Clinical trials: Severe Haemophilia B
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
Haemophilia A (Moderate or Severe), Hemophilia, Classic
A Study to Investigate the Course of Synovial Hypertrophy in Patients With Haemophilia A on Efanesoctocog Alfa Prophylaxis
The rationale for conducting this open-label phase 4 study is to assess whether once-weekly prophylaxis with efanesoctocog alfa (50 IU/kg) improves the disease course of existing synovial hypertrophy and prevents the risk of…
- Conditions
- Haemophilia A (Moderate or Severe), Hemophilia, Classic, Haemophilia, Hemophilia
- Trial name
- NCT06752850
- Testing
- Efanesoctocog alfa
- Where
- 10 sites in Italy, Norway and 2 more
- Volunteers
- 37 people
- Sponsor
- Swedish Orphan Biovitrum
Severe Haemophilia B
Dosage and PD Study of Eftrenonacog-alfa
The purpose of this study is to evaluate the performance of different methods for measuring factor IX activity levels in haemophilia B patients treated with eftrenonacog-alfa and assess its pharmacodynamics (PD) in a real-life…
- Trial name
- BIOPAL (NCT04590950)
- Testing
- Non interventional study
- Where
- France
- Volunteers
- 15 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Severe Haemophilia A
Gene Therapy Study in Severe Haemophilia A Patients (270-201)
This study is being conducted by BioMarin Pharmaceutical Inc. as an open label, dose escalation study in order to determine the safety and efficacy of valoctocogene roxaparvovec (an Adenovirus-Associated Virus based gene therapy…
- Trial name
- NCT02576795
- Testing
- valoctocogene roxaparvovec
- Where
- 5 sites in United Kingdom
- Volunteers
- 15 people
- Sponsor
- BioMarin Pharmaceutical
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
Severe Haemophilia
Quality of Life and Adjustment Among Siblings of Children and Adolescents With Severe Hemophilia
Severe hemophilia is a rare and chronic disease characterized by spontaneous bleedings from early childhood, which may lead to various complications especially in joints.
- Trial name
- FRATHEMO (NCT03299699)
- Where
- France
- Volunteers
- 30 people
- Sponsor
- Assistance Publique Hopitaux De Marseille
Severe Haemophilia
Occupational Integration of Adults With Severe Haemophilia in France: a Study Based on the FranceCoag Cohort
Severe haemophilia and subsequent arthropathy result in joint pain, disability and impaired health-related quality of life. Improvements in haemophilia care over the last decades might lead to expectation of a near-normal quality…
- Trial name
- INTHEMO (NCT03301376)
- Where
- France
- Volunteers
- 590 people
- Sponsor
- Assistance Publique Hopitaux De Marseille
Severe Haemophilia A
Assess the Efficacy and Safety of Personalized Prophylaxis Human-cl rhFVIII in Patients With Severe Haemophilia A
The rationale of this study is to further fine-tune and individualize prophylactic treatment of patients with severe Haemophilia A with the goal of keeping the trough FVIII level above 1% between doses.
- Trial name
- NCT02256917
- Testing
- Human cl rhFVIII
- Where
- 30 sites in United States, Canada and 7 more
- Volunteers
- 58 people
- Sponsor
- Octapharma
Severe Haemophilia A
Efficacy and Cost Effectiveness of Pharmacokinetic Dosing in Haemophilia A
Patients with severe Haemophilia A need prophylactic factor VIII to reduce their risk of joint and soft tissue bleeds and to prevent or reduce joint damage.
- Trial name
- NCT02697370
- Testing
- Pharmacokinetic based dosage change
- Where
- United Kingdom
- Volunteers
- 20 people
- Sponsor
- Hampshire Hospitals NHS Foundation Trust
Severe Haemophilia A
Assess the Safety and Efficacy of Individually Tailored Prophylaxis With Human-cl rhFVIII in Patients With Severe Haemophilia A
To compare the number of breakthrough bleeds under tailored prophylaxis with Human cell line recombinant factor FVIII (Human-cl rhFVIII) with the historical bleeding rate from patients who received Human-cl rhFVIII as on demand…
- Trial name
- NCT01863758
- Testing
- Human-cl rhFVIII
- Where
- 20 sites in Austria, Bulgaria and 6 more
- Volunteers
- 66 people
- Sponsor
- Octapharma
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
Severe Haemophilia A
Subclinical Joint Bleeding in Irish Adults With Severe Haemophilia A on Personalised Prophylaxis Regimens
This trial is designed to assess if there is evidence of subclinical joint bleeding on MRI/X-Ray in adults with severe Haemophilia A while on standard and/or pharmacokinetically tailored prophylaxis regimens.
- Trial name
- PERSONAL (NCT02314325)
- Testing
- ADVATE [Antihemophilic Factor (Recombinant)]
- Where
- Ireland
- Volunteers
- 20 people
- Sponsor
- St. James's Hospital, Ireland
Severe Haemophilia A With Inhibitors
Case-control Pilot Study of the Immune Modulating Effect of FEIBA on Patients With Haemophilia A and Inhibitors
This study aims to evaluate the immunomodulatory effect of FEIBA® in patients with severe haemophilia A and inhibitors.
- Trial name
- NCT02018393
- Where
- Spain
- Volunteers
- 20 people
- Sponsor
- Hospital Universitario La Paz