Observational Registry of NovoSeven® Used as On-demand Treatment of Bleeds in Patients With Haemophilia A and B With Inhibitors (ONE)

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Novo Nordisk A/S
ClinicalTrials.gov Identifier:
NCT00703911
First received: June 19, 2008
Last updated: August 6, 2014
Last verified: August 2014
Results First Received: July 29, 2011  
Study Type: Observational
Study Design: Time Perspective: Prospective
Conditions: Congenital Bleeding Disorder
Haemophilia A With Inhibitors
Haemophilia B With Inhibitors
Intervention: Drug: activated recombinant human factor VII

  Participant Flow
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Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
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Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
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Reporting Groups
  Description
Activated Recombinant Human Factor VII Male patients above 2 years of age with haemophilia A or B who have developed inhibitors and have been prescribed on-demand treatment of activated recombinant human factor VII at any dose for treatment of mild to moderate spontaneous bleeds

Participant Flow:   Overall Study
    Activated Recombinant Human Factor VII  
STARTED     102  
COMPLETED     92  
NOT COMPLETED     10  
Death                 1  
Physician Decision                 6  
Lost to Follow-up                 1  
Withdrawal by Subject                 2  



  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.
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Reporting Groups
  Description
Activated Recombinant Human Factor VII Male patients above 2 years of age with haemophilia A or B who have developed inhibitors and have been prescribed on-demand treatment of activated recombinant human factor VII at any dose for treatment of mild to moderate spontaneous bleeds

Baseline Measures
    Activated Recombinant Human Factor VII  
Number of Participants  
[units: participants]
  102  
Age  
[units: years]
Mean ± Standard Deviation
  23.3  ± 16.37  
Age, Customized  
[units: participants]
 
2 - 12 years     35  
above 12 - 17 years     14  
18 years or above     53  
Gender  
[units: participants]
 
Female     0  
Male     102  
Region of Enrollment  
[units: participants]
 
Algeria     17  
Austria     3  
Belgium     4  
Czech Republic     1  
France     16  
Germany     4  
Italy     16  
Netherlands     1  
Poland     9  
Portugal     1  
Saudi Arabia     2  
South Africa     8  
Sweden     1  
Turkey     6  
United Kingdom     7  
Venezuela     6  
Peak inhibitor titre  
[units: participants]
 
At least 5 Bethesda Units     91  
Less than 5 Bethesda Units     11  
Outcome of immune tolerance induction (ITI) treatment  
[units: participants]
 
None     66  
Unsuccessful     20  
Successful, with inhibitor recurrence     5  
Ongoing ITI     11  
Estimated number of bleeds for the 6 months preceding enrolment in registry  
[units: participants]
 
None     9  
1 - 6 (1 or less per month)     65  
6 - 12 (1 - 2 per month)     13  
12 - 18 (2 - 3 per month)     12  
18 (more than 3 per month)     2  
Data missing     1  
Primary treatment pattern for the 6 months preceding enrolment in registry  
[units: participants]
 
On-demand     88  
Prophylaxis     13  
Data missing     1  
Primary treatment type and dose for the 6 months preceding enrolment in registry  
[units: participants]
 
Multiple dose 90 mcg/kg NovoSeven®     44  
Single dose 270 mcg/kg NovoSeven®     28  
Other dose regimen NovoSeven®     17  
Other bypassing agent (e.g., FEIBA)     13  



  Outcome Measures
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1.  Primary:   Percentage of Bleed Treatments Resulting in Effective Bleed Resolution (All Bleed Episodes)   [ Time Frame: within 9 hours of first injection ]

2.  Primary:   Percentage of Bleed Treatments Resulting in Effective Bleed Resolution (Spontaneous Bleed Episodes)   [ Time Frame: within 9 hours of first injection ]

3.  Primary:   Percentage of Bleed Treatments Resulting in Effective Pain Relief (All Bleed Episodes)   [ Time Frame: within 9 hours of first injection ]

4.  Primary:   Percentage of Bleed Treatments Resulting in Effective Pain Relief (Spontaneous Bleed Episodes)   [ Time Frame: within 9 hours of first injection ]

5.  Secondary:   Percentage of Bleed Treatments Resulting in Effective Haemostasis (Cessation of Bleeds) by Time Point (All Bleed Episodes)   [ Time Frame: 1 hour, 3 hours and 6 hours, respectively, after first injection ]

6.  Secondary:   Percentage of Bleed Treatments Resulting in Effective Haemostasis (Cessation of Bleeds) by Time Point (Spontaneous Bleed Episodes)   [ Time Frame: 1 hour, 3 hours and 6 hours, respectively, after first injection ]

7.  Secondary:   Percentage of Bleed Treatments Resulting in Effective Pain Relief by Time Point (All Bleed Episodes)   [ Time Frame: 1 hour, 3 hours and 6 hours, respectively, after first injection ]

