Clinical trials: Moderate Hemophilia 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
Hemophilia A
Phase 3/4 Study of a Recombinant Protein-Free Factor VIII (rAHF-PFM): Comparison of Continuous Infusion Versus Intermittent Bolus Infusion in Hemophilia A Subjects Undergoing Major Orthopedic Surgery
The purpose of this study is to compare the hemostatic efficacy and safety of continuous infusion versus intermittent bolus infusion in the peri- and post-operative setting, employing rAHF-PFM, a recombinant antihemophilic factor…
- Trial name
- NCT00357656
- Testing
- Recombinant Protein-Free Factor VIII (rAHF-PFM)
- Where
- 36 sites in United States, Austria and 12 more
- Volunteers
- 85 people
- Sponsor
- Baxalta now part of Shire
Hemophilia A, Hemophilia B
A Clinical Trial of Study Medicine (Marstacimab) in Pediatric Patients With Hemophilia A or Hemophilia B
The purpose of this clinical trial is to learn about the safety and effects of the study medicine (called marstacimab) for the potential treatment of hemophilia in pediatric patients.
- Trial name
- BASIS KIDS (NCT05611801)
- Testing
- marstacimab
- Where
- 63 sites in United States, Argentina and 21 more
- Volunteers
- 100 people
- Sponsor
- Pfizer
Hemophilia A
Comparison of Different Prophylaxis Regimens for Moderate to Severe Hemophilia A Pediatric Patients
Title: Comparison of different prophylaxis regimens for hemophilia A pediatric patients Principal Investigator: Assistant Professor Darintr Sosothikul, MD Research Question: Does different factor VIII dosage effect outcome of…
- Trial name
- NCT02727647
- Testing
- FVIII
- Where
- Thailand
- Volunteers
- 16 people, healthy volunteers welcome
- Sponsor
- Chulalongkorn University
Hemophilia A
Retrospective Chart Review to Evaluate Safety and Tolerability of ADVATE Among Previously Untreated Patients in China With Moderate to Severe Hemophilia A
The purpose of this post-marketing safety study is to evaluate the safety, immunogenicity, and effectiveness of ADVATE in previously untreated patients (PUPs) in China with moderate to severe hemophilia A.
- Trial name
- NCT02634723
- Testing
- Octocog alfa (recombinant human coagulation factor VIII)
- Where
- 6 sites in China
- Volunteers
- 21 people
- Sponsor
- Baxalta now part of Shire
Congenital Bleeding Disorder, Haemophilia A
Research Study to Look at Side Effects During Regular Injection With Factor VIII Medicine Named Turoctocog Alfa for a 8 Weeks Period
This study will test the well-known medicine turoctocog alfa for any side effects. The purpose is to test turoctocog alfa for any side effects in the Indian population. The participants will get turoctocog alfa.
- Trial name
- guardian 10 (NCT03449342)
- Testing
- turoctocog alfa
- Where
- 9 sites in India
- Volunteers
- 60 people
- Sponsor
- Novo Nordisk A/S
Hemophilia A, Hemophilia B
Study of the Efficacy and Safety PF-06741086 in Adult and Teenage Participants With Severe Hemophilia A or Moderately Severe to Severe Hemophilia B
Treatment with PF-06741086 is anticipated to demonstrate a clinically relevant advantage and/or a major contribution to patient care in comparison to current methods of treatment for hemophilia A or B because it works differently…
- Trial name
- NCT03938792
- Testing
- PF-06741086
- Where
- 62 sites in United States, Bulgaria and 18 more
- Volunteers
- 188 people
- Sponsor
- Pfizer
Moderate and Severe Haemophilia
HEAD-US SCORING SYSTEM: Assessment of the Real-world Impact of Ultrasound
Ultrasound represents a promising technique for the assessment of joint health in persons with haemophilia (PWH) by non-imaging specialists.
- Trial name
- NCT03237273
- Testing
- HEAD-US scoring system
- Volunteers
- 200 people
- Sponsor
- Guy's and St Thomas' NHS Foundation Trust
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
Severe Hereditary Factor VIII Deficiency Disease Without Inhibitor, Moderate Hereditary Factor VIII Deficiency Disease Without Inhibitor
An Observational Study to Evaluate Physical Activity, Bleeding Incidence and Health Related Quality of Life, in Participants With Haemophilia A Without Inhibitors Receiving Standard of Care Treatment
This multicenter, non-interventional, prospective study will collect information about activity status, bleeds, health-related quality of life (HRQoL), health status, and safety in participants with moderate or severe haemophilia…
- Conditions
- Severe Hereditary Factor VIII Deficiency Disease Without Inhibitor, Moderate Hereditary Factor VIII Deficiency Disease Without Inhibitor, Haemophilia A
- Trial name
- POWER (NCT04165135)
- Testing
- Standard of Care for Haemophilia A
- Where
- 17 sites in Italy
- Volunteers
- 103 people
- Sponsor
- Hoffmann-La Roche
Hemophilia
Canadian CHO-KLAT/H-FIT Study - Quality of Life of, and Burden of Caring for, Persons With Hemophilia
The primary goal of this observational study is to determine if: health-related quality of life of boys with moderate/severe hemophilia A who are followed in Canadian pediatric hemophilia treatment centres is significantly…
- Trial name
- NCT06191068
- Where
- Canada
- Volunteers
- 190 people
- Sponsor
- The Hospital for Sick Children
Hemophilia A, Factor VIII
Personalized Prophylactic Treatment With Advate® in Severe or Moderate Haemophilia A Patients
Haemophilia A is an inherited bleeding disorder caused by a deficiency of factor VIII (FVIII). Patients with severe hemophilia A have a FVIII plasma concentration less than1 IU/dL and experience spontaneous and trauma-induced…
- Trial name
- NCT02622646
- Testing
- Recombinant VIII factor
- Where
- Spain
- Volunteers
- 25 people
- Sponsor
- Hospital Universitario La Fe
Hemophilia B
Study of AAV5-hFIX in Severe or Moderately Severe Haemophilia B
This is an open-label, extension study enrolling patients who have successfully completed all assessments in Study CT-AMT-060-01 (Years 1-5).
