A Study to Evaluate Prospective Efficacy and Safety Data of Current FIX Prophylaxis Replacement T… (NCT03587116) | Clinical Trial Compass
CompletedPhase 3
A Study to Evaluate Prospective Efficacy and Safety Data of Current FIX Prophylaxis Replacement Therapy in Adult Hemophilia B Subjects (FIX:C≤2%) or Current FVIII Prophylaxis Replacement Therapy in Adult Hemophilia A Subjects (FVIII:C≤1%)
United States, Australia, Belgium212 participantsStarted 2018-07-26
Plain-language summary
To establish baseline prospective efficacy data of current FIX prophylaxis replacement therapy in the usual care setting of hemophilia B subjects, who are negative for nAb to AAV-Spark100, prior to the Phase 3 gene therapy study.
To establish baseline prospective efficacy data of current FVIII prophylaxis replacement therapy in the usual care setting of hemophilia A subjects, who are negative for nAb to AAV6, prior to the Phase 3 gene therapy study.
The enrollment for hemophilia A participants is completed. At this time participants are only being enrolled for hemophilia B cohort.
Who can participate
Age range
18 Years – 64 Years
Sex
MALE
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
. Evidence of a signed and dated informed consent document indicating that the participant has been informed of all pertinent aspects of the study.
. Willing and able to comply with scheduled visits, FIX prophylaxis treatment plan, laboratory tests and other study procedures.
. Males ≥ 18 and \<65 years of age with moderately severe to severe hemophilia B and documented FIX activity (≤2%) prior to baseline visit.
. Previous experience with FIX therapy (≥50 documented exposure days to a FIX protein product such as recombinant, plasma-derived or extended half-life FIX product).
. Participants on FIX prophylaxis replacement therapy (recombinant, plasma-derived or extended half-life FIX product) must have the intention to remain on FIX prophylaxis replacement therapy for the duration of the study.
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Annualized Bleeding Rate (ABR) for Treated Bleeds and All Bleeds During Prospective Data Collection Period in Hemophilia B Participants: Efficacy Analysis Set
Timeframe: During prospective data collection period (from baseline visit of the study [Day 1] up to end of study follow-up [maximum follow-up: 1269 days])
2
ABR for Treated Bleeds and All Bleeds During Prospective Data Collection Period in Hemophilia B Participants: Per-protocol Analysis Set
Timeframe: During prospective data collection period (from baseline visit of the study [Day 1] up to end of study follow-up [maximum follow-up: 1269 days])
3
ABR for Treated Bleeds and All Bleeds During Prospective Data Collection Period in Hemophilia B Participants: Protocol Amendment 5 Analysis Set
Timeframe: During prospective data collection period (from baseline visit of the study [Day 1] up to end of study follow-up [maximum follow-up: 1269 days])
4
ABR for Treated Bleeds and All Bleeds During Retrospective Data Collection Period in Hemophilia B Participants: Protocol Amendment 5 Analysis Set
Timeframe: During retrospective data collection period (12 months before screening collected in the hemophilia history case report form [CRF])
5
ABR for Treated Bleeds and All Bleeds From the Combined Retrospective and Prospective Data Collection Period in Hemophilia B Participants: Protocol Amendment 5 Analysis Set
. No known hypersensitivity to FIX replacement product.
. No history of FIX inhibitor (clinical or laboratory-based assessment) defined as a titer
. Evidence of a signed and dated informed consent document indicating that the participant has been informed of all pertinent aspects of the study.
Exclusion criteria
. Anti-AAV-Spark100 neutralizing antibody titer above the established threshold performed by a central laboratory during screening in hemophilia B subjects or Anti-AAV6 neutralizing antibody titer above the established threshold performed by a central laboratory during screening in hemophilia A subjects.
. Lack of participant compliance with documentation of bleeds and/or prophylaxis replacement therapy administration.
. If there is no documentation regarding hepatitis status, as defined below, within the last 12 months prior to screening for hepatitis B and 6 months prior to screening for hepatitis C, then participants will be required to have the following hepatitis testing performed at screening:
. Hepatitis B screening (acute and chronic):
. Hepatitis C (acute or chronic):
. Currently on antiviral therapy for hepatitis B or C.
. Significant liver disease, as defined by pre-existing diagnosis of portal hypertension, splenomegaly, or hepatic encephalopathy.
. Documented serological evidence of human immunodeficiency virus HIV-1 or HIV-2 with Cluster of Differentiation 4 positive (CD4+) cell count ≤200 mm3 within the last 12 months prior to screening. Participants who are HIV positive and stable, have an adequate CD4 count (\>200/mm3) and undetectable viral load (\<50 gc/mL) documented within the preceding 12 months, and are on an antiretroviral drug regimen are eligible to enroll. Participants who have not been tested within the prior 12 months of screening will need to be tested for HIV status at screening.
Timeframe: From start of retrospective data collection period (12 months before screening collected in hemophilia history CRF) up to end of prospective data collection follow-up of period (maximum follow-up:1269 days), for a total of approximately 4.5 years
6
ABR for Treated Bleeds and All Bleeds During Prospective Data Collection Period in Hemophilia A Participants: Efficacy Analysis Set
Timeframe: During prospective data collection period (from baseline visit of the study [Day 1] up to end of study follow-up [maximum follow-up: 948 days])
7
ABR for Treated Bleeds and All Bleeds During Prospective Data Collection Period in Hemophilia A Participants: Per-protocol Analysis Set
Timeframe: During prospective data collection period (from baseline visit of the study [Day 1] up to end of study follow-up [maximum follow-up: 948 days])
8
ABR for Treated Bleeds and All Bleeds During Prospective Data Collection Period in Hemophilia A Participants: Protocol Amendment 5 Analysis Set
Timeframe: During prospective data collection period (from baseline visit of the study [Day 1] up to end of study follow-up [maximum follow-up: 948 days])
9
ABR for Treated Bleeds and All Bleeds During Retrospective Data Collection Period in Hemophilia A Participants: Protocol Amendment 5 Analysis Set
Timeframe: During retrospective data collection period (12 months before screening collected in the hemophilia history CRF)
10
ABR for Treated Bleeds and All Bleeds From the Combined Retrospective and Prospective Data Collection Period in Hemophilia A Participants: Protocol Amendment 5 Analysis Set
Timeframe: From start of retrospective data collection period (12 months before screening collected in hemophilia history CRF) up to end of prospective data collection follow-up of period (maximum follow-up:948 days), for a total of approximately 3.6 years