Clinical trials: Factor VIII Deficiency
Healthy Volunteers, Haemophilia A With or Without Inhibitors
A Research Study Investigating Mim8 in People With Haemophilia A
This study is investigating how Mim8 works in people with haemophilia A, who either have inhibitors or do not have inhibitors. Mim8 is a new medication that will be used for prevention of bleeding episodes.
- Trial name
- FRONTIER1 (NCT04204408)
- Testing
- NNC0365-3769 (Mim8)
- Where
- 40 sites in United States, Austria and 10 more
- Volunteers
- 275 people, healthy volunteers welcome
- Sponsor
- Novo Nordisk A/S
Severe Hemophilia A
Nuwiq for Perioperative Management Of Patients With Haemophilia A on Emicizumab Regular Prophylaxis Study
Recombinant factor VIII for the prevention of bleeding in patients with severe haemophilia A undergoing major surgery while receiving emicizumab prophylaxis
- Trial name
- NuPOWER (NCT05935358)
- Testing
- Nuwiq
- Where
- 18 sites in United States, Croatia and 9 more
- Volunteers
- 28 people
- Sponsor
- Octapharma
Hemophilia A
EFFEKT - Efficacy and Safety of Long-term Treatment With KOGENATE Bayer/FS
The aim of this international prospective, non-interventional post-marketing surveillance study is to obtain data on treatment procedures, long-term safety and efficacy and patient acceptance of KOGENATE Bayer in treatment of…
- Trial name
- NCT00874926
- Testing
- Recombinant Factor VIII (Kogenate FS, BAY14-2222)
- Where
- 17 sites in Bahrain, Bosnia and Herzegovina and 15 more
- Volunteers
- 405 people
- Sponsor
- Bayer
Hemophilia A
Study of Pharmacokinetics, Efficacy, and Safety of a Recombinant and Protein-Free Factor VIII (rAHF-PFM) in Pediatric Patients With Hemophilia A
The purpose of this study is to evaluate whether rAHF-PFM is effective and safe in the treatment of children with hemophilia A. The study consists of 2 parts.
- Trial name
- NCT00157040
- Testing
- Antihemophilic factor, recombinant, manufactured protein-free
- Where
- 12 sites in United States, Canada and 1 more
- Volunteers
- 50 people
- Sponsor
- Baxalta now part of Shire
Haemophilia A
BAY81-8973 Pediatric Safety and Efficacy Trial
The primary objective was to evaluate the safety and efficacy of the treatment with BAY81-8973 for prophylaxis and treatment of breakthrough bleeds in children with severe hemophilia A.
- Trial name
- NCT01311648
- Testing
- Recombinant Factor VIII (Kovaltry, BAY81-8973)
- Where
- 43 sites in United States, Argentina and 15 more
- Volunteers
- 94 people
- Sponsor
- Bayer
Congenital Bleeding Disorder, Haemophilia A With Inhibitors
Safety and Efficacy of 3 Different Doses of Long Acting Factor VII in Haemophilia A or B Patients With Inhibitors
This trial is conducted in Asia, Europe, Japan and North America. The aim of this clinical trial is to investigate the safety and the efficacy of a prophylactic treatment option with long acting coagulation factor VII (LA-rFVIIa)…
- Conditions
- Congenital Bleeding Disorder, Haemophilia A With Inhibitors, Haemophilia B With Inhibitors
- Trial name
- NCT00951405
- Testing
- activated recombinant human factor VII, long acting
- Where
- 25 sites in United States, Brazil and 9 more
- Volunteers
- 23 people
- Sponsor
- Novo Nordisk A/S
Haemophilia A
An Open Study to Investigate the Safety and Efficacy of Optivate® in Haemophilia A Patients Undergoing Surgery.
The main objective of the study is to investigate the safety and efficacy of Optivate®, administered in appropriate dosage to present bleeding and achieve haemostasis in patients with Haemophilia A undergoing surgery.
- Trial name
- NCT02250482
- Testing
- Optivate® (Human Coagulation Factor VIII)
- Where
- 8 sites in Poland, United Kingdom
- Sponsor
- Bio Products Laboratory
Hemophilia
LR769 in Congenital Hemophilia Patients With Inhibitors Undergoing Elective Surgery or Invasive Procedures
The purpose of this study, PerSept 3, is to evaluate LR769 for the prevention of excessive bleeding and achievement of hemostasis in congenital hemophilia A or B patients who have inhibitors to Factor VIII or Factor IX , are aged…
- Trial name
- PerSept3 (NCT02548143)
- Testing
- Coagulation Factor VIIa (Recombinant)
- Where
- 9 sites in United States, Mexico and 4 more
- Volunteers
- 12 people
- Sponsor
- Laboratoire français de Fractionnement et de Biotechnologies
Mild Hereditary Factor VIII Deficiency Disease Without Inhibitor, Moderate Hereditary Factor VIII Deficiency Disease Without Inhibitor
A Study to Evaluate the Safety, Efficacy, Pharmacokinetics and Pharmacodynamics of Emicizumab in Participants With Mild or Moderate Hemophilia A Without FVIII Inhibitors
This is a multicenter, open-label, single-arm study designed to evaluate the safety, efficacy, pharmacokinetics, and pharmacodynamics of emicizumab in participants with mild or moderate hemophilia A without inhibitors against…
- Conditions
- Mild Hereditary Factor VIII Deficiency Disease Without Inhibitor, Moderate Hereditary Factor VIII Deficiency Disease Without Inhibitor, Hemophilia A
- Trial name
- HAVEN 6 (NCT04158648)
- Testing
- Emicizumab
- Where
- 22 sites in United States, Belgium and 8 more
- Volunteers
- 73 people
- Sponsor
- Hoffmann-La Roche
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
Haemophilia A
Efficacy and Safety Evaluation Study of SCT800 in Previously Untreated Hemophilia A Patients.
