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An Open-label Safety, Efficacy and Pharmacokinetic Study of a Recombinant FVIII Compared to Recombinant Human Antihemophilic FVIII in Patients With Severe Hemophilia A

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
CSL Behring
ClinicalTrials.gov Identifier:
NCT01486927
First received: November 19, 2011
Last updated: June 24, 2016
Last verified: June 2016
Results First Received: June 24, 2016  
Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment;   Masking: Open Label;   Primary Purpose: Treatment
Condition: Hemophilia A
Interventions: Biological: rVIII-SingleChain
Biological: Octocog alfa

  Participant Flow
  Hide Participant Flow

Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
This multicenter, multinational study enrolled subjects at 54 participating study centers in the United States, Japan, Europe, Australia, Canada, Lebanon, Malaysia, Philippines, Russian Federation, South Africa, and Ukraine.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
Screening took place 4 to 28 days prior to first dose of study product (rVIII-SingleChain). A total of 204 subjects were screened, 29 of these did not fulfill all eligibility criteria and were therefore screening failures. A total of 175 subjects were enrolled; 174 subjects were exposed to treatment with rVIII-SingleChain.

Reporting Groups
  Description
Recombinant Factor VIII (rFVIII) In Part 1 of the study (a single-sequence crossover pharmacokinetic [PK] analysis), 27 subjects received a single injection of octocog alfa followed by a single injection of rVIII-SingleChain. Twenty-six of the 27 subjects from Part 1 then entered Part 2 of the study where they were assigned either to an on-demand or prophylaxis regimen with repeat injections of rVIII-SingleChain until they reached 50 exposure days (EDs). In Part 3 of the study, 148 additional subjects were enrolled and were assigned either to an on-demand or prophylaxis regimen with repeat injections of rVIII-SingleChain until they reached 50 EDs; 64 of these subjects participated in additional PK analyses. Overall, 174 subjects received rVIII-SingleChain as either on-demand or prophylaxis regimens, and 13 subjects participated in the surgical substudy.

Participant Flow:   Overall Study
    Recombinant Factor VIII (rFVIII)
STARTED   174 
COMPLETED   161 
NOT COMPLETED   13 
Knee surgery (prior to surgery substudy)                1 
Physician Decision                1 
50 Exposure days not reached                2 
Withdrawal by Subject                8 
Subject did not reach 6 mo of treatment                1 



  Baseline Characteristics
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Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
No text entered.

Reporting Groups
  Description
Recombinant Factor VIII (rFVIII) In Part 1 of the study (a single-sequence crossover PK analysis), 27 subjects received a single injection of octocog alfa followed by a single injection of rVIII-SingleChain. Twenty-six of the 27 subjects from Part 1 then entered Part 2 of the study where they were assigned either to an on-demand or prophylaxis regimen with repeat injections of rVIII-SingleChain until they reached 50 EDs. In Part 3 of the study, 148 additional subjects were enrolled and were assigned either to an on-demand or prophylaxis regimen with repeat injections of rVIII-SingleChain until they reached 50 EDs; 64 of these subjects participated in additional PK analyses. Overall, 174 subjects received rVIII-SingleChain as either on-demand or prophylaxis regimens, and 13 subjects participated in the surgical substudy.

Baseline Measures
   Recombinant Factor VIII (rFVIII) 
Overall Participants Analyzed 
[Units: Participants]
 174 
Age 
[Units: Years]
Mean (Standard Deviation)
 31.3  (11.77) 
Age, Customized 
[Units: Participants]
 
Adolescents (12-17 years)   14 
Adults (18-65 years)   160 
Gender 
[Units: Participants]
 
Female   0 
Male   174 
Type of FVIII product used before enrollment [1] 
[Units: Participants]
 
Plasma-derived Product   83 
Recombinant Product   91 
[1] Type of FVIII product used by the subjects before enrollment into the study. This could have been a plasma-derived FVIII product or a recombinant FVIII product.
Treatment modality of FVIII therapy before enrollment [1] 
[Units: Participants]
 
Prophylaxis   82 
On-demand   92 
[1] Treatment modality of FVIII therapy before enrollment, ie, routine prophylaxis or on-demand treatment. If a subject used both modalities, only the most recent one was counted.


