Clinical trials: Factor IX Deficiency
Congenital Bleeding Disorder, Haemophilia A
Observational Study Describing the Usual Clinical Practice Use of NovoSeven® in the Home Treatment of Joint Bleeds in Patients With Haemophilia A or B and Inhibitors
This trial is conducted in Africa and Asia. The aim of this study is to evaluate the efficacy of home treatment of joint bleeds (haemarthrosis) with NovoSeven® (activated recombinant human factor VII) in patients with haemophilia…
- Conditions
- Congenital Bleeding Disorder, Haemophilia A, Haemophilia B
- Trial name
- NCT01234545
- Testing
- activated recombinant human factor VII
- Where
- 5 sites in Algeria, Morocco and 3 more
- Volunteers
- 35 people
- Sponsor
- Novo Nordisk A/S
Congenital Bleeding Disorder, Haemophilia B
Safety of 40K Pegylated Recombinant Factor IX in Non-Bleeding Patients With Haemophilia B
This trial is conducted in Europe, Japan and the United States of America (USA). The aim of this clinical trial is to investigate the safety and pharmacokinetics (the determination of the concentration of the administered…
- Trial name
- NCT00956345
- Testing
- nonacog beta pegol
- Where
- 19 sites in United States, Denmark and 6 more
- Volunteers
- 18 people
- Sponsor
- Novo Nordisk A/S
Congenital Bleeding Disorder, Haemophilia A
Observational Study on Safety of Room Temperature Stable NovoSeven® in Patients With Haemophilia A or B
This study is conducted in Europe and Asia. The aim of this observational study is to monitor antibody formation towards the room temperature stable formulation of NovoSeven® (activated recombinant human factor VII).
- Conditions
- Congenital Bleeding Disorder, Haemophilia A, Haemophilia B
- Trial name
- NCT01220141
- Testing
- activated recombinant human factor VII
- Where
- 14 sites in Argentina, Austria and 12 more
- Volunteers
- 51 people
- Sponsor
- Novo Nordisk A/S
Congenital Bleeding Disorder, Haemophilia A With Inhibitors
Safety and Efficacy of Activated Recombinant Human Factor VII in Haemophilia Patients With Inhibitors During and After Major Surgery
This trial is conducted in North America. The aim of this trial is to compare the safety and efficacy of activated recombinant human factor VII in patients with haemophilia A or B undergoing major surgical procedures.
- Conditions
- Congenital Bleeding Disorder, Haemophilia A With Inhibitors, Haemophilia B With Inhibitors
- Trial name
- NCT01561391
- Testing
- activated recombinant human factor VII, factor IX, factor VIII
- Where
- 14 sites in United States
- Volunteers
- 36 people
- Sponsor
- Novo Nordisk A/S
Congenital Bleeding Disorder, Haemophilia A
Haemophilia Patients With Inhibitors Being Treated for Acute Joint Bleeds
This trial is conducted in Africa, Asia, Europe, Japan, and North and South America. The aim of this trial is to evaluate the safety and efficacy of activated recombinant human factor VII analogue (vatreptocog alfa (activated))…
- Conditions
- Congenital Bleeding Disorder, Haemophilia A, Haemophilia B
- Trial name
- NCT00486278
- Testing
- eptacog alfa (activated), vatreptacog alfa (activated)
- Where
- 45 sites in United States, Argentina and 16 more
- Volunteers
- 51 people
- Sponsor
- Novo Nordisk A/S
Factor IX Deficiency
Natural History Study of Factor IX Treatment and Complications
This study will examine two groups of subjects with factor IX (FIX) deficiency: 1) those with a current or history of inhibitors to FIX, and; 2) groups of two or more affected brothers, with or without inhibitors.
