Trial of AAV5-hFIX in Severe or Moderately Severe Hemophilia B
NCT02396342 · Status: COMPLETED · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 10
Last updated 2022-06-27
Summary
This study evaluates how safe gene therapy treatment with AAV5-hFIX is in adult patients with severe or moderately severe hemophilia B and severe bleeding type.
Conditions
- Hemophilia B
Interventions
- GENETIC
-
AAV5-hFIX
AAV5hFIX gene therapy
Sponsors & Collaborators
-
CSL Behring
lead INDUSTRY
Principal Investigators
-
uniQure Clinical Trials · UniQure Biopharma B.V.
Study Design
- Allocation
- NON_RANDOMIZED
- Purpose
- TREATMENT
- Masking
- NONE
- Model
- SEQUENTIAL
Eligibility
- Min Age
- 18 Years
- Sex
- MALE
- Healthy Volunteers
- No
Timeline & Regulatory
- Start
- 2015-06-10
- Primary Completion
- 2021-04-15
- Completion
- 2021-04-15
Countries
- Denmark
- Germany
- Netherlands
Study Locations
More Related Trials
-
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
NCT03861273 ·Status: ACTIVE_NOT_RECRUITING ·Phase: PHASE3
-
An Observational Cohort Study to Characterize the Effectiveness and Safety of HEMGENIX® in Patients With Hemophilia B
NCT06008938 ·Status: RECRUITING
-
Long-term Follow-up Study of Male Adults With Hemophilia B Previously Treated With Etranacogene Dezaparvovec (CSL222)
NCT05962398 ·Status: ENROLLING_BY_INVITATION
-
Ascending Dose Study of Genome Editing by Zinc Finger Nuclease Therapeutic SB-FIX in Subjects With Severe Hemophilia B
NCT02695160 ·Status: TERMINATED ·Phase: PHASE1
-
Safety of a New Type of Treatment Called Gene Transfer for the Treatment of Severe Hemophilia B
NCT00076557 ·Status: TERMINATED ·Phase: PHASE1/PHASE2
-
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)
NCT06003387 ·Status: RECRUITING ·Phase: PHASE3
-
A Study to Evaluate Prospective Efficacy and Safety Data of Current FIX Prophylaxis Replacement Therapy in Adult Hemophilia B Subjects (FIX:C≤2%) or Current FVIII Prophylaxis Replacement Therapy in Adult Hemophilia A Subjects (FVIII:C≤1%)
NCT03587116 ·Status: COMPLETED ·Phase: PHASE3
-
Gene Therapy Study in Severe Hemophilia A Patients With Antibodies Against AAV5
NCT03520712 ·Status: TERMINATED ·Phase: PHASE1/PHASE2
-
Study of Recombinant Factor IX Fc Fusion Protein (rFIXFc) in Participants With Hemophilia B
NCT01027364 ·Status: COMPLETED ·Phase: PHASE3
-
Dose Confirmation Trial of AAV5-hFIXco-Padua
NCT03489291 ·Status: COMPLETED ·Phase: PHASE2
-
Open-Label Single Ascending Dose of Adeno-associated Virus Serotype 8 Factor IX Gene Therapy in Adults With Hemophilia B
NCT01687608 ·Status: ACTIVE_NOT_RECRUITING ·Phase: PHASE1/PHASE2
-
Patient Interview Study to Explore the Impact of Gene Therapy in Hemophilia A & B
NCT06634836 ·Status: WITHDRAWN
-
Phase 3, Open-label, Single-dose Study of CSL222 in Adolescent Male Subjects (≥ 12 to < 18 Years of Age) With Severe or Moderately Severe Hemophilia B
NCT07080905 ·Status: RECRUITING ·Phase: PHASE3
-
LTFU for Gene Transfer Subjects With Hemophilia B
NCT00515710 ·Status: COMPLETED
-
HOPE-B: Trial of AMT-061 in Severe or Moderately Severe Hemophilia B Patients
NCT03569891 ·Status: COMPLETED ·Phase: PHASE3
-
A Phase 2/ 3 Trial to Evaluate the Efficacy and Safety of BAY86-6150
NCT01625390 ·Status: COMPLETED ·Phase: PHASE2/PHASE3
-
Long-Term Safety and Efficacy of rFIXFc in the Prevention and Treatment of Bleeding Episodes in Previously Treated Participants With Hemophilia B
NCT01425723 ·Status: COMPLETED ·Phase: PHASE3
-
A Study of Recombinant AAV2/6 Human Factor 8 Gene Therapy SB-525 (PF-07055480) in Subjects With Severe Hemophilia A
NCT03061201 ·Status: COMPLETED ·Phase: PHASE2
-
A Phase 3 Open-label Interventional Study of Intravenous Recombinant Coagulation Factor VIII Fc-von Willebrand Factor-XTEN Fusion Protein, Efanesoctocog Alfa (BIVV001), in Patients With Severe Hemophilia A
NCT04161495 ·Status: COMPLETED ·Phase: PHASE3
-
A Trial Investigating Safety and Efficacy of Treatment With BAY94-9027 in Severe Hemophilia A
NCT01580293 ·Status: COMPLETED ·Phase: PHASE2/PHASE3
-
Lentiviral FIX Gene Therapy
NCT03961243 ·Status: RECRUITING ·Phase: PHASE1
-
Gene Therapy Study in Severe Haemophilia A Patients (270-201)
NCT02576795 ·Status: COMPLETED ·Phase: PHASE1/PHASE2
-
Long-term Safety and Efficacy of Efanesoctocog Alfa (BIVV001) in Previously Treated Patients With Hemophilia A
NCT04644575 ·Status: ACTIVE_NOT_RECRUITING ·Phase: PHASE3
-
A Safety and Efficacy Extension Study of a Recombinant Fusion Protein Linking Coagulation Factor IX With Albumin (rIX-FP) in Patients With Hemophilia B
NCT02053792 ·Status: COMPLETED ·Phase: PHASE3
-
Trial to Evaluate the Effect of Secondary Prophylaxis With rFVIII Therapy in Severe Hemophilia A Adult and/or Adolescent Subjects Compared to That of Episodic Treatment
NCT00623480 ·Status: COMPLETED ·Phase: PHASE3