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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT04272554




Registration number
NCT04272554
Ethics application status
Date submitted
10/02/2020
Date registered
17/02/2020
Date last updated
4/11/2022

Titles & IDs
Public title
AAV Gene Therapy Screening/Observational Protocol (ECLIPSE)
Scientific title
Screening/Observational Protocol to Determine Patient Eligibility for Inclusion in AAV Gene Therapy Clinical Trials (ECLIPSE)
Secondary ID [1] 0 0
FLT-01
Universal Trial Number (UTN)
Trial acronym
ECLIPSE
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Hemophilia B 0 0
Blood Coagulation Disorders, Inherited 0 0
Blood Coagulation Disorder 0 0
Haematologic Disease 0 0
Condition category
Condition code
Cardiovascular 0 0 0 0
Diseases of the vasculature and circulation including the lymphatic system
Blood 0 0 0 0
Clotting disorders
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Blood 0 0 0 0
Clotting disorders
Blood 0 0 0 0
Haematological diseases

Intervention/exposure
Study type
Observational [Patient Registry]
Patient registry
Target follow-up duration
Target follow-up type
Description of intervention(s) / exposure
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Bleeding episodes
Timepoint [1] 0 0
6 - 24 months
Primary outcome [2] 0 0
Factor IX replacement therapy consumption
Timepoint [2] 0 0
6 - 24 months
Secondary outcome [1] 0 0
To screen Haemophilia B patients for neutralising antibodies to a novel AAV vector (AAVS3).
Timepoint [1] 0 0
6 - 24 months
Secondary outcome [2] 0 0
To characterise baseline clinical parameters related to Haemophilia B.
Timepoint [2] 0 0
6 - 24 months

Eligibility
Key inclusion 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.
Minimum age
16 Years
Maximum age
No limit
Sex
Males
Can healthy volunteers participate?
No
Key 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.

Study design
Purpose
Duration
Selection
Timing
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
14/02/2020
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
10/10/2022
Sample size
Target
Accrual to date
Final
37
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Royal Children's Hospital, Parkville - Melbourne
Recruitment hospital [2] 0 0
The Alfred Hospital - Melbourne
Recruitment hospital [3] 0 0
Calvary Mater Hospital - Newcastle
Recruitment hospital [4] 0 0
Royal Prince Alfred Hospital - Sydney
Recruitment hospital [5] 0 0
Westmead Hospital - Sydney
Recruitment postcode(s) [1] 0 0
- Melbourne
Recruitment postcode(s) [2] 0 0
- Newcastle
Recruitment postcode(s) [3] 0 0
- Sydney
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Arizona
Country [2] 0 0
United States of America
State/province [2] 0 0
California
Country [3] 0 0
United States of America
State/province [3] 0 0
Florida
Country [4] 0 0
United States of America
State/province [4] 0 0
Illinois
Country [5] 0 0
United States of America
State/province [5] 0 0
Indiana
Country [6] 0 0
United States of America
State/province [6] 0 0
Michigan
Country [7] 0 0
United States of America
State/province [7] 0 0
Nebraska
Country [8] 0 0
United States of America
State/province [8] 0 0
New York
Country [9] 0 0
United States of America
State/province [9] 0 0
North Carolina
Country [10] 0 0
United States of America
State/province [10] 0 0
Ohio
Country [11] 0 0
United States of America
State/province [11] 0 0
Tennessee
Country [12] 0 0
United States of America
State/province [12] 0 0
Washington
Country [13] 0 0
United States of America
State/province [13] 0 0
Wisconsin
Country [14] 0 0
Austria
State/province [14] 0 0
Vienna
Country [15] 0 0
Canada
State/province [15] 0 0
Hamilton
Country [16] 0 0
Canada
State/province [16] 0 0
London
Country [17] 0 0
Canada
State/province [17] 0 0
Moncton
Country [18] 0 0
Canada
State/province [18] 0 0
Saskatoon
Country [19] 0 0
Canada
State/province [19] 0 0
Winnipeg
Country [20] 0 0
France
State/province [20] 0 0
Bron
Country [21] 0 0
France
State/province [21] 0 0
Paris
Country [22] 0 0
Germany
State/province [22] 0 0
Frankfurt
Country [23] 0 0
Germany
State/province [23] 0 0
Homburg
Country [24] 0 0
Ireland
State/province [24] 0 0
Dublin
Country [25] 0 0
Italy
State/province [25] 0 0
Firenze
Country [26] 0 0
Italy
State/province [26] 0 0
Genova
Country [27] 0 0
Italy
State/province [27] 0 0
Milan
Country [28] 0 0
Italy
State/province [28] 0 0
Torino
Country [29] 0 0
Netherlands
State/province [29] 0 0
Groningen
Country [30] 0 0
Netherlands
State/province [30] 0 0
Rotterdam
Country [31] 0 0
Netherlands
State/province [31] 0 0
Utrecht
Country [32] 0 0
South Africa
State/province [32] 0 0
Cape Town
Country [33] 0 0
South Africa
State/province [33] 0 0
Johannesburg
Country [34] 0 0
Turkey
State/province [34] 0 0
Istanbul
Country [35] 0 0
Turkey
State/province [35] 0 0
Izmir
Country [36] 0 0
Turkey
State/province [36] 0 0
Samsun
Country [37] 0 0
Turkey
State/province [37] 0 0
Seyhan
Country [38] 0 0
United Kingdom
State/province [38] 0 0
Kent
Country [39] 0 0
United Kingdom
State/province [39] 0 0
Birmingham
Country [40] 0 0
United Kingdom
State/province [40] 0 0
Cardiff
Country [41] 0 0
United Kingdom
State/province [41] 0 0
Glasgow
Country [42] 0 0
United Kingdom
State/province [42] 0 0
London
Country [43] 0 0
United Kingdom
State/province [43] 0 0
Manchester
Country [44] 0 0
United Kingdom
State/province [44] 0 0
Newcastle

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Freeline Therapeutics
Address
Country

Ethics approval
Ethics application status

Summary
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.
Trial website
https://clinicaltrials.gov/study/NCT04272554
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Pratima Chowdary, Dr
Address 0 0
The Royal Free Hospital
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT04272554