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

NCT ID: NCT02172950

Last Updated: 2021-10-27

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE3
• Total Enrollment: 246 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2014-10-13
• Study Completion Date: 2021-01-19

Brief Summary

This multicenter, open-label, phase 3 extension study will investigate the safety and efficacy of rVIII-SingleChain for prophylaxis and on-demand treatment of bleeding episodes in at least 200 previously treated patients (PTPs) with severe congenital hemophilia A and previous exposure to FVIII products who achieve at least 100 exposure days (EDs) to rVIII-SingleChain in this study, as well as in previously untreated patients (PUPs) with no previous exposure to any FVIII product who achieve at least 50 EDs to rVIII-SingleChain in this study. A substudy (open to both PTPs and PUPs) will investigate the use of rVIII-SingleChain in surgery. A substudy (open to PUPs who develop an inhibitor to rVIII-SingleChain) will investigate the use of rVIII-SingleChain in immune tolerance induction (ITI) therapy.

Conditions

Hemophilia A; Severe Hemophilia A

Study Design

• Allocation Method: NON_RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Previously treated patients (PTPs)

The investigator will assign subjects to either prophylaxis or on-demand treatment regimens for rVIII-SingleChain by intravenous injection. The investigator will determine the rVIII-SingleChain dose and dosing schedule for the subject based upon the subject's pharmacokinetic (PK) profile, rVIII-SingleChain PK data, previous FVIII treatment regimen, and bleeding phenotype, if available.

Group Type: EXPERIMENTAL

rVIII-SingleChain

Intervention Type: BIOLOGICAL

Recombinant single-chain coagulation factor VIII

Previously untreated patients (PUPs)

The investigator will assign subjects to either prophylaxis or on-demand treatment regimens for rVIII-SingleChain by intravenous injection. The investigator will determine the rVIII-SingleChain dose and dosing schedule at their discretion, taking into consideration the World Federation of Hemophilia (WFH) guidelines, the type of bleeding episode, location of the bleeding, subject's age, and other disease characteristics.

Group Type: EXPERIMENTAL

rVIII-SingleChain

Intervention Type: BIOLOGICAL

Recombinant single-chain coagulation factor VIII

Interventions

rVIII-SingleChain

Recombinant single-chain coagulation factor VIII

Intervention Type: BIOLOGICAL

Other Intervention Names

CSL627

Eligibility Criteria

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.

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

• 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 8400213

San Diego, California, United States

Study Site 8400241

Aurora, Colorado, United States

Study Site 8400118

Hartford, Connecticut, United States

Study Site 8400116

Miami, Florida, United States

Study Site 8400184

Chicago, Illinois, United States

Study Site 8400204

New Orleans, Louisiana, United States

Study Site 8400240

Dallas, Texas, United States

Study Site 8400041

Houston, Texas, United States

Study Site 8400154

Milwaukee, Wisconsin, United States

Study Site 0360014

Melbourne, , Australia

Study Site 0360028

Nedlands, , Australia

Study Site 0360031

Perth, , Australia

Study Site 0400012

Graz, , Austria

Study Site 0400003

Linz, , Austria

Study Site 0400001

Vienna, , Austria

Study Site 0400002

Vienna, , Austria

Study Site 1240022

Saint John, , Canada

Study Site 2030017

Hradec Králové, , Czechia

Study Site 2500015

Brest, , France

Study Site 2500017

Le Kremlin-Bicêtre, , France

Study Site 2500028

Lille, , France

Study Site 2500018

Nantes, , France

Study Site 2500002

Paris, , France

Study Site 2680001

Tbilisi, , Georgia

Study Site 2760034

Bonn, , Germany

Study Site 2760091

Frankfurt, , Germany

Study Site 2760087

Giessen, , Germany

Study Site 2760066

Hanover, , Germany

Study Site 3480007

Debrecen, , Hungary

Study Site 3720002

Dublin, , Ireland

Study Site 3800023

Milan, , Italy

Study Site 3920031

Hyōgo, , Japan

Study Site 3920029

Nagoya, , Japan

Study Site 3920064

Okayama, , Japan

Study Site 3920033

Saitama, , Japan

Study Site 3920025

Tokyo, , Japan

Study Site 4220007

Beirut, , Lebanon

Study Site 4580001

Kuala Lumpur, , Malaysia

Study Site 5280006

Amsterdam-Zuidoost, , Netherlands

Study Site 5280008

Nijmegen, , Netherlands

Study Site 5280007

Utrecht, , Netherlands

Study Site 6080001

Cebu City, , Philippines

Study Site 6080002

Davao City, , Philippines

Study Site 6160013

Gdansk, , Poland

Study Site 6160038

Krakow, , Poland

Study Site 6160035

Rzeszów, , Poland

Study Site 6160014

Wroclaw, , Poland

Study Site 6200001

Porto, , Portugal

Study Site 6420030

Bucharest, , Romania

Study Site 6420037

Timișoara, , Romania

Study Site 7100001

Parktown, , South Africa

Study Site 7240008

A Coruña, , Spain

Study Site 7240021

Barcelona, , Spain

Study Site 7240007

Madrid, , Spain

Study Site 7240023

Valencia, , Spain

Study Site 7560010

Lucerne, , Switzerland

Study Site 7640001

Bangkok, , Thailand

Study Site 7640005

Bangkok, , Thailand

Study Site 7640002

Chiang Mai, , Thailand

Study Site 7640004

Khon Kaen, , Thailand

Study Site 7640003

Songkhla, , Thailand

Study Site 8040007

Dnipropetrovsk, , Ukraine

Study Site 8040005

Lviv, , Ukraine

Study Site 8260008

London, , United Kingdom

Countries

Turkey (Türkiye); United States; Australia; Austria; Canada; Czechia; France; Georgia; Germany; Hungary; Ireland; Italy; Japan; Lebanon; Malaysia; Netherlands; Philippines; Poland; Portugal; Romania; South Africa; Spain; Switzerland; Thailand; Ukraine; United Kingdom

Provided Documents

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

2013-003262-13

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

CSL627_3001

Identifier Type: -

Identifier Source: org_study_id