Immune ThrombocytopeniaNCT02298075
Response Rate and Duration After Discontinuation Thrombopoietin Receptor Agonists Primary Immune Thrombocytopenia
An observational study of Immune Thrombocytopenia, run by Gruppo Italiano Malattie EMatologiche dell'Adulto at 13 sites in Italy.
Trial summary
The primary objective of the study is the evaluation of sustained response rate after discontinuation of treatment with TPO-RAs, Eltrombopag and Romiplostim, in persistent or chronic pITP patients who failed one or more therapy lines, splenectomy included. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Immune Thrombocytopenia
- What is being tested?
- Eltrombopag and Romiplostin
- How many people
- 148 participants
- Where
- 13 sitesItaly
- Study dates
- Apr 2016to 17 Apr 2019
- Phase
- Observational
- 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
Study population: Patients affected by primary immune thrombocytopenia (pITP)
Who may be able to join
- Signed written informed consent according to ICH/EU/GCP and national local laws;
- Persistent or chronic pITP adult patients who have been treated with TPO-RAs therapy after failure of one or more therapy lines, splenectomy included;
- Age > 18 years.
Who cannot join
- Active malignancy;
- Active malignancy;
- HCVAb, HIVAb, HBsAg, HBcAb seropositive status;
- Chronic liver disease;
- Treatment with Rituximab less than 8 weeks before TPO-RAs start;
- Recent splenectomy (less than 8 weeks) before TPO-RAs start. Active malignancy;
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- HCVAb, HIVAb, HBsAg, HBcAb seropositive status;
- Chronic liver disease;
- Treatment with Rituximab less than 8 weeks before TPO-RAs start;
- Recent splenectomy (less than 8 weeks) before TPO-RAs start. HCVAb, HIVAb, HBsAg, HBcAb seropositive status;
- Chronic liver disease;
- Treatment with Rituximab less than 8 weeks before TPO-RAs start;
- Recent splenectomy (less than 8 weeks) before TPO-RAs start.
Trial locations and contacts
13 sites in 1 country.
13 sites in 1 country
Italy13 sites
S.O.C. di Medicina Interna B - Ospedale - Cardinal Massaia di Asti
Asti
Istituto di Ematologia "Lorenzo e A. Seragnoli"
Bologna
Unità di Onco-Ematologia - Azienda Ospedaliera - Garibaldi
Catania
Unità di Ricerca e di Malattie del sangue - Ematologia San Luca Vecchio Pad. 16 - 1° Piano
Florence
Clinica Medica III - Padiglione ex isolamento - Azienda Ospedale Università San Martino
Genova
ASL Le/1 P.O. Vito Fazzi - U.O. di Ematologia ed UTIE
Lecce
Azienda Ospedaliera "S.Gerardo"
Monza
Ematologia - Ospedale San Carlo
Potenza
Unità Operativa Complessa di Ematologia - Arcispedale S. Maria Nuova
Reggio Emilia
Università Cattolica del Sacro Cuore - Policlinico A. Gemelli
Roma
U.O.C. Ematologia - Ospedale S. Eugenio
Rome
Università degli studi "Sapienza" di Roma
Rome
Ematologia - Dipartimento di Medicina Clinica e Sperimentale
Sassari
About this study
Official title: Response Rate and Response Duration After Discontinuation of Treatment With Thrombopoietin Receptor Agonists in Patients Affected by Primary Immune Thrombocytopenia (pITP): Retrospective Study
The primary objective of the study is the evaluation of sustained response rate after discontinuation of treatment with TPO-RAs, Eltrombopag and Romiplostim, in persistent or chronic pITP patients who failed one or more therapy lines, splenectomy included.
Read the detailed description
Increasing evidence exists in the literature on the persistent response after TPO-RAs discontinuation. However, the available data consist of description of case reports from different patients' series. At this time, systematic data collections on this topic do not exist and the real incidence of such persistent response is unknown. Therefore, because of the increasing interest in this new and challenging therapeutic field, also for the therapeutic implications, we would like to investigate the impact of TPO- RAs on the achievement of sustained and persistent responses after their discontinuation in the Italian adult patients affected by pITP who have been referred to the GIMEMA Centers.