- Trial name
- B-Natural (NCT02502409)
- Testing
- Standard care with blood and urine sample collection
- Where
- United States
- Volunteers
- 550 people
- Sponsor
- Skane University Hospital
Hemophilia B
Safety of a New Type of Treatment Called Gene Transfer for the Treatment of Severe Hemophilia B
In this study a modified virus called adeno-associated virus (AAV) will be used to transfer a normal gene for human clotting factor IX into patients with severe hemophilia B (AAV human Factor IX vector).
- Trial name
- NCT00076557
- Testing
- Adeno-Associated Viral with Human Factor IX
- Where
- 3 sites in United States
- Volunteers
- 15 people
- Sponsor
- Avigen
Hemophilia B
Post-Marketing Surveillance (Usage Results Study) of RIXUBIS in Adult and Pediatric Patients With Haemophilia B in South Korea
Primary: To characterize the safety of RIXUBIS when used under normal clinical care in South Korea. Secondary: To describe hemostatic effectiveness in subjects receiving RIXUBIS under normal clinical care in South Korea.
- Trial name
- NCT02922231
- Testing
- RIXUBIS
- Where
- 5 sites in South Korea
- Volunteers
- 57 people
- Sponsor
- Baxalta now part of Shire
Hemophilia A With Inhibitor, Hemophilia B With Inhibitor
Prospective, Non-interventional Study to Evaluate Immunogenicity of AryoSeven
This study evaluates the immunogenicity of the biosimilar rFVIIa (AryoSeven) in subjects receiving AryoSeven in real-life clinical practice.
- Conditions
- Hemophilia A With Inhibitor, Hemophilia B With Inhibitor, Factor VII Deficiency, Glanzmann's Thrombasthenia
- Trial name
- NCT03372993
- Where
- 2 sites in Iran
- Volunteers
- 200 people
- Sponsor
- AryoGen Pharmed Co.
Congenital Bleeding Disorder, Haemophilia A With Inhibitors
Efficacy and Safety of NNC 0078-0000-0007 in Patients With Congenital Haemophilia and Inhibitors
This trial is conducted globally. The purpose of this trial is to confirm the efficacy and safety of NNC 0078-0000-0007 in patients with congenital haemophilia and inhibitors.
- Conditions
- Congenital Bleeding Disorder, Haemophilia A With Inhibitors, Haemophilia B With Inhibitors
- Trial name
- adept™2 (NCT01392547)
- Testing
- vatreptacog alfa (activated), eptacog alfa (activated)
- Where
- 32 sites in United States, Austria and 17 more
- Volunteers
- 72 people
- Sponsor
- Novo Nordisk A/S
Prothrombin Complex Factor Deficiency
Efficacy and Safety of Prothromplex Total (Prothrombin Complex Concentrate) in Oral Anticoagulant Reversal
The purpose of the study is to assess the efficacy and safety of Prothromplex Total as a treatment for the immediate reversal of oral anticoagulant therapy with vitamin K antagonists in patients with acquired deficiency of…
- Trial name
- NCT01159210
- Testing
- Prothrombin complex concentrate (coagulation factors IX, II, VII and X in combination)
- Where
- 6 sites in Austria, Hungary
- Volunteers
- 61 people
- Sponsor
- Baxalta now part of Shire
Haemophilia B
A Study Following Males With Haemophilia B on Prophylaxis With Refixia/REBINYN
This study will collect information on side effects and how well Refixia/REBINYN works during long-term treatment (prophylaxis) in males with haemophilia B.
- Trial name
- NCT03745924
- Testing
- Nonacog beta pegol
- Where
- 30 sites in Austria, Belgium and 11 more
- Volunteers
- 60 people
- Sponsor
- Novo Nordisk A/S
Congenital Bleeding Disorder, Haemophilia A
Investigating Safety and Pharmacokinetics of 2 Different Single Doses of NNC128-0000-2011 in Haemophilia A or B Patients
This trial is conducted in Europe and Japan. The aim of this trial is to assess the safety and pharmacokinetics (the rate at which the body eliminates the trial drug) of single doses of NNC128-0000-2011, when administered i.v.
