Status:
COMPLETED
AAV Gene Therapy Screening/Observational Protocol (ECLIPSE)
Lead Sponsor:
Spur Therapeutics
Conditions:
Hemophilia B
Blood Coagulation Disorders, Inherited
Eligibility:
MALE
16+ years
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 prospec...
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 Freel...
Eligibility Criteria
Inclusion
- Inclusion Criteria
- Male participants, ≥ 16 years of age.
- 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.
- Interested in participation in future gene therapy clinical studies.
- 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
- Continuous routine FIX prophylaxis, OR
- On demand FIX treatment
- If receiving prophylaxis, participant has been on stable and adequate prophylaxis for at least 2 months prior to enrolment.
- Exclusion Criteria:
- Documented evidence of liver fibrosis and/ or liver dysfunction
- Prior treatment with a gene transfer medicinal product.
- Known presence or history of neutralising anti-human FIX antibodies (inhibitors)
- Previously established serological evidence of HIV-1
- 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
- Participants at high risk of thromboembolic events (history of arterial or venous thromboembolism
- Known coagulation disorder other than Haemophilia B
- Known history of an allergic reaction or anaphylaxis to Factor IX products or known uncontrolled allergic conditions
- Known history of allergy to corticosteroids or to tacrolimus or any other macrolide
- Known medical condition that would require chronic administration of corticosteroids (excluding topical formulations)
- History of alcohol or drug dependence.
- Planned surgical procedure within the next 12 months requiring prophylactic FIX treatment.
- 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.
Exclusion
Key Trial Info
Start Date :
February 14 2020
Trial Type :
OBSERVATIONAL
Allocation :
ACTUAL
End Date :
October 10 2022
Estimated Enrollment :
37 Patients enrolled
Trial Details
Trial ID
NCT04272554
Start Date
February 14 2020
End Date
October 10 2022
Last Update
November 4 2022
Active Locations (56)
Enter a location and click search to find clinical trials sorted by distance.
1
Phoenix Childrens Hospital
Phoenix, Arizona, United States, 85016
2
Children's Hospital of Los Angeles
Los Angeles, California, United States, 90027
3
University of California - Davis
Sacramento, California, United States, 95817
4
University of South Florida
Tampa, Florida, United States, 33612