Study To Compare On-Demand Treatment To A Prophylaxis Regimen Of BeneFIX In Subjects With Moderately Severe to Severe Hemophilia B

Hemophilia B Clinical Trial

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%)

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01335061
• Other study ID #: B1821010
• Secondary ID: 3090A1-33062011-
• Status: Completed
• Phase: Phase 3
• First received: March 30, 2011
• Last updated: May 12, 2015
• Start date: September 2011
• Est. completion date: April 2014

Study information

• Verified date: May 2015
• Source: Pfizer
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Food and Drug Administration
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 25
• Est. completion date: April 2014
• Est. primary completion date: April 2014
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 12 Years to 65 Years

Eligibility

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.

Study Design

Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Hemophilia A
• Hemophilia B

Intervention

Biological:
• Nonacog alfa
Period 1: During on-demand period, dosing at the discretion of investigator.
• Nonacog alfa
Period 2: During the prophylaxis period, 100 IU/kg once weekly

Locations

Country	Name	City	State
Bulgaria	UMBAL Sveti Georgi, Klinika po hematologia	Plovdiv
Canada	The Ottawa Hospital	Ottawa	Ontario
Croatia	University Hospital Center Zagreb	Zagreb
Korea, Republic of	Eulji University Hospital	Daejeon
Malaysia	National Blood Centre	Kuala Lumpur
Malaysia	Hospital Tengku Ampuan Afzan	Kuantan	Pahang
Mexico	Instituto Biomedico de Investigacion A.C.	Aguascalientes
Mexico	Hospital y Clinica OCA	Monterrey	Nuevo Leon
Poland	Nzoz Triclinium	Warszawa
Poland	Klinika Hematologii, Nowotworow Krwi i Transplantacji Szpiku	Wroclaw
Singapore	Singapore General Hospital	Singapore
Turkey	Hacettepe Universitesi Tip Fakultesi Ic Hastaliklari Anabilim Dali Hematoloji Bolumu	Ankara
Turkey	Ege Universitesi Tip Fakultesi	Bornova/Izmir
Turkey	Istanbul Universitesi Cerrahpasa Tip Fakultesi Ic Hastaliklari Anabilim Dali Hematoloji Bilim Dali	Fatih	Istanbul
Turkey	Gaziantep Universitesi Tip Fakultesi Sahinbey Arastirma ve Uygulama Hastanesi Hematoloji Poliklinigi	Gaziantep
Turkey	Erciyes Universitesi Tip Fakultesi	Kayseri
Turkey	Erciyes Universitesi Tip Fakultesi Hastaneleri M. Kemal Dedeman Onkoloji Hastanesi	KAYSERI, Erciyes

Sponsors (1)

Lead Sponsor	Collaborator
Pfizer

Countries where clinical trial is conducted

Bulgaria,  Canada,  Croatia,  Korea, Republic of,  Malaysia,  Mexico,  Poland,  Singapore,  Turkey, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Annualized Number of Bleeding Episodes.	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.	2 years	No
Secondary	Response to On-Demand Treatment for All Bleeding Episodes.	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.	2 years	No
Secondary	Number of Nonacog Alfa, Recombinant Factor IX (BeneFIX) Infusions Used to Treat Each Bleeding Episode.	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.	2 years	No
Secondary	Number of Breakthrough (Spontaneous/Non-Traumatic) Bleeds Within 48 Hours of a Prophylaxis Dose of BeneFIX.	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.	2 years	No
Secondary	Average Infusion Dose.	The mean dose by per infusion by weight (IU/kg) was reported for both prophylaxis and on demand infusions	2 years	No
Secondary	Total Factor Consumption.	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.	2 years	No
Secondary	Incidence of Less Than Expected Therapeutic Effect (LETE)	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.	2 years	No

External Links

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