Apotransferrin in Patients With β-thalassemia

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

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.

Study Overview

• Status: Terminated
• Conditions: β-thalassemia Intermedia
• Intervention / Treatment: Biological: human apotransferrin

• Study Type: Interventional
• Enrollment (Actual): 10
• Phase: Phase 2

Contacts and Locations

Study Locations

• Netherlands
  • Noord-Holland
    • Amsterdam-Zuidoost, Noord-Holland, Netherlands, 1100 DD
      • Academic Medical Centre

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (ADULT, OLDER_ADULT)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• 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.

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: TREATMENT
• Allocation: NA
• Interventional Model: SINGLE_GROUP
• Masking: NONE

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
EXPERIMENTAL: human apotransferrin; Patients will receive an intravenous dose of human apotransferrin every two weeks for 14-18 weeks.	Biological: human apotransferrin; Intravenous infusions

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Erythropoiesis	Change of haemoglobin level and/or or change of number of RBC units transfused/week	17 weeks

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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
Ctrough	Ctrough calculated from serum transferrin levels	predose and postdose 5 minutes, 2 hours and 1, 4, 7, 14 days
Cmin	Cmin calculated from serum transferrin levels	predose and postdose 5 minutes, 2 hours and 1, 4, 7, 14 days
tmax	tmax calculated from serum transferrin levels	predose and postdose 5 minutes, 2 hours and 1, 4, 7, 14 days
Cmax	Cmax calculated from serum transferrin levels	predose and postdose 5 minutes, 2 hours and 1, 4, 7, 14 days
AUCτ	AUCτ calculated from serum transferrin levels	predose and postdose 5 minutes, 2 hours and 1, 4, 7, 14 days
Ctrough	Ctrough calculated from serum transferrin levels	predose
Adverse events	Number of adverse events	17 weeks

Collaborators and Investigators

• Sponsor: Prothya Biosolutions
• Investigators: Principal Investigator: Bart Biemond, MD, PhD, Academic Medical Centre

Study record dates

Study Major Dates

• Study Start (ACTUAL): March 21, 2019
• Primary Completion (ACTUAL): March 31, 2022
• Study Completion (ACTUAL): March 31, 2022

Study Registration Dates

• First Submitted: May 29, 2019
• First Submitted That Met QC Criteria: June 19, 2019
• First Posted (ACTUAL): June 20, 2019

Study Record Updates

• Last Update Posted (ACTUAL): August 15, 2022
• Last Update Submitted That Met QC Criteria: August 11, 2022
• Last Verified: August 1, 2022

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Hematologic Diseases; Genetic Diseases, Inborn; Anemia; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Hemoglobinopathies; Thalassemia; beta-Thalassemia
• Other Study ID Numbers: MD2014.01

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No