Hemophilia B Clinical Trial
Official title:
Six-month lead-in Study to Collect Prospective Efficacy and Safety Data of Current Replacement Therapy in Adult Hemophilia B Patients -- An Observational Survey Analysis Study
This is a prospective, open, multicenter, observational lead-in study，to collect prospective efficacy and safety data of current replacement therapy in adult hemophilia B patients.
Hemophilia B is a genetic bleeding disorder caused by pathogenic variants (eg, mutations, deletion) in the FIX gene. HB patients have frequent and potentially life-threatening bleeding and often develop progressive physical disability and pain from chronic haemarthropathy. Current replacement therapy needs regular treatment in the life-long time, bringing heavy economic and social burdens.VGB-R04 is a novel AAV vector carrying a high specific activity factor IX variant. This is a prospective, open, multicenter, observational lead-in study. To evaluate the efficacy and safety of current treatment regiments in patients with moderate to severe HB (FIX:C activity ≤2%). The efficacy and safety data collected in this study may be used as baseline data for subsequent related clinical trials of VGB-R04. All subjects in this study will provide informed consent and then undergo screening assessments up to 4 weeks. All subjects will undergo 26 weeks of efficacy and safety observation .Eligible subjects will be invited to a key clinical study（phase 2 study）of VGB-R04,and then encouraged to enroll in an Long-term follow-up study to evaluate the long-term safety of VGB-R04 for a total of five years. ;
| Status | Clinical Trial | Phase | |
|---|---|---|---|
| Completed |
NCT04072237 -
Study of Coagulation Faction VIIa Variant Marzeptacog Alfa (Activated) in Adult Subjects With Hemophilia
|
Phase 1 | |
| Completed |
NCT02199717 -
An Institutional Pilot Study to Investigate Physical Activity Patterns in Boys With Hemophilia
|
N/A | |
| Completed |
NCT01662531 -
A Safety, Efficacy and Pharmacokinetics Study of a Recombinant Fusion Protein Linking Coagulation Factor IX With Albumin (rIX-FP) in Children With Hemophilia B
|
Phase 3 | |
| Completed |
NCT01335061 -
Study To Compare On-Demand Treatment To A Prophylaxis Regimen Of BeneFIX In Subjects With Moderately Severe to Severe Hemophilia B
|
Phase 3 | |
| Completed |
NCT01217255 -
Comparing the Burden of Illness of Hemophilia in the Developing and the Developed World
|
||
| Completed |
NCT00037557 -
Study Evaluating rFIX; BeneFIX in Severe Hemophilia B
|
Phase 3 | |
| Completed |
NCT02554773 -
An Open-label Extension Study of an Investigational Drug, Fitusiran, in Patients With Moderate or Severe Hemophilia A or B
|
Phase 1/Phase 2 | |
| Terminated |
NCT02807753 -
The Hemophilia Ultrasound Project
|
||
| Active, not recruiting |
NCT03901755 -
An International Study to Evaluate the Real-world Effectiveness and Usage of Alprolix in Patients With Haemophilia B
|
||
| Not yet recruiting |
NCT05980377 -
Patterns of Hemophilia Care in Assiut Children Patients
|
||
| Recruiting |
NCT05687474 -
Baby Detect : Genomic Newborn Screening
|
||
| Terminated |
NCT03248141 -
Understanding Hemophilia A and B Drug Dosage Administration Patterns
|
||
| Terminated |
NCT01460147 -
Osteoporosis and MRI Study in Hemophilia
|
N/A | |
| Completed |
NCT03818529 -
ATHN 8: Previously Untreated Patients (PUPs) Matter Study
|
||
| Completed |
NCT02571569 -
A Single Escalating Dose and Multiple Dose Study of BAY 1093884 in Subjects With Severe Hemophilia Types A or B, With or Without Inhibitors
|
Phase 1 | |
| Terminated |
NCT01620801 -
Hemophilia B Gene Therapy With AAV8 Vector
|
Phase 1 | |
| Completed |
NCT01233440 -
Safety and Pharmacokinetic Study of a Recombinant Coagulation Factor IX Albumin Fusion Protein in Subjects With Hemophilia B
|
Phase 1 | |
| Active, not recruiting |
NCT04135300 -
Gene Therapy for Chinese Hemophilia B
|
N/A | |
| Enrolling by invitation |
NCT03655223 -
Early Check: Expanded Screening in Newborns
|
||
| Terminated |
NCT00947193 -
Study of Ataluren (PTC124) in Hemophilia A and B
|
Phase 2 |