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Trial record 1 of 1 for:    NCT01335061
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Study To Compare On-Demand Treatment To A Prophylaxis Regimen Of BeneFIX In Subjects With Moderately Severe to Severe Hemophilia B

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ClinicalTrials.gov Identifier: NCT01335061
Recruitment Status : Completed
First Posted : April 13, 2011
Results First Posted : May 4, 2015
Last Update Posted : June 8, 2015
Sponsor:
Information provided by (Responsible Party):
Pfizer

Study Type Interventional
Study Design Allocation: Non-Randomized;   Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Hemophilia B
Intervention Biological: Nonacog alfa
Enrollment 25
Recruitment Details Participation included 25 enrolled participants from 15 study centers and 9 countries.
Pre-assignment Details The duration of participation was approximately 86 weeks, consisting of a Screening period (1 day to 4 weeks), Period 1 (on-demand treatment for 26 weeks), Period 2 (prophylaxis therapy for 52 weeks), and a follow-up safety period (4 weeks).
Arm/Group Title All Participants
Hide Arm/Group Description The data for all the participants is presented.
Period Title: Overall Study
Started 25
Completed 25
Not Completed 0
Arm/Group Title All Participants
Hide Arm/Group Description The data for all the participants is presented.
Overall Number of Baseline Participants 25
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 25 participants
31.3  (12.6)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 25 participants
Female
0
   0.0%
Male
25
 100.0%
1.Primary Outcome
Title Annualized Number of Bleeding Episodes.
Hide Description The annualized bleed rate (ABR) or the annualized number of bleeding episodes per year, will be derived for each participant for each treatment period by using the following formula: ABR = number of bleeds / (Days on treatment period / 365.25) The number of bleeds for the ABR calculation includes all bleeds requiring treatment with factor IX product during the time on treatment.
Time Frame 2 years
Hide Outcome Measure Data
Hide Analysis Population Description
The efficacy analysis set (EAS) was used for the primary efficacy analyses with respect to ABR. It includes all participants who participated in at least one day of the routine prophylaxis period (ie, in the study through at least Visit 4).
Arm/Group Title On-Demand Therapy Prophylaxis Therapy
Hide Arm/Group Description:
Participants were treated for the bleeding events at the discretion of the study physician according to BeneFIX label.
The prophylaxis regimen of approximately 100 IU/kg once weekly was initiated at Visit 4.
Overall Number of Participants Analyzed 25 25
Mean (Standard Deviation)
Unit of Measure: Number of bleeds per year
32.9  (17.4) 3.6  (4.6)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection On-Demand Therapy, Prophylaxis Therapy
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.0001
Comments [Not Specified]
Method Paired t-test
Comments [Not Specified]
2.Secondary Outcome
Title Response to On-Demand Treatment for All Bleeding Episodes.
Hide Description Assessment scores on a 4-point Response Scale for an on-demand bleeding episode, as assessed by participant/caregiver or investigator/qualified staff. The 4-point scale assessments are Excellent, Good, Moderate or No response. Responses to number of observations were noted.
Time Frame 2 years
Hide Outcome Measure Data
Hide Analysis Population Description
The safety analysis set (SAS) was any participant who received at least one dose of BeneFIX, including the dose given during the enrollment visit (Visit 2) for the factor IX (FIX) recovery study. Follow-up infusion was only required for 18 participants.
Arm/Group Title On-Demand Therapy-First Infusion On-Demand Therapy-Follow-up Infusions
Hide Arm/Group Description:
Participants were treated for the bleeding events at the discretion of the study physician according to BeneFIX label.
Participants were treated for the bleeding events at the discretion of the study physician according to BeneFIX label.
Overall Number of Participants Analyzed 25 18
Overall Number of Units Analyzed
Type of Units Analyzed: Bleeds
 507 152
Measure Type: Number
Unit of Measure: Number of observations with response
Excellent 271 39
Good 177 80
Moderate 55 33
No response 3 0
Data not recorded 1 0
3.Secondary Outcome
Title Number of Nonacog Alfa, Recombinant Factor IX (BeneFIX) Infusions Used to Treat Each Bleeding Episode.
Hide Description The number of study drug infusions administered to treat a bleed will be calculated by adding the initial (on-demand) infusion to any subsequent (on-demand) infusions for the same bleed (same bleed start date/time). The number of infusions needed to treat a bleed will be classified into the following categories: 1, 2, 3, 4 and >4 infusions. If there were more than one bleed location (e.g., ankle and joint) with identical bleed start date and time, it was treated as one bleed occurrence.
Time Frame 2 years
Hide Outcome Measure Data
Hide Analysis Population Description
The safety analysis set (SAS) was any participant who received at least one dose of BeneFIX, including the dose given during the enrollment visit (Visit 2) for the factor IX (FIX) recovery study.
Arm/Group Title All Population
Hide Arm/Group Description:
The data for all the participants is presented.
