A Phase 2 interventional study of Human apotransferrin in Congenital Atransferrinemia, sponsored by Prothya Biosolutions. Active, not recruiting at 3 sites in 3 countries. Per ClinicalTrials.gov, last updated 2026-09-16.
Sponsored by Prothya Biosolutions · Phase 2, Interventional, and Treatment
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.
1,810 studies on the registry are indexed under Anemia; 215 are open to participants now.
This study's enrollment of 5 is below the median of 117 across 1,336 interventional studies indexed under Anemia.
Browse Anemia studies →Prothya Biosolutions is the lead sponsor of 15 studies on the registry; none are open to participants now.
Counted across the registry records on this site, refreshed daily.
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Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
intravenous apotransferrin every 4-8 weeks
Drug: Human apotransferrin
intravenous infusion
Hemoglobin
Hemoglobin within normal range
Time frame: 15 year
pharmacokinetics of transferrin
serum transferrin levels measured on several time points before and after infusion
Time frame: first infusion and year 3
iron overload in organs
Elimination of iron overload (liver, heart)
Time frame: 15 years
Iron overload
Serum Ferritin within normal values
Time frame: 15 years
Hematocrit
Hematocrit within normal values
Time frame: 15 years
Erythrocytes
Erythrocytes within normal values
Time frame: 15 years
transferrin levels
Effective transferrin levels
Time frame: 15 years
adverse drug reactions
number of adverse drug reactions
Time frame: 15 years
Ran from Dec 2010 to primary completion in Mar 2022, 135 months.
Of 58 industry-sponsored phase 2 interventional studies under Anemia, half reached primary completion within 23 months of starting (the median), counting those still running; a quarter within 13 and three quarters within 40. This study's 135 months is above that.
Counting every sponsor, half of 88 studies reached it within 28 months; a quarter within 16 and three quarters within 56.
How these figures are worked out, and which studies are left out of them.
This study is active, not recruiting, as verified in Sep 2026. You cannot join it, but the record below documents what was studied.
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Prothya Biosolutions