- Conditions
- Immune Thrombocytopenia
- Being tested
- Eltrombopag, Romiplostin
- Keywords
- Immune thrombocytopenia, Primary, Thrombopoietin receptor agonist
- Medical subject headings
- Purpura, Thrombocytopenic, Idiopathic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
EltrombopagDrug
Also called Thrombopoietin Receptor Agonists (TPO-RAs)
Investigation of the impact of TPO-RAs on the achievement of sustained and persistent response after discontinuation in adult patients affected by pITP.
RomiplostinDrug
Also called Thrombopoietin Receptor Agonists (TPO-RAs)
Investigation of the impact of TPO-RAs on the achievement of sustained and persistent response after discontinuation in adult patients affected by pITP.
What is being measured
The primary outcome is the main question the study is designed to answer.
Estimation of sustained response rate after discontinuation of treatment with TPO-RAs in terms of patients still alive in Complete Response or R at more than 4 weeks after discontinuation.
Measured: One year from start of recruitment.
Secondary outcomes (9)
Frequency of the type of response in patients treated with TPO-RAs in absence of concomitant or rescue medication, in order to estimate the rate of Response (R) and Complete Response (CR).
Measured: One year from start of recruitment.
Estimation of the suspension criteria in terms of frequency of discontinuation causes for each center.
Measured: One year from start of recruitment.
Estimation of the duration of sustained response in terms of patients alive in CR or R at 6 and 12 months after sustained response achievement.
Measured: One year from start of recruitment.
Estimation of incidence of relapse and loss of response at 6 and 12 months after sustained response achievement.
Measured: One year from start of recruitment.
Estimation of treatment duration starting from the date of first dose of TPO-RAs.
Measured: One year from start of recruitment.
Description of Platelet levels and TPO-RAs dosage at stop.
Measured: One year from start of recruitment.
Description of patients' characteristics in term of: biological and clinical features, disease duration at the beginning of TPO-RAs, number and type of therapy lines preceding TPO-RAs treatment, platelet levels at TPO-RAs start.
Measured: One year from start of recruitment.
Assessment of an association between previous splenectomy and sustained response after discontinuation of treatment with TPO-RAs.
Measured: One year from start of recruitment.
Description of concomitant therapies since TPO-RAs start in terms of type and duration of therapies.
Measured: One year from start of recruitment.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Nov 2014 (estimated)
- Study startApr 2016
- Primary completion17 Apr 2019
- Study completion17 Apr 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Immune ThrombocytopeniaStudies 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
- Observational
- Observational model
- Other
- Time perspective
- Retrospective
- Enrolment
- 148 participants (actual)
Sponsor and investigators
- Lead sponsor
- Gruppo Italiano Malattie EMatologiche dell'Adulto
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Maria Gabriella Mazzucconi, Study chair, Policlinico Umberto I, Hematology Department.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02298075
- Sponsor's protocol ID
- ITP0714
- Status last verified
- Apr 2025
- First submitted
- 17 Nov 2014
- Last updated
- 29 Apr 2025
- Results posted
- No
- Data monitoring committee
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 29 Apr 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Response Rate and Response Duration After Discontinuation of Treatment With Thrombopoietin Receptor Agonists in Patients Affected by Primary Immune Thrombocytopenia (pITP): Retrospective Study
- ClinicalTrials.gov ID
- NCT02298075
- Acronym / sponsor ID
- ITP0714
- Conditions
- Immune Thrombocytopenia
- Keywords
- Immune thrombocytopenia, Primary, Thrombopoietin receptor agonist
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 148 participants (actual)
- Lead sponsor
- Gruppo Italiano Malattie EMatologiche dell'AdultoUniversity, hospital or other
- Study start
- Apr 2016
- Primary completion
- 17 Apr 2019 (actual)
- Study completion
- 17 Apr 2019 (actual)
- First posted
- 21 Nov 2014 (estimated)
- 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 the study is the evaluation of sustained response rate after discontinuation of treatment with TPO-RAs, Eltrombopag and Romiplostim, in persistent or chronic pITP patients who failed one or more therapy lines, splenectomy included.
