A Study of SerpinPC in Participants with Hemophilia B (HemB) with Inhibitors (NCT05789537) | Clinical Trial Compass
TerminatedPhase 2
A Study of SerpinPC in Participants with Hemophilia B (HemB) with Inhibitors
Stopped: Study terminated due to business and strategic decision
United States, Armenia, Australia3 participantsStarted 2023-07-28
Plain-language summary
The purpose of the study is to evaluate the efficacy, safety, tolerability and pharmacokinetic (PK) profile of prophylactic SerpinPC in participants with Hemophilia B with inhibitors, as part of the SerpinPC registrational program.
Who can participate
Age range
12 Years – 65 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
. Male participants greater than or equal to (\>=) 12 and less than or equal to (\<=) 65 years of age at the time of informed consent.
. Capable of providing written informed consent (adolescent assent and parental/guardian/legal representative consent when appropriate) for participation and having the opportunity to discuss the study with the Investigator or delegate.
. Historically documented HemB (defined as factor IX \<=0.05 international unit/Milliliter (IU/mL) \[\<=5 percent (%)\]).
. Participants who are currently in a prophylaxis program must be willing to stop prophylaxis (including episodic prophylaxis for sporting events) before the first dose of SerpinPC.
. Historical or ongoing Factor IX inhibitor with bypass agents based on medical records or laboratory reports.
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 up to Week 24
. Documented ABR of 6 in the 12 months before screening (participants not on prophylaxis regimen) or documented ABR of ≥2 for participants on prophylaxis regimen
. At least 12 weeks of prospective documentation of bleeding episodes in the AP-0105 (NCT05605678) non-interventional study before SerpinPC dosing, or willing to complete a 12-week observational period (at minimum) in AP-0103.
. No bleeding in the 7 days before Baseline (the prospective observation period can be extended by 10 days if there is an ongoing active bleed).
Exclusion criteria
0. Adequate hematologic function, defined as a platelet count of \>=100,000/microliters (mcL) (\>=100\*10\^9/L) and hemoglobin level of \>=10 grams/deciliter(g/dL) (\>=100 g/L or \>= 6.206 millimoles per liter (mmol/L) at Screening and Pre-dosing visits.
1. Adequate hepatic function, defined as a total bilirubin level of \<=1.5\*upper limit of normal (ULN) (excluding Gilbert syndrome) and aspartate aminotransferase and/or alanine aminotransferase of \<=3\*ULN at Screening and Pre-dosing visits; no clinical signs or known laboratory or radiographic evidence consistent with cirrhosis of the liver.
2. Adequate renal function, defined as a serum creatinine level of \<=2.0\*ULN at Screening and Pre-dosing visits.
3. Able to use a diary to document bleeding events and medication usage.
4. Sexually active participants with a partner who could become pregnant should agree to use effective contraception for the duration of the study.
. Known severe thrombophilia (defined as antithrombin deficiency and/or protein S deficiency and/or protein C deficiency).
. Participant with previous factor IX inhibitor who responded to immune tolerance induction and remains on prophylactic factor concentrate.
. Previous deep vein thrombosis, pulmonary embolism, myocardial infarction, or stroke.