bleeding disorder hemophilia
Conditions
Interventions
None listed
Sponsors
Freeline Therapeutics
Eligibility
Age
12 Years to 99 Years
Inclusion criteria
Inclusion criteria: 1) Male participants >= 16 years of age. 2) Able to give 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 (
Exclusion criteria
Exclusion criteria: 1) Documented evidence of liver fibrosis and/or liver dysfunction (including, but not limited to, persistently elevated alanine aminotransaminase, aspartate aminotransferase, and/or billirubin > 1.5 x upper limit of normal). 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. Negative viral assays in 2 samples, collected at least 6 months apart, will be required to be considered negative. 6) Participants at high risk of thromboembolic events (history of arterial or venous thromboembolism [e.g. deep vein thrombosis, pulmonary embolism, non-haemorrhagic stroke, arterial embolus] and those with acquired thrombophilia. Participants with a history of atrial fibrillation). 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, ophthalmological, haematological, immunological, gastrointestinal, endocrine, pulmonary, neurological, cerebral or psychiatric disease, malignancy or any other psychological disorder.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Primary Data: Bleeding episodes based on a prospective data capture period. • Date and time of bleed onset and resolution. • Location of bleed. • Type of bleed (spontaneous, injury). • Severity of bleed. • Concomitant treatments for bleed, if any. • Response to treatment Factor IX concentrate consumption based on a prospective data capture period. • FIX concentrate. • Dose. • Dates of administration. • Reason for administration (prevention, bleeding episode). • Frequency of administration. • Total doses. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary Data: Presence/absence of neutralising antibodies to AAVS3. Baseline clinical parameters related to Haemophilia B: • Target joint number measured by Target Joint Assessment at enrolment. • Health Resource Utilisation (usage assessed during Monthly Contact Visits/Calls). • FIX activity levels (obtained as part of patient standard care, if available). | — |
Countries
Netherlands
Outcome results
None listed