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

ClinicalTrials.gov Identifier:

NCT00703911

First received: June 19, 2008

Last updated: December 3, 2012

Last verified: December 2012

History of Changes

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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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 ]

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2. Primary:

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

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3. Primary:

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

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4. Primary:

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

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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 ]

Show Outcome Measure 5

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 ]

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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 ]

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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 ]

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9. Secondary:

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

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10. Secondary:

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

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11. Secondary:

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

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12. Secondary:

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

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13. Secondary:

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

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14. Secondary:

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

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15. Secondary:

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

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16. Secondary:

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

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17. Secondary:

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

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18. Secondary:

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

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19. Secondary:

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

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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) ]

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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) ]

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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 ]

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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 ]

Show Outcome Measure 23

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 ]

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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 ]

Show Outcome Measure 25

Serious Adverse Events

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Other Adverse Events

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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:

	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.
	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.
	Other disclosure agreement that restricts the right of the PI to discuss or publish trial results after the trial is completed. Restriction Description: If an investigator wishes to publish information from the study, a copy of the manuscript must be provided to Novo Nordisk for review at least 60 days before submission for publication or presentation. The investigator will recognize the integrity of a multi-centre study by not publishing data derived from the individual site until the combined results from the completed registry have been published in full, or the sponsor confirms there will be no multi-centre registry publication.

Limitations and Caveats

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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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
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:	December 3, 2012
Health Authority:	Poland: The Office for Reg. of Medicinal Products, Medical Devices and Biocidal Products - Central Register of Clinical Trials Netherlands: The Central Committee on Research Involving Human Subjects (CCMO) 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 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

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