Brief Title
AAV Gene Therapy Screening/Observational Protocol (ECLIPSE)
Official Title
Screening/Observational Protocol to Determine Patient Eligibility for Inclusion in AAV Gene Therapy Clinical Trials (ECLIPSE)
Brief 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.
Study Type
Observational [Patient Registry]
Primary Outcome
Bleeding episodes
Secondary Outcome
To screen Haemophilia B patients for neutralising antibodies to a novel AAV vector (AAVS3).
Condition
Hemophilia B
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Estimated Enrollment
37
Start Date
February 14, 2020
Completion Date
October 10, 2022
Primary Completion Date
October 10, 2022
Eligibility Criteria
Inclusion Criteria
1. Male participants, ≥ 16 years of age.
2. Able to give full informed consent or obtain full informed consent/assent (according
to local regulations) and/or obtain full informed consent from the participant's
legally acceptable representative (as appropriate), and able to understand and comply
with all requirements of the study, including diary completion.
3. Interested in participation in future gene therapy clinical studies.
4. Subjects with Haemophilia B with known severe or moderately severe FIX deficiency (≤2%
of normal circulating FIX activity) for which the subject is either on
1. Continuous routine FIX prophylaxis, OR
2. On demand FIX treatment
5. If receiving prophylaxis, participant has been on stable and adequate prophylaxis for
at least 2 months prior to enrolment.
Exclusion Criteria:
1. Documented evidence of liver fibrosis and/ or liver dysfunction
2. Prior treatment with a gene transfer medicinal product.
3. Known presence or history of neutralising anti-human FIX antibodies (inhibitors)
4. Previously established serological evidence of HIV-1
5. Documented active hepatitis B or C, and HBsAg or HCV RNA viral load positivity,
respectively, or currently on antiviral therapy for hepatitis B or C
6. Participants at high risk of thromboembolic events (history of arterial or venous
thromboembolism
7. Known coagulation disorder other than Haemophilia B
8. Known history of an allergic reaction or anaphylaxis to Factor IX products or known
uncontrolled allergic conditions
9. Known history of allergy to corticosteroids or to tacrolimus or any other macrolide
10. Known medical condition that would require chronic administration of corticosteroids
(excluding topical formulations)
11. History of alcohol or drug dependence.
12. Planned surgical procedure within the next 12 months requiring prophylactic FIX
treatment.
13. Known active severe infection (including documented COVID-19 infection), or any other
significant concurrent, uncontrolled medical condition evaluated by the investigator
to interfere with adherence to the protocol procedures or with tolerance to gene
therapy in a future treatment study including, but not limited to, renal, hepatic,
cardiovascular, opthalmological, hematological, immunological, gastrointestinal,
endocrine, pulmonary, neurological, cerebral or psychiatric disease, malignancy or any
other psychological disorder.
Gender
Male
Ages
16 Years - N/A
Accepts Healthy Volunteers
No
Contacts
Pratima Chowdary, Dr, ,
Location Countries
Australia
Location Countries
Australia
Administrative Informations
NCT ID
NCT04272554
Organization ID
FLT-01
Responsible Party
Sponsor
Study Sponsor
Freeline Therapeutics
Study Sponsor
Pratima Chowdary, Dr, Principal Investigator, The Royal Free Hospital
Verification Date
November 2022