Overall Number of Participants Analyzed 25
Overall Number of Units Analyzed
Type of Units Analyzed: Bleeds
 507
Measure Type: Number
Unit of Measure: Number of bleeds requiring infusion
Number of Infusion: 1 416
Number of Infusions: 2 69
Number of Infusions: 3 9
Number of Infusions: 4 3
Number of Infusions: >4 10
4.Secondary Outcome
Title Number of Breakthrough (Spontaneous/Non-Traumatic) Bleeds Within 48 Hours of a Prophylaxis Dose of BeneFIX.
Hide Description The number of spontaneous, non-traumatic breakthrough bleeds within 48 hours following a prophylaxis dose of BeneFIX were summarized. If there was more than one bleed location (eg, ankle and joint) with identical bleed start date and time, it was treated as one bleed occurrence.
Time Frame 2 years
Hide Outcome Measure Data
Hide Analysis Population Description
The safety analysis set (SAS) was any participant who received at least one dose of BeneFIX, including the dose given during the enrollment visit (Visit 2) for the factor IX (FIX) recovery study. Three participants experienced 1 spontaneous bleeding episode each within 48 hours of a previous prophylaxis infusion.
Arm/Group Title All Participants
Hide Arm/Group Description:
The data for all the participants is presented.
Overall Number of Participants Analyzed 25
Overall Number of Units Analyzed
Type of Units Analyzed: Breakthrough bleeds
 3
Mean (Standard Deviation)
Unit of Measure: Number of breakthrough bleeds
1.0  (0.0)
5.Secondary Outcome
Title Average Infusion Dose.
Hide Description The mean dose by per infusion by weight (IU/kg) was reported for both prophylaxis and on demand infusions
Time Frame 2 years
Hide Outcome Measure Data
Hide Analysis Population Description
The safety analysis set (SAS) was any participant who received at least one dose of BeneFIX, including the dose given during the enrollment visit (Visit 2) for the factor IX (FIX) recovery study.
Arm/Group Title On-Demand Therapy Prophylaxis Therapy
Hide Arm/Group Description:
Participants were treated for the bleeding events at the discretion of the study physician according to BeneFIX label.
The prophylaxis regimen of approximately 100IU/kg once weekly was initiated at Visit 4.
Overall Number of Participants Analyzed 25 25
Mean (Standard Deviation)
Unit of Measure: IU/Kg
52  (16) 99  (2)
6.Secondary Outcome
Title Total Factor Consumption.
Hide Description The total amount (IU) infused for each infusion recorded were summed to calculate the total factor consumption for each participant. For each infusion, IU/kg was calculated, using the most recently recorded weight measurement and the total factor consumption, divided by number of infusions, and was summarized similarly to average infusion dose (IU). Annualized TFC by weight was reported. Annualized TFC by weight = (Total IU/kg / treatment interval duration)*365.25.
Time Frame 2 years
Hide Outcome Measure Data
Hide Analysis Population Description
The safety analysis set (SAS) was any participant who received at least one dose of BeneFIX, including the dose given during the enrollment visit (Visit 2) for the factor IX (FIX) recovery study.
Arm/Group Title On-Demand Therapy Prophylaxis Therapy
Hide Arm/Group Description:
Participants were treated for the bleeding events at the discretion of the study physician according to BeneFIX label.
The prophylaxis regimen of approximately 100IU/kg once weekly was initiated at Visit 4.
Overall Number of Participants Analyzed 25 25
Mean (Standard Deviation)
Unit of Measure: IU/Kg
707  (519) 4985  (233)
7.Secondary Outcome
Title Incidence of Less Than Expected Therapeutic Effect (LETE)
Hide Description The following criteria are the definitions for LETE in this study: 1. LETE in the On-Demand Setting: LETE occurs in the on-demand setting if 2 successive "No Response" ratings are recorded after 2 successive BeneFIX drug infusions in the absence of confounding factors. 2. LETE in the Prophylaxis Setting: LETE occurs in the prophylaxis setting if there is a spontaneous bleed within 48 hours (≤ 48 hours) after a regularly scheduled prophylactic dose of BeneFIX in the absence of confounding factors. 3. LETE (Low Recovery): LETE can also be lower than expected recovery of FIX in the opinion of the investigator following infusion of BeneFIX in the absence of confounding factors. Each reported occurrence of low recovery LETE was listed.
Time Frame 2 years
Hide Outcome Measure Data
Hide Analysis Population Description
The safety analysis set (SAS) was any participant who received at least one dose of BeneFIX, including the dose given during the enrollment visit (Visit 2) for the factor IX (FIX) recovery study.
Arm/Group Title All Participants
Hide Arm/Group Description:
The data for all the participants is presented.
Overall Number of Participants Analyzed 25
Measure Type: Number
Unit of Measure: Percentage of occurence
LETE in On-Demand setting 0
LETE in prophylaxis setting 0
LETE (Low recovery) 0
Time Frame Until follow-up 82 weeks +/- 4 days.
