An Open-Label, Multicenter Evaluation of the Long-Term Safety and Efficacy of Recombinant Human Coagulation Factor VIII Fusion Protein (rFVIIIFc) in the Prevention and Treatment of Bleeding Episodes in Previously Treated Subjects With Hemophilia A.…
ID
Source
Brief title
Condition
- Coagulopathies and bleeding diatheses (excl thrombocytopenic)
Synonym
Research involving
Sponsors and support
Intervention
Outcome measures
Primary outcome
Occurence of inhibitor development
Secondary outcome
Secondary:
The annualized number of bleeding episodes (spontaneous and traumatic) per
subject
The annualized number of spontaneous joint bleeding episodes per subject
The total number of days of exposure per subject per year
The consumption of rFVIIIFc as total dose per kg per subject per year
Physician*s global assessment of subject's response to his treatment using a
4-point scale
Subject*s assessment of response to treatment of bleeding episodes using a
4-point scale
Background summary
To evaluate the long-term safety of rFVIIIFc for prevention and on-demand
treatment of bleeding episodes in subjects with hemophilia A, and to allow
subjects from the A-LONG pivotal study (997HA301), the pediatric study
(8HA02PED), or any other rFVIIIFc study to continue treatment with rFVIIIFc.
Subjects will continue in this study for up to 4 years or until rFVIIIFc is
commercially available in the applicable participating country.
Subjects will follow a tailored or personalized prophylactic regiment chosen by
the study doctor. Once the child is 12 years old or older also other options
can be chosed such as once-weekly modified prophylaxis or on-demand regimen
based on the clinical profile of the subject and by the trough or peak
(recovery) values, if needed, as observed in the preceding study. Subjects are
allowed to change treatment regimens (for example, from prophylaxis, including
modified prophylaxis, to on-demand, or from on-demand to prophylaxis) during
the study.
Study objective
An Open-Label, Multicenter Evaluation of the Long-Term Safety and Efficacy of
Recombinant Human Coagulation Factor VIII Fusion Protein (rFVIIIFc) in the
Prevention and Treatment of Bleeding Episodes in Previously Treated Subjects
With Hemophilia A.
The primary objective of the study is to evaluate the long-term safety of
rFVIIIFc in subjects with hemophilia A.
Study design
This is an open-label, multicenter, long-term study of intravenous (IV)
administration of rFVIIIFc in previously treated patients with hemophilia A,
who have completed the A-LONG study (997HA301), the pediatric study (8HA02PED),
or any other trial with rFVIIIFc. Treatment will be administered in tailored or
personalized prophylaxis, once-weekly modified prophylaxis or on-demand
regimens.
Intervention
Study drug: Recombinant Human Coagulation Factor VIII Fusion Protein (rFVIIIFc)
per IV administration
Study burden and risks
Benefit: A possible benefit may be the need for less frequent injections to
manage his hemophilia. What we learn from this
study may lead to improved understanding of how the study drug works when taken
for several years by people with
hemophilia A.
Risks: The examinations done during the study may cause discomfort. Taking
blood from a vein may cause bruising,
localized bleeding, infection, faintness, and a small amount of pain from the
needle puncture.
Side effects known to occur with other Factor VIII products may also occur with
the study drug. Side effects that have
occurred with other FVIII products include symptoms of itching, rash, local
reactions at the injection site (such as burning
and temporary redness), unusual taste in the mouth, fever, headache, discomfort
or swelling around the vein from which
blood was drawn, bleeding/bruising at the injection site, and Factor VIII
inhibitor formation.
Innovation House, Norden Road 70
Maidenhead, Berkshire SL6 4AY
GB
Innovation House, Norden Road 70
Maidenhead, Berkshire SL6 4AY
GB
Listed location countries
Age
Inclusion criteria
1. Ability to understand the purpose and risks of the study and provide
signed and dated informed consent and authorization to use protected
health information (PHI) in accordance with national and local subject
privacy regulations. Parental or guardian consent is required for subjects
who are less than 18 years of age or unable to give consent, or as
applicable per local laws. Subjects who are less than 18 years of age
may provide assent in addition to the parental/guardian consent, if
appropriate.
2. Subjects who have completed the studies 997HA301, 8HA02PED, or
other Phase 3 studies with rFVIIIFc.
Exclusion criteria
1. Confirmed positive high-titer inhibitor test (*5.00 BU/mL)
2. Current enrollement in any other study.
3. Inability to comply with study requirements.
4. Other unspecified reasons that, in the opinion of the Investigator or Biogen Idec, make the subject unsuitalbe for enrollment.
Design
Recruitment
Medical products/devices used
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 |
|---|---|
| EudraCT | EUCTR2011-003072-37-NL |
| CCMO | NL43600.042.13 |
| Other | NTC01454739 |