An Open-label Safety and Efficacy Study of Recombinant FVIII in Patients With Severe Hemophilia A

Phase 1

• Conditions: Hemophilia ASevere hemophilia A; MedDRA version: 20.0Level: LLTClassification code 10060613Term: Hemophilia A (Factor VIII)System Organ Class: 100000004850; Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

• Registration Number: EUCTR2013-003262-13-GB
• Lead Sponsor: CSL Behring GmbH

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2014-05-06
• Last Update Posted: 30 April 2018

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: Male
• Target Recruitment: 250

Inclusion Criteria

PTPs:
• Males of any age who have been diagnosed with severe congenital hemophilia A (FVIII activity levels < 1%) and who participated in a previous CSL-sponsored clinical study with rVIII-SingleChain.
• Males 0 to <65 years age who have been diagnosed with severe congenital hemophilia A (FVIII activity levels < 1%), who have at least 50 EDs to any FVIII product, and who are not currently enrolled in a CSL-sponsored clinical study with rVIII-SingleChain.

PUPs:
• Males 0 to <18 years of who have been diagnosed with severe congenital hemophilia A (FVIII activity levels < 1%)
• No prior exposure to any Factor VIII product (with the exception of short-term use of blood products).

ITI substudy:
• PUPs who have developed a confirmed inhibitor to rVIII-SingleChain in the main study.
Are the trial subjects under 18? yes
Number of subjects for this age range: 143
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 131
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 1

Exclusion Criteria

• Known or suspected hypersensitivity to rVIII-SingleChain or to any excipients of rVIII-SingleChain or Chinese hamster ovary (CHO) proteins.
• Currently receiving a therapy not permitted during the study.
• Serum creatinine > 2 x upper limit of normal, alanine aminotransferase or aspartate aminotransferase > 5 x upper limit of normal at Screening (if specified)
• Any first-order family (eg, siblings) history of FVIII inhibitors
• For PTPs not rolling over directly from a CSL-sponsored clinical study with rVIII-SingleChain: any history of or current FVIII inhibitors

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified