β-thalassemia IntermediaPhase 2NCT03993613
Apotransferrin in Patients With β-thalassemia
A Phase 2 study testing human apotransferrin in people with β-thalassemia Intermedia, run by Prothya Biosolutions at 1 site in Netherlands.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- β-thalassemia Intermedia
- What is being tested?
- human apotransferrin
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 1 siteNetherlands
- Study dates
- 21 Mar 2019to 31 Mar 2022
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country.
Netherlands1 site
Academic Medical Centre
Amsterdam-Zuidoost, North Holland, 1100 DD
About this study
Official title: Efficacy and Safety of Human Apotransferrin in Patients With β-thalassemia Intermedia
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.
- Conditions
- β-thalassemia Intermedia
- Being tested
- human apotransferrin
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
human apotransferrinBiological
Intravenous infusions
| Study group | Role | What they receive |
|---|---|---|
| human apotransferrin | Experimental | Patients will receive an intravenous dose of human apotransferrin every two weeks for 14-18 weeks. Receives: Biological: human apotransferrin |
What is being measured
The primary outcome is the main question the study is designed to answer.
Erythropoiesis
Change of haemoglobin level and/or or change of number of RBC units transfused/week
Measured: 17 weeks
Secondary outcomes (12)
Change from baseline in serum iron
Measured: 17 weeks
Change from baseline in change plasma levels of advanced glycation end products
Measured: 17 weeks
Change in spleen size
Measured: at baseline and at 16 weeks
Change from baseline in reticulocyte count
Measured: 17 weeks
Change from baseline in erythropoietin levels
Measured: 17 weeks
Ctrough
Ctrough calculated from serum transferrin levels
Measured: predose and postdose 5 minutes, 2 hours and 1, 4, 7, 14 days
Cmin
Cmin calculated from serum transferrin levels
Measured: predose and postdose 5 minutes, 2 hours and 1, 4, 7, 14 days
tmax
tmax calculated from serum transferrin levels
Measured: predose and postdose 5 minutes, 2 hours and 1, 4, 7, 14 days
Cmax
Cmax calculated from serum transferrin levels
Measured: predose and postdose 5 minutes, 2 hours and 1, 4, 7, 14 days
AUCτ
AUCτ calculated from serum transferrin levels
Measured: predose and postdose 5 minutes, 2 hours and 1, 4, 7, 14 days
Ctrough
Ctrough calculated from serum transferrin levels
Measured: predose
Adverse events
Number of adverse events
Measured: 17 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Jun 2019
- Study start21 Mar 2019
- Primary completion31 Mar 2022
- Study completion31 Mar 2022
Why it stopped: no availability of IMP for this study
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for β-thalassemia IntermediaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 10 participants (actual)
Sponsor and investigators
- Lead sponsor
- Prothya Biosolutions
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Bart Biemond, MD, PhD, Principal investigator, Academic Medical Centre
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03993613
- Sponsor's protocol ID
- MD2014.01
- Status last verified
- Aug 2022
- First submitted
- 29 May 2019
- Last updated
- 15 Aug 2022
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 15 Aug 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Efficacy and Safety of Human Apotransferrin in Patients With β-thalassemia Intermedia
- ClinicalTrials.gov ID
- NCT03993613
- Acronym / sponsor ID
- MD2014.01
- Conditions
- β-thalassemia Intermedia
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: no availability of IMP for this study
- Enrolment
- 10 participants (actual)
- Lead sponsor
- Prothya BiosolutionsIndustry (pharma, biotech, devices)
- Study start
- 21 Mar 2019 (actual)
- Primary completion
- 31 Mar 2022 (actual)
- Study completion
- 31 Mar 2022 (actual)
- First posted
- 20 Jun 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| human apotransferrin | Experimental | Biological: human apotransferrinPatients will receive an intravenous dose of human apotransferrin every two weeks for 14-18 weeks. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- 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.
Key exclusion criteria (5 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| human apotransferrinIntravenous infusions | Biological | human apotransferrin |
Primary outcome
Erythropoiesis
Time frame: 17 weeks
Change of haemoglobin level and/or or change of number of RBC units transfused/week
12 secondary outcomes
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
Cmin
Time frame: predose and postdose 5 minutes, 2 hours and 1, 4, 7, 14 days
tmax
Time frame: predose and postdose 5 minutes, 2 hours and 1, 4, 7, 14 days
Cmax
Time frame: predose and postdose 5 minutes, 2 hours and 1, 4, 7, 14 days
AUCτ
Time frame: predose and postdose 5 minutes, 2 hours and 1, 4, 7, 14 days
Ctrough
Time frame: predose
Adverse events
Time frame: 17 weeks
Results and safety
The study was stopped early (reason given: no availability of IMP for this study). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Aug 2022.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Bart Biemond, MD, PhD principal investigator
Academic Medical Centre
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing human apotransferrin in people with β-thalassemia Intermedia, run by Prothya Biosolutions at 1 site in Netherlands.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 15 Aug 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Netherlands. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was stopped early (reason given: no availability of IMP for this study). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Erythropoiesis (time frame: 17 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 15 Aug 2022.