8.  Secondary:   Percentage of Bleed Treatments Resulting in Effective Pain Relief by Time Point (Spontaneous Bleed Episodes)   [ Time Frame: 1 hour, 3 hours and 6 hours, respectively, after first injection ]

9.  Secondary:   Total Number of Injections (All Bleed Episodes)   [ Time Frame: individual bleed episode ]

10.  Secondary:   Total Number of Injections (Spontaneous Bleed Episodes)   [ Time Frame: individual bleed episode ]

11.  Secondary:   Total Exposure (Cumulative Dose) to Activated Recombinant Human Factor VII (All Bleed Episodes)   [ Time Frame: individual bleed episode ]

12.  Secondary:   Total Exposure (Cumulative Dose) to Activated Recombinant Human Factor VII (Spontaneous Bleed Episodes)   [ Time Frame: individual bleed episode ]

13.  Secondary:   Percentage of Patients Reporting Satisfaction With Symptom Relief (All Bleed Episodes)   [ Time Frame: duration of bleed episode ]

14.  Secondary:   Percentage of Bleed Treatments Resulting in Patient Satisfaction With Symptom Relief (Spontaneous Bleed Episodes)   [ Time Frame: duration of bleed episode ]

15.  Secondary:   Percentage of Bleed Treatments Resulting in Patient Satisfaction With Ease of Use (All Bleed Episodes)   [ Time Frame: duration of bleed episode ]

16.  Secondary:   Percentage of Bleed Treatments Resulting in Patient Satisfaction With Ease of Use (Spontaneous Bleed Episodes)   [ Time Frame: duration of bleed episode ]

17.  Secondary:   Overall Time to Cessation of Bleed/Achievement of Haemostasis (All Bleed Episodes)   [ Time Frame: duration of bleed episode ]

18.  Secondary:   Overall Time to Cessation of Bleed/Achievement of Haemostasis (Spontaneous Bleed Episodes)   [ Time Frame: duration of bleed episode ]

19.  Secondary:   Overall Time to Cessation/Achievement of Haemostasis (Spontaneous Bleed Episodes)   [ Time Frame: duration of bleed episode ]

20.  Secondary:   Childrens' Health Related Quality of Life (Haemo-QoL): Overall Score   [ Time Frame: Baseline (week 0) and and registry discontinuation (up to 28 months) ]

21.  Secondary:   Adults' Health Related Quality of Life (Haemo-QoL-A): Overall Score   [ Time Frame: Baseline (week 0) and and registry discontinuation (up to 28 months) ]

22.  Other Pre-specified:   Percentage of Bleed Treatments Resulting in Effective Haemostasis (Cessation of Bleed) by Dose Level (All Bleed Episodes)   [ Time Frame: within 9 hours after first injection ]

23.  Other Pre-specified:   Percentage of Bleed Treatments Resulting in Effective Haemostasis (Cessation of Bleed) by Dose Level (Spontaneous Bleed Episodes)   [ Time Frame: within 9 hours after first injection ]

24.  Other Pre-specified:   Percentage of Bleed Treatments Resulting in Effective Pain Relief by Initial Dose (All Bleed Episodes)   [ Time Frame: within 9 hours after first injection ]

25.  Other Pre-specified:   Percentage of Bleed Treatments Resulting in Effective Pain Relief by Initial Dose (Spontaneous Bleed Episodes)   [ Time Frame: within 9 hours after first injection ]


  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: Public Access to Clinical Trials
Organization: Novo Nordisk A/S
e-mail: clinicaltrials@novonordisk.com


No publications provided


Responsible Party: Novo Nordisk A/S
ClinicalTrials.gov Identifier: NCT00703911     History of Changes
Other Study ID Numbers: F7HAEM-3507
Study First Received: June 19, 2008
Results First Received: July 29, 2011
Last Updated: August 6, 2014
Health Authority: Algeria: Ministry of Health
Austria: The Austrian Agency for Health and Food Safety (AGES)
Belgium: Federal Agency for Medicines and Healthcare Products
Czech Republic: State Institute for Drug Control
France: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis)
Germany: Federal Institute for Drugs and Medicinal Devices (BfarM)
Italy: AIFA, National Medicines Agency
Netherlands: The Central Committee on Research Involving Human Subjects (CCMO)
Poland: The Office for Reg. of Medicinal Products, Medical Devices and Biocidal Products - Central Register of Clinical Trials
Portugal: INFARMED
Saudi Arabia: Ministry of Health
South Africa: Medicines Control Council
Sweden: Medical Products Agency
Turkey: Ministry of Health Drug and Pharmaceutical Department
United Kingdom: Medicines and Healthcare Regulatory Authority (MHRA)
Venezuela: Ministry of Health