Phase 3
N=84
Pharmacokinetic, Efficacy, and Safety Study of Recombinant Factor VIII Single Chain (rVIII-SingleChain) in Children With Severe Hemophilia A
Congenital Hemophilia A
Bottom Line
View on ClinicalTrials.gov: NCT02093897 ↗Enrolled (actual)
84
Serious AEs
10.7%
Results posted
Jan 2017
Primary outcome: Primary: Treatment Success — 96.3 Percentage of treated bleeding events
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- rVIII-SingleChain (Biological)
- Age
- Pediatric
- Sex
- Male
- Sponsor
- CSL Behring
- Primary completion
- Aug 2015
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Treatment Success |
96.3 | — |
| SECONDARY Annualized Bleeding Rate |
78.56; 3.69 | — |
| SECONDARY Percentage of Bleeding Episodes Requiring 1, 2, 3, or More Than 3 Infusions of rVIII-SingleChain to Achieve Hemostasis. |
85.9; 9.8; 2.3; 2.0 | — |
| SECONDARY Consumption of rVIII-SingleChain - IU/kg Per Subject Per Month |
202; 378 | — |
| SECONDARY Consumption of rVIII-SingleChain - IU/kg Per Subject Per Year |
2429; 4541 | — |
| SECONDARY Consumption of rVIII-SingleChain - IU/kg Per Bleeding Event |
25.9; 37.0 | — |
| SECONDARY Consumption of rVIII-SingleChain (On-demand Regimen) - Number of Infusions Per Subject Per Month |
7.58 | — |
| SECONDARY Consumption of rVIII-SingleChain (On-demand Regimen) - Number of Infusions Per Subject Per Year |
90.95 | — |
| SECONDARY Incremental Recovery |
1.63 | — |
| SECONDARY Half-life (t1/2) of rVIII-SingleChain |
10.3 | — |
| SECONDARY Area Under the Concentration Curve (AUC) |
1050 | — |
| SECONDARY Clearance (Cl) of rVIII-SingleChain |
4.86 | — |
| SECONDARY Number of Subjects With Inhibitor Formation to rVIII-SingleChain |
— | — |
Summary
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 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 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.
Eligibility Criteria
Inclusion Criteria
- Diagnosis of severe hemophilia A defined as 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.
Data sourced from ClinicalTrials.gov (NCT02093897). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.