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出境医 / 临床实验 / Apotransferrin in Patients With β-thalassemia
Apotransferrin in Patients With β-thalassemia
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.
| Condition or disease | Intervention/treatment | Phase |
|---|---|---|
| β-thalassemia Intermedia | Biological: human apotransferrin | Phase 2 |
Study Design
| Study Type : | Interventional (Clinical Trial) |
| Estimated Enrollment : | 12 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 |
| Estimated Primary Completion Date : | March 1, 2021 |
| Estimated Study Completion Date : | March 1, 2021 |
Arms and Interventions
| 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
Primary Outcome Measures :
- Erythropoiesis [ Time Frame: 17 weeks ]Change of haemoglobin level and/or or change of number of RBC units transfused/week
Secondary Outcome Measures :
- Change from baseline in serum iron [ Time Frame: 17 weeks ]
- Change from baseline in change plasma levels of advanced glycation end products [ Time Frame: 17 weeks ]
- Change in spleen size [ Time Frame: at baseline and at 16 weeks ]
- Change from baseline in reticulocyte count [ Time Frame: 17 weeks ]
- Change from baseline in erythropoietin levels [ Time Frame: 17 weeks ]
- Ctrough [ Time Frame: predose and postdose 5 minutes, 2 hours and 1, 4, 7, 14 days ]Ctrough calculated from serum transferrin levels
- Cmin [ Time Frame: predose and postdose 5 minutes, 2 hours and 1, 4, 7, 14 days ]Cmin calculated from serum transferrin levels
- tmax [ Time Frame: predose and postdose 5 minutes, 2 hours and 1, 4, 7, 14 days ]tmax calculated from serum transferrin levels
- Cmax [ Time Frame: predose and postdose 5 minutes, 2 hours and 1, 4, 7, 14 days ]Cmax calculated from serum transferrin levels
- AUCτ [ Time Frame: predose and postdose 5 minutes, 2 hours and 1, 4, 7, 14 days ]AUCτ calculated from serum transferrin levels
- Ctrough [ Time Frame: predose ]Ctrough calculated from serum transferrin levels
- Adverse events [ Time Frame: 17 weeks ]Number of adverse events
Eligibility Criteria
| 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
Contacts
| Contact: Ilona Kleine Budde, PhD | +31205123537 | i.kleinebudde@sanquin.nl | |
| Contact: Roel Romij | hemat.trial@amc.uva.nl |
Locations
| 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
| Principal Investigator: | Bart Biemond, MD, PhD | Academic Medical Centre |
| Tracking Information | |||||||||
|---|---|---|---|---|---|---|---|---|---|
| First Submitted Date ICMJE | May 29, 2019 | ||||||||
| First Posted Date ICMJE | June 20, 2019 | ||||||||
| Last Update Posted Date | October 23, 2020 | ||||||||
| Actual Study Start Date ICMJE | March 21, 2019 | ||||||||
| Estimated Primary Completion Date | March 1, 2021 (Final data collection date for primary outcome measure) | ||||||||
| Current Primary Outcome Measures ICMJE |
Erythropoiesis [ Time Frame: 17 weeks ] Change of haemoglobin level and/or or change of number of RBC units transfused/week
|
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| Original Primary Outcome Measures ICMJE | Same as current | ||||||||
| Change History | |||||||||
| Current Secondary Outcome Measures ICMJE |
|
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| Original Secondary Outcome Measures ICMJE | Same as current | ||||||||
| Current Other Pre-specified Outcome Measures | Not Provided | ||||||||
| Original Other Pre-specified Outcome Measures | Not Provided | ||||||||
| Descriptive Information | |||||||||
| Brief Title ICMJE | Apotransferrin in Patients With β-thalassemia | ||||||||
| Official Title ICMJE | Efficacy and Safety of Human Apotransferrin in Patients With β-thalassemia Intermedia | ||||||||
| 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. | ||||||||
| Detailed Description | Not Provided | ||||||||
| Study Type ICMJE | Interventional | ||||||||
| Study Phase ICMJE | Phase 2 | ||||||||
| Study Design ICMJE | Allocation: N/A Intervention Model: Single Group Assignment Masking: None (Open Label) Primary Purpose: Treatment |
||||||||
| Condition ICMJE | β-thalassemia Intermedia | ||||||||
| Intervention ICMJE | Biological: human apotransferrin
Intravenous infusions
|
||||||||
| Study Arms ICMJE | Experimental: human apotransferrin
Patients will receive an intravenous dose of human apotransferrin every two weeks for 14-18 weeks.
Intervention: Biological: human apotransferrin
|
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| Publications * | Not Provided | ||||||||
|
* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline. |
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| Recruitment Information | |||||||||
| Recruitment Status ICMJE | Recruiting | ||||||||
| Estimated Enrollment ICMJE |
12 | ||||||||
| Original Estimated Enrollment ICMJE | Same as current | ||||||||
| Estimated Study Completion Date ICMJE | March 1, 2021 | ||||||||
| Estimated Primary Completion Date | March 1, 2021 (Final data collection date for primary outcome measure) | ||||||||
| Eligibility Criteria ICMJE |
Inclusion Criteria:
Exclusion Criteria:
|
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| Sex/Gender ICMJE |
|
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| Ages ICMJE | 18 Years and older (Adult, Older Adult) | ||||||||
| Accepts Healthy Volunteers ICMJE | No | ||||||||
| Contacts ICMJE |
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| Listed Location Countries ICMJE | Netherlands | ||||||||
| Removed Location Countries | |||||||||
| Administrative Information | |||||||||
| NCT Number ICMJE | NCT03993613 | ||||||||
| Other Study ID Numbers ICMJE | MD2014.01 | ||||||||
| Has Data Monitoring Committee | No | ||||||||
| U.S. FDA-regulated Product |
|
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| IPD Sharing Statement ICMJE | Not Provided | ||||||||
| Responsible Party | Sanquin Plasma Products BV | ||||||||
| Study Sponsor ICMJE | Sanquin Plasma Products BV | ||||||||
| Collaborators ICMJE | Not Provided | ||||||||
| Investigators ICMJE |
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| PRS Account | Sanquin Plasma Products BV | ||||||||
| Verification Date | October 2020 | ||||||||
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ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP |
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