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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. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03993613
Recruitment Status : Terminated (no availability of IMP for this study)
First Posted : June 20, 2019
Last Update Posted : August 15, 2022
Sponsor:
Information provided by (Responsible Party):
Prothya Biosolutions

Study Description
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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

Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 10 participants
Allocation: N/A
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
Actual Primary Completion Date : March 31, 2022
Actual Study Completion Date : March 31, 2022

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Thalassemia

Arms and Interventions
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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


Outcome Measures
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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


Eligibility Criteria
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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
Contacts and Locations
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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


Locations
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Netherlands
Academic Medical Centre
Amsterdam-Zuidoost, Noord-Holland, Netherlands, 1100 DD
Sponsors and Collaborators
Prothya Biosolutions
Investigators
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Principal Investigator: Bart Biemond, MD, PhD Academic Medical Centre
More Information
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Responsible Party: Prothya Biosolutions
ClinicalTrials.gov Identifier: NCT03993613    
Other Study ID Numbers: MD2014.01
First Posted: June 20, 2019    Key Record Dates
Last Update Posted: August 15, 2022
Last Verified: August 2022

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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