To evaluate the efficacy of rVIII-SingleChain in the treatment of major andminor bleeding events based on the investigator*s 4-point assessment scale
ID
Source
Brief title
Condition
- Blood and lymphatic system disorders congenital
Synonym
Research involving
Sponsors and support
Intervention
Outcome measures
Primary outcome
The primary objective of the study is:
* To evaluate the efficacy of rVIII-SingleChain in the treatment of major and
minor bleeding events based on the investigator*s 4-point assessment scale
Secondary outcome
The secondary objectives of the study are:
* To evaluate the annualized bleeding rate during prophylaxis treatment
* To evaluate the annualized bleeding rate during on-demand treatment
* To evaluate the proportion of bleeding episodes requiring 1, 2, 3, or >3
infusions of rVIII-SingleChain to achieve hemostasis
* To evaluate the consumption of rVIII-SingleChain
Background summary
Hemophilia A is a rare but serious X-linked recessive bleeding disorder that
affects males and is
characterized by a deficiency of clotting factor VIII (FVIII). Individuals with
severe hemophilia often bleed spontaneously, that is without any known trauma
or other cause.
This bleeding is predominantly into the joints and muscle.
Study objective
To evaluate the efficacy of rVIII-SingleChain in the treatment of major and
minor bleeding events based on the investigator*s 4-point assessment scale
Study design
This is an international, multicenter, open-label study to assess the efficacy,
safety, and PK
profile of rVIII-SingleChain in pediatric subjects with severe hemophilia A
(FVIII
concentration <1%).
Intervention
rVIII-SingleChain is supplied as a lyophilized powder to be reconstituted with
diluent
(Sterile Water for Injection). After reconstitution, rVIII-SingleChain is
administered as an
IV infusion.
Study burden and risks
The associated benefit of treatment with rVIII-SingleChain outweighs any
potential risks for subjects participating in the study.
Emil-von-Behring-Str. 76
Marburg 35041
DE
Emil-von-Behring-Str. 76
Marburg 35041
DE
Listed location countries
Age
Inclusion criteria
* Diagnosis of severe hemophilia A defined as < 1% Factor VIII (FVIII) concentration (FVIII:C) documented in medical records, ;* Males <12 years of age,;* Subjects who have received > 50 EDs with a FVIII product, ;* Prior PK data (at least incremental recovery and half-life) from previous FVIII exposure for subjects participating in the PK part
Exclusion criteria
* Any history of or current FVIII inhibitors;* Use of an Investigational Medical Product (IMP) within 30 days prior to the first rVIII-SingleChain administration, ;* Administration of any cryoprecipitate, whole blood or plasma within 30 days prior to administration of rVIII-SingleChain, ;* Known hypersensitivity (allergic reaction or anaphylaxis) to any FVIII product or hamster protein, ;* Subject currently receiving IV immunomodulating agents such as immunoglobulin or chronic systemic corticosteroid treatment, ;* Subject with serum aspartate aminotransferase (AST) or serum alanine aminotransferase (ALT) values >5 times (x) the upper limit of normal (ULN) at Screening, ;* Subjects with serum creatinine values >2 x ULN at Screening, ;* Evidence of thrombosis, including deep vein thrombosis, stroke, pulmonary embolism, myocardial infarction and arterial embolus within 3 months before Day 1,;* Experienced life-threatening bleeding episode or had major surgery or an orthopedic surgical procedure during the 3 months before rVIII-SingleChain administration.;Participated in another interventional clinical study within 30days days before the first administration of rVIII-SingleChain or at any time during the study.;* Alcohol, drug or medication abuse within 1 year before the study.;* Currently receiving a therapy not permitted during the study;* Known or suspected hypersensitivity to rVIII-SingleChain or to any excipients of rVIII-SingleChain;* Any issue that, in the opinion of the investigator, would render the subject unsuitable for participation in the study.
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 | EUCTR2012-001336-65-NL |
| CCMO | NL47150.018.13 |