Clinical trials: Coagulation Protein Disorders
Hemophilia A
China ADVATE PTP Study
The purpose of this study is to assess efficacy, safety and pharmacokinetics of ADVATE in the treatment and prevention of bleeding episodes (BEs)
- Trial name
- NCT02170402
- Testing
- Octocog alfa (recombinant human coagulation factor VIII)
- Where
- 11 sites in China
- Volunteers
- 82 people
- Sponsor
- Baxalta now part of Shire
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
Hemophilia
Phase III Clinical Trial of STSP-0601 for Injection in Hemophilia Patients
This study will assess the efficacy of multiple-dose of STSP-0601 for the treatment of bleeding episodes in hemophilia A or B patients with inhibitor
- Trial name
- NCT06922045
- Testing
- STSP-0601 for Injection
- Where
- 18 sites in China
- Volunteers
- 40 people
- Sponsor
- Jiangsu BioJeTay Biotechnology Co., Ltd.
Von Willebrand Disease, Type 3
A Study to Assess the Efficacy and Safety of Emicizumab in Participants With Type 3 Von Willebrand Disease
This is a Phase III, multicenter, open-label clinical study designed to evaluate the efficacy, safety, pharmacokinetics, and pharmacodynamics of emicizumab prophylaxis in participants aged 1 month and above, who have been…
- Trial name
- WILL-EMI (NCT06998524)
- Testing
- Emicizumab, von Willebrand Factor (VWF) Concentrates, Factor VIII (FVIII) Concentrates
- Where
- 29 sites in United States, Belgium and 12 more
- Volunteers
- 75 people
- Sponsor
- Hoffmann-La Roche
Hemophilia A
An Open Label Study to Determine the Safety and Efficacy of Replacement Factor VIII Protein (Known as rFVIIIFc) in Previously Untreated Males With Severe Hemophilia A
The primary objective of the study was to evaluate the safety of rFVIIIFc (BIIB031) in previously untreated participants (PUPs) with severe hemophilia A.
- Trial name
- NCT02234323
- Testing
- rFVIIIFc
- Where
- 71 sites in United States, Australia and 12 more
- Volunteers
- 108 people
- Sponsor
- Bioverativ, a Sanofi company
Hemophilia B
A Phase 1/2 Study of SHP648, an Adeno-Associated Viral Vector for Gene Transfer in Hemophilia B Subjects
The purpose of this study is to evaluate the safety and dose escalation of SHP648 an adeno-associated viral vector for gene transfer in hemophilia B participants.
- Trial name
- NCT04394286
- Testing
- SHP648
- Where
- 2 sites in Spain, Turkey (Türkiye)
- Volunteers
- 2 people
- Sponsor
- Baxalta now part of Shire
Hemophilia A
A 48-Month Study to Evaluate Long-Term Effectiveness of Elocta on Joint Health
This is a 48-month observational, prospective, multicentre study. The overall aim of the study is to evaluate the long-term effectiveness of Elocta treatment on joint health in patients treated prophylactically with Elocta in a…
- Trial name
- A-MORE (NCT04293523)
- Testing
- ELOCTA
- Where
- 53 sites in Czechia, Estonia and 12 more
- Volunteers
- 427 people
- Sponsor
- Swedish Orphan Biovitrum
Congenital Bleeding Disorder, Haemophilia A
Safety and Pharmacokinetics of NNC 0129-0000-1003 in Subjects With Haemophilia A
This trial is conducted globally. The aim of this clinical trial is to investigate the safety and pharmacokinetics (the determination of the concentration of the administered medication in blood over time) of NNC 0129-0000-1003…
- Trial name
- pathfinder™1 (NCT01205724)
- Testing
- turoctocog alfa pegol
- Where
- 18 sites in United States, Germany and 5 more
- Volunteers
- 27 people
- Sponsor
- Novo Nordisk A/S
Hemophilia A
Efficacy and Safety Study of a Recombinant Protein-Free Manufactured Factor VIII (rAHF-PFM) in Previously Untreated Hemophilia A Patients
The purpose of this study is to evaluate whether Antihemophilic factor, recombinant, manufactured protein-free (rAHF-PFM) is effective and safe in the treatment of hemophilia A patients who have not been treated with factor VIII…
- Trial name
- NCT00157157
- Testing
- Recombinant Antihemophilic Factor Manufactured and Formulated without Added Human or Animal Proteins (rAHF-PFM)
- Where
- 35 sites in United States, Austria and 8 more
- Volunteers
- 66 people
- Sponsor
- Baxalta now part of Shire
Hemophilia
First-in-Human and Proof-of-Mechanism Study of ARC19499 Administered to Hemophilia Patients
The purpose of this study is to examine the safety, tolerability and the way the body handles various single and multiple doses of ARC19499 in patients with hemophilia.
- Trial name
- NCT01191372
- Testing
- ARC19499
- Where
- United Kingdom
- Volunteers
- 17 people
- Sponsor
- Baxalta now part of Shire
Hemophilia B
RIXUBIS Drug Use-Result Survey (Japan)
The purpose of this survey is to understand the following items observed in the actual clinical use of RIXUBIS. 1. Unexpected adverse drug reactions 2. Occurrence of adverse drug reactions in the actual clinical use 3.
