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Apotransferrin in Atransferrinemia

NCT01797055 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 5

Last updated 2025-08-03

No results posted yet for this study

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.

Conditions

  • Congenital Atransferrinemia

Interventions

DRUG

Human apotransferrin

intravenous infusion

Sponsors & Collaborators

  • Prothya Biosolutions

    lead INDUSTRY

Principal Investigators

  • Cristina R Diaz de Heredia Rubio, PhD, MD · Vall d'Hebron, Spain

  • Katja Moser, MD · klinikum Aschaffenburg, Germany

  • Raffaella Mariani, MD, PhD · AO San Gerardo Monza, Italy

  • Alberto Piperno, MD, PhD · AO San Gerardo Monza, Italy

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2010-12-31
Primary Completion
2022-03-31
Completion
2028-01-31

Countries

  • Germany
  • Italy
  • Spain

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT01797055 on ClinicalTrials.gov