Study of AAV5-hFIX in Severe or Moderately Severe Haemophilia B

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

ACTIVE_NOT_RECRUITING

Total Enrollment

9 participants

Study Classification

OBSERVATIONAL

Study Start Date

2021-03-18

Study Completion Date

2026-05-31

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

This is an open-label, extension study enrolling patients who have successfully completed all assessments in Study CT-AMT-060-01 (Years 1-5). Assessment phase will begin at Visit 36 (the first clinical visit in this extension study, approximately 5.5 years after the initial dosing visit Study CT-AMT-060-01) and go to Visit 45 (10-years post-dosing in Study CT-AMT-060-01).

Related Clinical Trials

Explore similar clinical trials based on study characteristics and research focus.

Trial of AAV5-hFIX in Severe or Moderately Severe Hemophilia B

NCT02396342

Hemophilia B

COMPLETED PHASE1/PHASE2

HOPE-B: Trial of AMT-061 in Severe or Moderately Severe Hemophilia B Patients

NCT03569891

Hemophilia B

COMPLETED 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

Hemophilia B

COMPLETED PHASE3

Gene Therapy Study in Severe Hemophilia A Patients With Antibodies Against AAV5

NCT03520712

Hemophilia A Gene Therapy Clotting Disorders +1 more

TERMINATED PHASE1/PHASE2

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

Hemophilia B Hemophilia A

COMPLETED PHASE3

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Hemophilia B

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

CSL220

AAV5 containing a codon-optimized human factor IX gene

AAV5-hFIX

Intervention Type GENETIC

AAV5 containing a codon-optimized human factor IX gene

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

AAV5-hFIX

AAV5 containing a codon-optimized human factor IX gene

Intervention Type GENETIC

Other Intervention Names

Discover alternative or legacy names that may be used to describe the listed interventions across different sources.

CSL220

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

Inclusion Criteria

* Subjects with congenital hemophilia B who completed Study CTAMT-060-01
* Able to provide informed consent following receipt of verbal and written information about the trial.

Minimum Eligible Age

18 Years

Eligible Sex

MALE

Accepts Healthy Volunteers

No