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 range12 Years – 65 Years
SexMALE
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.
✓. 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.
What they're measuring
1
Annualized Bleeding Rate (ABR) for Treated Bleeds up to Week 24
✕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.