- Conditions
- Congenital Bleeding Disorder, Haemophilia A, Haemophilia B
- Trial name
- NCT01288391
- Testing
- NNC 0128-0000-2011
- Where
- 3 sites in Germany, Japan and 1 more
- Volunteers
- 15 people
- 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
Congenital Bleeding Disorder, Haemophilia A With Inhibitors
Efficacy of NovoSeven® in Bleeding Prophylaxis in Hemophilia
This trial is conducted in Africa, Asia, Europe, South America, and the United States of America (USA). The purpose of this study is to evaluate the effectiveness of secondary prophylactic treatment with NovoSeven® in haemophilia…
- Conditions
- Congenital Bleeding Disorder, Haemophilia A With Inhibitors, Haemophilia B With Inhibitors
- Trial name
- NCT00108758
- Testing
- activated recombinant human factor VII
- Where
- 37 sites in United States, Argentina and 10 more
- Volunteers
- 23 people
- Sponsor
- Novo Nordisk A/S
Haemophilia A, Haemophilia B
A Study to Evaluate the Real-world Usage and Effectiveness of Elocta and Alprolix in Patients With Haemophilia A or B
Elocta (rFVIIIFc) and Alprolix (rFIXFc) are recombinant extended half-life coagulation factor products. The purpose of this non-interventional study is to describe the real-world usage and effectiveness of Elocta and Alprolix in…
- Trial name
- PREVENT (NCT03055611)
- Testing
- ELOCTA, ALPROLIX
- Where
- 22 sites in Germany
- Volunteers
- 201 people
- Sponsor
- Swedish Orphan Biovitrum
Congenital Bleeding Disorder, Haemophilia A With Inhibitors
Non-Interventional Study of NovoSeven® Used in Patients With Haemophilia A and B With Inhibitors When Undergoing Surgery
This study is conducted in Japan. The aim of this non-interventional study is to investigate the safety and effectiveness of treatment with eptacog alpha (NovoSeven®) when undergoing surgery under normal clinical practice…
- Conditions
- Congenital Bleeding Disorder, Haemophilia A With Inhibitors, Haemophilia B With Inhibitors
- Trial name
- NCT01579968
- Testing
- eptacog alfa (activated)
- Where
- Japan
- Volunteers
- 86 people
- Sponsor
- Novo Nordisk A/S
Hematologic Disorder, Bleeding Disorder
ATHN Transcends: A Natural History Study of Non-Neoplastic Hematologic Disorders
In parallel with the growth of ATHN's clinical studies, the number of new therapies for all blood disorders is increasing significantly.
- Conditions
- Hematologic Disorder, Bleeding Disorder, Connective Tissue Disorder, Hemophilia
- Trial name
- NCT04398628
- Where
- 76 sites in United States
- Volunteers
- 3,000 people
- Sponsor
- American Thrombosis and Hemostasis Network
Haemophilia A, Haemophilia B
Post-Marketing Surveillance (All Case Surveillance) on Treatment With Alhemo® in Patients With Haemophilia A or Haemophilia B With Inhibitors
The purpose of the study is to investigate the safety and effectiveness of Alhemo® in all participants under real-world clinical practice in Japan. Participants will get Alhemo® as prescribed by the study doctor.
- Trial name
- NCT06285071
- Testing
- Concizumab
- Where
- 11 sites in Japan
- Volunteers
- 23 people
- Sponsor
- Novo Nordisk A/S
Hemophilia A, Hemophilia B
An Institutional Pilot Study to Investigate Physical Activity Patterns in Boys With Hemophilia
This pilot project was developed to investigate subjective and objective data related to the patterns of physical activity participation among hemophilia patients (FVIII or FIX) between the ages of 5 and 18 years.
- Conditions
- Hemophilia A, Hemophilia B, Factor VIII Deficiency, Factor IX Deficiency
- Trial name
- HEPS (NCT02199717)
- Testing
- Accelerometer
- Where
- Canada
- Volunteers
- 66 people
- Sponsor
- The Hospital for Sick Children