AAV Gene Therapy Screening/Observational Protocol (ECLIPSE)

NCT04272554 | Completed

• 1 other identifier: FLT-01
• Study Type: observational
• Target: 37
• Locations: 11 countries
• Sites: 51

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.

Conditions

Hemophilia B; Blood Coagulation Disorders, Inherited; Blood Coagulation Disorder; Haematologic Disease

Outcome Measures

Primary Outcomes (2)

• Bleeding episodes

  Bleeding episode data recorded during the study

  6 - 24 months

• Factor IX replacement therapy consumption

  Factor IX replacement therapy data recorded during the study

  6 - 24 months

Secondary Outcomes (2)

• To screen Haemophilia B patients for neutralising antibodies to a novel AAV vector (AAVS3).

  6 - 24 months

• To characterise baseline clinical parameters related to Haemophilia B.

  6 - 24 months

Eligibility Criteria

• Age: 16 Years+
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Haemophila B patients

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.

Sponsors & Collaborators

• Spur Therapeutics: lead

Study Sites (56)

Phoenix Childrens Hospital

Phoenix, Arizona, 85016, United States

Location

Children's Hospital of Los Angeles

Los Angeles, California, 90027, United States

Location

University of California - Davis

Sacramento, California, 95817, United States

Location

University of South Florida

Tampa, Florida, 33612, United States

Location

Rush University Medical Center

Chicago, Illinois, 60612, United States

Location

Indiana Hemophilia and Thrombosis Center

Indianapolis, Indiana, 46260, United States

Location

University of Michigan

Ann Arbor, Michigan, 48109, United States

Location

University of Nebraska Medical Center

Omaha, Nebraska, 68198, United States

Location

Hemophilia Center of Western New York

Buffalo, New York, 14209, United States

Location

University of North Carolina (UNC)

Chapel Hill, North Carolina, 27599, United States

Location

East Carolina University

Greenville, North Carolina, 27599, United States

Location

University Hospitals Cleveland Medical Center

Cleveland, Ohio, 44106, United States

Location

Nationwide Childrens Hospital

Columbus, Ohio, 43205, United States

Location

St. Jude Children's Research Hospital

Memphis, Tennessee, 38105, United States

Location

Vanderbilt University Medical Center

Nashville, Tennessee, 37212, United States

Location

Lombardi Comprehensive Cancer Center at Georgetown University

Georgetown, Washington, 20057, United States

Location

Blood Center of Wisconsin

Milwaukee, Wisconsin, 53226, United States

Location

Royal Children's Hospital, Parkville

Melbourne, Australia

Location

The Alfred Hospital

Melbourne, Australia

Location

Calvary Mater Hospital

Newcastle, Australia

Location

Royal Prince Alfred Hospital

Sydney, Australia

Location

Westmead Hospital

Sydney, Australia

Location

Medical University of Vienna

Vienna, Austria

Location

McMaster University Medical Centre

Hamilton, Canada

Location

Lawson Health Research Institute

London, Canada

Location

The Moncton Hospital

Moncton, Canada

Location

Saskatoon City Hospital

Saskatoon, Canada

Location

University of Alberta Hospital

Winnipeg, Canada

Location

Hôpital Louis Pradel - HCL

Bron, France

Location

CHU Bicetre

Paris, France

Location

Hopital Necker-Enfants Malades - AP-HP

Paris, France

Location

Universitaetsklinikum Frankfurt - Klinikum der Johann Wolfgang Goethe Universitaet

Frankfurt, Germany

Location

Universitaetsklinikum des Saarlandes und Medizinische Fakultaet der Universitaet des Saarlandes

Homburg, Germany

Location

St James Hospital

Dublin, Ireland

Location

Azienda Ospedaliero-Universitaria Careggi

Florence, Italy

Location

Istituto Giannina Gaslini

Genova, Italy

Location

Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico di Milano

Milan, Italy

Location

Citta della Salute e della Scienza di Torino - Ospedale le Molinette

Torino, Italy

Location

Groningen UMC

Groningen, Netherlands

Location

Erasmus MC

Rotterdam, Netherlands

Location

Universitair Medisch Centrum Utrecht

Utrecht, Netherlands

Location

University of Cape Town Clinical Research Center

Cape Town, South Africa

Location

Charlotte Maxeke Johannesburg Academic Hospital

Johannesburg, South Africa

Location

Istanbul University Faculty of Medicine

Istanbul, Turkey (Türkiye)

Location

Ege University Medical Faculty

Izmir, Turkey (Türkiye)

Location

Samsun Ondokuz Mayis University Medical Faculty

Samsun, Turkey (Türkiye)

Location

Özel Acibadem Adana Hastanesi

Seyhan, Turkey (Türkiye)

Location

The Haemophilia and Thrombosis Centre

Canterbury, Kent, United Kingdom

Location

Queen Elizabeth Hospital

Birmingham, United Kingdom

Location

Cardiff University Hospital

Cardiff, United Kingdom

Location

Glasgow Royal Infirmary

Glasgow, United Kingdom

Location

Hammersmith Hospital

London, United Kingdom

Location

Royal Free London NHS Foundation Tust

London, United Kingdom

Location

St Thomas' Hospital

London, United Kingdom

Location

Manchester Royal Infirmary

Manchester, United Kingdom

Location

Newcastle Hemophilia Comprehensive Care Centre

Newcastle, United Kingdom

Location

Biospecimen

Retention: NONE RETAINED

Pharmacogenomic and research biomarker samples will be collected from consenting patients

MeSH Terms

Conditions

Hemophilia B; Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases

Condition Hierarchy (Ancestors)

Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, X-Linked

Study Officials

• Pratima Chowdary, Dr

  The Royal Free Hospital

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: CASE ONLY
• Time Perspective: OTHER
• Target Duration: 24 Months
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 10, 2020
• First Posted: February 17, 2020
• Study Start: February 14, 2020
• Primary Completion: October 10, 2022
• Study Completion: October 10, 2022
• Last Updated: November 4, 2022

Record last verified: 2022-11

Data Sharing

• IPD Sharing: Will not share

Locations

IE (1); CA (5); GB (9); TU (4); ZA (2); FR (3); IT (4); US (17); DE (2); NL (3); AU (1)