Trial record 1 of 1 for:
NCT01335061
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 |
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Recruitment Status :
Completed
First Posted : April 13, 2011
Results First Posted : May 4, 2015
Last Update Posted : June 8, 2015
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Sponsor:
Pfizer
Information provided by (Responsible Party):
Pfizer
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Brief Summary:
The purpose of this study will be to determine if a once-weekly prophylaxis regimen of BeneFIX in subjects with moderately severe to severe Hemophilia B is safe and effective.
| Condition or disease | Intervention/treatment | Phase |
|---|---|---|
| Hemophilia B | Biological: Nonacog alfa | Phase 3 |
| Study Type : | Interventional (Clinical Trial) |
| Actual Enrollment : | 25 participants |
| Allocation: | Non-Randomized |
| Intervention Model: | Single Group Assignment |
| Masking: | None (Open Label) |
| Primary Purpose: | Treatment |
| Official Title: | A Multicenter, Open-label Study To Compare On-demand Treatment To A Prophylaxis Regimen Of Nonacog-alfa (Benefix) In Subjects With Moderately Severe To Severe Hemophilia B (Fix:c</=2%) |
| Study Start Date : | September 2011 |
| Actual Primary Completion Date : | April 2014 |
| Actual Study Completion Date : | April 2014 |
Resource links provided by the National Library of Medicine
MedlinePlus Genetics related topics:
Hemophilia
Drug Information available for:
Nonacog alfa
| Arm | Intervention/treatment |
|---|---|
| BeneFIX |
Biological: Nonacog alfa
Period 1: During on-demand period, dosing at the discretion of investigator.
Other Name: BeneFIX Biological: Nonacog alfa Period 2: During the prophylaxis period, 100 IU/kg once weekly
Other Name: BeneFIX |
Primary Outcome Measures :
- Annualized Number of Bleeding Episodes. [ Time Frame: 2 years ]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.
Secondary Outcome Measures :
- Response to On-Demand Treatment for All Bleeding Episodes. [ Time Frame: 2 years ]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.
- Number of Nonacog Alfa, Recombinant Factor IX (BeneFIX) Infusions Used to Treat Each Bleeding Episode. [ Time Frame: 2 years ]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.
- Number of Breakthrough (Spontaneous/Non-Traumatic) Bleeds Within 48 Hours of a Prophylaxis Dose of BeneFIX. [ Time Frame: 2 years ]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.
- Average Infusion Dose. [ Time Frame: 2 years ]The mean dose by per infusion by weight (IU/kg) was reported for both prophylaxis and on demand infusions
- Total Factor Consumption. [ Time Frame: 2 years ]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.
- Incidence of Less Than Expected Therapeutic Effect (LETE) [ Time Frame: 2 years ]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.
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| Ages Eligible for Study: | 12 Years to 65 Years (Child, Adult, Older Adult) |
| Sexes Eligible for Study: | Male |
| Accepts Healthy Volunteers: | No |
Criteria
Inclusion Criteria:
- Documented history of moderately-severe to severe hemophilia B (FIX activity </=2%).
- Male subjects, aged 12 years to 65 years.
- Subjects with at least 100 exposure days (EDs) to factor IX products.
- Subjects with a minimum of 12 bleeding episodes, 6 of which must be joint bleeds, in the 12-month period before the Screening visit.
Exclusion Criteria:
- Subjects who have received FIX as a primary or secondary prophylaxis regimen within the last 12 months prior to the Screening visit.
- Subjects who have had major surgery or an orthopedic surgical procedure within the past 3 months prior to Screening visit.
- Subjects for which major surgery or orthopedic surgery is planned within the duration of study participation.
- Subjects with a past history of, or current FIX inhibitor, defined as >ULN (upper limit of normal) of the reporting laboratory.
- Subjects with a known hypersensitivity to any FIX product or hamster protein.
Information from the National Library of Medicine
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01335061
Locations
Show 17 study locations
Sponsors and Collaborators
Pfizer
Investigators
| Study Director: | Pfizer CT.gov Call Center | Pfizer |
Additional Information:
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
| 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 Posted: | April 13, 2011 Key Record Dates |
| Results First Posted: | May 4, 2015 |
| Last Update Posted: | June 8, 2015 |
| Last Verified: | May 2015 |
Keywords provided by Pfizer:
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Hemophilia B on-demand prophylaxis |
nonacog alfa BeneFIX FIX |
Additional relevant MeSH terms:
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Hemophilia A Hemophilia B Blood Coagulation Disorders, Inherited Blood Coagulation Disorders Hematologic Diseases |
Coagulation Protein Disorders Hemorrhagic Disorders Genetic Diseases, Inborn Genetic Diseases, X-Linked |