Clinical trials: Coagulation Protein Disorders
Hemophilia A, Congenital
Alphanate in Immune Tolerance Induction Therapy
This is a multicenter, multinational, prospective, single-arm, nonrandomized, open-label study, planned in of approximately 25 male participants with congenital hemophilia A who will receive their first (primary) immune tolerance…
- Trial name
- NCT03095287
- Testing
- Alphanate
- Where
- 20 sites in United States, Canada and 4 more
- Volunteers
- 2 people
- Sponsor
- Grifols Therapeutics LLC
Severe Hemophilia A
To Evaluate the Safety and Efficacy of GS1191-0445 Injection in the Treatment of Severe Hemophilia A
This study is a single-arm, open-label study evaluating the safety and efficacy of GS1191-0445 injection as a single dose in Chinese subjects with severe hemophilia A.
- Trial name
- NCT07548411
- Testing
- GS1191-0445 injection
- Where
- 2 sites in China
- Volunteers
- 7 people
- Sponsor
- Gritgen Therapeutics Co., Ltd.
Congenital Bleeding Disorder, Haemophilia A
Safety and Efficacy of Turoctocog Alfa During Long-Term Treatment of Severe and Moderately Severe Haemophilia A
This study is conducted in Europe, and North and South America. The aim of this study is to provide additional documentation of the immunogenicity, and obtain additional clinical data, of turoctocog alfa in the setting of normal…
- Trial name
- guardian™ 5 (NCT02035384)
- Testing
- turoctocog alfa
- Where
- 41 sites in United States, Austria and 13 more
- Volunteers
- 69 people
- Sponsor
- Novo Nordisk A/S
Hemophilia A
BAX 855 Continuation
To continue the evaluation of the safety and efficacy of BAX 855 for prophylaxis and treatment of bleeding episodes in adult and pediatric previously treated patients (PTPs) aged ≤ 75 years of age with severe hemophilia A.
- Trial name
- NCT01945593
- Testing
- BAX855
- Where
- 89 sites in United States, Australia and 21 more
- Volunteers
- 218 people
- Sponsor
- Baxalta now part of Shire
Hemophilia A, Hemophilia B
Study of Coagulation Faction VIIa Variant Marzeptacog Alfa (Activated) in Adult Subjects With Hemophilia
This multi-center, open label Phase 1 study will evaluate the pharmacokinetics, pharmacodynamics, and safety of a single IV dose of MarzAA followed by ascending single SC doses of MarzAA in adult subjects with moderate or severe…
- Conditions
- Hemophilia A, Hemophilia B, Hemophilia A With Inhibitor, Hemophilia B With Inhibitor
- Trial name
- NCT04072237
- Testing
- MarzAA (marzeptacog alfa [activated])
- Where
- 5 sites in Bulgaria, Russia
- Volunteers
- 11 people
- Sponsor
- Catalyst Biosciences
Hemophilia B
Efficacy and Safety of CSL222 (Etranacogene Dezaparvovec) Gene Therapy in Adults With Hemophilia B With Pretreatment Adeno-associated Virus Serotype 5 (AAV5) Neutralizing Antibodies (Nabs)
The purpose of this study is to assess the risk of bleeding due to failure of expected pharmacological action of CSL222 in adults with severe or moderately severe hemophilia B with detectable pretreatment AAV5 Nabs.
- Trial name
- NCT06003387
- Testing
- CSL222 (AAV5-hFIXco-Padua)
- Where
- 32 sites in United States, Australia and 13 more
- Volunteers
- 35 people
- Sponsor
- CSL Behring
Hemophilia A
Efficacy and Safety Study of SCT800 for On-demand Treatment With in Patients With Hemophilia A
In this trial safety and efficacy of SCT800 (B-domain deleted recombinant factor VIII) is being evaluated in 50 subjects, 12 to 65 years of age, with moderate to severe Hemophilia A.
- Trial name
- NCT02921945
- Testing
- SCT800
- Where
- China
- Volunteers
- 50 people
- Sponsor
- Sinocelltech Ltd.
Hemophilia A
Comparative Pharmacokinetic Study of Two Different Strengths of BAY14-2222
This is a single dose, open-label, randomized, crossover study in subjects with severe Hemophilia A to compare the bioavailability of 2 different strengths of Kogenate FS (BAY 14-2222).
- Trial name
- NCT01653639
- Testing
- Recombinant Factor VIII (Kogenate FS, BAY14-2222)
- Where
- Bulgaria
- Volunteers
- 18 people
- Sponsor
- Bayer
Hemophilia A
Study of a Gene Therapy Treatment for Hemophilia A
The purpose of this study is to evaluate the efficacy of SPK-8011 in preventing bleed episodes compared with FVIII prophylaxis in participants with hemophilia A without FVIII inhibitors on routine FVIII prophylaxis.
- Trial name
- KEYSTONE 1 (NCT06297486)
- Testing
- SPK-8011
- Where
- 27 sites in United States
- Sponsor
- Spark Therapeutics, Inc.
