AAV Gene Therapy Screening/Observational Protocol (ECLIPSE) (ECLIPSE)
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT04272554|
Recruitment Status : Recruiting
First Posted : February 17, 2020
Last Update Posted : February 5, 2021
|Condition or disease|
|Hemophilia B Blood Coagulation Disorders, Inherited Blood Coagulation Disorder Haematologic Disease|
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.
|Study Type :||Observational [Patient Registry]|
|Estimated Enrollment :||200 participants|
|Target Follow-Up Duration:||24 Months|
|Official Title:||Screening/Observational Protocol to Determine Patient Eligibility for Inclusion in AAV Gene Therapy Clinical Trials (ECLIPSE)|
|Actual Study Start Date :||February 14, 2020|
|Estimated Primary Completion Date :||August 1, 2026|
|Estimated Study Completion Date :||August 1, 2026|
- Bleeding episodes [ Time Frame: 6 - 24 months ]Bleeding episode data recorded during the study
- Factor IX replacement therapy consumption [ Time Frame: 6 - 24 months ]Factor IX replacement therapy data recorded during the study
- To screen Haemophilia B patients for neutralising antibodies to a novel AAV vector (AAVS3). [ Time Frame: 6 - 24 months ]AAV antibody assay result
- To characterise baseline clinical parameters related to Haemophilia B. [ Time Frame: 6 - 24 months ]Target joint data and health reasource utilisation
Biospecimen Retention: None Retained
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04272554
|Contact: Clinical Operations||+44 1438 firstname.lastname@example.org|
|Principal Investigator:||Pratima Chowdary, Dr||The Royal Free Hospital|