Status:
COMPLETED
Evaluation of a Recombinant Factor IX Product, APVO101, in Previously-Treated Pediatric Patients With Hemophilia B
Lead Sponsor:
Medexus Pharma, Inc.
Conditions:
Hemophilia B
Eligibility:
All Genders
Up to 11 years
Phase:
PHASE3
Brief 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.
Detailed Description
Study APVO101-903 is a Phase 3/4, single arm, open-label clinical trial. The purpose of the study is to evaluate pharmacokinetics (PK), safety, and efficacy of APVO101 prophylaxis in severe or moderat...
Eligibility Criteria
Inclusion
- Age: \< 11.5 years of age at the time of the first dose and \< 12 years throughout the Treatment Phase of the study (for at least 50 ED).
- Informed consent: subject's parent or legal guardian written Institutional Review Board (IRB)/Ethics Committee (EC)-approved informed consent. An assent form (IRB/EC-approved) will be obtained, when required by local regulations/guidelines.
- Willingness and ability to make the required study visits, and follow instructions while enrolled in the study (for at least 50 ED; approximately 6 months).
- Documented severe or moderately severe hemophilia B diagnosis (factor IX activity ≤ 2 IU/dL); in addition, severity may be indicated by the occurrence of one or more joint bleeding episode(s) at any point in the child's medical history requiring infusion(s) to replace factor IX.
- Subjects must be on prophylaxis or switch to a prophylaxis regimen for the duration of the study.
- Previously treated patients with a minimum of 50 ED (as documented and determined by the investigator) to a preparation/blood components containing factor IX.
- Willingness to adhere to the 4-day washout period of any factor IX replacement therapy prior to PK evaluation. In case of previous exposure to a factor IX product with a prolonged half-life, a washout period of 3 half-lives is required in order to achieve steady state factor IX level prior to exposure to APVO101.
- Immunocompetent (CD4 count \> 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
- 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.
Key Trial Info
Start Date :
January 16 2020
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
July 4 2022
Estimated Enrollment :
21 Patients enrolled
Trial Details
Trial ID
NCT03855280
Start Date
January 16 2020
End Date
July 4 2022
Last Update
May 16 2024
Active Locations (11)
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1
Centro Estadual de Hemopterapia e Hematologia do Espirito Santo
Vitória, Espírito Santo, Brazil, 29040-090
2
Universidade Estadual de Campinas - Centro de Hematologia e Hemoterapia
Campinas, Brazil, 13083-878
3
Hospital das Clínicas da Faculdade de Medicina de Ribeirão Preto da Universidade de São Paulo
Ribeirão Preto, Brazil, 14051-140
4
JSC K Eristavi National Center for Experimental and Clinical Surgery
Tbilisi, Georgia, 0159