Brief Title
A Long-Term Follow-Up Study of Haemophilia B Patients Who Have Undergone Gene Therapy
Official Title
An Open-Label, Multicentre, Long-Term Follow-Up Study to Investigate the Safety and Durability of Response Following Dosing of a Novel Adeno-Associated Viral Vector (FLT180a) in Patients With Haemophilia B
Brief Summary
Severe haemophilia B (HB) is a bleeding disorder where a protein made by the body to help make blood clot is either partly or completely missing. This protein is called a clotting factor; with severe HB, levels of clotting Factor IX (nine; FIX) are very low and affected individuals can suffer life threatening bleeding episodes. HB mainly affects boys and men (approximately one in every 30,000 males). Current treatment for HB involves intravenous infusions of FIX as regular treatment (prophylaxis) or 'on demand' treatment. On demand treatment is highly effective at stopping bleeding but cannot fully reverse long-term damage that follows after a bleed. Regular treatment can prevent bleeding; however it is invasive for patients and also expensive. This clinical study aims to investigate the long-term safety and durability of FIX activity in participants who have been dosed with a new gene therapy product (FLT180a) in earlier clinical studies. Following administration, FLT180a results in production of FIX in the participants' liver cells which is then released into the blood stream. The aim is to have the participants' own body produce levels of FIX that allow for clotting to occur as normal as would be seen in a non-HB individual. This would remove the need for prophylaxis or on demand treatment following just a single administration of FLT180a. Up to 50 participants who have already been administered with FLT180a in the EU and US will take part in this study. Participants will be followed up in this trial until they have reached 15 years after being dosed. Participants will undergo procedures including physical examinations, join assessments, blood tests and liver ultrasounds. Participants will also need to complete a diary to document occurrence of bleeding episodes and record the amount of Factor IX concentrate they receive. Patient reports outcomes including measures of Quality of Life, disability and physical activity will also be collected.
Study Phase
Phase 1/Phase 2
Study Type
Interventional
Primary Outcome
Primary Safety Measurement
Secondary Outcome
Secondary Safety Measurement
Condition
Hemophilia B
Intervention
FLT180a
Study Arms / Comparison Groups
FLT180a
Description: Participants who have received gene therapy vector (FLT180a)
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Biological
Estimated Enrollment
10
Start Date
July 10, 2018
Completion Date
December 31, 2035
Primary Completion Date
December 31, 2035
Eligibility Criteria
Inclusion Criteria: - Patients who have previously received FLT180a within a clinical study. - Able to give full informed consent and able to comply with all requirements of the study including long-term follow-up for the time frame the study requires. - Willing to practice barrier contraception until at least three consecutive semen samples after vector administration are negative for vector sequences. Exclusion Criteria: N/A
Gender
Male
Ages
18 Years - N/A
Accepts Healthy Volunteers
No
Contacts
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Location Countries
United Kingdom
Location Countries
United Kingdom
Administrative Informations
NCT ID
NCT03641703
Organization ID
FLT180a-04
Responsible Party
Sponsor
Study Sponsor
Freeline Therapeutics
Study Sponsor
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Verification Date
January 2020