunteers welcome
- Sponsor
- Hoffmann-La Roche
Healthy Volunteers
A Study of Marstacimab to Compare Prefilled Pen (PFP) Device to Prefilled Syringe (PFS) Device
The goal in this study is to show that there are not significant differences in biologic activity of the study drug when administered using either the prefilled pen and prefilled syringe.
- Trial name
- NCT04832139
- Testing
- Marstacimab PFP, Marstacimab PFS
- Where
- Belgium
- Volunteers
- 22 people, healthy volunteers welcome
- Sponsor
- Pfizer
Hemophilia
Restricting Blood Flow in Improving Muscle Strength in Patients With Hemophilic Arthropathy
Background. The main physical sequela of patients with hemophilia is the development of a progressive, degenerative intra-articular lesion, known as hemophilic arthropathy).
- Trial name
- NCT05039008
- Testing
- Blood flow restriction
- Where
- Spain
- Volunteers
- 23 people, healthy volunteers welcome
- Sponsor
- Investigación en Hemofilia y Fisioterapia
Hemophilia A, Hemophilia B
Gaining Insight Into the Complexity of Pain in Patients With Haemophilia
Joint pain has been reported as a major problem in people with haemophilia (PwH). Therefore, haemophilia in adults seems clinically more related to a musculoskeletal disorder than a bleeding disorder, with many patients reporting…
- Trial name
- NCT05606640
- Testing
- Biopsychological pain assessment
- Where
- 2 sites in Belgium
- Volunteers
- 104 people, healthy volunteers welcome
- Sponsor
- Universiteit Antwerpen
Hemophilia A
Trial of Rituximab Versus Oral Cyclophosphamide to Eradicate or Suppress Autoimmune Anti-Factor VIII Antibodies in Acquired Hemophilia A
The purpose of this study is to evaluate the rate of response when administering rituximab to suppress or eliminate the anti-body in a patient's blood that inhibits the effectiveness of their factor replacement product compared…
- Trial name
- NCT00306670
- Testing
- Rituxan, prednisone
- Volunteers
- 2 people, healthy volunteers welcome
- Sponsor
- Georgetown University
Hemophilia
Predictors of Physical Activity in Adult Patients With Hemophilic Arthropathy
Introduction. The development of hemophilic arthropathy causes degenerative joint damage that leads to functional impairment, limiting physical activity and causing disability in patients with hemophilia. Objectives.
- Trial name
- NCT06960499
- Testing
- Surveys
- Where
- Spain
- Volunteers
- 88 people, healthy volunteers welcome
- Sponsor
- Universidad Católica San Antonio de Murcia
Hereditary Factor VIII Deficiency Disease With Inhibitor
A Comparison Study of Bypassing Agent Therapy With and Without Tranexamic Acid in Haemophilia A Patients With Inhibitor
Activated prothrombin complex concentrate (aPCC) and recombinant activated factor VII (rFVIIa) are the only two drugs that are available to treat bleeds in haemophilia A patients with high titer inhibitors.
- Trial name
- BPATXAS (NCT01800435)
- Testing
- aPCC, aPCC + TXA, rFVIIa, rFVIIa + TXA
- Where
- Norway
- Volunteers
- 6 people, healthy volunteers welcome
- Sponsor
- Oslo University Hospital
Hemophilia
Motor Proficiency of People With Bleeding Disorders Using the BOT-2 (TM)
The purpose of this study is to find out if gross motor skills of children and young adults with bleeding disorders are different from those without bleeding disorders.
- Trial name
- NCT02279199
- Where
- 2 sites in United States
- Volunteers
- 45 people, healthy volunteers welcome
- Sponsor
- Oregon Health and Science University