Phase 3
N=21
Evaluation of a Recombinant Factor IX Product, APVO101, in Previously-Treated Pediatric Patients With Hemophilia B
Hemophilia B
Bottom Line
View on ClinicalTrials.gov: NCT03855280 ↗Enrolled (actual)
21
Serious AEs
9.5%
Results posted
May 2024
Primary outcome: Primary: Annualized Bleeding Rate (ABR) — 2.93 bleeding episodes per year
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- APVO101 (Drug)
- Age
- Pediatric
- Sex
- All
- Sponsor
- Medexus Pharma, Inc.
- Primary completion
- Jul 2022
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Annualized Bleeding Rate (ABR) |
2.93 | — |
| PRIMARY Annualized Bleeding Rate (ABR) Overall |
2.34 | — |
| SECONDARY Concentration (Cmax) |
60.1 | — |
| SECONDARY Area Under the Curve (0-inf) |
1170 | — |
| SECONDARY Mean Residence Time (MRT) |
20 | — |
| SECONDARY Terminal Half-Life (t 1/2) |
16.3 | — |
| SECONDARY Clearance (CL) |
6.8 | — |
| SECONDARY Volume of Distribution at Steady-State (Vdss) |
134 | — |
| SECONDARY Incremental Recovery (IR) |
0.790 | — |
| SECONDARY Subject Rating of APVO101 Efficacy - Evaluated at the Bleeding Episode Level (Treatment Phase) |
10; 5; 0.0; 0.0; 7; 2 | — |
| SECONDARY Subject Rating of APVO101 Efficacy - Evaluated at the Bleeding Episode Level (Overall) |
28; 13; 0.0; 0.0; 9; 2 | — |
| SECONDARY Investigator Rating of APVO101 Prophylaxis Efficacy (Treatment Phase) |
80; 1; 0 | — |
| SECONDARY Investigator Rating of APVO101 Prophylaxis Efficacy (Overall) |
167; 3; 0 | — |
| SECONDARY Investigator Rating of APVO101 Efficacy for Control and Management of Bleeding Episodes (Treatment Phase) |
16; 0; 0 | — |
| SECONDARY Investigator Rating of APVO101 Efficacy for Control and Management of Bleeding Episodes (Overall) |
35; 0; 0 | — |
Summary
Phase 3/4, single arm, open-label study to evaluate PK, safety, and efficacy of APVO101 prophylaxis in severe or moderately severe hemophilia B subjects < 12 years of age.
Eligibility Criteria
Inclusion Criteria
- Age: 400/mm3) and not receiving immune modulating or chemotherapeutic agents.
- Platelet count at least 150,000/mm3.
- Liver function: alanine transaminase (ALT) and aspartate transaminase (AST) ≤ 2 times the upper limit of the normal range.
- Total bilirubin ≤ 1.5 times the upper limit of the normal range.
- Renal function: serum creatinine ≤ 1.25 times the upper limit of the normal range.
- Hemoglobin ≥ 7 g/dL.
Exclusion Criteria
- History of factor IX inhibitor ≥ 0.6 Bethesda Units (BU); confirmed by the screening result.
- Existence of another coagulation disorder.
- Evidence of thrombotic disease, fibrinolysis, or disseminated intravascular coagulation (DIC).
- Use of an investigational drug within 30 days prior to study entry.
- Previous use of APVO101.
- Use of medications that could impact hemostasis, such as aspirin.
- Known hypersensitivity to the active substance or to any of the excipients in the investigational products.
- Known allergic reaction to hamster proteins.
- History of poor compliance, geographic isolation, unreliable transportation, a serious medical or social condition, or any other circumstance that, in the opinion of the investigator, would interfere with participation or compliance with the study protocol.
- History of adverse reaction to either plasma-derived factor IX or recombinant factor IX that interfered with the subject's ability to treat bleeding episodes with a factor IX product.
- History of any medical condition that would impact the efficacy evaluation and/or safety evaluation of the study product.
Data sourced from ClinicalTrials.gov (NCT03855280). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.