Pharmacokinetic, Efficacy, and Safety Study of Recombinant Factor VIII Single Chain (rVIII-SingleChain) in Children With Severe Hemophilia A

NCT ID: NCT02093897

Last Updated: 2017-01-27

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE3
• Total Enrollment: 84 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2014-03-31
• Study Completion Date: 2015-08-31

Brief 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 < 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 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.

Conditions

Congenital Hemophilia A

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

rVIII-SingleChain

Subjects will be assigned to either an on-demand or prophylaxis regimen and will receive rVIII-SingleChain as an intravenous (IV) infusion. Subjects assigned to a prophylaxis regimen will be treated with 15 to 50 IU/kg of rVIII-SingleChain every second day or 2 to 3 times per week, or at the investigator's discretion, based on available PK data, the FVIII treatment regimen used before enrollment and/or the subject's bleeding phenotype. The dose for on-demand treatment of a bleeding episode is based on the recommendations of the World Federation of Hemophilia (WFH), with a minimum dose of 15 IU/kg. All subjects were to be treated for a minimum of 50 EDs. For the PK evaluation, the subjects will receive a single IV dose of 50 IU/kg of rVIII-SingleChain on Day 1 at the start of the PK evaluation period.

Group Type: EXPERIMENTAL

rVIII-SingleChain

Intervention Type: BIOLOGICAL

Interventions

rVIII-SingleChain

Intervention Type: BIOLOGICAL

Other Intervention Names

Recombinant Factor VIII single chain; CSL627

Eligibility Criteria

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.

• Maximum Eligible Age: 11 Years
• Eligible Sex: MALE
• Accepts Healthy Volunteers: No

Sponsors

CSL Behring

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Program Director

Role: STUDY_DIRECTOR

CSL Behring

Locations

Study Site

Aurora, Colorado, United States

Study Site

Chicago, Illinois, United States

Study Site

Melbourne, Victoria, Australia

Study Site

Linz, , Austria

Study Site

Vienna, , Austria

Study Site

Brest, , France

Study Site

Le Kremlin-Bicêtre, , France

Study Site

Lille, , France

Study Site

Nantes, , France

Study Site

Paris, , France

Study Site

Tbilisi, , Georgia

Study Site

Bonn, , Germany

Study Site

Bremen, , Germany

Study Site

Frankfurt am Main, , Germany

Study Site

Hanover, , Germany

Study Site

Milan, , Italy

Study Site

Beirut, , Lebanon

Study Site

Kuala Lumpur, , Malaysia

Study Site

Amsterdam, , Netherlands

Study Site

Njmegen, , Netherlands

Study Site

Utrecht, , Netherlands

Study Site

Cebu City, , Philippines

Study Site

Davao City, , Philippines

Study Site

Rzeszów, , Poland

Study Site

Porto, , Portugal

Study Site

Timișoara, , Romania

Study Site

Madrid, , Spain

Study Site

Lucerne, , Switzerland

Study Site

Bangkok, , Thailand

Study Site

Bangkok, , Thailand

Study Site

Chiang Mai, , Thailand

Study Site

Khon Kaen, , Thailand

Study Site

Songkhla, , Thailand

Study Site

Adana, , Turkey (Türkiye)

Study Site

Istanbul, , Turkey (Türkiye)

Study Site

Izmir, , Turkey (Türkiye)

Study Site

Lviv, , Ukraine

Countries

United States; Australia; Austria; France; Georgia; Germany; Italy; Lebanon; Malaysia; Netherlands; Philippines; Poland; Portugal; Romania; Spain; Switzerland; Thailand; Turkey (Türkiye); Ukraine

Other Identifiers

2012-001336-65

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

CSL627_3002

Identifier Type: -

Identifier Source: org_study_id