Myelodysplastic Syndromes (MDS)NCT06764511
Evaluation of Response to Biosimilar Erythropoietin Alfa Therapy in Anemic Patients With Myelodysplastic Syndrome
An observational study of Myelodysplastic Syndromes (MDS), run by IRCCS Azienda Ospedaliero-Universitaria di Bologna at 1 site in Italy.
Trial summary
The primary objective of this study is to describe the response to treatment with biosimilar EPO alpha in MDS patients who had already been treated with "originator" EPO alpha and were responsive, and in patients who started treatment with biosimilar EPO alpha Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Myelodysplastic Syndromes (MDS)
- How many people
- 60 participants
- Where
- 1 siteItaly
- Study dates
- 30 Aug 2024to 30 Apr 2025
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
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
- Adult, Older adult
Study population: All patients with MDS who started treatment with biosimilar EPO alpha at the "Seràgnoli" Institute, U.O. of Hematology of IRCCS AOU of Bologna during the period from 01 Jun 2018 to 31 Dec 2021 will be enrolled in the study. Specifically, the population will consist of * patients who initiated treatment after having already been treated with "originator" EPO alfa and responsive to treatment at the time of initiation of biosimilar EPO alfa, and * patients who started treatment without previous treatment with biosimilar EPO alfa
Who may be able to join
- MDS patients with symptomatic anemia (pre-treatment hemoglobin < 10g/dL) who started treatment with biosimilar EPO alpha, diagnosed according to the WHO 2016 classification, and characterized by very low, low, or intermediate IPSS-R risk and a pre-treatment serum EPO level < 500 U/L, who started treatment with biosimilar EPO alpha at the U. O. of Hematology of IRCCS AOUBO during the period from 01/06/2018 to 31/12/2021.
- Age ≥ 18 years at the time of enrollment
- Acquisition of informed consent to study participation and data processing
Who cannot join
- - Presence of other possible contributory causes of anemia (e.g., anemia from chronic inflammatory disease, hemolysis, hemorrhage)
- Poor compliance with treatment
- Very impaired general clinical condition (ECOG performance status > 3)
- Concomitant treatment with antineoplastic cytotoxic drugs
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Italy1 site, 1 recruiting
- Recruiting
IRCCS Azienda Ospedaliera -Universitaria di Bologna
Bologna, 40138
About this study
Official title: Valutazione Della Risposta Alla Terapia Con Eritropoietina Alfa Biosimilare Nei Pazienti Anemici Affetti da Sindrome Mielodisplastica
The primary objective of this study is to describe the response to treatment with biosimilar EPO alpha in MDS patients who had already been treated with "originator" EPO alpha and were responsive, and in patients who started treatment with biosimilar EPO alpha
Read the detailed description
To date, therapy with erythropoiesis-stimulating agents (ESAs) has been shown to be effective in the treatment of symptomatic anemia in patients with Myelodysplastic Syndrome ( MDS) at low to intermediate risk, with the percentage of responsive patients ranging from 15 to 63%, depending on the characteristics of the patients treated, and averaging 45%.
Significant improvement in survival and quality of life was observed in treatment-responsive patients compared with non-responsive patients.
Recently, biosimilar ESAs have been introduced into clinical practice, and in patients with renal failure anemia and anemia associated with antineoplastic chemotherapy, their efficacy and safety have been demonstrated.
However, data on the efficacy and safety of biosimilar ESAs in patients with MDS are scarce to date, and efficacy and safety data for the purpose of their approval have been mainly extrapolated from studies conducted in patients with anemia from renal failure and anemia from antineoplastic chemotherapy. In these conditions bone marrow erythropoiesis is reduced but not qualitatively impaired as in MDS.
These considerations may justify a retrospective study examining the efficacy and safety of biosimilar EPO alpha in a real-life setting in anemic MDS patients.
- Conditions
- Myelodysplastic Syndromes (MDS)
- Medical subject headings
- Myelodysplastic Syndromes
What is being measured
The primary outcome is the main question the study is designed to answer.
Response and maintenance of ORR
response to treatment (ORR) (assessed according to the response criteria established by the International Working Group (23) in patients who initiated treatment with biosimilar EPO alfa
- maintenance of ORR (assessed according to the response criteria established by the International Working Group (23) from the date of biosimilar drug initiation), in patients previously on treatment and responsive to "originator" EPO alfa who were subsequently treated with biosimilar EPO alfa
Measured: 6 months from the start of the study
Secondary outcomes (1)
A measure of the frequency of major clinical outcomes in two groups of patients who had different modes of treatment administration with biosimilar EPO alpha
- DOR to EPO alpha: to biosimilar drug only in patients who started treatment with biosimilar EPO alpha; to "originator" drug + biosimilar in patients already on treatment and responsive to "originator" EPO alpha who were subsequently treated with biosimilar EPO alpha ; 2) incidence of adverse events in year/person (in both patient groups) according to NCI-CTCAE v.5.0 ; 3) number of patients who discontinued treatment with the biosimilar drug due to intolerance, loss of response, disease progression (DP), evolution into AML, or death (both groups) ; 4) number of patients who resumed the originator drug due to intolerance or failure to respond to the biosimilar drug ; 5) ORR and incidence of adverse events of patients who resumed treatment with "originator" EPO alfa after discontinuation of the biosimilar drug ; 6) incidence of evolution into AML in year/person (both groups) ; 7) OS and causes of death (both groups)
Measured: 6 months from the start of the study
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Jan 2025
- Study start30 Aug 2024
- Primary completion30 Apr 2025 (estimated)
- Study completion30 Apr 2025 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Myelodysplastic Syndromes (MDS)Studies of the same condition, open and under way.
