Hemophilia BECLIPSENCT04272554
AAV Gene Therapy Screening/Observational Protocol (ECLIPSE)
An observational study of Hemophilia B and Blood Coagulation Disorders, Inherited, run by Spur Therapeutics at 56 sites in 12 countries.
Trial 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… Read more about this study
- Who is it for?
- Ages 16 years and older, men only
- Conditions the study is for
- Hemophilia B, Blood Coagulation Disorders, Inherited, Blood Coagulation Disorder
- How many people
- 37 participants
- Where
- 56 sitesin 12 countries
- Study dates
- 14 Feb 2020to 10 Oct 2022
- How long will I take part?
- 24 Months
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 16 years and older
- Men only
- No healthy volunteers
- Child, Adult, Older adult
Study population: Haemophila B patients
Who may be able to join
- 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.
Who cannot join
- 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
Show all 13Show fewer
- 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.
Trial locations and contacts
56 sites in 12 countries.
56 sites in 12 countries
United States17 sites
University of Michigan
Ann Arbor, Michigan, 48109
Hemophilia Center of Western New York
Buffalo, New York, 14209
University of North Carolina (UNC)
Chapel Hill, North Carolina, 27599
Rush University Medical Center
Chicago, Illinois, 60612
University Hospitals Cleveland Medical Center
Cleveland, Ohio, 44106
Nationwide Childrens Hospital
Columbus, Ohio, 43205
Lombardi Comprehensive Cancer Center at Georgetown University
Georgetown, Washington, 20057
East Carolina University
Greenville, North Carolina, 27599
Indiana Hemophilia and Thrombosis Center
Indianapolis, Indiana, 46260
Children's Hospital of Los Angeles
Los Angeles, California, 90027
St. Jude Children's Research Hospital
Memphis, Tennessee, 38105
Blood Center of Wisconsin
Milwaukee, Wisconsin, 53226
Vanderbilt University Medical Center
Nashville, Tennessee, 37212
University of Nebraska Medical Center
Omaha, Nebraska, 68198
Phoenix Childrens Hospital
Phoenix, Arizona, 85016
University of California - Davis
Sacramento, California, 95817
University of South Florida
Tampa, Florida, 33612
United Kingdom9 sites
Queen Elizabeth Hospital
Birmingham
The Haemophilia and Thrombosis Centre
Canterbury, Kent
Cardiff University Hospital
Cardiff
Glasgow Royal Infirmary
Glasgow
Hammersmith Hospital
London
Royal Free London NHS Foundation Tust
London
St Thomas' Hospital
London
Manchester Royal Infirmary
Manchester
Newcastle Hemophilia Comprehensive Care Centre
Newcastle
Australia5 sites
Royal Children's Hospital, Parkville
Melbourne
The Alfred Hospital
Melbourne
Calvary Mater Hospital
Newcastle
Royal Prince Alfred Hospital
Sydney
Westmead Hospital
Sydney
Canada5 sites
McMaster University Medical Centre
Hamilton
Lawson Health Research Institute
London
The Moncton Hospital
Moncton
Saskatoon City Hospital
Saskatoon
University of Alberta Hospital
Winnipeg
Italy4 sites
Azienda Ospedaliero-Universitaria Careggi
Florence
Istituto Giannina Gaslini
Genova
Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico di Milano
Milan
Citta della Salute e della Scienza di Torino - Ospedale le Molinette
Torino
Turkey (Türkiye)4 sites
Istanbul University Faculty of Medicine
Istanbul
Ege University Medical Faculty
Izmir
Samsun Ondokuz Mayis University Medical Faculty
Samsun
Özel Acibadem Adana Hastanesi
Seyhan
France3 sites
Hôpital Louis Pradel - HCL
Bron
CHU Bicetre
Paris
Hopital Necker-Enfants Malades - AP-HP
Paris
Netherlands3 sites
Groningen UMC
Groningen
Erasmus MC
Rotterdam
Universitair Medisch Centrum Utrecht
Utrecht
Germany2 sites
Universitaetsklinikum Frankfurt - Klinikum der Johann Wolfgang Goethe Universitaet
Frankfurt
Universitaetsklinikum des Saarlandes und Medizinische Fakultaet der Universitaet des Saarlandes
Homburg
South Africa2 sites
University of Cape Town Clinical Research Center
Cape Town
Charlotte Maxeke Johannesburg Academic Hospital
Johannesburg
About this study
Official title: Screening/Observational Protocol to Determine Patient Eligibility for Inclusion in AAV Gene Therapy Clinical Trials (ECLIPSE)
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.
