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Apotransferrin in Patients With β-thalassemia
Efficacy and Safety of Human Apotransferrin in Patients With β-thalassemia Intermedia
1 other identifier
interventional
10
1 country
1
Brief Summary
The aim of the trial is to study the effect of apotransferrin administration in patients suffering from β-thalassemia intermedia in order to restore the erythropoiesis as reflected by enhanced haemoglobin levels or reduced transfusion dependency.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_2
Started Mar 2019
Typical duration for phase_2
1 active site
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
Study Start
First participant enrolled
March 21, 2019
CompletedFirst Submitted
Initial submission to the registry
May 29, 2019
CompletedFirst Posted
Study publicly available on registry
June 20, 2019
CompletedPrimary Completion
Last participant's last visit for primary outcome
March 31, 2022
CompletedStudy Completion
Last participant's last visit for all outcomes
March 31, 2022
CompletedAugust 15, 2022
August 1, 2022
3 years
May 29, 2019
August 11, 2022
Conditions
Outcome Measures
Primary Outcomes (1)
Erythropoiesis
Change of haemoglobin level and/or or change of number of RBC units transfused/week
17 weeks
Secondary Outcomes (12)
Change from baseline in serum iron
17 weeks
Change from baseline in change plasma levels of advanced glycation end products
17 weeks
Change in spleen size
at baseline and at 16 weeks
Change from baseline in reticulocyte count
17 weeks
Change from baseline in erythropoietin levels
17 weeks
- +7 more secondary outcomes
Study Arms (1)
human apotransferrin
EXPERIMENTALPatients will receive an intravenous dose of human apotransferrin every two weeks for 14-18 weeks.
Interventions
Eligibility Criteria
You may qualify if:
- 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.
You may not qualify if:
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (1)
Academic Medical Centre
Amsterdam-Zuidoost, North Holland, 1100 DD, Netherlands
Study Officials
- PRINCIPAL INVESTIGATOR
Bart Biemond, MD, PhD
Academic Medical Centre
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- NA
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
May 29, 2019
First Posted
June 20, 2019
Study Start
March 21, 2019
Primary Completion
March 31, 2022
Study Completion
March 31, 2022
Last Updated
August 15, 2022
Record last verified: 2022-08