This is an Open Label, Uncontrolled Study to Evaluate the the Safety and Efficacy of SCT800 for Prophylaxis Treatment in Severe Previously Untreated Hemophilia Patients.
- Trial name
- NCT07437404
- Testing
- SCT800 prophylaxis and treatment for bleeding events
- Where
- China
- Volunteers
- 36 people
- Sponsor
- Sinocelltech Ltd.
Congenital Bleeding Disorder, Haemophilia A
Safety, Tolerability, and Pharmacokinetics Study of Turoctocog Alfa Pegol Injected Under the Skin in Patients With Haemophilia A
The trial is conducted in Asia, Europe and North America. The aim of the study is to evaluate the safety of administration under the skin of turoctocog alfa pegol (SC N8-GP) in patients with severe haemophilia A.
- Trial name
- alleviate 1 (NCT02994407)
- Testing
- turoctocog alfa pegol
- Where
- 19 sites in United States, Austria and 4 more
- Volunteers
- 50 people
- Sponsor
- Novo Nordisk A/S
Hemophilia A
Study of Modified Recombinant Factor VIII (OBI-1) in Subjects With Congenital Hemophilia A
This study is to test whether the study drug (OBI-1) is safe and effective for the treatment of serious bleeding episodes in people with congenital hemophilia A.
- Trial name
- NCT01434511
- Testing
- OBI-1
- Where
- 3 sites in United States, South Africa and 1 more
- Volunteers
- 1 people
- Sponsor
- Baxalta now part of Shire
Hemophilia A With Inhibitor
A Study to Evaluate the Safest Dose Range for FEIBA in Hemophilia A Patients With Inhibitors on Emicizumab
Hemophilia A is a severe, life-long, genetic bleeding disorder characterized by a deficiency of factor VIII (FVIII), a crucial cofactor of the coagulation system.
- Trial name
- NCT04205175
- Testing
- Feiba
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- Children's Hospital Los Angeles
Factor VIII Deficiency
Efanesoctocog Alfa Prophylaxis in Patients With Hemophilia A With Synovial Hypertrophy
The objective of the study is to assess the improvement of synovial hypertrophy during the 12 months of efanesoctocog alfa prophylaxis once per week (QW) in joints with existing evidence of synovial hypertrophy in participants…
- Trial name
- SYNOVIIIUS (NCT06941870)
- Testing
- Efanesoctocog alfa
- Where
- 14 sites in United States, Canada and 2 more
- Volunteers
- 35 people
- Sponsor
- Sanofi
Congenital Bleeding Disorder, Haemophilia A
Impact of Pain on Functional Impairment and Quality of Life in Adults With Hemophilia
This study is conducted in the United States of America (USA). The aim of the study is to assess the impact of pain on functional impairment and quality of life in adult persons with hemophilia (PWH) with and without inhibitors…
- Conditions
- Congenital Bleeding Disorder, Haemophilia A, Haemophilia A With Inhibitors, Haemophilia B
- Trial name
- NCT01988532
- Testing
- No treatment given
- Where
- United States
- Volunteers
- 381 people
- Sponsor
- Novo Nordisk A/S
Congenital Bleeding Disorder, Haemophilia A
Study Investigating Novoeight®/NovoEight® (Turoctocog Alfa) in Mexican Haemophilia A Patients
The trial is conducted in North America. The aim of the trial is to assess the safety of turoctocog alfa under conditions of routine clinical care in patients with haemophilia A in Mexico
- Trial name
- NCT03179748
- Testing
- turoctocog alfa
- Where
- Mexico
- Volunteers
- 10 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
Hemophilia A With Inhibitors, Hemophilia B With Inhibitors
A Phase III Study on the Safety, Pharmacokinetics and Efficacy of Coagulation Factor VIIa
The purpose of the study is to assess the safety, efficacy and pharmacokinetics of 2 separate dose regimens (75µg/kg and 225 µg/kg) of Coagulation Factor VIIa (Recombinant) for the treatment of bleeding episodes in hemophilia A…
- Trial name
- PERSEPT2 (NCT02448680)
- Testing
- Coagulation FVIIa (Recombinant)
- Where
- 9 sites in United States, Bulgaria and 4 more
- Volunteers
- 25 people
- Sponsor
- Laboratoire français de Fractionnement et de Biotechnologies
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