NCT01797055

Dose Escalating Study to Evaluate Pharmacokinetics, Efficacy and Safety of Apotransferrin in Atransferrinemia Patients

Active, enrolled Phase 2 Last updated 30 July 2025

What this trial tests

Phase 2 trial testing Human apotransferrin in Congenital Atransferrinemia in 5 participants. Participants enrolled and being followed up; not accepting new ones.

Timeline

1 December 2010

Primary endpoint
31 March 2022

1 January 2028

Quick facts

Lead sponsor	Prothya Biosolutions
Phase	Phase 2
Status	Active, enrolled
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	5
Start date	1 December 2010
Primary completion	31 March 2022
Estimated completion	1 January 2028
Sites	3 locations across Germany, Italy, Spain

Drugs / interventions tested

• Human apotransferrin — full drug profile →

Conditions studied

• Congenital Atransferrinemia — all drugs for Congenital Atransferrinemia →

Sponsor

Prothya Biosolutions — full company profile →

Who can join

Eligibility, any sex, with Congenital Atransferrinemia. Patients with the condition only — healthy volunteers not accepted.

What's being measured

Primary outcomes are the specific endpoints the trial is designed to prove or disprove.

• Hemoglobin
  Time frame: 15 year
  Hemoglobin within normal range
• pharmacokinetics of transferrin
  Time frame: first infusion and year 3
  serum transferrin levels measured on several time points before and after infusion
• iron overload in organs
  Time frame: 15 years
  Elimination of iron overload (liver, heart)

Sponsor's own description

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.

Publications & conference data

2 peer-reviewed publications reference this trial (live from Europe PMC):

1. Transferrin-mediated iron sequestration as a novel therapy for bacterial and fungal infections.
   Bruhn KW, Spellberg B. · · 2015 · cited 37× · PMID 26261881 · DOI 10.1016/j.mib.2015.07.005
2. What NIR photodynamic activation offers molecular targeted nanomedicines: Perspectives into the conundrum of tumor specificity and selectivity.
   Bhandari C, Guirguis M, Savan NA, Shrivastava N, et al · · 2021 · cited 27× · PMID 33552231 · DOI 10.1016/j.nantod.2020.101052