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Apotransferrin in Patients With β-thalassemia

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03993613
Recruitment Status : Recruiting
First Posted : June 20, 2019
Last Update Posted : June 20, 2019
Sponsor:
Information provided by (Responsible Party):
Sanquin Plasma Products BV

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.

Condition or disease Intervention/treatment Phase
β-thalassemia Intermedia Biological: human apotransferrin Phase 2

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 12 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Efficacy and Safety of Human Apotransferrin in Patients With β-thalassemia Intermedia
Actual Study Start Date : March 21, 2019
Estimated Primary Completion Date : May 1, 2020
Estimated Study Completion Date : May 1, 2021

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Thalassemia

Arm Intervention/treatment
Experimental: human apotransferrin
Patients will receive one intravenous loading dose of human apotransferrin on Day -1 followed by the maintenance dose every two weeks from Day 0 onwards for 14 weeks.
Biological: human apotransferrin
Intravenous infusions




Primary Outcome Measures :
  1. Erythropoiesis [ Time Frame: 17 weeks ]
    Change of haemoglobin level and/or or change of number of RBC units transfused/week


Secondary Outcome Measures :
  1. Change from baseline in serum iron [ Time Frame: 17 weeks ]
  2. Change from baseline in change plasma levels of advanced glycation end products [ Time Frame: 17 weeks ]
  3. Change in spleen size [ Time Frame: at baseline and at 16 weeks ]
  4. Change from baseline in reticulocyte count [ Time Frame: 17 weeks ]
  5. Change from baseline in erythropoietin levels [ Time Frame: 17 weeks ]
  6. Ctrough [ Time Frame: predose and postdose 5 minutes, 2 hours and 1, 4, 7, 14 days ]
    Ctrough calculated from serum transferrin levels

  7. Cmin [ Time Frame: predose and postdose 5 minutes, 2 hours and 1, 4, 7, 14 days ]
    Cmin calculated from serum transferrin levels

  8. tmax [ Time Frame: predose and postdose 5 minutes, 2 hours and 1, 4, 7, 14 days ]
    tmax calculated from serum transferrin levels

  9. Cmax [ Time Frame: predose and postdose 5 minutes, 2 hours and 1, 4, 7, 14 days ]
    Cmax calculated from serum transferrin levels

  10. AUCτ [ Time Frame: predose and postdose 5 minutes, 2 hours and 1, 4, 7, 14 days ]
    AUCτ calculated from serum transferrin levels

  11. Ctrough [ Time Frame: predose ]
    Ctrough calculated from serum transferrin levels

  12. Adverse events [ Time Frame: 17 weeks ]
    Number of adverse events



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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03993613


Contacts
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Contact: Ilona Kleine Budde, PhD +31205123537 i.kleinebudde@sanquin.nl
Contact: Roel Romij hemat.trial@amc.uva.nl

Locations
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Netherlands
Academic Medical Centre Recruiting
Amsterdam-Zuidoost, Noord-Holland, Netherlands, 1100 DD
Contact: Bart J Biemond, PhD, MD    +31 20 566 5785    b.j.biemond@amc.uva.nl   
Principal Investigator: Bart J Biemond, MD,PhD         
Sponsors and Collaborators
Sanquin Plasma Products BV
Investigators
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Principal Investigator: Bart Biemond, MD, PhD Academic Medical Centre

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Responsible Party: Sanquin Plasma Products BV
ClinicalTrials.gov Identifier: NCT03993613    
Other Study ID Numbers: MD2014.01
First Posted: June 20, 2019    Key Record Dates
Last Update Posted: June 20, 2019
Last Verified: June 2019

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Thalassemia
beta-Thalassemia
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Anemia
Hematologic Diseases
Hemoglobinopathies
Genetic Diseases, Inborn