Clinical trials: Severe Hemophilia A
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 A Without Inhibitor
HemFitBit, an Observational Study of Physical Activity in People With Moderate and Severe Haemophilia A
The study aims to describe physical activity (PA) levels in young people with haemophilia A in Norway compared with non-haemophilia controls, and to identify factors influencing PA.
- Trial name
- NCT04181697
- Testing
- PA measurement with Fitbit Charge 3, PA measurement with Fitbit and ActiGraph GT3X
- Where
- Norway
- Volunteers
- 40 people
- Sponsor
- Oslo University Hospital
Severe Hemophilia A Without Inhibitor
Clinical Outcomes of Low Dose PK-guided EHL FVIII Concentrates Versus Standard Prophylaxis in Severe Haemophilia A
Individualised pharmacokinetic (PK)-guided dosing of extended half-life (EHL) FVIII concentrates prophylaxis may reduce hemophilia A bleeding events than previous prophylactic regimen.
- Trial name
- NCT05281185
- Testing
- Extended half-life FVIII concentrates with PK-guided dosing, standard half-life FVIII concentrates with weight-based dosing
- Where
- Thailand
- Volunteers
- 15 people
- Sponsor
- Chulalongkorn 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)
Hemophilia A
Relative Bioavailability Of Two Formulations Of Moroctocog Alfa (AF-CC)
This study is being conducted to compare how moroctocog alfa (AF-CC) acts in the body when administered as 2 different dose presentations.
- Trial name
- NCT01579903
- Testing
- moroctocog alfa (AF-CC)
- Where
- 3 sites in Bulgaria, Hungary and 1 more
- Volunteers
- 16 people
- Sponsor
- Pfizer
Hemophilia B, Allergic Reactions
Study to Describe the Allergic Reactions to Factor IX in Patients With Hemophilia B
Retrospective, multicenter study in patients with hemophilia B. This study will be placed at 20 to 50 hemophilia treatment centers in North America and the EU, and is designed to estimate the frequency of Class II and III…
- Trial name
- NCT00195221
- Where
- 38 sites in United States, Austria and 7 more
- Volunteers
- 166 people
- Sponsor
- Wyeth is now a wholly owned subsidiary of Pfizer
Hemophilia A Patient
Efanesoctocog Prophylaxis Or The Optimization Of Treatment: Real-World Experience Of Taylorizing Clotting Therapy In Hemophilia A
Prophylactic management of severe Hemophilia A (HA) without inhibitors has historically relied on frequent intravenous infusions of standard or EHL FVIII concentrates or on subcutaneous emicizumab administered weekly to monthly…
- Trial name
- E-PROTECT (NCT07830693)
- Testing
- Study questionnaires, Clinical/pharmacological data collection
- Where
- 8 sites in France
- Volunteers
- 553 people
- Sponsor
- Nantes University Hospital
Severe Hemophilia A With an Inhibitor
The FEIBA NovoSeven Comparative Study
FENOC is a prospective, open-label, randomized, cross-over, multi-center study to investigate and compare the hemostatic effect and cost-efficacy of two different by-passing agents in the treatment of joint hemorrhages in…
- Trial name
- NCT00166309
- Testing
- FEIBA and NovoSeven
- Where
- Sweden
- Volunteers
- 60 people
- Sponsor
- Skane University Hospital
Hemophilia A, Hemophilia B
A Study of Fitusiran (ALN-AT3SC) in Severe Hemophilia A and B Patients Without Inhibitors
Primary Objective: -To evaluate the efficacy of fitusiran compared to on-demand treatment with factor concentrates, as determined by the frequency of bleeding episodes.
- Trial name
- NCT03417245
- Testing
- fitusiran, factor concentrates
- Where
- 64 sites in United States, Australia and 17 more
- Volunteers
- 120 people
- Sponsor
- Genzyme, a Sanofi Company
Haemophilia A
TGA (Thrombin Generation Assay) and Prophylaxis in Haemophilia
Haemophilic arthropathy is one of the major complications of severe haemophilia. In order to maintain plasma clotting factor activity levels above 1% and avoid spontaneous joint bleeds and other serious bleeding events…
- Trial name
- HOPE (NCT02781766)
- Testing
- Thrombin generation measurement.
