Six Month lead-in Study to Evaluate Prospective Efficacy and Safety Data of Current FIX Prophylaxis Replacement Therapy in Adult Hemophilia B Subjects (FIX:C≤2%)
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|ClinicalTrials.gov Identifier: NCT03587116|
Recruitment Status : Recruiting
First Posted : July 16, 2018
Last Update Posted : July 8, 2019
|Condition or disease||Intervention/treatment||Phase|
|Hemophilia B||Drug: Standard of Care FIX Replacement therapy||Phase 3|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||110 participants|
|Intervention Model:||Single Group Assignment|
|Intervention Model Description:||The data obtained from this 6 month lead-in study will serve as the control group for the subsequent Phase 3 gene therapy study.|
|Masking:||None (Open Label)|
|Official Title:||AN OPEN-LABEL, NON-INVESTIGATIONAL PRODUCT, MULTI-CENTER, LEAD-IN STUDY TO EVALUATE AT LEAST 6 MONTHS OF PROSPECTIVE EFFICACY AND SELECTED SAFETY DATA OF FACTOR IX (FIX) PROPHYLAXIS REPLACEMENT THERAPY IN THE USUAL CARE SETTING OF MODERATELY SEVERE TO SEVERE ADULT HEMOPHILIA B SUBJECTS (FIX:C≤2%) WHO ARE NEGATIVE FOR NEUTRALIZING ANTIBODIES (NAB) TO ADENO-ASSOCIATED VIRUS VECTOR (AAV)-SPARK100|
|Actual Study Start Date :||July 26, 2018|
|Estimated Primary Completion Date :||November 17, 2021|
|Estimated Study Completion Date :||November 17, 2021|
|Standard of Care FIX replacement therapy||
Drug: Standard of Care FIX Replacement therapy
There is no investigational product being administered. Subjects will be administering their own standard of care FIX replacement therapy.
- Annualized bleeding rate (ABR) [ Time Frame: 6 months ]The annualized bleeding rate (ABR) per subject will be calculated as the number of bleeds over number of days subject received FIX prophylaxis replacement therapy from baseline visit (Day 1) to end of study x 365.25 days. ABR will be summarized using descriptive statistics (n, mean, standard deviation, median, Q1, Q3, minimum, maximum).
- Incidence of serious adverse events [ Time Frame: 6 months ]The primary safety analysis will be performed on all subjects that sign the informed consent document and are subsequently identified as nAb negative and are enrolled (complete baseline visit) into the study.
- Events of special interest (ESI):inhibitor against FIX, thrombotic events, and FIX hypersensitivity reactions [ Time Frame: 6 months ]Frequency and percentage of these ESI events will be summarized by event. In addition any events leading to discontinuation from the study will be described.
- Annualized infusion rate (AIR) [ Time Frame: 6 months ]The annualized infusion rate (AIR) per subject will be calculated as the number of infusions received over number of days subject received FIX prophylaxis replacement therapy from baseline visit (Day 1) to end of study x 365.25 days. AIR will be summarized using descriptive statistics (n, mean, standard deviation, median, Q1, Q3, minimum, maximum).
- Dose and total factor consumption [ Time Frame: 6 months ]The total factor IX replacement therapy consumption and the corresponding dose will be descriptively summarized by the categories of the replacement therapy, where appropriate. Infusion diary (electronic infusion diary) of the factor IX replacement therapy will be listed.
- Number of bleeding events (spontaneous and/or traumatic) [ Time Frame: 6 months ]The number of bleeding episodes will be summed up by spontaneous, traumatic and overall as defined as any bleed occurring >72 hours after stopping treatment from the original bleed for which treatment was initiated or a bleed occurring at a different site from the original bleed regardless of the time from last injection.
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): NCT03587116
|Contact: Pfizer CT.gov Call Center||1-800-718-1021||ClinicalTrials.gov_Inquiries@pfizer.com|
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|Study Director:||Pfizer CT.gov Call Center||Pfizer|