Study To Compare On-Demand Treatment To A Prophylaxis Regimen Of BeneFIX In Subjects With Moderately Severe to Severe Hemophilia B
This study is ongoing, but not recruiting participants.
Sponsor:
Pfizer
Information provided by (Responsible Party):
Pfizer
ClinicalTrials.gov Identifier:
NCT01335061
First received: March 30, 2011
Last updated: May 22, 2013
Last verified: May 2013
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Purpose
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 | Intervention | Phase |
|---|---|---|
|
Hemophilia B |
Biological: Nonacog alfa |
Phase 3 |
| Study Type: | Interventional |
| Study Design: | Allocation: Non-Randomized Endpoint Classification: Safety/Efficacy Study Intervention Model: Single Group Assignment Masking: 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%) |
Resource links provided by NLM:
Genetics Home Reference related topics:
hemophilia
Drug Information available for:
Factor IX
U.S. FDA Resources
Further study details as provided by Pfizer:
Primary Outcome Measures:
- Annualized number of bleeding episodes [ Time Frame: Up to 2 years ] [ Designated as safety issue: No ]
Secondary Outcome Measures:
- Assessment scores on a 4-point Response Scale for an on-demand bleeding episode, as assessed by subject/caregiver or investigator/qualified staff. The 4 point scale assessments are Excellent, Good, Moderate or No response. [ Time Frame: Up to 2 years ] [ Designated as safety issue: No ]
- The number of BeneFIX infusions used to treat each bleed. [ Time Frame: Up to 2 years ] [ Designated as safety issue: No ]
- The number of breakthrough (spontaneous/non-traumatic) bleeds within 48 hours of a prophylaxis dose of BeneFIX . [ Time Frame: Up to 2 years ] [ Designated as safety issue: No ]
- The average infusion dose and total factor consumption. [ Time Frame: Up to 2 years ] [ Designated as safety issue: No ]
- The number of subjects with incidences of less-than-expected therapeutic effect (LETE) in the absence of confounding factors. There are 3 circumstances in which LETE can occur. In the on-demand or prophylaxis settings and for low recovery of Factor IX. [ Time Frame: Up to 2 years ] [ Designated as safety issue: No ]
| Estimated Enrollment: | 24 |
| Study Start Date: | September 2011 |
| Estimated Study Completion Date: | April 2014 |
| Estimated Primary Completion Date: | April 2014 (Final data collection date for primary outcome measure) |
| Arms | Assigned Interventions |
|---|---|
| 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
|
Eligibility| Ages Eligible for Study: | 12 Years to 65 Years |
| Genders 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.
Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01335061
Locations
| Bulgaria | |
| UMBAL Sveti Georgi, Klinika po hematologia | |
| Plovdiv, Bulgaria, 4002 | |
| Canada, Ontario | |
| The Ottawa Hospital | |
| Ottawa, Ontario, Canada, K1H 8L6 | |
| Croatia | |
| University Hospital Center Zagreb | |
| Zagreb, Croatia, 10000 | |
| Korea, Republic of | |
| Eulji University Hospital | |
| Daejeon, Korea, Republic of, 302-799 | |
| Malaysia | |
| Hospital Tengku Ampuan Afzan | |
| Kuantan, Pahang, Malaysia, 25200 | |
| National Blood Centre | |
| Kuala Lumpur, Malaysia, 50400 | |
| Mexico | |
| Hospital y Clinica OCA | |
| Monterrey, Nuevo Leon, Mexico, 64000 | |
| Instituto Biomedico de Investigacion A.C. | |
| Aguascalientes, Mexico, 20127 | |
| Poland | |
| Nzoz Triclinium | |
| Warszawa, Poland, 02-797 | |
| Klinika Hematologii, Nowotworow Krwi i Transplantacji Szpiku | |
| Wroclaw, Poland, 50-367 | |
| Singapore | |
| Singapore General Hospital | |
| Singapore, Singapore, 169608 | |
| Turkey | |
| Hacettepe Universitesi Tip Fakultesi Ic Hastaliklari ABD Hematoloji Bolumu | |
| Ankara, Turkey, 06100 | |
| Cukurova Universitesi Tip Fakultesi | |
| Balcali/Adana, Turkey, 01330 | |
| Ege Universitesi Tip Fakultesi | |
| Bornova/Izmir, Turkey, 35100 | |
| Erciyes Universitesi Tip Fakultsi Ic Hastaliklari ABD Hematoloji BD | |
| Erciyes, Turkey | |
| Gaziantep Universitesi Tip Fakultesi Sahinbey Arastirma ve Uygulama Hastanesi Hematoloji Poliklinigi | |
| Gaziantep, Turkey, 27300 | |
| Istanbul Universitesi Cerrahpasa Tip Fakultesi Ic Hastaliklari Anabilim Dali Hematoloji Bilim Dali | |
| Istanbul, Turkey, 34098 | |
| Erciyes Universitesi Tip Fakultesi | |
| Kayseri, Turkey, 38039 | |
Sponsors and Collaborators
Pfizer
Investigators
| Study Director: | Pfizer CT.gov Call Center | Pfizer |
More Information
Additional Information:
No publications provided
| Responsible Party: | Pfizer |
| ClinicalTrials.gov Identifier: | NCT01335061 History of Changes |
| Other Study ID Numbers: | B1821010, 3090A1-3306 |
| Study First Received: | March 30, 2011 |
| Last Updated: | May 22, 2013 |
| Health Authority: | United States: Food and Drug Administration |
Keywords provided by Pfizer:
|
Hemophilia B on-demand prophylaxis |
nonacog alfa BeneFIX FIX |
Additional relevant MeSH terms:
|
Hemophilia B Hemophilia A Blood Coagulation Disorders, Inherited Blood Coagulation Disorders Hematologic Diseases |
Coagulation Protein Disorders Hemorrhagic Disorders Genetic Diseases, Inborn Genetic Diseases, X-Linked |
ClinicalTrials.gov processed this record on June 18, 2013