- Where
- 7 sites in France
- Volunteers
- 50 people
- Sponsor
- Hospices Civils de Lyon
Hemophilia B
A Study to Evaluate the Efficacy and Safety of Factor IX Gene Therapy With PF-06838435 in Adult Males With Moderately Severe to Severe Hemophilia B
This study will evaluate the efficacy and safety of PF-06838435 (a gene therapy drug) in adult male participants with moderately severe to severe hemophilia B (participants that have a Factor IX circulating activity of 2% or…
- Trial name
- BENEGENE-2 (NCT03861273)
- Testing
- PF-06838435/ fidanacogene elaparvovec
- Where
- 65 sites in United States, Australia and 14 more
- Volunteers
- 51 people
- Sponsor
- Pfizer
Hemophilia A
A Safety, Tolerability, and Pharmacokinetics Study of a Single Intravenous Injection of Recombinant Coagulation Factor VIII Fc - Von Willebrand Factor - XTEN Fusion Protein (rFVIIIFc-VWF-XTEN) (BIVV001) in Previously Treated Adults With Severe Hemophilia A (EXTEN-A)
The primary purpose was to assess the safety and tolerability of a single intravenous (IV) administration of BIVV001 in adult previously treated patients (PTPs) with severe hemophilia A.
- Trial name
- NCT03205163
- Testing
- Advate (Low Dose), Advate (High Dose), BIVV001 (Low Dose)
- Where
- 10 sites in United States, Japan
- Volunteers
- 16 people
- Sponsor
- Bioverativ, a Sanofi company
Hemophilia
Comparison of Efficacy, Safety and Costs of Recombinant FVIII Products Between On-demand and Secondary Prophylaxis Groups in Haemophilia A Patients
The project is an observational, multi-central, prospective, non-interventional and open-label data collection study on secondary prophylaxis with recombinant FVIII products in adolescents and adults with severe hemophilia A…
- Trial name
- NCT01817868
- Testing
- Recombinant Factor VIII (Kogenate, BAY14-2222)
- Where
- Turkey (Türkiye)
- Volunteers
- 73 people
- Sponsor
- Bayer
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
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
Hemophilia A
Efficacy and Safety of ADVATE Standard Prophylaxis to Hemophilia A
Hemophilia A is an X-linked recessive, congenital bleeding disorder caused by deficient or defective coagulation factor VIII (FVIII).
- Trial name
- NCT02280265
- Testing
- Recombinant Human Coagulation Factor VIII for injection
- Volunteers
- 15 people
- Sponsor
- Nanjing Medical University
Hemophilia B
Hemophilia B Gene Therapy With AAV8 Vector
Hemophilia B is a bleeding disease in males due to very low levels of coagulation factor IX (FIX) in the blood. The current treatment is intravenous injection of FIX clotting factor concentrates, in response to bleeding.
- Trial name
- NCT01620801
- Testing
- AAV8-hFIX19
- Where
- 3 sites in United States, Australia
- Volunteers
- 4 people
- Sponsor
- Spark Therapeutics, Inc.
Hemophilia
A Phase 1b Study to Assess the Safety, Tolerability, PK and PD of MG1113 in Hemophilia Patient
The purpose of this study is to assess the safety and tolerability, pharmacokinetics and pharmacodynamics of subcutaneous MG1113 in the multiple ascending dose study in patients with severe hemophilia.
- Trial name
- NCT05493631
- Testing
- MG1113
- Where
- South Korea
- Volunteers
- 15 people
- Sponsor
- GC Biopharma Corp
Hemophilia A
Safety and Efficacy of BAY94-9027 in Previously Treated Male Children With Haemophilia A
Hemophilia A is an inherited blood disorder in which one protein, Factor VIII, needed to form blood clots is missing or not present in sufficient levels.
- Trial name
- NCT01775618
- Testing
- BAY94-9027
- Where
- 37 sites in United States, Argentina and 15 more
- Volunteers
- 73 people
- Sponsor
- Bayer