Pharmacokinetic, Efficacy, and Safety Study of Recombinant Factor VIII Single Chain (rVIII-SingleChain) in Children With Severe Hemophilia A
| Status: | Completed |
|---|---|
| Conditions: | Hematology |
| Therapuetic Areas: | Hematology |
| Healthy: | No |
| Age Range: | Any - 11 |
| Updated: | 4/21/2016 |
| Start Date: | March 2014 |
| End Date: | August 2015 |
A Phase III Open-label Pharmacokinetic, Efficacy and Safety Study of rVIII-SingleChain in a Pediatric Population With Severe Hemophilia A
This is an international, multicenter, open-label study to assess the efficacy, safety, and
pharmacokinetic (PK) profile of rVIII-SingleChain in pediatric patients with severe
hemophilia A. A minimum of 25 previously treated subjects ≥ 6 to < 12 years of age and at
least 25 subjects < 6 years of age who have undergone > 50 exposure days (EDs) with a
previous Factor VIII (FVIII) product are planned to be enrolled. Subjects will be assigned
to either an on-demand or prophylaxis treatment regimen for the treatment of bleeding
episodes and will receive rVIII-SingleChain at a dose to be determined by the investigator.
Hemostatic efficacy will be assessed by the subject/caregiver and the investigator who will
assess overall efficacy by a 4-point scale.
pharmacokinetic (PK) profile of rVIII-SingleChain in pediatric patients with severe
hemophilia A. A minimum of 25 previously treated subjects ≥ 6 to < 12 years of age and at
least 25 subjects < 6 years of age who have undergone > 50 exposure days (EDs) with a
previous Factor VIII (FVIII) product are planned to be enrolled. Subjects will be assigned
to either an on-demand or prophylaxis treatment regimen for the treatment of bleeding
episodes and will receive rVIII-SingleChain at a dose to be determined by the investigator.
Hemostatic efficacy will be assessed by the subject/caregiver and the investigator who will
assess overall efficacy by a 4-point scale.
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
- Investigator believes that the subject is willing and able to adhere to all protocol
requirements. Investigator believes that the subject's parent(s) or legally
acceptable representative(s) is / are willing and able to adhere to all protocol
requirements.
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.
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