Clinical trials: Classic Hemophilia
Hemophilia
Movement Visualization in Patients With Hemophilic Arthropathy
Background. The recurrence of hemarthrosis in patients with hemophilia favors the development of an intra-articular, degenerative and progressive lesion (hemophilic arthropathy).
- Trial name
- NCT04237766
- Testing
- Experimental group
- Volunteers
- 140 people, healthy volunteers welcome
- Sponsor
- Real Fundación Victoria Eugenia
Trisomy 13 Syndrome, Arthrogryposis Congenita Multiplex With Intestinal Atresia
Palliative Care Needs of Children With Rare Diseases and Their Families
The palliative care needs of family caregivers of children with rare diseases and their children are largely unmet, including the need for support to prepare for future medical decision making.
- Conditions
- Trisomy 13 Syndrome, Arthrogryposis Congenita Multiplex With Intestinal Atresia, Asparagine Synthetase Deficiency, CHARGE Syndrome
- Trial name
- FACE-Rare (NCT06938542)
- Testing
- Family Centered pediatric palliative care for family caregivers of children with rare diseases.
- Where
- United States
- Volunteers
- 480 people, healthy volunteers welcome
- Sponsor
- Children's National Research Institute
Immune Thrombocytopenia, Thrombotic Thrombocytopenic Purpura
Longitudinal Cohort of Thrombosis and Hemostasis Diseases
This is a multicenter, prospective, longitudinal, observational cohort study to investigate thrombosis and hemostasis diseases in Chinese patients.
- Conditions
- Immune Thrombocytopenia, Thrombotic Thrombocytopenic Purpura, Hemophilia A, Acquired, Disseminated Intravascular Coagulation
- Trial name
- NCT06727669
- Where
- 5 sites in China
- Volunteers
- 3,000 people, healthy volunteers welcome
- Sponsor
- Peking University People's Hospital
Haemophilia A
A Study to Compare the Blood Levels of Mim8 in Healthy Men When Administered With the DV3407-C1 Pen Injector or a Syringe and Cartridge
Novo Nordisk is developing the study medicine Mim8 for the treatment of haemophilia A. The study aims to show similar levels of Mim8 in blood when using a new pen injector, called DV3407-C1 pen injector, and when using a syringe…
- Trial name
- NCT05681845
- Testing
- NNC0365-3769 (Mim8)
- Where
- Germany
- Volunteers
- 205 people, healthy volunteers welcome
- Sponsor
- Novo Nordisk A/S
Hemophilia
Assessment of Proprioceptive and Functional Characteristics in Patients With Hemophilia
Analyze and evaluate the skeletal muscle of patients affected with hemophilia in the Region of Murcia. Describe the periarticular muscular deficit of the lower limbs, depending on the degree of injury: acute and chronic, or…
- Trial name
- HE-BALANCE (NCT02165592)
- Where
- Spain
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- Universidad Católica San Antonio de Murcia
Hemophilia A
Phase 3 Study for Efficacy and Safety Outcomes Data in Japanese Patients With Severe Hemophilia A
This Phase III clinical study will evaluate the safety and effectiveness of valoctocogene roxaparvovec in Japanese patients with severe hemophilia A.
- Trial name
- GENEr8-JPN (NCT06224907)
- Testing
- Valoctocogene roxaparvovec
- Where
- 4 sites in Japan
- Volunteers
- 6 people, healthy volunteers welcome
- Sponsor
- BioMarin Pharmaceutical
Haemophilia A
A Study Looking at How Different Doses of Study Medicine (Inno8) Works in the Body of Healthy Men
This study will test how different doses of study medicine (Inno8) work in the healthy men. The purpose of this study is to prove safety of Inno8 in healthy men, which will support further development of Inno8 in people with…
- Trial name
- VOYAGER1 (NCT06649630)
- Testing
- NNC0442-0344 A
- Where
- 2 sites in Germany
- Volunteers
- 95 people, healthy volunteers welcome
- Sponsor
- Novo Nordisk A/S
Haemophilia
Movement Visualization in Pain Management in Patients With Hemophilic Arthropathy.
Background. The recurrence of hemarthrosis in patients with hemophilia favors the development of a progressive, degenerative intra-articular lesion (hemophilic arthropathy).
- Trial name
- HeMirror (NCT04549402)
- Testing
- Mirror Therapy group, Video group
- Where
- Spain
- Volunteers
- 70 people, healthy volunteers welcome
- Sponsor
- Investigación en Hemofilia y Fisioterapia
Hemophilia A and B, Periodontitis
Effect of Non-Surgical Periodontal Treatment on Oral Health in Hemophilia Patients
This study aimed to evaluate the effect of non-surgical periodontal treatment (NSPT) on oral health-related quality of life in patients with hemophilia using the Oral Health Impact Profile-14 (OHIP-14) questionnaire.
- Conditions
- Hemophilia A and B, Periodontitis, Hemophilia
- Trial name
- NCT07691437
- Testing
- Non-surgical periodontal treatment
- Where
- Turkey (Türkiye)
- Volunteers
- 146 people, healthy volunteers welcome
- Sponsor
- Karadeniz Technical University
Congenital Bleeding Disorder, Haemophilia A
A Multi-centre, Open Labelled, Multiple Dosing Trial Investigating Safety, Pharmacokinetics and Pharmacodynamics of NNC 0172-2021 Administered Subcutaneously to Healthy Male Subjects and Haemophilia Subjects
This trial is conducted in Europe. The aim of this trial is to investigate safety, pharmacokinetics (the exposure of the trial drug in the body) and pharmacodynamics (the effect of the investigated drug on the body) of NNC…
- Conditions
- Congenital Bleeding Disorder, Haemophilia A, Healthy
- Trial name
- explorer™2 (NCT01631942)
- Testing
- NNC172-2021
- Where
- 6 sites in Austria, France and 3 more
- Volunteers
- 4 people, healthy volunteers welcome
- Sponsor
- Novo Nordisk A/S
Hemophilia A
Biomarkers Serum Collection Methodology Pilot Study
The purpose of this study is to compare the standard collection of whole blood in tiger top tubes for serum collection versus a collection method of added FVIII to whole blood samples in the hemophilia population for routine…
- Trial name
- NCT01318707
- Where
- United States
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- Georgetown University
Hemophilia
Risk Models to Optimise Prophylaxis Schedules in Children With Haemophilia
The MOrPH study is designed to identify optimal prophylaxis schedules for children with haemophilia. This involves development of combined pharmacokinetic and pharmacodynamic models.
