Apotransferrin in Patients With β-thalassemia
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|ClinicalTrials.gov Identifier: NCT03993613|
Recruitment Status : Terminated (no availability of IMP for this study)
First Posted : June 20, 2019
Last Update Posted : August 15, 2022
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|Condition or disease||Intervention/treatment||Phase|
|β-thalassemia Intermedia||Biological: human apotransferrin||Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||10 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Efficacy and Safety of Human Apotransferrin in Patients With β-thalassemia Intermedia|
|Actual Study Start Date :||March 21, 2019|
|Actual Primary Completion Date :||March 31, 2022|
|Actual Study Completion Date :||March 31, 2022|
Experimental: human apotransferrin
Patients will receive an intravenous dose of human apotransferrin every two weeks for 14-18 weeks.
Biological: human apotransferrin
- Erythropoiesis [ Time Frame: 17 weeks ]Change of haemoglobin level and/or or change of number of RBC units transfused/week
- Change from baseline in serum iron [ Time Frame: 17 weeks ]
- Change from baseline in change plasma levels of advanced glycation end products [ Time Frame: 17 weeks ]
- Change in spleen size [ Time Frame: at baseline and at 16 weeks ]
- Change from baseline in reticulocyte count [ Time Frame: 17 weeks ]
- Change from baseline in erythropoietin levels [ Time Frame: 17 weeks ]
- Ctrough [ Time Frame: predose and postdose 5 minutes, 2 hours and 1, 4, 7, 14 days ]Ctrough calculated from serum transferrin levels
- Cmin [ Time Frame: predose and postdose 5 minutes, 2 hours and 1, 4, 7, 14 days ]Cmin calculated from serum transferrin levels
- tmax [ Time Frame: predose and postdose 5 minutes, 2 hours and 1, 4, 7, 14 days ]tmax calculated from serum transferrin levels
- Cmax [ Time Frame: predose and postdose 5 minutes, 2 hours and 1, 4, 7, 14 days ]Cmax calculated from serum transferrin levels
- AUCτ [ Time Frame: predose and postdose 5 minutes, 2 hours and 1, 4, 7, 14 days ]AUCτ calculated from serum transferrin levels
- Ctrough [ Time Frame: predose ]Ctrough calculated from serum transferrin levels
- Adverse events [ Time Frame: 17 weeks ]Number of adverse events
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|Ages Eligible for Study:||18 Years and older (Adult, Older Adult)|
|Sexes Eligible for Study:||All|
|Accepts Healthy Volunteers:||No|
- Non-transfusion dependent β-thalassemia intermedia, defined as patients with microcytic anaemia in combination with an elevated HbA2 (>2.5%) and a haemoglobin of <6.2 mmol/L, or transfusion dependent β-thalassemia treated with a regular transfusion schedule.
- Age above≥ 17 years.
- Adequate renal and hepatic function tests
- WHO performance 0, 1 or 2.
- Signed informed consent.
- Known with allergic reactions against human plasma or plasma products.
- Concurrent severe and/or uncontrolled medical condition (e.g. uncontrolled diabetes, infection, hypertension, pulmonary disease).
- Cardiac dysfunction as defined by: myocardial infarction within the last 6 months of study entry, unstable angina, or unstable cardiac arrhythmias.
- Pregnant or lactating females.
- Known with IgA deficiency with anti-IgA antibodies
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03993613
|Academic Medical Centre|
|Amsterdam-Zuidoost, Noord-Holland, Netherlands, 1100 DD|
|Principal Investigator:||Bart Biemond, MD, PhD||Academic Medical Centre|
|Responsible Party:||Prothya Biosolutions|
|Other Study ID Numbers:||
|First Posted:||June 20, 2019 Key Record Dates|
|Last Update Posted:||August 15, 2022|
|Last Verified:||August 2022|
|Studies a U.S. FDA-regulated Drug Product:||No|
|Studies a U.S. FDA-regulated Device Product:||No|
Anemia, Hemolytic, Congenital
Genetic Diseases, Inborn