Read the 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.
- Conditions
- Hemophilia B, Blood Coagulation Disorders, Inherited, Blood Coagulation Disorder, Haematologic Disease
- Medical subject headings
- Hemophilia B, Blood Coagulation Disorders, Inherited, Blood Coagulation Disorders, Hematologic Diseases
What is being measured
The primary outcome is the main question the study is designed to answer.
Bleeding episodes
Bleeding episode data recorded during the study
Measured: 6 - 24 months
Factor IX replacement therapy consumption
Factor IX replacement therapy data recorded during the study
Measured: 6 - 24 months
Secondary outcomes (2)
To screen Haemophilia B patients for neutralising antibodies to a novel AAV vector (AAVS3).
AAV antibody assay result
Measured: 6 - 24 months
To characterise baseline clinical parameters related to Haemophilia B.
Target joint data and health reasource utilisation
Measured: 6 - 24 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Feb 2020
- Study start14 Feb 2020
- Primary completion10 Oct 2022
- Study completion10 Oct 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hemophilia BStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Case only
- Time perspective
- Other
- Enrolment
- 37 participants (actual)
- Target duration
- 24 Months
Sponsor and investigators
- Lead sponsor
- Spur Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Pratima Chowdary, Dr, Principal investigator, The Royal Free Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04272554
- Sponsor's protocol ID
- FLT-01
- Acronym
- ECLIPSE
- Status last verified
- Nov 2022
- First submitted
- 10 Feb 2020
- Last updated
- 4 Nov 2022
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 4 Nov 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Screening/Observational Protocol to Determine Patient Eligibility for Inclusion in AAV Gene Therapy Clinical Trials (ECLIPSE)
- ClinicalTrials.gov ID
- NCT04272554
- Acronym / sponsor ID
- ECLIPSE / FLT-01
- Conditions
- Hemophilia B, Blood Coagulation Disorders, Inherited, Blood Coagulation Disorder, Haematologic Disease
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 37 participants (actual)
- Lead sponsor
- Spur TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- 14 Feb 2020 (actual)
- Primary completion
- 10 Oct 2022 (actual)
- Study completion
- 10 Oct 2022 (actual)
- First posted
- 17 Feb 2020 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
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.
Study design
- Observational model
- Case only
- Time perspective
- Other
- Target follow-up
- 24 Months
Who may qualify
- Ages
- 16 years and older
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Haemophila B patients
Key inclusion criteria (7 listed)
- 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
- If receiving prophylaxis, participant has been on stable and adequate prophylaxis for at least 2 months prior to enrolment.
Key exclusion criteria (13 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Bleeding episodes
Time frame: 6 - 24 months
Bleeding episode data recorded during the study
Factor IX replacement therapy consumption
Time frame: 6 - 24 months
Factor IX replacement therapy data recorded during the study
2 secondary outcomes
To screen Haemophilia B patients for neutralising antibodies to a novel AAV vector (AAVS3).
Time frame: 6 - 24 months
To characterise baseline clinical parameters related to Haemophilia B.
Time frame: 6 - 24 months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 Nov 2022.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Pratima Chowdary, Dr principal investigator
The Royal Free Hospital
56 sites in 12 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Hemophilia B and Blood Coagulation Disorders, Inherited, run by Spur Therapeutics at 56 sites in 12 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 13 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 4 Nov 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 56 sites in United States, United Kingdom, Australia, Canada, Italy and 7 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Bleeding episodes (time frame: 6 - 24 months). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 4 Nov 2022.