- More trials run by IRCCS Azienda Ospedaliero-Universitaria di BolognaThe sponsor's other registered studies.
- 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
- Observational
- Observational model
- Cohort
- Time perspective
- Retrospective
- Enrolment
- 60 participants (estimated)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Stefania Paolini, MD, Principal investigator, IRCCS Azienda Ospedaliero-Universitaria di Bologna
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06764511
- Sponsor's protocol ID
- EPO BIOSIM
- Status last verified
- Dec 2024
- First submitted
- 2 Jan 2025
- Last updated
- 8 Jan 2025
- 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 8 Jan 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Valutazione Della Risposta Alla Terapia Con Eritropoietina Alfa Biosimilare Nei Pazienti Anemici Affetti da Sindrome Mielodisplastica
- ClinicalTrials.gov ID
- NCT06764511
- Acronym / sponsor ID
- EPO BIOSIM
- Conditions
- Myelodysplastic Syndromes (MDS)
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 60 participants (estimated)
- Lead sponsor
- IRCCS Azienda Ospedaliero-Universitaria di BolognaUniversity, hospital or other
- Study start
- 30 Aug 2024 (actual)
- Primary completion
- 30 Apr 2025 (estimated)
- Study completion
- 30 Apr 2025 (estimated)
- First posted
- 8 Jan 2025 (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 primary objective of this study is to describe the response to treatment with biosimilar EPO alpha in MDS patients who had already been treated with "originator" EPO alpha and were responsive, and in patients who started treatment with biosimilar EPO alpha
Detailed description
To date, therapy with erythropoiesis-stimulating agents (ESAs) has been shown to be effective in the treatment of symptomatic anemia in patients with Myelodysplastic Syndrome ( MDS) at low to intermediate risk, with the percentage of responsive patients ranging from 15 to 63%, depending on the characteristics of the patients treated, and averaging 45%.
Significant improvement in survival and quality of life was observed in treatment-responsive patients compared with non-responsive patients.
Recently, biosimilar ESAs have been introduced into clinical practice, and in patients with renal failure anemia and anemia associated with antineoplastic chemotherapy, their efficacy and safety have been demonstrated.
However, data on the efficacy and safety of biosimilar ESAs in patients with MDS are scarce to date, and efficacy and safety data for the purpose of their approval have been mainly extrapolated from studies conducted in patients with anemia from renal failure and anemia from antineoplastic chemotherapy. In these conditions bone marrow erythropoiesis is reduced but not qualitatively impaired as in MDS.
These considerations may justify a retrospective study examining the efficacy and safety of biosimilar EPO alpha in a real-life setting in anemic MDS patients.
Study design
- Observational model
- Cohort
- Time perspective
- Retrospective
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Age groups
- Adult, Older adult
- Study population
- All patients with MDS who started treatment with biosimilar EPO alpha at the "Seràgnoli" Institute, U.O. of Hematology of IRCCS AOU of Bologna during the period from 01 Jun 2018 to 31 Dec 2021 will be enrolled in the study. Specifically, the population will consist of * patients who initiated treatment after having already been treated with "originator" EPO alfa and responsive to treatment at the time of initiation of biosimilar EPO alfa, and * patients who started treatment without previous treatment with biosimilar EPO alfa
Key inclusion criteria (3 listed)
- MDS patients with symptomatic anemia (pre-treatment hemoglobin < 10g/dL) who started treatment with biosimilar EPO alpha, diagnosed according to the WHO 2016 classification, and characterized by very low, low, or intermediate IPSS-R risk and a pre-treatment serum EPO level < 500 U/L, who started treatment with biosimilar EPO alpha at the U. O. of Hematology of IRCCS AOUBO during the period from 01/06/2018 to 31/12/2021.
- Age ≥ 18 years at the time of enrollment
- Acquisition of informed consent to study participation and data processing
Key exclusion criteria (4 listed)
- - Presence of other possible contributory causes of anemia (e.g., anemia from chronic inflammatory disease, hemolysis, hemorrhage)
- Poor compliance with treatment
- Very impaired general clinical condition (ECOG performance status > 3)
- Concomitant treatment with antineoplastic cytotoxic drugs
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Response and maintenance of ORR
Time frame: 6 months from the start of the study
response to treatment (ORR) (assessed according to the response criteria established by the International Working Group (23) in patients who initiated treatment with biosimilar EPO alfa - maintenance of ORR (assessed according to the response criteria established by the International Working Group (23) from the date of biosimilar drug initiation), in patients previously on treatment and responsive to "originator" EPO alfa who were subsequently treated with biosimilar EPO alfa
1 secondary outcome
A measure of the frequency of major clinical outcomes in two groups of patients who had different modes of treatment administration with biosimilar EPO alpha
Time frame: 6 months from the start of the study
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Jan 2025.
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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.
Stefania Paolini, MD principal investigator
IRCCS Azienda Ospedaliero-Universitaria di Bologna
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Myelodysplastic Syndromes (MDS), run by IRCCS Azienda Ospedaliero-Universitaria di Bologna at 1 site in Italy.
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 3 inclusion and 4 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: looking for participants now, as of the sponsor's last update on 8 Jan 2025. 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 Italy. 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.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Stefania Paolini, MD (+390512143680, stefania.paolini@unibo.it), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Response and maintenance of ORR (time frame: 6 months from the start of the study). 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 8 Jan 2025.