The Sponsor 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…
ID
Source
Brief title
Condition
- Coagulopathies and bleeding diatheses (excl thrombocytopenic)
Synonym
Research involving
Sponsors and support
Intervention
Outcome measures
Primary outcome
Primary Data:
Bleeding episodes based on a prospective data capture period.
• Date and time of bleed onset and resolution.
• Location of bleed.
• Type of bleed (spontaneous, injury).
• Severity of bleed.
• Concomitant treatments for bleed, if any.
• Response to treatment
Factor IX concentrate consumption based on a prospective data capture period.
• FIX concentrate.
• Dose.
• Dates of administration.
• Reason for administration (prevention, bleeding episode).
• Frequency of administration.
• Total doses.
Secondary outcome
Secondary Data:
Presence/absence of neutralising antibodies to AAVS3.
Baseline clinical parameters related to Haemophilia B:
• Target joint number measured by Target Joint Assessment at enrolment.
• Health Resource Utilisation (usage assessed during Monthly Contact
Visits/Calls).
• FIX activity levels (obtained as part of patient standard care, if
available).
Background summary
People with Haemophilia B have very low levels of Factor IX (a protein that is
needed for making blood clot properly), and those with the most severe form
have frequent bleeding episodes. Where bleeding occurs into joints, this can
cause long-term disability and where bleeds occur in a closed space, such as
the brain, they can have catastrophic consequences. The current treatment for
Haemophilia B is regular (2-3 times per week) intravenous injection (injection
into a vein) of Factor IX concentrate to prevent bleeding or on demand
treatment when a bleeding episode occurs, for the lifetime of the individual.
Both of these modes of treatment have associated limitations and
inconveniences.
Study objective
The Sponsor 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 gene therapy study of the Sponsor.
Study design
Participants providing consent will attend an enrolment visit either remotely
or at the study site to complete eligibility evaluations and receive
instruction for completing the study diary. Demographic data and relevant
information to characterise the patient*s HB disease status will be captured. A
blood sample will be drawn at a convenient timepoint during the study to assess
the participant*s AAV neutralising antibody (NAb) status.
Study burden and risks
This is a prospective data collection study and does not confer any additional
risk beyond the normal course of the disease and routine treatment practices.
Patient management will be in line with normal standards of care according to
the physician treating the patient. No treatment intervention will occur as
part of this study.
Stevenage Bioscience Catalyst, Gunnels Wood Road N/A
Stevenage, Hertfordshire SG1 2FX
GB
Stevenage Bioscience Catalyst, Gunnels Wood Road N/A
Stevenage, Hertfordshire SG1 2FX
GB
Listed location countries
Age
Inclusion criteria
1) Male participants >= 16 years of age.
2) Able to give 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
a) Continuous routine FIX prophylaxis*, OR
b) On demand FIX treatment*
5) If receiving prophylaxis, participant has been on stable** and adequate¶
prophylaxis for at least 2 months prior to enrolment
*Continuous routine prophylaxis is defined as the intent of treating with an a
priori defined frequency of infusions (e.g., twice weekly, once every two
weeks, etc.) as documented in the medical records3.
* Enrollment of on demand patients is competitive up to a maximum of 10
patients.
** Stable denotes subject has been on prophylaxis with no change in either the
FIX product used, the dose administered or the regimen in the 2 months prior to
enrolment.
¶ Adequate prophylaxis for purposes of this protocol means an annualised
bleeding rate (ABR) of <=8 in the year preceding enrolment.
Exclusion criteria
1) Documented evidence of liver fibrosis and/or liver dysfunction (including,
but not limited to, persistently elevated alanine aminotransaminase, aspartate
aminotransferase, and/or billirubin > 1.5 x upper limit of normal).
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. Negative viral assays in 2 samples, collected at least 6 months apart, will
be required to be considered negative.
6) Participants at high risk of thromboembolic events (history of arterial or
venous thromboembolism [e.g. deep vein thrombosis, pulmonary embolism,
non-haemorrhagic stroke, arterial embolus] and those with acquired
thrombophilia. Participants with a history of atrial fibrillation).
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, ophthalmological, haematological,
immunological, gastrointestinal, endocrine, pulmonary, neurological, cerebral
or psychiatric disease, malignancy or any other psychological disorder.
Design
Recruitment
Followed up by the following (possibly more current) registration
No registrations found.
Other (possibly less up-to-date) registrations in this register
No registrations found.
In other registers
| Register | ID |
|---|---|
| CCMO | NL74976.041.20 |
| Other | NTC04272554 |