Congenital hemophilia A MedDRA version: 17.0 Level: LLT Classification code 10060613 Term: Hemophilia A (Factor VIII) System Organ Class: 100000004850
Conditions
Interventions
Product Name: rVIII-SingleChain
Product Code: CSL627
Pharmaceutical Form: Powder and solvent for solution for injection/infusion
INN or Proposed INN: Lonoctocog alfa
CAS Number: 1388129-63-2
Current S
Sponsors
CSL Behring GmbH
Eligibility
Sex/Gender
Male
Inclusion 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. Are the trial subjects under 18? yes Number of subjects for this age range: 75 F.1.2 Adults (18-64 years) no F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main 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.;Secondary Objective: • 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 • To evaluate the pharmacokinetic (PK) profile of rVIII-SingleChain • To assess the rate of inhibitor formation to rVIII-SingleChain • To assess the safety of rVIII-SingleChain with regard to adverse events (AEs), laboratory parameters, physical examination, and vital signs (blood pressure, heart rate, temperature, and respiratory rate) ;Primary end point(s): Treatment success ;Timepoint(s) of evaluation of this end point: Up to two years | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): • Annualized bleeding rate • Proportion of bleeding episodes requiring 1, 2, 3, or more than 3 infusions of rVIII-SingleChain to achieve hemostasis. • Consumption of rVIII-SingleChain • The number of subjects with inhibitor formation to rVIII-SingleChain PK: • Incremental recovery • Half-life (t1/2) of rVIII-SingleChain • Area under the concentration curve (AUC) • Clearance (Cl) of rVIII-SingleChain ;Timepoint(s) of evaluation of this end point: Up to two years; for PK endpoints, 0 to 2 days after infusion. | — |
Countries
Australia, Austria, Chile, European Union, France, Georgia, Hungary, Ireland, Korea, Democratic People's Republic of, Malaysia, Netherlands, Philippines, Poland, Portugal, Russian Federation, South Africa, Spain, Switzerland, Thailand, Turkey, Ukraine, United States
Contacts
Public ContactClin.Trial Registration Coordinator
CSL Behring GmbH
Outcome results
None listed