Not provided
| ID | Type | Description | Link |
|---|---|---|---|
| NCT05568719 | Registry Identifier | ClinicalTrials.gov |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
A study to learn about the long-term safety and efficacy of giroctocogene fitelparvovec or fidanacogene elaparvovec in patients with hemophilia A or hemophilia B respectively, who have received treatment through prior participation in a Pfizer-sponsored clinical trial. Data collection and participant visits will be based on standard of care.
Not provided
Not provided
Not provided
Not provided
Not provided
| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Hemophilia A / giroctocogene fitelparvovec | Other | Participants have received treatment with giroctocogene fitelparvovec in a previous study and are not receiving any investigational product in this study |
|
| Hemophilia B / fidanacogene elaparvovec | Other | Participants have received treatment with fidanacogene elaparvovec in a previous study and are not receiving any investigational product in this study |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Testing of hepatic AAV Vector integration | Diagnostic Test | Evaluation of AAV vector integration in participants for whom a sample of liver has been obtained through biopsy or surgical resection when clinically indicated |
| Measure | Description | Time Frame |
|---|---|---|
| Incidence of thromboembolic events | Day 1 to 10 years | |
| Incidence of factor inhibitor development | FIX inhibitor development was defined as an inhibitor titer >= 0.6 Bethesda units per milliliter (BU/mL). | Day 1 to 10 years |
| Incidence of hepatic malignancy | Day 1 to 10 years | |
| Incidence of liver abnormalities | Day 1 to 10 years | |
| Factor activity level | Factor activity level will be reported. Factor levels may be measured using different assay methods including a one-stage assay or by chromogenic substrate assay and a second one-stage assay. | Day 1 to 10 years |
| Measure | Description | Time Frame |
|---|---|---|
| Total ABR (treated or untreated; (excluding bleeds related to surgery) | ABR (Annual Bleed Rate): number of bleeding episodes per year. This includes treated and untreated bleeds. The ABR or the annualized number of bleeding episodes per year, will be derived for each participant for each observation period by using the following formula: ABR = (Number of bleeds / Days in observation period) x 365.25 days/year. |
Not provided
Inclusion Criteria:
-Only participants who received investigational giroctocogene fitelparvovec or fidanacogene eleparvovec and were enrolled in a Pfizer-sponsored study (C0371002, C0371003, C0371005, C3731001, C3731003) are eligible.
Exclusion Criteria:
-None
Not provided
Not provided
Not provided
Not provided
| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Pfizer CT.gov Call Center | Contact | 1-800-718-1021 | ClinicalTrials.gov_Inquiries@pfizer.com |
| Name | Affiliation | Role |
|---|---|---|
| Pfizer CT.gov Call Center | Pfizer | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| The Regents of the University of California||UC Davis Health | Recruiting | Rancho Cordova | California | 95670 | United States |
| Label | URL |
|---|---|
| To obtain contact information for a study center near you, click here. | View source |
Not provided
Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical\_trials/trial\_data\_and\_results/data\_requests.
Not provided
Not provided
Not provided
Not provided
Non-investigational study
Not provided
Not provided
Not provided
Not provided
| Day 1 to 10 years |
| Incidence of and time from vector infusion to resumption of prophylaxis | Describe incidence of resumption of prophylaxis resumption and the time (in days) to resumption of prophylaxis after receiving vector infusion. | Day 1 to 10 years |
| AIR of exogenous factor (excluding infusions related to surgery) | The AIR or the annualized number of FIX infusions per year, will be derived for each participant for each observation period by using the following formula: AIR = (Number of FIX infusions / Days in observation period) x 365.25 days/year. | Day 1 to 10 years |
| Consumption of exogenous factor (excluding infusions related to surgery) | The annualized TFC in international units (IU) will be derived for each participant for each observation period using the following formula: Annualized TFC = (Total units of FIX infused (IU)/ Days in observation period) x 365.25 days/year | Day 1 to 10 years |
| Incidence of Non-hepatic malignancy | Day 1 to 10 years |
| Incidence of Auto-immune disorders | Day 1 to 10 years |