Adverse Event Reporting Description Five participants experienced six SAEs since one participant experienced the same event twice (nephrolithiasis).
 
Arm/Group Title On-Demand Therapy Prophylaxis Therapy
Hide Arm/Group Description Participants were treated for the bleeding events at the discretion of the study physician according to BeneFIX label. The prophylaxis regimen of approximately 100IU/kg once weekly was initiated at Visit 4.
All-Cause Mortality
On-Demand Therapy Prophylaxis Therapy
Affected / at Risk (%) Affected / at Risk (%)
Total   --/--   --/-- 
Hide Serious Adverse Events
On-Demand Therapy Prophylaxis Therapy
Affected / at Risk (%) Affected / at Risk (%)
Total   1/25 (4.00%)   4/25 (16.00%) 
General disorders     
Pain * 1  0/25 (0.00%)  1/25 (4.00%) 
Infections and infestations     
Varicella * 1  0/25 (0.00%)  1/25 (4.00%) 
Investigations     
Blood pressure decreased * 1  1/25 (4.00%)  0/25 (0.00%) 
Neoplasms benign, malignant and unspecified (incl cysts and polyps)     
Lipoma * 1  0/25 (0.00%)  1/25 (4.00%) 
Renal and urinary disorders     
Nephrolithiasis * 1  0/25 (0.00%)  1/25 (4.00%) 
*
Indicates events were collected by non-systematic assessment
1
Term from vocabulary, MedDRA version 17.0
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
On-Demand Therapy Prophylaxis Therapy
Affected / at Risk (%) Affected / at Risk (%)
Total   16/25 (64.00%)   20/25 (80.00%) 
Gastrointestinal disorders     
Toothache * 1  3/25 (12.00%)  5/25 (20.00%) 
Gastritis * 1  1/25 (4.00%)  2/25 (8.00%) 
Abdominal pain upper * 1  1/25 (4.00%)  2/25 (8.00%) 
General disorders     
Pyrexia * 1  4/25 (16.00%)  4/25 (16.00%) 
Local swelling * 1  1/25 (4.00%)  3/25 (12.00%) 
Infections and infestations     
Upper respiratory tract infection * 1  4/25 (16.00%)  5/25 (20.00%) 
Pharyngitis * 1  2/25 (8.00%)  3/25 (12.00%) 
Nasopharyngitis * 1  3/25 (12.00%)  1/25 (4.00%) 
Influenza * 1  2/25 (8.00%)  1/25 (4.00%) 
Injury, poisoning and procedural complications     
Inappropriate schedule of drug administration * 1  0/25 (0.00%)  6/25 (24.00%) 
Drug dose omission * 1  0/25 (0.00%)  4/25 (16.00%) 
Medication error * 1  0/25 (0.00%)  3/25 (12.00%) 
Underdose * 1  0/25 (0.00%)  3/25 (12.00%) 
Wrong dose administration * 1  0/25 (0.00%)  3/25 (12.00%) 
Head injury * 1  0/25 (0.00%)  2/25 (8.00%) 
Musculoskeletal and connective tissue disorders     
Arthralgia * 1  5/25 (20.00%)  5/25 (20.00%) 
Joint swelling * 1  3/25 (12.00%)  2/25 (8.00%) 
Back pain * 1  1/25 (4.00%)  3/25 (12.00%) 
Neck pain * 1  0/25 (0.00%)  2/25 (8.00%) 
Pain in extremity * 1  2/25 (8.00%)  1/25 (4.00%) 
Nervous system disorders     
Headache * 1  8/25 (32.00%)  4/25 (16.00%) 
Respiratory, thoracic and mediastinal disorders     
Cough * 1  0/25 (0.00%)  2/25 (8.00%) 
*
Indicates events were collected by non-systematic assessment
1
Term from vocabulary, MedDRA version 17.0
[Not Specified]
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.
Pfizer has the right to review disclosures, requesting a delay of less than 60 days. Investigator will postpone single center publications until after disclosure of pooled data (all sites), less than 12 months from study completion/termination at all participating sites. Investigator may not disclose previously undisclosed confidential information other than study results.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Pfizer ClinicalTrials.gov Call Center
Organization: Pfizer, Inc.
Phone: 1-800-718-1021
EMail: ClinicalTrials.gov_Inquiries@pfizer.com
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Layout table for additonal information
Responsible Party: Pfizer
ClinicalTrials.gov Identifier: NCT01335061    
Other Study ID Numbers: B1821010
3090A1-3306 ( Other Identifier: Alias Study Number )
2011-000520-15 ( EudraCT Number )
First Submitted: March 30, 2011
First Posted: April 13, 2011
Results First Submitted: March 18, 2015
Results First Posted: May 4, 2015
Last Update Posted: June 8, 2015