The effect and safety of human apotransferrin in patients with ß-thalassemia

Efficacy and Safety of human apotransferrin in patients with ß-thalassemia - Apotransferrin in patients with ß-thalassemia

Status

Active, not recruiting

Phases

Phase 2

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2014-001936-12-NL

Enrollment

Registered

2018-11-28

Start date

2019-02-11

Completion date

Unknown

Last updated

2022-04-19

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Beta thalassemia intermedia MedDRA version: 20.1 Level: LLT Classification code 10054660 Term: Thalassemia beta System Organ Class: 100000004850 MedDRA version: 20.1 Level: LLT Classification code 10062923 Term: Thalassemia intermedia System Organ Class: 100000004850

Interventions

Product Name: human apotransferrin Product Code: apotransferrin Pharmaceutical Form: Solution for infusion INN or Proposed INN: Human Apotransferrin Other descriptive name: APOTRANSFERRIN Concentratio

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: 1. Non-transfusion dependent ß-thalassemia intermedia, defined as patients with microcytic anemia in combination with an elevated HbA2 (>2.5%) and a hemoglobin of =18 years 3. Adequate renal and hepatic function tests as indicated by the following laboratory values: •Serum creatinine =1.0 mg/dl (= 88.7 µmol/L); if serum creatinine >1.0 mg/dl (>88.7 µmol/L), then the glomerular filtration rate (GFR) must be >60 ml/min/1.73 m2 as calculated by the Modification of Diet in Renal Disease equation where the predicted GFR (ml/min/1.73 m2) = 186 x (Serum Creatinine in mg/dl) -1.154 x (age in years) -0.203 x (0.742 if patient is female) x (1.212 if patient is black) NOTE: if serum creatinine is measured in µmol/L, recalculate it in mg/dl according to the equation: 1 mg/dl = 88.7 µmol/L) and used above mentioned formula. • Aspartate transaminase (AST)/alanine transaminase (ALT) =2.5 × ULN • Alkaline phosphatase (AP) = 2.5 × ULN 4. WHO performance 0, 1 or 2. 5. Signed informed consent. Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 12 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range

Exclusion criteria

Exclusion criteria: 1.Known with allergic reactions against human plasma or plasma products. 2.Concurrent severe and/or uncontrolled medical condition (e.g. uncontrolled diabetes, infection, hypertension, pulmonary disease). 3.Cardiac dysfunction as defined by: myocardial infarction within the last 6 months of study entry, unstable angina, or unstable cardiac arrhythmias. 4.Pregnant or lactating females. 5.Known with IgA deficiency with anti-IgA antibodies

Design outcomes

Primary

Measure	Time frame
Main Objective: To study the effect of apotransferrin administration in patients with ß thalassemia intermedia on erythropoiesis as reflected by hemoglobin level or transfusion dependency. ;Secondary Objective: Secondary objectives are the effect of apotransferrin on transferrin plasma levels, iron metabolism, oxidative stress, and erythropoiesis. Moreover the safety of apotransferrin transfusion will be studied.;Primary end point(s): Hematological response defined as the change from baseline of Hb level, expressed as mmol/L and %, in NTDßTI and TDßTI patients or change from baseline of number of RBC in TDßTI patients;Timepoint(s) of evaluation of this end point: Ad 1: Day 0, week 2, 4, 6, 8, 10, 12 , 14, 16 in NTDßTI and TDßTI patients, and in TDßTI patients week 18 and 20 as well Ad 2: study period of 20 weeks	—

Measure

Time frame

Main Objective: To study the effect of apotransferrin administration in patients with ß thalassemia intermedia on erythropoiesis as reflected by hemoglobin level or transfusion dependency. ;Secondary Objective: Secondary objectives are the effect of apotransferrin on transferrin plasma levels, iron metabolism, oxidative stress, and erythropoiesis. Moreover the safety of apotransferrin transfusion will be studied.;Primary end point(s): Hematological response defined as the change from baseline of Hb level, expressed as mmol/L and %, in NTDßTI and TDßTI patients or change from baseline of number of RBC in TDßTI patients;Timepoint(s) of evaluation of this end point: Ad 1: Day 0, week 2, 4, 6, 8, 10, 12 , 14, 16 in NTDßTI and TDßTI patients, and in TDßTI patients week 18 and 20 as well Ad 2: study period of 20 weeks

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Secondary

Measure	Time frame
Secondary end point(s): 1. Increase of > 0.9 mmol/l (1.5 g/dl) in NTDßTI and TDßTI (before transfusion) 2. The reduction of at least 50% of the number of RBC units transfused/week from baseline in TDßTI 3. Pharmacokinetics of transferrin 4. Increase iron metabolism as reflected by serum iron, hepcidin, NTBI levels and iron saturation. 5. Oxidative stress as assessed by plasma levels of advanced glycation end products (AGEs) 6. The effect on markers of erythropoiesis like reticulocyte count, erythropoietin levels and red cell indices 7. All adverse events (number, type) will be analysed regarding to causality, seriousness, outcome and expectedness. ;Timepoint(s) of evaluation of this end point: Ad 1: Day 0, week 2, 4, 6, 8, 10, 12 , 14, 16 and for TDßTI week 18, 20 as well Ad 2: study period of 20 weeks Ad 3: before every infusion, and 5 minutes, 2 hours and 1, 4, 7, and 14 days after the stop of infusion no.4, week 16 (NTDßTI) and week 20 (TDßTI) and subsequently till transferrin levels are back to normal Add 4-6: Day 0, week 2, 4, 6, 8, 10, 12 , 14, 16 and for TDßTI week 18, 20 as well Ad 7: study period of 16 weeks (NTDßTI) or 20 weeks (TDßTI) or till transferrin levels are back to normal.	—

Measure

Time frame

Secondary end point(s): 1. Increase of > 0.9 mmol/l (1.5 g/dl) in NTDßTI and TDßTI (before transfusion) 2. The reduction of at least 50% of the number of RBC units transfused/week from baseline in TDßTI 3. Pharmacokinetics of transferrin 4. Increase iron metabolism as reflected by serum iron, hepcidin, NTBI levels and iron saturation. 5. Oxidative stress as assessed by plasma levels of advanced glycation end products (AGEs) 6. The effect on markers of erythropoiesis like reticulocyte count, erythropoietin levels and red cell indices 7. All adverse events (number, type) will be analysed regarding to causality, seriousness, outcome and expectedness. ;Timepoint(s) of evaluation of this end point: Ad 1: Day 0, week 2, 4, 6, 8, 10, 12 , 14, 16 and for TDßTI week 18, 20 as well Ad 2: study period of 20 weeks Ad 3: before every infusion, and 5 minutes, 2 hours and 1, 4, 7, and 14 days after the stop of infusion no.4, week 16 (NTDßTI) and week 20 (TDßTI) and subsequently till transferrin levels are back to normal Add 4-6: Day 0, week 2, 4, 6, 8, 10, 12 , 14, 16 and for TDßTI week 18, 20 as well Ad 7: study period of 16 weeks (NTDßTI) or 20 weeks (TDßTI) or till transferrin levels are back to normal.

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Countries

Netherlands

Contacts

Public ContactIlona Kleine Budde

Sanquin Plasma Products BV

i.kleinebudde@sanquin.nl+31205123537

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026