Missing Iron-Carrying protein replaced in rare blood disorder
NCT ID NCT01797055
First seen Sep 16, 2026 · Last updated Sep 17, 2026 · Updated 1 time
Summary
Atransferrinemia is a rare inherited condition in which the body cannot make enough transferrin, a protein that moves iron through the blood. Without it, iron builds up in organs and red blood cells cannot form properly. Researchers are testing intravenous infusions of apotransferrin, a human plasma protein, in a small group of patients to see how the body processes it and whether it improves hemoglobin levels and reduces iron overload in the liver and heart.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- apotransferrin, a human plasma protein that carries iron in the blood
- What this could lead to
- If it works, apotransferrin replacement could become the first treatment that corrects the underlying protein deficiency in atransferrinemia, potentially easing anemia and organ iron overload.
- What could go wrong
- This is a tiny Phase 2 study with only a handful of participants, so results may not apply broadly. Infusions of plasma-derived proteins can trigger allergic reactions, and the trial excludes people with known plasma allergies or anti-IgA antibodies.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
-
Phase 2
Tests whether the treatment actually works, and watches for side effects, in a larger group.
- Participants
-
5 people
The number who actually took part.
- Started
-
Dec 2010
- Expected to finish
-
Jan 2028
An estimate. End dates often move.
- Lead sponsor
-
A company
The lead sponsor is a pharmaceutical, biotech, or medical-device company.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
-
Children (under 18), adults (18 to 64) and older adults (65 and over)
- Sex
-
Anyone
- Healthy volunteers
-
Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
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
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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AO San Gerardo
Monza, 20900, Italy
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Klinikum Aschaffenburg
Aschaffenburg, Germany
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Vall d'Hebron Hospital
Barcelona, Spain