Clinical trials: Hemophilia a
Septic Shock, Sepsis
Effects of Anisodamine on Sublingual Microcirculation and Vascular Waterfall Phenomenon in Patients With Septic Shock
This prospective, multicenter, single-arm, open-label interventional pilot study aims to evaluate the short-term physiological effects of intravenous anisodamine on sublingual microcirculation and vascular-waterfall parameters in…
- Conditions
- Septic Shock, Sepsis, Critical Illness
- Trial name
- ANISO-MICRO (NCT07657702)
- Testing
- Anisodamine intravenous infusion
- Where
- China
- Volunteers
- 20 people
- Sponsor
- First Affiliated Hospital of Wannan Medical College
Hemophilia B
Trial of AAV5-hFIX in Severe or Moderately Severe Hemophilia B
This study evaluates how safe gene therapy treatment with AAV5-hFIX is in adult patients with severe or moderately severe hemophilia B and severe bleeding type.
- Trial name
- NCT02396342
- Testing
- AAV5-hFIX
- Where
- 7 sites in Denmark, Germany and 1 more
- Volunteers
- 10 people
- Sponsor
- CSL Behring
Congenital Bleeding Disorder, Haemophilia A
Intra-subject Variability Following Administrations of Activated Recombinant Human Factor VII in Haemophilia Patients in a Non-bleeding State
This trial is conducted in Asia and Europe. The aim of this trial is to evaluate the intra-subject variability of thromboelastographic parameters (TEG® and ROTEM®) following two administrations of activated recombinant human…
- Conditions
- Congenital Bleeding Disorder, Haemophilia A, Haemophilia B
- Trial name
- NCT01562457
- Testing
- activated recombinant human factor VII
- Where
- 3 sites in France, Germany and 1 more
- Volunteers
- 30 people
- Sponsor
- Novo Nordisk A/S
Hemophilia Arthropathy
Pain and Joint Status With Psychosocial Factors in Patients With Hemophilia
Background. The recurrence of hemarthrosis in patients with congenital coagulopathies favors the development of a progressive, degenerative intra-articular lesion (hemophilic arthropathy) that mainly affects the knees, ankles and…
- Trial name
- NCT04715100
- Testing
- Observational group
- Where
- Spain
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- Investigación en Hemofilia y Fisioterapia
Severe Hemophilia A, Severe Hemophilia A With Inhibitor
Dynamics of the Anti-factor VIII Antibody Signature During Treatment With Emicizumab
The goal of this observational study is to learn about the changes of antibodies and inhibitors against the coagulation factor VIII in patients with severe hemophilia A receiving emicizumab therapy.
- Conditions
- Severe Hemophilia A, Severe Hemophilia A With Inhibitor, Severe Hemophilia A Without Inhibitor
- Trial name
- NAVIGATE (NCT05802836)
- Testing
- no interventions
- Where
- Germany
- Volunteers
- 100 people
- Sponsor
- Christoph Königs
Sonography, Haemophilia
Sonography and Haemophilia
The aim of this study is to evaluate the possibility of establishing a patient-based ultrasound monitoring of the knee (representing a target joint accessible with any hand) with a handheld device: The following questions will be…
- Trial name
- NCT04550988
- Testing
- feasibility study
- Where
- Germany
- Volunteers
- 5 people
- Sponsor
- University Hospital, Bonn
Hemophilia A, Hemophilia B
A Single Escalating Dose and Multiple Dose Study of BAY 1093884 in Subjects With Severe Hemophilia Types A or B, With or Without Inhibitors
Investigate the safety, tolerability and pharmacokinetics of BAY1093884 after Intravenous (IV) and subcutaneous (SC) administration of increasing single doses and SC administration of multiple doses.
- Trial name
- NCT02571569
- Testing
- BAY1093884
- Where
- 10 sites in Bulgaria, Germany and 3 more
- Volunteers
- 32 people
- Sponsor
- Bayer
Hemophilia A
Real World Use of Emicizumab in Infants and Children Ages 0-3 Years With Hemophilia A
Hemophilia A (HA) is a rare congenital bleeding disorder characterized by coagulation factor VIII deficiency. In severe HA, defined as plasma FVIII clotting activity < 1%, bleeding may frequently occur spontaneously, most…
- Trial name
- NCT05248594
- Testing
- HEMLIBRA
- Where
- 10 sites in United States
- Volunteers
- 71 people
- Sponsor
- Montefiore Medical Center
Hemophilia A
Study to Learn More About the Physical Activity Level of Patients Suffering From Hemophilia A Treated With Damoctocog Alfa Pegol (LIFE ACTIVE Study)
In this study researcher want to learn more about the change of physical activity of hemophilia A patients treated with damoctocog alfa pegol.
- Trial name
- LIFE-ACTIVE (NCT04091386)
- Testing
- Damoctocog alfa pegol (Jivi, BAY94-9027)
- Where
- 8 sites in United States, Belgium and 5 more
- Volunteers
- 10 people
- Sponsor
- Bayer
Severe Hemophilia A
Hemophilia Adult Prophylaxis Study: Factor VIII in Severe Hemophilia A
The purpose of this pilot R34 trial is to determine the feasibility of a large single dose Phase III study of hemophilia adult prophylaxis comparing once weekly with thrice-weekly recombinant factor VIII.
