Apotransferrin in Atransferrinemia

July 30, 2025 updated by: Prothya Biosolutions

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

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.

Study Overview

Status

Active, not recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

5

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Germany
      • Aschaffenburg, Germany
        • Klinikum Aschaffenburg
  • Italy
      • Monza, Italy, 20900
        • AO San Gerardo
  • Spain
      • Barcelona, Spain
        • Vall d'Hebron Hospital

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  • Established diagnosis of atransferrinemia, defined as serum levels of transferrin below 40 mg/dl
  • Informed consent

Exclusion Criteria:

  • Known with allergic reactions against human plasma or plasma products
  • Having detectable anti-immunoglobulin A antibodies

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: human apotransferrin
intravenous apotransferrin every 4-8 weeks
Drug: Human apotransferrin
intravenous infusion

What is the study measuring?

Primary Outcome Measures

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

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Iron overload
Time Frame: 15 years
Serum Ferritin within normal values
15 years
Hematocrit
Time Frame: 15 years
Hematocrit within normal values
15 years
Erythrocytes
Time Frame: 15 years
Erythrocytes within normal values
15 years

Other Outcome Measures

Outcome Measure
Measure Description
Time Frame
transferrin levels
Time Frame: 15 years
Effective transferrin levels
15 years
adverse drug reactions
Time Frame: 15 years
number of adverse drug reactions
15 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Prothya Biosolutions

Investigators

  • Principal Investigator: Alberto Piperno, MD, PhD, AO San Gerardo Monza, Italy
  • Principal Investigator: Cristina R Diaz de Heredia Rubio, PhD, MD, Vall d'Hebron, Spain
  • Principal Investigator: Katja Moser, MD, klinikum Aschaffenburg, Germany
  • Principal Investigator: Raffaella Mariani, MD, PhD, AO San Gerardo Monza, Italy

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

December 1, 2010

Primary Completion (Actual)

March 31, 2022

Study Completion (Estimated)

January 1, 2028

Study Registration Dates

First Submitted

February 20, 2013

First Submitted That Met QC Criteria

February 20, 2013

First Posted (Estimated)

February 22, 2013

Study Record Updates

Last Update Posted (Actual)

August 3, 2025

Last Update Submitted That Met QC Criteria

July 30, 2025

Last Verified

July 1, 2025

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • MD2009.04

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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