Clinical trials: Congenital Bleeding Disorder
Hemophilia A
Evaluation of Patient and Physician Reported Reasons for Switching Factor VIII Replacement Therapies
This US study will assess hemophilia A patient characteristics, health history and reasons for switching or not switching from both patient/caregiver and physician perspectives.
- Trial name
- PARkER (NCT03405337)
- Testing
- FVIII products, Conventional FVIII replacement therapies
- Where
- United States
- Volunteers
- 160 people
- Sponsor
- Bayer
Von Willebrand Disease (VWD), Von Willebrand Disease (VWD), Type 1
A Study of Bleeding and Treatment in Participants With Von Willebrand Disease
The purpose of this screening study is to accumulate information regarding bleeding events, quality of life, and the social and clinical impact of bleeds in participants with Von Willebrand Disease (VWD).
- Conditions
- Von Willebrand Disease (VWD), Von Willebrand Disease (VWD), Type 1, Von Willebrand Disease (VWD), Type 2, Von Willebrand Disease (VWD), Type 3
- Trial name
- NCT06610201
- Testing
- Clinical outcomes of patients with VWD, Type 1, Clinical outcomes of patients with VWD, Type 2A, Type 2M, Type 2N, or Type 3
- Where
- 17 sites in United States, Australia and 1 more
- Volunteers
- 200 people
- Sponsor
- Hemab ApS
Hemophilia A
Evaluation of SCT800 in Prophylaxis Therapy on Previous Treated Patients With Severe Hemophilia A
This study is a multi-center, open-label, single-arm trial to evaluate the efficacy,safety and pharmacokinetics of SCT800 in regular prophylaxis and perioperative treatment in patients (≥12 years old) with severe hemophilia A who…
- Trial name
- NCT03815318
- Testing
- Recombinant Human Coagulation FVIII
- Where
- China
- Volunteers
- 73 people
- Sponsor
- Sinocelltech Ltd.
Severe Hemophilia A
Study to Investigate the Long-term Efficacy and Safety of Human-cl rhFVIII in Previously Treated Patients (PTPs)
The purpose of the study is to study the long-term efficacy, safety and tolerability of Human-cl rhFVIII in previously treated patients with severe hemophilia A.
- Trial name
- NCT01341912
- Testing
- Human-cl rhFVIII
- Where
- 2 sites in United States, Bulgaria
- Volunteers
- 3 people
- Sponsor
- Octapharma
Pregnancy Complications, Maternal Death
Nutrition Optimalization Among Pregnant Women to Improve Maternal and Neonatal Outcome in DKI Jakarta
The goal of this MONAS Study is to learn about comprehensive monitoring and nutritional intervention among pregnant women in order to improve maternal and neonatal outcomes. The main questions it aims to answer are: 1.
- Conditions
- Pregnancy Complications, Maternal Death, Preeclampsia, Intrauterine Infection
- Trial name
- MONAS (NCT06268509)
- Testing
- Nutritional panel laboratory examination, Pregnancy supplements (other than provided by Ministry of Health), Fetomaternal ultrasound
- Where
- Indonesia
- Volunteers
- 245 people
- Sponsor
- Dr Cipto Mangunkusumo General Hospital
Hemophilia, Hemophilia A
ATHN 8: Previously Untreated Patients (PUPs) Matter Study
This is a multi-center cohort study of approximately 250 previously untreated patients (PUPs) with congenital moderate to severe hemophilia A or B in a network of up to 50 US Hemophilia Treatment Centers (HTCs).
- Conditions
- Hemophilia, Hemophilia A, Hemophilia B, Hemophilia A With Inhibitor
- Trial name
- NCT03818529
- Where
- 27 sites in United States
- Volunteers
- 237 people
- Sponsor
- American Thrombosis and Hemostasis Network
Craniosynostoses, Hypovolemia
Hemodynamic Monitoring During Craniosynostosis Surgery: Comparing Traditional and Newer Technology Monitors (CRASY-PRAM)
Hemodynamic evaluation during pediatric anesthesia is essential to care management. Intraoperative cardiovascular instability is frequent in major surgeries, and appropriate monitoring is necessary to ensure safe anesthetic…
- Conditions
- Craniosynostoses, Hypovolemia, Hemorrhage, Hemodynamic Instability
- Trial name
- CRASY-PRAM (NCT06263075)
- Testing
- Intraoperative monitoring
- Where
- Italy
- Volunteers
- 30 people
- Sponsor
- IRCCS Azienda Ospedaliero-Universitaria di Bologna
Hemophilia, Hemophilia A
Improved Factor VIII Inhibitor Evaluation
Commercial one and two-stage factor VIII assays may not detect some clinically significant inhibitor antibodies. The purpose of the proposed study is to standardize and validate a platelet-based factor VIII activity assay with…
- Trial name
- IFIE (NCT02726139)
- Where
- United States
- Sponsor
- Boston VA Research Institute, Inc.
Penile Infection, Penile Haematoma
Evaluation of the Acceptability and Safety of the ShangRing Device for Male Circumcision in Shinyanga, Tanzania
The goal of this observational study was to evaluate the safety and acceptability of the ShangRing device for male circumcision among men in Shinyanga, Tanzania.
- Conditions
- Penile Infection, Penile Haematoma, Penile Bleeding, Deformity
- Trial name
- NCT06273696
- Testing
- ShangRing
- Where
- 2 sites in Tanzania
- Volunteers
- 575 people, healthy volunteers welcome
- Sponsor
- IntraHealth International
Hemophilia A
A Study to Test a Medicine (Fitusiran) for Preventing Bleeds in People With Severe Hemophilia Who Previously Received Preventive Treatment With Emicizumab
This is an exploratory, single group, Phase 4, study to assess treatment with fitusiran prophylaxis after switching from emicizumab prophylaxis.
