Hemophilia B Clinical Trial
Official title:
Screening/Observational Protocol to Determine Patient Eligibility for Inclusion in AAV Gene Therapy Clinical Trials (ECLIPSE)
Freeline is developing adeno-associated virus (AAV) vector based gene therapies for a number of diseases and is actively advancing a programme in Haemophilia B (HB). This study aims to collect prospective data to characterise bleeding events and Factor IX (FIX) concentrate consumption in HB patients that can be used as baseline for participants who elect to participate in a subsequent Freeline gene therapy study. The study will also screen participants for antibodies to a novel AAV vector to assess their suitability for inclusion in a Freeline gene therapy treatment study.
This is a prospective study to collect baseline disease characteristics in patients with HB that are required to establish eligibility for treatment with a novel AAV gene therapy in a subsequent Freeline gene therapy treatment study. Participants providing consent will attend an enrolment visit to complete eligibility evaluations, collect health and demographic information and receive instruction for completing the study diary. Participants will then complete a diary recording bleeding episodes and FIX usage until they enrol into a gene therapy treatment study. A blood sample will be drawn at a convenient timepoint during the study to assess the participant's AAV neutralising antibody (NAb) status. No treatment intervention will occur as part of this study. ;
| 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 |