Apotransferrin in Atransferrinemia
Phase 2
Active, not recruiting
- Conditions
- Congenital Atransferrinemia
- Interventions
- Registration Number
- NCT01797055
- Lead Sponsor
- Prothya Biosolutions
- Brief 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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ACTIVE_NOT_RECRUITING
- Sex
- All
- Target Recruitment
- 5
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 & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description human apotransferrin Human apotransferrin intravenous apotransferrin every 4-8 weeks
- Primary Outcome Measures
Name Time Method pharmacokinetics of transferrin first infusion and year 3 serum transferrin levels measured on several time points before and after infusion
Hemoglobin 10 year Hemoglobin within normal range
iron overload in organs 10 years Elimination of iron overload (liver, heart)
- Secondary Outcome Measures
Name Time Method Iron overload 10 years Serum Ferritin within normal values
Hematocrit 10 years Hematocrit within normal values
Erythrocytes 10 years Erythrocytes within normal values
Trial Locations
- Locations (3)
AO San Gerardo
🇮🇹Monza, Italy
Klinikum Aschaffenburg
🇩🇪Aschaffenburg, Germany
Vall d'Hebron Hospital
🇪🇸Barcelona, Spain