- Trial name
- NCT06145373
- Testing
- Fitusiran (SAR439774), Clotting factor concentrates (CFC) or bypassing agents (BPA), Antithrombin concentrate (ATIIIC)
- Where
- 3 sites in United States, Taiwan
- Volunteers
- 20 people
- Sponsor
- Sanofi
Congenital Bleeding Disorder, Haemophilia A
Safety and Efficacy of Turoctocog Alfa in Haemophilia A Subjects
This trial is conducted in Asia, Europe, and North and South America. The trial consists of a main trial and a sub-trial. The main trial investigates safety and efficacy of turoctocog alfa (recombinant factor VIII, rFVIII (N8))…
- Trial name
- guardian™1 (NCT00840086)
- Testing
- turoctocog alfa
- Where
- 63 sites in United States, Brazil and 16 more
- Volunteers
- 150 people
- Sponsor
- Novo Nordisk A/S
Hemophilia B with Inhibitor, Hemophilia B
A Study of SerpinPC in Participants with Hemophilia B (HemB) with Inhibitors
The purpose of the study is to evaluate the efficacy, safety, tolerability and pharmacokinetic (PK) profile of prophylactic SerpinPC in participants with Hemophilia B with inhibitors, as part of the SerpinPC registrational…
- Trial name
- PRESent-3 (NCT05789537)
- Testing
- SerpinPC
- Where
- 20 sites in United States, Armenia and 9 more
- Volunteers
- 3 people
- Sponsor
- ApcinteX Ltd
Afibrinogenemia, Congenital
Quality of Life in Patients With Congenital Afibrinogenemia
The aim of this observational study is to evaluate the quality of life in patients with congenital afibrinogenemia using the Haemo-QoL SF for kids and the Haem-A-QoL for adult patients.
- Trial name
- QualyAFIB (NCT03484065)
- Testing
- Questionnaire quality of life
- Where
- 28 sites in United States, Algeria and 19 more
- Volunteers
- 250 people
- Sponsor
- University Hospital, Geneva
Sickle Cell Disease, Thalassemia
Unrelated Hematopoietic Stem Cell Transplantation(HSCT) for Genetic Diseases of Blood Cells
This is a clinical trial of bone marrow transplantation for patients with the diagnosis of a genetic disease of blood cells that do not have an HLA-matched sibling donor.
- Conditions
- Sickle Cell Disease, Thalassemia, Anemia, Granuloma
- Trial name
- NCT00730314
- Testing
- Hematopoietic stem cell transplantation
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- Children's Hospital Los Angeles
Von Willebrand Diseases
The Lived Experience of People With Von Willebrand Disease
von Willebrand disease (vWD) is reported to be the most common bleeding disorder, with prevalence estimated at 1% of the general population.
- Trial name
- VWD360 (NCT06064643)
- Testing
- Online Survey, Qualitative Interview, Bleed Diary
- Where
- United Kingdom
- Volunteers
- 611 people
- Sponsor
- Haemnet
Hemophilia A
Satisfaction Regarding the Dispensing of Emicizumab (HEMLIBRA®) for Patients With Haemophilia A in France
Haemophilia A (HA) is a rare constitutional haemorrhagic disease whose drug management is based on the use of chronic lifelong replacement therapy.
- Trial name
- PASODOBLEDEMI2 (NCT05450640)
- Testing
- satisfaction survey
- Where
- France
- Volunteers
- 176 people
- Sponsor
- Hospices Civils de Lyon
Von Willebrand Disease, Type 3
An Observational Study of Participants With Type 3 Von Willebrand Disease on Prophylactic Standard-of-Care Treatment
This non-interventional study (NIS) is designed to collect information on the effectiveness and safety of treatment received in routine clinical care, as well as measure the health-related quality of life (HRQoL) of participants…
- Trial name
- WILL-EMI NIS (NCT06883240)
- Testing
- Von Willebrand Factor Concentrates, Von Willebrand Factor Concentrates and Factor VIII Concentrates, Factor VIII Concentrates
- Where
- 26 sites in United States, Belgium and 12 more
- Volunteers
- 44 people
- Sponsor
- Hoffmann-La Roche
Protein C Deficiency
A Study of Freeze-dried Human Protein C Concentrate (TAK-662) in Participants With Congenital Protein C Deficiency
This study is conducted in Japan of Freeze-dried Human Protein C Concentrate (TAK-662) used to treat participants with congenital protein C deficiency.
- Trial name
- NCT06590974
- Testing
- Freeze-dried Human Protein C Concentrate
- Where
- Japan
- Volunteers
- 7 people
- Sponsor
- Takeda
Hemophilia A
Efficacy and Safety of Recombinant Factor VIII (GreenGene) in Patients With Hemophilia A
The purpose of this study is to study the evaluation of efficacy and safety of GreenGene (Recombinant Factor VIII).
- Trial name
- NCT01568580
- Testing
- GreenGene
- Volunteers
- 71 people
- Sponsor
- Green Cross Corporation
Hemophilia B
Evaluation of a Recombinant Factor IX Product, APVO101, in Previously-Treated Pediatric Patients With Hemophilia B
Phase 3/4, single arm, open-label study to evaluate PK, safety, and efficacy of APVO101 prophylaxis in severe or moderately severe hemophilia B subjects < 12 years of age.
- Trial name
- NCT03855280
- Testing
- APVO101
- Where
- 11 sites in Brazil, Georgia and 4 more
- Volunteers
- 21 people
- Sponsor
- Medexus Pharma, Inc.