NCT04272554

AAV Gene Therapy Screening/Observational Protocol (ECLIPSE)

Screening/Observational Protocol to Determine Patient Eligibility for Inclusion in AAV Gene Therapy Clinical Trials (ECLIPSE)

• Status: Completed
• Phase: —
• Study type: Observational
• Enrollment: 37 (actual)
• Sponsor: Spur Therapeutics · Industry
• Sex: Male
• Age: 16 Years
• Healthy volunteers: Not accepted

Summary

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.

Detailed description

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.

Conditions

• Hemophilia B
• Blood Coagulation Disorders, Inherited
• Blood Coagulation Disorder
• Haematologic Disease

Timeline

• Start date: 2020-02-14
• Primary completion: 2022-10-10
• Completion: 2022-10-10
• First posted: 2020-02-17
• Last updated: 2022-11-04

Locations

56 sites across 12 countries: United States, Australia, Austria, Canada, France, Germany, Ireland, Italy, Netherlands, South Africa, Turkey (Türkiye), United Kingdom