- Trial name
- NCT05360706
- Testing
- AAV5-hFIX
- Where
- 6 sites in Germany, Netherlands
- Volunteers
- 9 people
- Sponsor
- CSL Behring
Haemophilia A (Moderate or Severe)
Study to Provide Continued Access to Treatment for Patients Completing a Previous Trial With Efanesoctocog Alfa
This is a multinational, prospective, open-label, roll-over study in patients with haemophilia A, ≥6 years of age, who have completed participation in any of the parental studies with efanesoctocog alfa; XTEND-ed study…
- Trial name
- LIBERTY (NCT06716814)
- Testing
- Efanesoctocog alfa
- Where
- 20 sites in Bulgaria, France and 5 more
- Volunteers
- 89 people
- Sponsor
- Swedish Orphan Biovitrum
Hemophilia B, Hemophilia B, Moderately Severe or Severe
BeCoMe-9: A Clinical Study of BE-101 for the Treatment of Adults With Moderately Severe or Severe Hemophilia B
The BeCoMe-9 Study (BE-101-01) is a Phase 1/2, first in human, multi-center, open-label, dose-escalation study to evaluate the safety and clinical activity of a single intravenous (IV) dose of BE-101 in adults with moderately…
- Trial name
- NCT06611436
- Testing
- BE-101
- Where
- 5 sites in United States
- Volunteers
- 5 people
- Sponsor
- Be Biopharma
Hemophilia A
A Gene Therapy Study of SPK-8011QQ in Adults With Severe or Moderately Severe Hemophilia A
This study will assess the safety and tolerability of SPK-8011QQ in adult males with moderately severe to severe hemophilia A.
- Trial name
- NCT07226206
- Testing
- SPK-8011QQ
- Where
- 6 sites in United States
- Volunteers
- 5 people
- Sponsor
- Hoffmann-La Roche
Hemophilia A
Efficacy and Safety Study of a Recombinant and Protein-Free Factor VIII (rAHF-PFM) in Pediatric Patients in Canada With Hemophilia A - A Continuation of Baxter Study 060101
The purpose of this study is to evaluate whether rAHF-PFM is safe and effective in the treatment of children with hemophilia A. The study is open to pediatric patients in Canada who completed Baxter Study 060101.
- Trial name
- NCT00189982
- Testing
- Antihemophilic Factor Manufactured and Formulated without Added Human or Animal Proteins (rAHF-PFM)
- Where
- Canada
- Volunteers
- 4 people
- Sponsor
- Baxalta now part of Shire
Hemophilia
Impact of Moderate Intensity Physical Activities on PK-guided EHL FVIII Concentrates Prophylaxis Severe HA Patients
To compare clinical outcomes before and after using combination between moderate to vigorous-intensity physical activities and pharmacokinetic-guided extended half-life FVIII concentrates prophylaxis in moderate to severe…
- Trial name
- NCT05728528
- Testing
- combination between moderate to vigorous-intensity physical activities and pharmacokinetic-guided extended half-life FVIII concentrates prophylaxis, pharmacokinetic-guided extended half-life FVIII concentrates prophylaxis alone
- Where
- Thailand
- Volunteers
- 13 people
- Sponsor
- Chulalongkorn University
Hemophilia A
Evaluate Efficacy and Safety of ADVATE in the Standard Prophylaxis Treatment of Severe or Moderately Severe Hemophilia A
Based on the successful pioneer work of Dr Nilsson and her colleagues in Sweden that started in the late 1950's, prophylaxis is recommend as the standard of care for boys with severe haemophilia by WHO and World Federation of…
- Trial name
- NCT02282410
- Testing
- ADVATE
- Volunteers
- 15 people
- Sponsor
- The Affiliated Nanjing Drum Tower Hospital of Nanjing University Medical School
Hemophilia B
A Study to Investigate the Safety and Effectiveness of a Coagulation Factor IX Gene Insertion Therapy (REGV131-LNP1265) in Pediatric, Adolescent and Adult Participants With Hemophilia B
Participants in this study have a genetic mutation, specifically in the coagulation (blood clotting) Factor 9 gene that causes severe or moderately severe hemophilia B.
- Trial name
- BEYOND-9 (NCT06379789)
- Testing
- REGV131, LNP1265
- Where
- 52 sites in United States, Australia and 7 more
- Volunteers
- 130 people
- Sponsor
- Regeneron Pharmaceuticals