  Outcome Measures
  Show All Outcome Measures

1.  Primary:   Treatment Success   [ Time Frame: Up to 24 months ]

2.  Primary:   Inhibitor Formation to FVIII   [ Time Frame: Up to 24 months ]

3.  Primary:   Annualized Spontaneous Bleeding Rate   [ Time Frame: Up to 24 months ]

4.  Primary:   Treatment Success During the Peri-operative Surgical Sub-study   [ Time Frame: From the start of surgery through the post-operative recovery (generally up to 14 days after surgery) ]

5.  Secondary:   AUC0-∞ (Part 1)   [ Time Frame: Before infusion and at up to 10 time points within 72 hours of infusion ]

6.  Secondary:   Cmax (Part 1)   [ Time Frame: Before infusion and at up to 10 time points within 72 hours of infusion ]

7.  Secondary:   Tmax (Part 1)   [ Time Frame: Before infusion and at up to 10 time points within 72 hours of infusion ]

8.  Secondary:   Half-life (t1/2) (Part 1)   [ Time Frame: Before infusion and at up to 10 time points within 72 hours of infusion. ]

9.  Secondary:   Mean Residence Time (MRT) (Part 1)   [ Time Frame: Before infusion and at up to 10 time points within 72 hours of infusion ]

10.  Secondary:   Clearance (Cl) (Part 1)   [ Time Frame: Before infusion and at up to 10 time points within 72 hours of infusion ]

11.  Secondary:   Volume of Distribution at Steady-state (Vss) (Part 1)   [ Time Frame: Before infusion and at up to 10 time points within 72 hours of infusion ]

12.  Secondary:   Incremental Recovery (Part 1)   [ Time Frame: At 30 minutes after infusion ]

13.  Secondary:   Annualized Bleeding Rate for Total Bleeds and Traumatic Bleeds   [ Time Frame: Up to 24 months ]

14.  Secondary:   Proportion of Bleeding Episodes Requiring 1, 2, 3 or > 3 Infusions of rVIII-SingleChain to Achieve Hemostasis   [ Time Frame: During the study (up to 24 months; assessed at Months 1, 2, 3, 4, 5, 6, 9, 12, 15, 18, 21 and 24) ]

15.  Other Pre-specified:   Incremental Recovery (Part 3)   [ Time Frame: At 30 minutes after infusion ]

16.  Other Pre-specified:   Volume of Distribution at Steady-state (Vss) (Part 3)   [ Time Frame: Before infusion and at up to 12 time points within 96 hours of infusion. ]

17.  Other Pre-specified:   Clearance (Cl) (Part 3)   [ Time Frame: Before infusion and at up to 12 time points within 96 hours of infusion. ]

18.  Other Pre-specified:   Mean Residence Time (MRT) (Part 3)   [ Time Frame: Before infusion and at up to 12 time points within 96 hours of infusion. ]

19.  Other Pre-specified:   Half-life (t1/2) (Part 3)   [ Time Frame: Before infusion and at up to 12 time points within 96 hours of infusion. ]

20.  Other Pre-specified:   Tmax (Part 3)   [ Time Frame: Before infusion and at up to 12 time points within 96 hours of infusion. ]

21.  Other Pre-specified:   Cmax (Part 3)   [ Time Frame: Before infusion and at up to 12 time points within 96 hours of infusion ]

22.  Other Pre-specified:   AUC0-∞ (Part 3)   [ Time Frame: Before infusion and at up to 12 time points within 96 hours of infusion ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats
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Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
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  More Information
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Certain Agreements:  
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
The agreement is:
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is less than or equal to 60 days. The sponsor cannot require changes to the communication and cannot extend the embargo.
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is more than 60 days but less than or equal to 180 days. The sponsor cannot require changes to the communication and cannot extend the embargo.


Results Point of Contact:  
Name/Title: Clinical Trial Disclosure Manager
Organization: CSL Behring
phone: Use email contact
e-mail: clinicaltrials@cslbehring.com


Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

Responsible Party: CSL Behring
ClinicalTrials.gov Identifier: NCT01486927     History of Changes
Other Study ID Numbers: CSL627_1001
2011-002393-23 ( EudraCT Number )
Study First Received: November 19, 2011
Results First Received: June 24, 2016
Last Updated: June 24, 2016