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AAV Gene Therapy Screening/Observational Protocol (ECLIPSE) (ECLIPSE)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT04272554
Recruitment Status : Recruiting
First Posted : February 17, 2020
Last Update Posted : February 5, 2021
Information provided by (Responsible Party):
Freeline Therapeutics

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 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.

Condition or disease
Hemophilia B Blood Coagulation Disorders, Inherited Blood Coagulation Disorder Haematologic Disease

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 Freeline gene therapy treatment study.

Participants providing consent will attend an enrolment visit to complete eligibility evaluations, collect health and demographic information and receive instruction for completing the study diary. Participants will then complete a diary recording bleeding episodes and FIX usage until they enrol into a gene therapy treatment study. A blood sample will be drawn at a convenient timepoint during the study to assess the participant's AAV neutralising antibody (NAb) status.

No treatment intervention will occur as part of this study.

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Study Type : Observational [Patient Registry]
Estimated Enrollment : 200 participants
Observational Model: Case-Only
Time Perspective: Other
Target Follow-Up Duration: 24 Months
Official Title: Screening/Observational Protocol to Determine Patient Eligibility for Inclusion in AAV Gene Therapy Clinical Trials (ECLIPSE)
Actual Study Start Date : February 14, 2020
Estimated Primary Completion Date : August 1, 2026
Estimated Study Completion Date : August 1, 2026

Resource links provided by the National Library of Medicine

Primary Outcome Measures :
  1. Bleeding episodes [ Time Frame: 6 - 24 months ]
    Bleeding episode data recorded during the study

  2. Factor IX replacement therapy consumption [ Time Frame: 6 - 24 months ]
    Factor IX replacement therapy data recorded during the study

Secondary Outcome Measures :
  1. To screen Haemophilia B patients for neutralising antibodies to a novel AAV vector (AAVS3). [ Time Frame: 6 - 24 months ]
    AAV antibody assay result

  2. To characterise baseline clinical parameters related to Haemophilia B. [ Time Frame: 6 - 24 months ]
    Target joint data and health reasource utilisation

Biospecimen Retention:   None Retained
Pharmacogenomic and research biomarker samples will be collected from consenting patients

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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Ages Eligible for Study:   16 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Haemophila B patients

Inclusion Criteria

  1. Male participants, ≥ 16 years of age.
  2. 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.
  3. Interested in participation in future gene therapy clinical studies.
  4. 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

    1. Continuous routine FIX prophylaxis, OR
    2. On demand FIX treatment
  5. If receiving prophylaxis, participant has been on stable and adequate prophylaxis for at least 2 months prior to enrolment.

Exclusion Criteria:

  1. Documented evidence of liver fibrosis and/ or liver dysfunction
  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
  6. Participants at high risk of thromboembolic events (history of arterial or venous thromboembolism
  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, opthalmological, hematological, immunological, gastrointestinal, endocrine, pulmonary, neurological, cerebral or psychiatric disease, malignancy or any other psychological disorder.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT04272554

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Contact: Clinical Operations +44 1438 906870

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Sponsors and Collaborators
Freeline Therapeutics
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Principal Investigator: Pratima Chowdary, Dr The Royal Free Hospital
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Responsible Party: Freeline Therapeutics Identifier: NCT04272554    
Other Study ID Numbers: FLT-01
First Posted: February 17, 2020    Key Record Dates
Last Update Posted: February 5, 2021
Last Verified: February 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Hemostatic Disorders
Hemophilia B
Blood Coagulation Disorders
Blood Coagulation Disorders, Inherited
Hematologic Diseases
Pathologic Processes
Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn
Genetic Diseases, X-Linked
Vascular Diseases
Cardiovascular Diseases