- Trial name
- NCT02937831
- Testing
- RIXUBIS
- Where
- 5 sites in Japan
- Volunteers
- 6 people
- Sponsor
- Baxalta now part of Shire
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
Hypofibrinogenemia, Congenital, Afibrinogenemia, Congenital
Human Fibrinogen Concentrate (FGTW) in Pediatric Patients With Congenital Fibrinogen Deficiency
The aim of the study is to evaluate clinical pharmacology, efficacy and safety of FGTW in pediatric patients with congenital fibrinogen deficiency.
- Trial name
- NCT02094430
- Testing
- biological: human fibrinogen concentrate
- Where
- 4 sites in France, Lebanon and 2 more
- Volunteers
- 16 people
- Sponsor
- Laboratoire français de Fractionnement et de Biotechnologies
Congenital Bleeding Disorder, Haemophilia A
A Multinational, Open-Label, Non-Controlled Trial on Safety, Efficacy and Pharmacokinetics of NNC 0129-0000-1003 in Previously Treated Paediatric Patients With Severe Haemophilia A
This trial is conducted globally. The aim of the trial is to investigate safety, efficacy and pharmacokinetics (the exposure of the trial drug in the body) of NNC 0129-0000-1003 (N8-GP) in children with severe haemophilia A who…
- Trial name
- pathfinder™5 (NCT01731600)
- Testing
- turoctocog alfa pegol
- Where
- 51 sites in United States, Brazil and 14 more
- Volunteers
- 68 people
- Sponsor
- Novo Nordisk A/S
Hemophilia A or B
Safety of KN057 Prophylaxis in Patients With Haemophilia A or B
The purposes of this open-label, multicenter III clinical trial are to evaluate the safety and efficacy of long-term preventive treatment with KN057 in Haemophilia A or B patients with or without inhibitors, and to assess the…
- Trial name
- NCT07545395
- Testing
- KN057
- Where
- China
- Volunteers
- 70 people
- Sponsor
- Suzhou Alphamab Co., Ltd.
Congenital Afibrinogenemia
Pharmacokinetics, Efficacy, and Safety of Human Plasma-Derived Fibrinogen (FIB Grifols) in Participants With Congenital Afibrinogenemia
The main objective of this study was to evaluate the pharmacokinetics (PK), efficacy, and safety of human plasma-derived fibrinogen concentrate FIB Grifols after a single-dose 70 milligrams/kilogram (mg/kg) body weight…
- Trial name
- IG0902 (NCT02281500)
- Testing
- Human Plasma-Derived Fibrinogen Concentrate
- Where
- 6 sites in United States, India and 2 more
- Volunteers
- 24 people
- Sponsor
- Grifols Therapeutics LLC
Hemophilia B
Evaluation of the Safety and Efficacy of Hemophilia B Gene Therapy Drug
This is a multi-center, Phase 1/2/3, single-arm, open-label, single-dose treatment clinical study to evaluate the safety, tolerability and efficacy of BBM-H901 injection in Hemophilia B subjects with ≤2 International unit per…
- Trial name
- NCT05203679
- Testing
- Single dose intravenous injection of BBM-H901
- Where
- 9 sites in China
- Volunteers
- 32 people
- Sponsor
- Shanghai Xinzhi BioMed Co., Ltd.
Hemophilic Arthropathy
Rofecoxib Efficacy and Safety Evaluation Trial in Hemophilic Arthropathy
This is a multicenter, randomized, double-blind study to evaluate the efficacy and safety of TRM-201 (rofecoxib) versus Placebo in the treatment of patients with hemophilic arthropathy (HA) over a 12-week period (Part I) that is…
- Trial name
- RESET-HA (NCT04684511)
- Testing
- TRM-201 (Rofecoxib)
- Where
- 46 sites in United States, Australia and 6 more
- Volunteers
- 2 people
- Sponsor
- Tremeau Pharmceuticals, Inc.
Hemophilia B
Long-term Follow-up Study of Male Adults With Hemophilia B Previously Treated With Etranacogene Dezaparvovec (CSL222)
The primary purpose of this study is to assess the long-term safety and efficacy in male adults with hemophilia B who were treated with CSL222 (CSL222) in parent studies CSL222_2001 (NCT03489291) or CSL222_3001 (NCT03569891).
- Trial name
- IX-TEND 3003 (NCT05962398)
- Testing
- AAV5-hFIXco-Padua
- Where
- 28 sites in United States, Belgium and 6 more
- Volunteers
- 56 people
- Sponsor
- CSL Behring
Hemophilia A
A Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of Prophylactic Emicizumab Versus no Prophylaxis in Hemophilia A Participants With Inhibitors
This multicenter, open-label study will evaluate the safety, efficacy and pharmacokinetics of prophylactic emicizumab treatment in participants previously treated with episodic or prophylactic bypassing agents.
- Trial name
- HAVEN 1 (NCT02622321)
- Testing
- Emicizumab, rFVIIa, aPCC
- Where
- 44 sites in United States, Australia and 12 more
- Volunteers
- 113 people
- Sponsor
- Hoffmann-La Roche