Apotransferrin in Patients With β-thalassemia

ß-thalassemia Intermedia Clinical Trial

Official title:

Efficacy and Safety of Human Apotransferrin in Patients With β-thalassemia Intermedia

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT03993613
• Other study ID #: MD2014.01
• Status: Terminated
• Phase: Phase 2
• Start date: March 21, 2019
• Est. completion date: March 31, 2022

Study information

• Verified date: August 2022
• Source: Prothya Biosolutions
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 10
• Est. completion date: March 31, 2022
• Est. primary completion date: March 31, 2022
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

Inclusion Criteria:

• Non-transfusion dependent ß-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 ß-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.

Exclusion Criteria:

• 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

Related Conditions & MeSH terms

• beta-Thalassemia
• ß-thalassemia Intermedia
• Thalassemia

Intervention

Biological:
• human apotransferrin
Intravenous infusions

Locations

Country	Name	City	State
Netherlands	Academic Medical Centre	Amsterdam-Zuidoost	Noord-Holland

Sponsors (1)

Lead Sponsor	Collaborator
Prothya Biosolutions

Country where clinical trial is conducted

Netherlands, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Erythropoiesis	Change of haemoglobin level and/or or change of number of RBC units transfused/week	17 weeks
Secondary	Change from baseline in serum iron		17 weeks
Secondary	Change from baseline in change plasma levels of advanced glycation end products		17 weeks
Secondary	Change in spleen size		at baseline and at 16 weeks
Secondary	Change from baseline in reticulocyte count		17 weeks
Secondary	Change from baseline in erythropoietin levels		17 weeks
Secondary	Ctrough	Ctrough calculated from serum transferrin levels	predose and postdose 5 minutes, 2 hours and 1, 4, 7, 14 days
Secondary	Cmin	Cmin calculated from serum transferrin levels	predose and postdose 5 minutes, 2 hours and 1, 4, 7, 14 days
Secondary	tmax	tmax calculated from serum transferrin levels	predose and postdose 5 minutes, 2 hours and 1, 4, 7, 14 days
Secondary	Cmax	Cmax calculated from serum transferrin levels	predose and postdose 5 minutes, 2 hours and 1, 4, 7, 14 days
Secondary	AUCt	AUCt calculated from serum transferrin levels	predose and postdose 5 minutes, 2 hours and 1, 4, 7, 14 days
Secondary	Ctrough	Ctrough calculated from serum transferrin levels	predose
Secondary	Adverse events	Number of adverse events	17 weeks