| Incidence of SAEs | An SAE was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly; development of a clinical thrombotic event; development of factor inhibitor; development of a hepatic malignancy; development of drug-related elevated hepatic transaminases that fail to improve with immunosuppressive regimens; occurrence of a malignancy with reasonable possibility of being related to study drug. | Day 1 to 10 years |
| All cause mortality | All-cause mortality was defined as the death due to any cause during the course of study. Incidence rate was defined as the total number of participants with admissible events divided by the total (for all qualifying participants) time at risk for the cohort/treatment group of interest. Incidence rate of all-cause deaths was reported in this outcome measure. | Day 1 to 10 years |
| EQ-5D-5L dimension and VAS scores | The EQ-5D-5L comprises a 5-item health status measure and a visual analog rating scale/feeling thermometer. Using the 5-dimensional Health State Classification, participants are asked to respond to five questions on different aspects of their health status that assess the following:
| Day 1 to 10 years |
| UC Davis Health | Recruiting | Sacramento | California | 95816 | United States |
| UC Davis Ambulatory Care Clinic | Recruiting | Sacramento | California | 95817 | United States |
| UC Davis Hemophilia Treatment Center | Recruiting | Sacramento | California | 95817 | United States |
| UC Davis Medical Center | Recruiting | Sacramento | California | 95817 | United States |
| UCSF Outpatient Hematology Clinic | Not yet recruiting | San Francisco | California | 94143 | United States |
| USF Health Morsani Center For Advanced Healthcare | Recruiting | Tampa | Florida | 33612 | United States |
| The University of South Florida Board of Trustees | Recruiting | Tampa | Florida | 33620 | United States |
| Mississippi Center For Advanced Medicine | Terminated | Madison | Mississippi | 39110 | United States |
| Cornell University||Joan & Sanford I. Weill Medical College | Recruiting | New York | New York | 10065 | United States |
| Weill Cornell Medical College-New York Presbyterian Hospital | Recruiting | New York | New York | 10065 | United States |
| The Childrens Hospital of Philadelphia Division of Hematology | Recruiting | Philadelphia | Pennsylvania | 19104 | United States |
| The Childrens Hospital of Philadelphia Division of Hematology | Recruiting | Philadelphia | Pennsylvania | 19146 | United States |
| Washington Institute for Coagulation | Not yet recruiting | Seattle | Washington | 98101 | United States |
| Royal Prince Alfred Hospital | Recruiting | Camperdown | New South Wales | 2050 | Australia |
| Sydney Local Health District (RPAH Zone) | Recruiting | Camperdown | New South Wales | 2050 | Australia |
| Unity Health Toronto, St. Michael's Hospital | Not yet recruiting | Toronto | Ontario | M5B 1W8 | Canada |
| Kyung Hee University Hospital at Gangdong | Not yet recruiting | Seoul | 05278 | South Korea |
| Skåne University Hospital | Not yet recruiting | Malmö | 21428 | Sweden |
| Taichung Veterans General Hospital | Not yet recruiting | Taichung | 40705 | Taiwan |
| National Taiwan University Hospital | Not yet recruiting | Taipei | 10041 | Taiwan |
| Ege Universitesi Hastanesi | Recruiting | Izmir | İ̇zmir | 35100 | Turkey (Türkiye) |
| Acibadem Adana Hospital | Not yet recruiting | Adana | 01130 | Turkey (Türkiye) |
| Background |
| Blanchette VS, Key NS, Ljung LR, Manco-Johnson MJ, van den Berg HM, Srivastava A; Subcommittee on Factor VIII, Factor IX and Rare Coagulation Disorders of the Scientific and Standardization Committee of the International Society on Thrombosis and Hemostasis. Definitions in hemophilia: communication from the SSC of the ISTH. J Thromb Haemost. 2014 Nov;12(11):1935-9. doi: 10.1111/jth.12672. Epub 2014 Sep 3. No abstract available. |
| 12709033 | Background | Blanchette VS, McCready M, Achonu C, Abdolell M, Rivard G, Manco-Johnson MJ. A survey of factor prophylaxis in boys with haemophilia followed in North American haemophilia treatment centres. Haemophilia. 2003 May;9 Suppl 1:19-26; discussion 26. doi: 10.1046/j.1365-2516.9.s1.12.x. |
| 18929522 | Background | Lillicrap D. Extending half-life in coagulation factors: where do we stand? Thromb Res. 2008;122 Suppl 4:S2-8. doi: 10.1016/S0049-3848(08)70027-6. |
| ID | Term |
|---|---|
| D006467 | Hemophilia A |
| D002836 | Hemophilia B |
| D006470 | Hemorrhage |
| ID | Term |
|---|---|
| D025861 | Blood Coagulation Disorders, Inherited |
| D001778 | Blood Coagulation Disorders |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D020147 | Coagulation Protein Disorders |
| D006474 | Hemorrhagic Disorders |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D040181 | Genetic Diseases, X-Linked |
| D010335 | Pathologic Processes |
| D013568 | Pathological Conditions, Signs and Symptoms |
Not provided
Not provided