Clinical Trial: Apotransferrin in Atransferrinemia
Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional
Official Title: Dose Escalating Study to Evaluate Pharmacokinetics, Efficacy and Safety of Apotransferrin in Atransferrinemia Patients
Brief Summary: Atransferrinemia is a very rare disorder, which is caused by a deficiency of the protein transferrin. No regular treatment is available for these patients. The objective of this study is to investigate the pharmacokinetics, efficacy and safety of Apotransferrin replacement therapy in atransferrinemia patients.
Detailed Summary:
Sponsor: Sanquin
Current Primary Outcome:
- Hemoglobulin [ Time Frame: 2 year ]
- pharmacokinetics [ Time Frame: first infusion ]serum transferrin levels measured on several time points before and after infusion
Original Primary Outcome: Hb normal [ Time Frame: 2 year ]
Current Secondary Outcome: iron overload in organs [ Time Frame: 2 years ]
Original Secondary Outcome: iron overload stable or decreased [ Time Frame: 2 years ]
Information By: Sanquin
Dates:
Date Received: February 20, 2013
Date Started: December 2010
Date Completion: January 2019
Last Updated: May 12, 2017
Last Verified: May 2017
