Study of Safety And Efficacy Of ReFacto AF In Previously Untreated Hemophilia A Patients In The Usual Care Setting
This study is currently recruiting participants.
Verified May 2013 by Pfizer
Sponsor:
Pfizer
Information provided by (Responsible Party):
Pfizer
ClinicalTrials.gov Identifier:
NCT00950170
First received: July 29, 2009
Last updated: May 21, 2013
Last verified: May 2013
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Purpose
Study to evaluate the safety and effectiveness of ReFacto AF for the treatment of severe hemophilia A in patients who have not yet received treatment for their hemophilia. Study subjects will be males less than 6 years old who have not taken any clotting factor or other blood products before the study. The safety and effectiveness of ReFacto AF will be determined in this study by tests and procedures done at the doctor's office.
| Condition | Intervention | Phase |
|---|---|---|
|
Hemophilia A |
Procedure: Laboratory Tests |
Phase 4 |
| Study Type: | Interventional |
| Study Design: | Allocation: Non-Randomized Endpoint Classification: Safety/Efficacy Study Intervention Model: Single Group Assignment Masking: Open Label |
| Official Title: | An Open-Label Study Of The Safety And Efficacy Of ReFacto AF In Previously Untreated Patients In Usual Care Settings |
Resource links provided by NLM:
Genetics Home Reference related topics:
hemophilia
MedlinePlus related topics:
Hemophilia
U.S. FDA Resources
Further study details as provided by Pfizer:
Primary Outcome Measures:
- Proportion of subjects who develop clinically significant FVIII inhibitors during the course of the study. [ Time Frame: 12 years (study duration) ] [ Designated as safety issue: Yes ]
Secondary Outcome Measures:
- Annualized bleeding rates. [ Time Frame: 12 years (study duration) ] [ Designated as safety issue: No ]
- Responses to the first on-demand treatment with Refacto AF for all new bleeds. [ Time Frame: 12 years (study duration) ] [ Designated as safety issue: No ]
- The number of ReFacto AF infusions to treat each new bleed. [ Time Frame: 12 years (study duration) ] [ Designated as safety issue: No ]
- The number of bleeds within 48 hours of a prophylaxis dose of ReFacto AF. [ Time Frame: 12 years (study duration) ] [ Designated as safety issue: No ]
- The average infusion dose and total factor consumption [ Time Frame: 12 years (study duration) ] [ Designated as safety issue: No ]
- The number of subjects who require dose escalation of their prescribed prophylaxis regimen during their participation in this study. [ Time Frame: 12 years (study duration) ] [ Designated as safety issue: No ]
- The incidence of less-than-expected therapeutic effect (LETE). [ Time Frame: 12 years (study duration) ] [ Designated as safety issue: No ]
| Estimated Enrollment: | 50 |
| Study Start Date: | February 2010 |
| Estimated Study Completion Date: | February 2022 |
| Estimated Primary Completion Date: | February 2022 (Final data collection date for primary outcome measure) |
| Arms | Assigned Interventions |
|---|---|
|
Experimental: 1
The investigator treats subjects with ReFacto AF in the usual care setting.
|
Procedure: Laboratory Tests
Blood draws to determine the level of factor VIII activity before and after dosing at appropriate visits, and blood draws to determine levels of factor VIII inhibitor (antibody to factor VIII).
Other Name: ReFacto AF
|
Detailed Description:
Regulatory Commitment
Eligibility| Ages Eligible for Study: | up to 5 Years |
| Genders Eligible for Study: | Male |
| Accepts Healthy Volunteers: | No |
Criteria
Inclusion Criteria:
- Male subjects <6 years of age with severe hemophilia A (FVIII:C <1%) based on clinical records, including newborns.
- No prior exposure to factor products or any blood products.
Exclusion Criteria:
- Presence of any bleeding disorder in addition to hemophilia A.
- Treatment with any investigational agent or device within the past 30 days.
- Any condition(s) that compromises the ability to collect study-related observations, or that poses a contraindication to study participation (these conditions include, but are not limited to, inadequate medical history to assure study eligibility; and expectation of poor adherence to study requirements).
Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00950170
Contacts
| Contact: Pfizer CT.gov Call Center | 1-800-718-1021 |
Locations
| France | |
| Pfizer Investigational Site | Not yet recruiting |
| Lille Cedex, France, 59037 | |
| Pfizer Investigational Site | Recruiting |
| Nantes, France, 44093 | |
| Germany | |
| Pfizer Investigational Site | Active, not recruiting |
| Bremen, Germany, 28177 | |
| Pfizer Investigational Site | Recruiting |
| Hannover, Germany, 30625 | |
| Pfizer Investigational Site | Not yet recruiting |
| Stuttgart, Germany, 70176 | |
| Italy | |
| Pfizer Investigational Site | Recruiting |
| Firenze, Italy, 50134 | |
| Netherlands | |
| Pfizer Investigational Site | Not yet recruiting |
| Groningen, Netherlands, 9713 GZ | |
| Romania | |
| Pfizer Investigational Site | Not yet recruiting |
| Bucharest, Romania, 011155 | |
| Spain | |
| Pfizer Investigational Site | Not yet recruiting |
| A Coruna, Spain, 15006 | |
| Pfizer Investigational Site | Not yet recruiting |
| Barcelona, Spain, 08035 | |
| Pfizer Investigational Site | Recruiting |
| Madrid, Spain, 28046 | |
| Pfizer Investigational Site | Not yet recruiting |
| Sevilla, Spain, 41013 | |
| Pfizer Investigational Site | Recruiting |
| Valencia, Spain, 46026 | |
| Sweden | |
| Pfizer Investigational Site | Not yet recruiting |
| Stockholm, Sweden, 171 76 | |
| Turkey | |
| Pfizer Investigational Site | Recruiting |
| Antalya, Turkey, 07059 | |
| Pfizer Investigational Site | Recruiting |
| Balcali/Adana, Turkey, 01330 | |
| Pfizer Investigational Site | Recruiting |
| Bornova/Izmir, Turkey, 35100 | |
| Pfizer Investigational Site | Recruiting |
| Fatih/Istanbul, Turkey, 34098 | |
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: | NCT00950170 History of Changes |
| Other Study ID Numbers: | 3082B2-4434, B1831006 |
| Study First Received: | July 29, 2009 |
| Last Updated: | May 21, 2013 |
| Health Authority: | Austria: Agency for Health and Food Safety |
Keywords provided by Pfizer:
|
ReFacto AF factor VIII inhibitors antibodies |
Additional relevant MeSH terms:
|
Hemophilia A Blood Coagulation Disorders, Inherited Blood Coagulation Disorders Hematologic Diseases Coagulation Protein Disorders Hemorrhagic Disorders |
Genetic Diseases, Inborn Factor VIII Coagulants Hematologic Agents Therapeutic Uses Pharmacologic Actions |
ClinicalTrials.gov processed this record on June 18, 2013