AAV Gene Therapy Screening/Observational Protocol (ECLIPSE)

Completed

Conditions studied: Hemophilia B, Blood Coagulation Disorders, Inherited, Blood Coagulation Disorder, Haematologic Disease

In brief

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.

Key facts

Study ID

NCT04272554

Run by

Spur Therapeutics

People needed

37

Starts

2020-02-14

Expected to finish

2022-10-10

Last updated by the study team

2022-11-04

Who can join

Age: 16 and older. Sex: male. Healthy volunteers: not accepted.

You may qualify if…

• 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.

You may not qualify if…

• 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.

Where it is running

• Children's Hospital of Los Angeles — Los Angeles, California, United States
• University of California - Davis — Sacramento, California, United States
• University of South Florida — Tampa, Florida, United States
• Rush University Medical Center — Chicago, Illinois, United States
• Indiana Hemophilia and Thrombosis Center — Indianapolis, Indiana, United States
• University of Michigan — Ann Arbor, Michigan, United States
• University of Nebraska Medical Center — Omaha, Nebraska, United States
• Hemophilia Center of Western New York — Buffalo, New York, United States
• University of North Carolina (UNC) — Chapel Hill, North Carolina, United States
• East Carolina University — Greenville, North Carolina, United States
• University Hospitals Cleveland Medical Center — Cleveland, Ohio, United States
• Nationwide Childrens Hospital — Columbus, Ohio, United States
• St. Jude Children's Research Hospital — Memphis, Tennessee, United States
• Vanderbilt University Medical Center — Nashville, Tennessee, United States
• Lombardi Comprehensive Cancer Center at Georgetown University — Georgetown, Washington, United States
• Blood Center of Wisconsin — Milwaukee, Wisconsin, United States
• Royal Children's Hospital, Parkville — Melbourne, Australia
• The Alfred Hospital — Melbourne, Australia
• Calvary Mater Hospital — Newcastle, Australia
• Royal Prince Alfred Hospital — Sydney, Australia
• Westmead Hospital — Sydney, Australia
• Medical University of Vienna — Vienna, Austria
• McMaster University Medical Centre — Hamilton, Canada
• Lawson Health Research Institute — London, Canada
• Phoenix Childrens Hospital — Phoenix, Arizona, United States

Full record on ClinicalTrials.gov

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