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

• Register: EUCTR
• Last refreshed on: 19 April 2022
• Main ID: EUCTR2013-003262-13-ES
• Date of registration: 09/06/2014
• Prospective Registration: Yes
• Primary sponsor: CSL Behring GmbH
• Public title: An Open-label Safety and Efficacy Study of Recombinant FVIII in Patients With Severe Hemophilia A
• Scientific title: A Phase III Open Label, Multicenter, Extension Study to Assess the Safety and Efficacy of Recombinant Coagulation Factor VIII (rVIII-SingleChain, CSL627) in Subjects with Severe Hemophilia A
• Date of first enrolment: 30/06/2014
• Target sample size: 200
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2013-003262-13
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: no; Randomised: no; Open: yes; Single blind: no; Double blind: no; Parallel group: no; Cross over: no; Other: no; If controlled, specify comparator, Other Medicinial Product: Placebo: Other:
• Phase: Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): no Therapeutic confirmatory - (Phase III): yes Therapeutic use (Phase IV): no

Countries of recruitment

Australia, Austria, Canada, Chile, Czech Republic, European Union, Georgia, Germany, Hungary, Ireland, Italy, Japan, Korea, Democratic People's Republic of, Lebanon, Malaysia, Netherlands, Philippines, Poland, Portugal, Russian Federation, South Africa, Spain, Switzerland, Thailand, Turkey, Ukraine, United Kingdom, United States

Contacts

• Name: Clin.Trial Registration Coordinator
• Affiliation: CSL Behring GmbH

• Name: Clin.Trial Registration Coordinator
• Affiliation: CSL Behring GmbH

Key inclusion & exclusion criteria

Inclusion criteria:
? Males of any age who have been diagnosed with severe hemophilia A (FVIII activity levels < 1%)
? Participated in a previous CSL-sponsored clinical study with rVIII-SingleChain. "
Are the trial subjects under 18? yes
Number of subjects for this age range: 75
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 124
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.


Age minimum:
Age maximum:
Gender:
Female: no
Male: yes

Health Condition(s) or Problem(s) studied

Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

Hemophilia A Severe hemophilia A
MedDRA version: 17.0 Level: LLT Classification code 10060613 Term: Hemophilia A (Factor VIII) System Organ Class: 100000004850

Intervention(s)

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 Sponsor code: CSL627
Other descriptive name: rVIII-SingleChain
Concentration unit: IU international unit(s)
Concentration type: equal
Concentration number: 250-

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 Sponsor code: CSL627
Other descriptive name: rVIII-SingleChain
Concentration unit: IU international unit(s)
Concentration type: equal
Concentration number: 500-

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 Sponsor code: CSL627
Other descriptive name: rVIII-SingleChain
Concentration unit: IU international unit(s)
Concentration type: equal
Concentration number: 1000-

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 Sponsor code: CSL627
Other descriptive name: rVIII-SingleChain
Concentration unit: IU international unit(s)
Concentration type: equal
Concentration number: 2000-

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 Sponsor code: CSL627
Other descriptive name: rVIII-SingleChain
Concentration unit: IU international unit(s)
Concentration type: equal
Concentration number

Primary Outcome(s)

Main Objective: The primary objective of this study is to evaluate the safety of long term use of rVIII-SingleChain

Timepoint(s) of evaluation of this end point: At the closest visit after 100 EDs (up to three years).

Secondary Objective: The secondary objectives of the study are:
? To measure the incidence rate of inhibitor formation to FVIII after 10 EDs and after 50 EDs
? To collect and evaluate additional efficacy information on the prophylaxis and treatment of bleeding events
? To assess the hemostatic efficacy of rVIII-SingleChain for subjects who undergo surgery
? To characterize the safety profile of rVIII-SingleChain

Primary end point(s): The primary outcome measure is the incidence rate of inhibitor formation to FVIII over 100 EDs.

Secondary Outcome(s)

Secondary end point(s): ? Treatment success: Rate of treatment success where treatment success of a bleeding episode is defined as a rating of "excellent" or "good" on the investigator's clinical assessment of hemostatic efficacy 4-point scale "excellent, good, moderate or poor/no response".
? Annualized bleeding rate: The annualized bleeding rate for subjects taking prophylaxis and on-demand treatment regimens
? The proportion of bleeding episodes requiring 1, 2, 3, or > 3 infusions of rVIII-SingleChain to achieve hemostasis
? Consumption of rVIII-SingleChain - infusions: The number of rVIII-SingleChain infusions per month and per year
? Consumption of rVIII-SingleChain - IU/kg: rVIII-SingleChain consumption (IU/kg) per month and per year, and per event during prophylaxis, on-demand, and surgery.
? Hemostatic efficacy of rVIII-SingleChain for subjects who undergo surgery: The investigator will rate the efficacy of the rVIII-SingleChain treatment during surgery based on a hemostatic efficacy four point rating scale of "excellent, good, moderate or poor/no response".
? The incidence rate of inhibitor formation to FVIII

Timepoint(s) of evaluation of this end point: For secondary end points 1 to 5: Up to three years
For secondary end point 6: From the start of surgery through the post-operative recovery (generally up to 14 days after surgery)
For secondary end point 7: After 10 and after 50 exposure days

Secondary ID(s)

2013-003262-13-IT

CSL627_3001

Source(s) of Monetary Support

CSL Behring GmbH

Ethics review

Status: Approved
Approval date: 05/06/2014
Contact:

Results

• Results available: Yes
• Date Posted: 22/07/2021
• Date Completed: 19/01/2021
• URL: https://www.clinicaltrialsregister.eu/ctr-search/trial/2013-003262-13/results