Detailed description
Increasing evidence exists in the literature on the persistent response after TPO-RAs discontinuation. However, the available data consist of description of case reports from different patients' series. At this time, systematic data collections on this topic do not exist and the real incidence of such persistent response is unknown. Therefore, because of the increasing interest in this new and challenging therapeutic field, also for the therapeutic implications, we would like to investigate the impact of TPO- RAs on the achievement of sustained and persistent responses after their discontinuation in the Italian adult patients affected by pITP who have been referred to the GIMEMA Centers.
Study design
- Observational model
- Other
- Time perspective
- Retrospective
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Patients affected by primary immune thrombocytopenia (pITP)
Key inclusion criteria (3 listed)
- Signed written informed consent according to ICH/EU/GCP and national local laws;
- Persistent or chronic pITP adult patients who have been treated with TPO-RAs therapy after failure of one or more therapy lines, splenectomy included;
- Age > 18 years.
Key exclusion criteria (13 listed)
- Active malignancy;
- Active malignancy;
- HCVAb, HIVAb, HBsAg, HBcAb seropositive status;
- Chronic liver disease;
- Treatment with Rituximab less than 8 weeks before TPO-RAs start;
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 |
|---|---|---|
| EltrombopagAlso: Thrombopoietin Receptor Agonists (TPO-RAs)Investigation of the impact of TPO-RAs on the achievement of sustained and persistent response after discontinuation in adult patients affected by pITP. | Drug | – |
| RomiplostinAlso: Thrombopoietin Receptor Agonists (TPO-RAs)Investigation of the impact of TPO-RAs on the achievement of sustained and persistent response after discontinuation in adult patients affected by pITP. | Drug | – |
Primary outcome
Estimation of sustained response rate after discontinuation of treatment with TPO-RAs in terms of patients still alive in Complete Response or R at more than 4 weeks after discontinuation.
Time frame: One year from start of recruitment.
9 secondary outcomes
Frequency of the type of response in patients treated with TPO-RAs in absence of concomitant or rescue medication, in order to estimate the rate of Response (R) and Complete Response (CR).
Time frame: One year from start of recruitment.
Estimation of the suspension criteria in terms of frequency of discontinuation causes for each center.
Time frame: One year from start of recruitment.
Estimation of the duration of sustained response in terms of patients alive in CR or R at 6 and 12 months after sustained response achievement.
Time frame: One year from start of recruitment.
Estimation of incidence of relapse and loss of response at 6 and 12 months after sustained response achievement.
Time frame: One year from start of recruitment.
Estimation of treatment duration starting from the date of first dose of TPO-RAs.
Time frame: One year from start of recruitment.
Description of Platelet levels and TPO-RAs dosage at stop.
Time frame: One year from start of recruitment.
Description of patients' characteristics in term of: biological and clinical features, disease duration at the beginning of TPO-RAs, number and type of therapy lines preceding TPO-RAs treatment, platelet levels at TPO-RAs start.
Time frame: One year from start of recruitment.
Assessment of an association between previous splenectomy and sustained response after discontinuation of treatment with TPO-RAs.
Time frame: One year from start of recruitment.
Description of concomitant therapies since TPO-RAs start in terms of type and duration of therapies.
Time frame: One year from start of recruitment.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 Apr 2025.
- GIMEMA Foundation websiteLink provided by the sponsor.
- Individual participant dataSharing planned: No
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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.
Maria Gabriella Mazzucconi study chair
Policlinico Umberto I, Hematology Department.
13 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Immune Thrombocytopenia, run by Gruppo Italiano Malattie EMatologiche dell'Adulto at 13 sites 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 13 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: finished, as of the sponsor's last update on 29 Apr 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 13 sites 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Estimation of sustained response rate after discontinuation of treatment with TPO-RAs in terms of patients still alive in Complete Response or R at more than 4 weeks after discontinuation. (time frame: One year from start of recruitment.). 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 29 Apr 2025.