Acquired Factor X Deficiency
A Study Investigating Coagadex in the Treatment AFXD Associated With AL Amyloidosis
The purpose of this study is to evaluate the efficacy and safety of Coagadex in treating active bleeds and to manage peri-operative bleeding in participants with acquired factor X deficiency associated with AL amyloidosis.
- Trial name
- NCT06963216
- Testing
- Coagadex(R)
- Sponsor
- Kedrion S.p.A.
Hemophilia A
Colorado Adult Joint Assessment Scale (CAJAS) Validation
To obtain evidence of content validity and reliability of the Colorado Adult Joint Assessment Scale (CAJAS), a clinician-reported outcome (ClinRO) measure, as adapted for use in a population of adults with moderate to severe…
- Trial name
- CAJAS (NCT02284789)
- Testing
- Colorado Adult Joint Assessment Evaluation
- Where
- 4 sites in United States
- Volunteers
- 30 people
- Sponsor
- Bayer
Von Willebrand Disease (VWD)
A Study on Estimated Risks of Certain Adverse Events in Adults With Von Willebrand Disease (VWD) Treated With Veyvondi
The main aim of this study is to estimate the risks of certain adverse events in adults with Von Willebrand Disease treated with VEYVONDI. No study medicines will be provided to participants in this study.
- Trial name
- NCT05265078
- Where
- Germany
- Volunteers
- 102 people
- Sponsor
- Takeda
Hemophilia
Retrospective Experience Assessing the Real-World Utilization and Effectiveness of SEVENFACT®
Retrospective Experience Assessing the Real-World Utilization and Effectiveness of SEVENFACT® [coagulation factor VIIa (recombinant)-jncw] in the USA.
- Trial name
- NCT05904210
- Testing
- SEVENFACT®
- Where
- 8 sites in United States
- Volunteers
- 17 people
- Sponsor
- Laboratoire français de Fractionnement et de Biotechnologies
Hemophilia A or B With Inhibitors
FEIBA Reconstitution Volume Reduction and Faster Infusion Study (FEIBA STAR)
The purpose of this study is to: 1. To evaluate the tolerability and safety of infusing reduced volume Factor Eight Inhibitor Bypassing Activity (FEIBA) at the standard infusion rate of 2 U/kg/min 2.
- Trial name
- NCT02764489
- Testing
- FEIBA
- Where
- 3 sites in Croatia, North Macedonia and 1 more
- Volunteers
- 45 people
- Sponsor
- Baxalta now part of Shire
Congenital Bleeding Disorder, Haemophilia A
Evaluating the Pharmacokinetics of NovoEight® (Turoctocog Alfa) in Relation to BMI in Subjects With Haemophilia A
This trial is conducted globally. The aim of this trial is evaluating the pharmacokinetics (the exposure of the trial drug in the body) of NovoEight® (turoctocog alfa) in relation to BMI (body mass index) in subjects with…
- Trial name
- guardian ™9 (NCT02941354)
- Testing
- turoctocog alfa
- Where
- 15 sites in United States, Austria and 5 more
- Volunteers
- 35 people
- Sponsor
- Novo Nordisk A/S
Hemophilia A, Hemophilia B
An Open-label Extension Study of an Investigational Drug, Fitusiran, in Patients With Moderate or Severe Hemophilia A or B
Primary Objective: To evaluate the long-term safety and tolerability of fitusiran in male patients with moderate or severe hemophilia A or B Secondary Objectives: To investigate the long-term efficacy of fitusiran To characterize…
- Trial name
- NCT02554773
- Testing
- Fitusiran (SAR439774)
- Where
- 12 sites in United States, Bulgaria and 3 more
- Volunteers
- 34 people
- Sponsor
- Genzyme, a Sanofi Company
Hemophilia A
Prospective, Non-interventional Study to Evaluate the Effectiveness of Elocta Compared to Conventional Factor Products
The purpose of the study is to evaluate the effectiveness of Elocta compared to conventional factor products in the prophylactic treatment of patients with haemophilia A over a 24-month prospective period.
- Trial name
- NCT02976753
- Testing
- efmoroctocog alfa, Factor VIII
- Where
- 36 sites in Austria, Belgium and 7 more
- Volunteers
- 361 people
- Sponsor
- Swedish Orphan Biovitrum
Hemophilia A
ADVATE/ ADYNOVI Hemophilia A Outcome Database (AHEAD)
The purpose of the study is to document the natural history of hemophilia A disease and long-term outcomes in terms of effectiveness, safety and quality of life in participants receiving Antihemophilic Factor (Recombinant) …
- Trial name
- AHEAD (NCT02078427)
- Testing
- ADVATE, ADYNOVI
- Where
- 110 sites in Australia, Austria and 20 more
- Volunteers
- 951 people
- Sponsor
- Baxalta now part of Shire
Hemophilia A, Healthy Adult Participants
A First-in Human Study of ARD001 in Healthy Volunteers and People With Hemophilia A
This study is evaluating ARD001, an investigational medicine being developed for the treatment of hemophilia A. In Phase 1, healthy adult participants will receive a single dose of ARD001 or placebo to assess its safety and…
- Trial name
- NCT07787091
- Testing
- ARD001
- Where
- Singapore
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- Arditus Pte Ltd
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