- Trial name
- NCT01405742
- Testing
- rF.VIII
- Where
- 6 sites in United States
- Volunteers
- 4 people
- Sponsor
- University of Pittsburgh
Haemophilia
Determinants and Consequences of the Transition to Adulthood for Adolescents With Severe Haemophilia: TRANSHEMO 2, an Ancillary Study to the TRANSHEMO Project
Haemophilia is a rare genetic disorder which, in its severe form and in the absence of treatment, can be life-threatening. Since the 1960s and the introduction of coagulation factor concentrates, the life expectancy of people…
- Trial name
- TRANSHEMO2 (NCT07418827)
- Testing
- questionnaires
- Where
- 25 sites in France
- Volunteers
- 75 people
- Sponsor
- Assistance Publique Hopitaux De Marseille
Hemophilia A, Hemophilia B
An Open-label Extension Study of an Investigational Drug, Fitusiran, in Patients With Moderate or Severe Hemophilia A or B
Primary Objective: To evaluate the long-term safety and tolerability of fitusiran in male patients with moderate or severe hemophilia A or B Secondary Objectives: To investigate the long-term efficacy of fitusiran To characterize…
- Trial name
- NCT02554773
- Testing
- Fitusiran (SAR439774)
- Where
- 12 sites in United States, Bulgaria and 3 more
- Volunteers
- 34 people
- Sponsor
- Genzyme, a Sanofi Company
Severe Hemophilia A
Human Cell Line-derived Recombinant Factor VIII (Human-cl-rhFVIII) in Previously Untreated Patients
Investigate the inhibitor development rate of Human cl rhFVIII in previously untreated patients with severe Hemophilia A.
- Trial name
- NCT01712438
- Testing
- Human cl rhFVIII
- Where
- 37 sites in United States, Belarus and 15 more
- Volunteers
- 110 people
- Sponsor
- Octapharma
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
Hemophilia a, Hemophilia B
The Efficacy and Safety of SerpinPC in Participants with Severe Hemophilia a or Moderately Severe to Severe Hemophilia B
The purpose of the study is to evaluate the efficacy, safety, tolerability and pharmacokinetics of prophylactic SerpinPC administered subcutaneously (SC) to participants with severe hemophilia A (HemA) (with or without…
- Trial name
- PRESent-2 (NCT05789524)
- Testing
- SerpinPC
- Where
- 59 sites in United States, Armenia and 13 more
- Volunteers
- 60 people
- Sponsor
- ApcinteX Ltd
Hemophilia A, Hemophilia B
Fc factOrs and Real-World hemophiliA Patient-ReporteD Outcomes
The primary objective of the study is to evaluate the effectiveness of prophylactic treatment with recombinant Factor VIII Fc fusion protein (rFVIIIFc) and recombinant Factor IX Fc fusion protein (rFIXFc) therapy as assessed by…
- Trial name
- FORWARD (NCT02392156)
- Testing
- rFVIIIFc, rFIXFc, non-Fc FVIII replacement products
- Where
- 2 sites in United States
- Volunteers
- 3 people
- Sponsor
- Bioverativ Therapeutics Inc.
Hemophilia A
Manual Therapy in Hemophilic Arthropathy of the Ankle
Introduction: Haemophilic ankle arthropathy manifests as functional (deficit in muscle strength, mobility and proprioception), intra-articular degenerative alterations and chronic pain.
- Trial name
- NCT06816056
- Testing
- Manual Therapy
- Where
- Spain
- Volunteers
- 13 people, healthy volunteers welcome
- Sponsor
- Investigación en Hemofilia y Fisioterapia
Hemophilia A
Retrospective Study in Chinese Pediatric Hemophilia A Patients With rFⅧ Contained Regular Prophylaxis
To assess and describe real-life treatment choices for rFⅧ contained regular prophylaxis/bleeding prevention treatment in pediatric hemophilia patients in China (2007-2013)
- Trial name
- ReCARE (NCT02263066)
- Testing
- BAY14-2222_Kogenate-FS FVIII
- Where
- China
- Volunteers
- 181 people
- Sponsor
- Bayer
Hemophilia, Arthropathy Hemophilic
The Effects of Manual Therapy in Hemophilic Patients
Uncertainty about how to treat existing hemophilic arthropathy (HA) is the most important issue that will increase functionality and joint health.
- Trial name
- NCT04309903
- Testing
- Manual therapy
- Where
- Turkey (Türkiye)
- Volunteers
- 17 people
- Sponsor
- Necmettin Erbakan University
Hemophilia A
Study of a pd vWF/FVIII, Biostate®, in Subjects With Haemophilia A
The aim of this study are to assess the efficacy of Biostate® [Study Product (SP)] in subjects with Haemophilia A compare the pharmacokinetics of Biostate® [SP] with the previously marketed product Biostate® (here referred to as…
- Trial name
- NCT00879541
- Testing
- Biostate® [SP], Biostate® [RP]
- Where
- 14 sites in Bulgaria, North Macedonia and 2 more
- Volunteers
- 81 people
- Sponsor
- CSL Behring