NCT03855280

Evaluation of a Recombinant Factor IX Product, APVO101, in Previously-Treated Pediatric Patients With Hemophilia B

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 21 (actual)
• Sponsor: Medexus Pharma, Inc. · Industry
• Sex: All
• Age: 11 Years
• Healthy volunteers: Not accepted

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 moderately severe hemophilia B subjects \< 12 years of age. The study is designed to gather information in two age groups of previously treated (with a minimum of 50 previous ED to factor IX replacement therapy) pediatric patients, specifically those \< 6 years of age and 6 to \<12 years of age. Study APVO101-903 consists of three distinct phases: * PK Phase - PK evaluation will consist of administration of a single 75 ± 5 IU/kg dose, followed by factor IX activity and safety assessments up to 50 hours post-infusion. * Treatment Phase - subjects will receive APVO101 prophylaxis (starting prophylaxis dose to be determined based on APVO101 recovery; ideally within the recommended dose range: 35 - 75 IU/kg; twice weekly) for 50 ED (approximately 6 months). * Continuation Phase - subjects may continue to receive APVO101 prophylaxis (recommended dose range: 35 - 75 IU/kg; twice weekly) for an additional ≥ 50 ED.

Conditions

• Hemophilia B

Interventions

Type	Name	Description
DRUG	APVO101	Subjects will receive a single IV dose of APVO101 twice weekly or at a frequency of infusions as determined appropriate by the investigator for the particular study subject for a total of 50 ED. The starting prophylaxis dose will be based on APVO101 recovery from PK Phase assessments (only pre-infusion and 15-30 minute post-infusion samples).

Timeline

• Start date: 2020-01-16
• Primary completion: 2022-07-04
• Completion: 2022-07-04
• First posted: 2019-02-26
• Last updated: 2024-05-16
• Results posted: 2024-05-16

Locations

11 sites across 6 countries: Brazil, Georgia, Moldova, South Africa, Turkey (Türkiye), Ukraine

Regulatory

• FDA-regulated drug study