Clinical Trial: Study of Recombinant Factor IX Product, IB1001, in Previously Treated Pediatric Subjects With Hemophilia B

Study Status: Active, not recruiting
Recruit Status: Active, not recruiting
Study Type: Interventional

Official Title: Study of Recombinant Factor IX Product, IB1001, in Previously Treated Pediatric Subjects With Hemophilia B

Brief Summary: The Study's Primary Objective is to evaluate the pharmacokinetics, safety (acute effects associated with infusions, and inhibitor development) and efficacy (breakthrough bleeding and control of hemorrhaging during prophylaxis) of IB1001 in previously treated pediatric subjects with hemophilia B.

Detailed Summary:

Primary Objective(s):

To evaluate the pharmacokinetics, safety (acute effects associated with infusions, and inhibitor development) and efficacy (breakthrough bleeding and control of hemorrhaging during prophylaxis) of IB1001 in previously treated pediatric subjects with hemophilia B. Acute effects are defined as adverse events (AEs) which occur within the first 72 hours after the administration of a study product.

Secondary Objectives:

  1. To obtain tolerance and compliance information on pediatric subject response to an intravenously delivered recombinant factor IX product , IB1001
  2. To evaluate the safety of IB1001 during the 50 exposure days treatment course

Investigators/Study Centers:

This international study will be conducted at clinical sites located in the United States (USA), Europe, Mexico and India

Phase of Development: Phase 3/4

Planned Sample Size:

Up to 22 pediatric subjects (<12 years of age) will be enrolled in order to have 20 subjects complete the study.

Study Duration:

The study duration will be approximately 6 months per subject to include a minimum of 50 exposures to IB1001.

Study Design:

The study is a non-randomized, open label design that includes an initial pharmacokinetics evaluation in all participants, followed by six months (50 expos
Sponsor: Aptevo BioTherapeutics

Current Primary Outcome: Pharmacokinetics, Safety and Efficacy [ Time Frame: 50 exposure days ]

To evaluate the pharmacokinetics, safety (acute effects associated with infusions, and inhibitor development) and efficacy (breakthrough bleeding and control of hemorrhaging during prophylaxis) of IB1001 in previously treated pediatric subjects with hemophilia B. Acute effects are defined as adverse events (AEs) which occur within the first 72 hours after the administration of a study product.


Original Primary Outcome: Same as current

Current Secondary Outcome:

Original Secondary Outcome:

Information By: Aptevo Therapeutics

Dates:
Date Received: January 6, 2011
Date Started: November 2010
Date Completion: May 2017
Last Updated: March 30, 2017
Last Verified: March 2017