- Trial name
- MOrPH (NCT02585635)
- Volunteers
- 9 people, healthy volunteers welcome
- Sponsor
- Neuroscience Research Australia
Hemophilia, Hemophilia A
Viscoelastic Properties of Lower Extremity Muscles in Patients With Hemophilia
The aim of our study is to evaluate the viscoelastic properties of lower extremity muscles in patients with hemophilia A and hemophilia B secondarily to compare them with their healthy peers.
- Conditions
- Hemophilia, Hemophilia A, Hemophilia B
- Trial name
- NCT05981313
- Where
- Turkey (Türkiye)
- Volunteers
- 40 people, healthy volunteers welcome
- Sponsor
- Hasan Kalyoncu University
Severe Hemophilia A Without Inhibitor
A Study on the Bone-health Effectiveness of Applying Recombinant Factor VIII Fc (rFVIIIFc) to Patients With Hemophilia A (Prototype A)
Study Objectives* 1. Provide a systematic evaluation of the treatment outcomes in patients with hemophilia A 2. Emphasize the importance and clinical benefits of rFVIII-Fc in joint and bone health. 3.
- Trial name
- NCT05981274
- Testing
- serum biomarker.
- Where
- Taiwan
- Volunteers
- 45 people, healthy volunteers welcome
- Sponsor
- Tri-Service General Hospital (TSGH)
Cystic Fibrosis, Hemophilia A
Medication Adherence and Non-adherence in Adults With Rare Disease
The purpose of this study is to use the Medication Adherence Reasons Scale (MAR-Scale) to determine the extent of non-adherence to specific medications indicated to treat cystic fibrosis, hemophilia (A or B), idiopathic pulmonary…
- Conditions
- Cystic Fibrosis, Hemophilia A, Hemophilia B, Idiopathic Pulmonary Fibrosis
- Trial name
- NCT04541875
- Testing
- MAR-Scale
- Volunteers
- healthy volunteers welcome
- Sponsor
- Xperiome
Hemophilia A, Hemophilia A, Severe
Hemophilia A Research Program
This study longitudinally observes the intergenerational (mother-child) continuum in hemophilia A from pregnancy through early childhood.
- Conditions
- Hemophilia A, Hemophilia A, Severe, Factor VIII (FVIII), FVIII Deficiency
- Trial name
- HARP (NCT07414511)
- Where
- United States
- Volunteers
- 500 people, healthy volunteers welcome
- Sponsor
- University of Washington
Hemophilia
Haemophilia and Bone Loss - PHILEOS Study
Haemophilia is a rare bleeding disorder, characterized by factor VIII (HA) or factor IX (HB) deficiency. The absence or the reduction of fVIII or fIX result in impaired thrombin generation and clot formation, causing excessive…
- Trial name
- PHILEOS (NCT04384341)
- Testing
- Bone densitometry (BMD), Blood sampling for patients only
- Where
- 23 sites in Belgium, Croatia and 3 more
- Volunteers
- 448 people, healthy volunteers welcome
- Sponsor
- Centre Hospitalier Universitaire de Saint Etienne
Hemophilia, Healthy Children
Comparison of Pulmonary Function, Exercise Capacity, and Physical Activity Level Between Children With Hemophilia and Healthy Controls
Hemophilia is a rare X-linked congenital bleeding disorder characterized by deficiency of clotting factor VIII (hemophilia A) or deficiency of factor IX (hemophilia B) with complex diagnosis and management.
- Trial name
- NCT06636565
- Testing
- No intervention
- Where
- Turkey (Türkiye)
- Volunteers
- 34 people, healthy volunteers welcome
- Sponsor
- Hacettepe University
Healthy Participants, Hemophilia A
A Study of Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of SR604 in Two Participants Groups (Part A: Healthy Participants, and Part B: Participants With Hemophilia A or Hemophilia B or Factor VII Deficiency)
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamic (PD) of SR604 in healthy participants (Part A) and to evaluate the safety, tolerability, PK, PD, and efficacy of SR604…
- Conditions
- Healthy Participants, Hemophilia A, Hemophilia B, Factor VII Deficiency
- Trial name
- NCT06349473
- Testing
- SR604
- Where
- 10 sites in United States, Canada
- Volunteers
- 31 people, healthy volunteers welcome
- Sponsor
- Equilibra Bioscience LLC
Congenital Bleeding Disorder, Haemophilia A
Investigation of the Pharmacokinetics of NNC172-2021, at Two Different Dose Levels, in Healthy Japanese Subjects
This trial is conducted in Europe. The aim of this trial is to investigate the pharmacokinetics (how the trial drug is distributed in the body) of NNC172-2021 administered subcutaneously, at two different dose levels, in healthy…
- Conditions
- Congenital Bleeding Disorder, Haemophilia A, Haemophilia B, Healthy
- Trial name
- NCT01555749
- Testing
- NNC172-2021
- Where
- United Kingdom
- Volunteers
- 8 people, healthy volunteers welcome
- Sponsor
- Novo Nordisk A/S