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Apotransferrin in Patients With β-thalassemia

Primary Purpose

β-thalassemia Intermedia

Status

Terminated

Phase

Phase 2

Locations

Netherlands

Study Type

Interventional

Intervention

human apotransferrin

About this trial

This is an interventional treatment trial for β-thalassemia Intermedia

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

Academic Medical Centre

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

human apotransferrin

Arm Description

Patients will receive an intravenous dose of human apotransferrin every two weeks for 14-18 weeks.

Outcomes

Primary Outcome Measures

Erythropoiesis

Change of haemoglobin level and/or or change of number of RBC units transfused/week

Secondary Outcome Measures

Change from baseline in serum iron

Change from baseline in change plasma levels of advanced glycation end products

Change in spleen size

Change from baseline in reticulocyte count

Change from baseline in erythropoietin levels

Ctrough

Ctrough calculated from serum transferrin levels

Cmin

Cmin calculated from serum transferrin levels

tmax

tmax calculated from serum transferrin levels

Cmax

Cmax calculated from serum transferrin levels

AUCτ

AUCτ calculated from serum transferrin levels

Ctrough

Ctrough calculated from serum transferrin levels

Adverse events

Number of adverse events

Full Information

NCT ID

NCT03993613

First Posted

May 29, 2019

Last Updated

August 11, 2022

Sponsor

Prothya Biosolutions

1. Study Identification

Unique Protocol Identification Number

NCT03993613

Brief Title

Apotransferrin in Patients With β-thalassemia

Official Title

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

Study Type

Interventional

2. Study Status

Record Verification Date

August 2022

Overall Recruitment Status

Terminated

Why Stopped

no availability of IMP for this study

Study Start Date

March 21, 2019 (Actual)

Primary Completion Date

March 31, 2022 (Actual)

Study Completion Date

March 31, 2022 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Prothya Biosolutions

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

β-thalassemia Intermedia

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

10 (Actual)

8. Arms, Groups, and Interventions

Arm Title

human apotransferrin

Arm Type

Experimental

Arm Description

Patients will receive an intravenous dose of human apotransferrin every two weeks for 14-18 weeks.

Intervention Type

Biological

Intervention Name(s)

human apotransferrin

Intervention Description

Intravenous infusions

Primary Outcome Measure Information:

Title

Erythropoiesis

Description

Change of haemoglobin level and/or or change of number of RBC units transfused/week

Time Frame

17 weeks

Secondary Outcome Measure Information:

Title

Change from baseline in serum iron

Time Frame

17 weeks

Title

Change from baseline in change plasma levels of advanced glycation end products

Time Frame

17 weeks

Title

Change in spleen size

Time Frame

at baseline and at 16 weeks

Title

Change from baseline in reticulocyte count

Time Frame

17 weeks

Title

Change from baseline in erythropoietin levels

Time Frame

17 weeks

Title

Ctrough

Description

Ctrough calculated from serum transferrin levels

Time Frame