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Trial details imported from ClinicalTrials.gov

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




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

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
To screen Haemophilia B patients for antibodies to a novel AAV vector - Detection of neutralising antibodies (NAbs) to the vector serotype in blood
Timepoint [1] 0 0
6 - 18 months
Primary outcome [2] 0 0
Annualized Bleeding Rate (ABR) - Bleeding episode number and type will be recorded during the study
Timepoint [2] 0 0
6 - 18 months
Primary outcome [3] 0 0
Factor IX replacement therapy consumption - Factor IX replacement therapy dose and frequency will be recorded during the study
Timepoint [3] 0 0
6 - 18 months

Eligibility
Key inclusion criteria
1. Male adults aged 16 years of age or older.

2. Able to give full informed consent or obtain full informed consent from the patient's
legally acceptable representative (as appropriate) and able to comply with all
requirements of the trial.

3. Interested in participation in gene therapy clinical trials.

4. Confirmed diagnosis of Haemophila B defined as one of the following:

1. Documented severe Factor IX (FIX) deficiency with plasma FIX activity of < 1% of
normal or

2. Moderately severe FIX deficiency with plasma FIX activity level between = 1% and
= 2% and a severe bleeding phenotype defined by 1 of the following:

(i) On prophylaxis for a history of bleeding, or (ii) On-demand therapy with a current
history of 4 or more bleeding episodes/year on average over the past 3 years, or (iii)
Evidence of chronic haemophilic arthropathy (pain, joint destruction, and loss of
range of motion).
Minimum age
16 Years
Maximum age
No limit
Gender
Males
Can healthy volunteers participate?
No
Key exclusion criteria
1. Evidence of liver dysfunction (persistently elevated alanine aminotransaminase,
aspartate aminotransferase, bilirubin > 1.5 x upper limit of normal).

2. Uncontrolled glaucoma, diabetes mellitus, or hypertension.

3. Malignancy requiring treatment.

4. Patients with uncontrolled cardiac failure, unstable angina, or myocardial infarction
in the past 6 months.

5. Prior treatment with a gene transfer medicinal product.

6. Presence or history of neutralising anti-human FIX antibodies (inhibitors).

Study design
Purpose
Duration
Selection
Timing
Statistical methods / analysis

Recruitment
Recruitment status
Not yet recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
VIC
Recruitment hospital [1] 0 0
The Alfred Hospital - Melbourne
Recruitment hospital [2] 0 0
Royal Children's Hospital, Parkville - Melbourne
Recruitment postcode(s) [1] 0 0
3004 - Melbourne
Recruitment postcode(s) [2] 0 0
3052 - Melbourne

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) vectors for a number of diseases and is
actively advancing its Haemophilia B programme. The background prevalence of neutralising
antibodies to the vector capsid necessitates screening before eligibility for gene therapy
protocols can be fully assessed.

This study is being conducted to screen patients for antibodies to a novel AAV vector and
determine potential eligibility for gene therapy clinical trials.

This study will also be used to collect observational data relevant to the condition under
study. This data maybe used to establish a baseline for patients who go on to receive a gene
therapy on a subsequent clinical trial.
Trial website
https://clinicaltrials.gov/show/NCT04272554
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Huyen Tran, A/Prof
Address 0 0
The Alfred
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Clinical Operations
Address 0 0
Country 0 0
Phone 0 0
+44 1438 906870
Fax 0 0
Email 0 0
contact@freeline.life
Contact person for scientific queries

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