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

Completed · Phase 3

Conditions studied: Congenital Hemophilia A

In brief

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.

Key facts

Study ID

NCT02093897

Run by

CSL Behring

People needed

84

Starts

2014-03-01

Expected to finish

2015-08-01

Last updated by the study team

2017-01-27

Who can join

Age: any, up to 11. Sex: male. Healthy volunteers: not accepted.

You may qualify if…

• 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.

You may not qualify if…

• 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.

Where it is running

• 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

Full record on ClinicalTrials.gov

Trial information comes from ClinicalTrials.gov and is refreshed daily. TrialsForMe does not provide medical care and does not run the studies it lists.