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

Phase 1

• Conditions: Beta thalassemia intermedia; MedDRA version: 20.1Level: LLTClassification code 10054660Term: Thalassemia betaSystem Organ Class: 100000004850; MedDRA version: 20.1Level: LLTClassification code 10062923Term: Thalassemia intermediaSystem Organ Class: 100000004850; Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

• Registration Number: EUCTR2014-001936-12-NL
• Lead Sponsor: Sanquin Plasma Products BV

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2018-11-28
• Last Update Posted: 19 April 2022

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 12

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 <6.2 mmol/L, or transfusion dependent ß-thalassemia treated with a regular transfusion schedule.
2. Age >=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

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

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified