Inherited Platelet DisorderPhase 2NCT02422394
Eltrombopag for Inherited Thrombocytopenias
A Phase 2 study testing Eltrombopag in people with Inherited Platelet Disorder, run by Fondazione IRCCS Policlinico San Matteo di Pavia at 3 sites in Italy.
Trial summary
Inherited thrombocytopenias (ITs) are a heterogeneous group of disorders characterized by a reduced number of blood platelets and a consequent bleeding tendency that ranges from mild to life-threatening. Read more about this study
- Who is it for?
- Ages 16 to 70 years
- Conditions the study is for
- Inherited Platelet Disorder
- What is being tested?
- Eltrombopag
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 24 participants
- Where
- 3 sitesItaly
- Study dates
- Apr 2015to Mar 2018
- 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 16 to 70 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Phase 1:
- Patients with the following forms of inherited thrombocytopenias will be considered for enrolment:
- MYH9-related disorders (OMIM 155100, 605249, 153640, 153650)
- Bernard-Soulier Syndrome (OMIM 231200) deriving from monoallelic mutations (at variance with biallelic classical BSS, monoallelic form does not present defective platelet function)
- Wiskott-Aldrich syndrome (OMIM 301000).
- X-linked thrombocytopenia (OMIM 313900).
Show all 21Show fewer
- X-linked thrombocytopenia with thalassemia (OMIM 314050).
- Dyserythropoietic anemia with thrombocytopenia (OMIM 300367).
- ITGA2B/ITGB3-related thrombocytopenia (OMIM not available).
- ANKRD26-related thrombocytopenia (OMIM 188000).
- TUBB1-related thrombocytopenia (OMIM not available)
- ACTN1-related thrombocytopenia (OMIM not available)
- GFI1B-related thrombocytopenia (OMIM not available)
- CYCS-related thrombocytopenia (OMIM 612004)
- SLFN14-related thrombocytopenia (OMIM not available)
- Patients will have to fulfill all the following criteria:
- Age ≥ 16 years and ≤ 70 years
- Average platelet count during the previous year less than 80 x109/L
- Written informed consent
- Phase 2
- Patients with clinically relevant chronic or recurrent bleedings at baseline (grade 2-4 of the WHO bleeding scale) who: (i) completed the phase 1 without severe side effects and (ii) obtained reduction or remission of bleeding by Eltrombopag administration.
Who cannot join
- Phases 1 and 2
- Hypersensitivity to Eltrombopag or one of the excipients.
- History of thromboembolic events.
- Treatment with anti-platelet drugs or other drugs affecting platelet function and/or with anticoagulants.
- Concurrent diseases or conditions that significantly increase the risk of thromboembolic events.
- Moderate to severe liver failure (Child-Pugh score ≥ 5).
Show all 12Show fewer
- Altered renal function as defined by creatinine ≥ 2 mg/dL
- Previous or concurrent clonal disorders of the myeloid series (acute myeloid leukemias and myelodysplastic syndromes).
- Females who are pregnant or nursing (a negative pregnancy test is required before enrolment of fertile women).
- Formal refusal of any recommendations for a safe contraception.
- Alcohol or drug addiction.
- Any other disease or condition that by the advice of the responsible physician would make the treatment dangerous for the patient or would make the patient ineligible for this study, including physical, psychiatric, social and behavioral problems.
Trial locations and contacts
3 sites in 1 country.
Italy3 sites
Department of Internal Medicine, Hospital of Padova
Padova, 35128
Unit of Internal Medicine 3, IRCCS Policlinico San Matteo Foundation
Pavia, 27100
Section of Internal and Cardiovascular Medicine, Department of Medicine, University Hospital of Perugia
Perugia, 06126
About this study
Inherited thrombocytopenias (ITs) are a heterogeneous group of disorders characterized by a reduced number of blood platelets and a consequent bleeding tendency that ranges from mild to life-threatening. Thrombocytopenia is caused by genetic mutations and therefore is present throughout life and can be transmitted to the progeny. Some patients with severely reduced platelet count present spontaneous bleeding, which represents a major clinical problem: in fact, bleeding diathesis exposes these subjects to the risk of severe hemorrhages, affects their quality of life and often requires hospitalization and/or transfusions. Conversely, other patients with ITs have absent or mild spontaneous bleeding tendency. However, even these patients are at risk of major bleeding on the occasion of surgery or other invasive procedures. Therefore, the potential for hemorrhages on the occasion of invasive procedures represent a clinical problem for all patients affected by ITs.
Eltrombopag is a drug, available in tablets, which stimulates the production of platelets by the bone marrow. A previous study demonstrated that a short course of eltrombopag was effective in increasing platelet count in most patients with the MYH9-related disease (MYH9-RD), the most frequent form of IT. Eltrombopag was given for 3 to 6 weeks to 12 patients with MYH9-RD and platelet counts lower than 50 x10e9/L. Eleven patients responded to the drug and 8 of them obtained platelet counts higher than 100 x10e9/L or three times the baseline value. Remission of spontaneous bleeding was achieved by 8 of 10 patients and treatment was well tolerated in all the cases. Based on these findings, short-term eltrombopag courses have been successfully used for preparing for major surgery two patients with MYH9-RD and less than 20 x10e9 platelets/L.
Read more
The present study has two main objectives. - To verify if eltrombopag is effective in transiently increasing platelet count over 100 x 10e9/L and abolishing bleeding tendency in patients with different forms of IT.
To this end, eltrombopag will be given for 3-6 weeks to patients with different forms of IT. Eltrombopag will be administered at the dose of 50 mg/day for 3 weeks. After 3 weeks of treatment, the patients who will obtain a platelet count higher than 100 x10e9/L and complete remission of bleeding tendency will stop therapy. In the other cases, patients will be treated with eltrombopag at a higher dose (75 mg/day) for 3 additional weeks. This treatment schedule is called "Phase 1" of the study.
If the study will achieve this goal, short-term eltrombopag could be potentially used in the future to prepare these patients for surgery or other invasive procedures
- To verify if eltrombopag can be used to stably reduce spontaneous bleeding tendency for long periods of time in patients with clinically significant spontaneous hemorrhages.
To this end, patients with clinically significant spontaneous bleedings at baseline and who had their bleeding tendency reduced during the Phase 1 of the study without severe side effects, will be admitted to the "Phase 2" of the study.
During the Phase 2, patients will be treated with eltrombopag for 16 weeks. In order to determine the lowest dose of eltrombopag that is able to reduce or abolish their bleeding tendency, patients will start treatment with eltrombopag 25 mg/day for 4 weeks. Then, every 4 weeks, patients will be re-evaluated and the dosage of eltrombopag will be adjusted according to bleeding tendency and platelet count. The dosages of eltrombopag that can be used in the Phase 2 range from 12.5 to 75 mg/day.
Other objectives of the study are:
- to evaluate safety and tolerability of Eltrombopag in patients affected with ITs.
- to identify the dosages of Eltrombopag required for achieving the primary endpoints of Phases 1 and 2.
- to study the effects of Phase 2 treatment on patients' health-related quality of life (HR-QoL);
- to study the effects of treatment on some laboratory parameters related to platelet production and function.
All patients will be undergo a follow-up visit 30 days after completion of treatment.
Patients will be treated as outpatients. The evaluation of patients at enrollment and at each subsequent on-treatment and post-treatment visits includes: medical history; physical examination; evaluation of bleeding tendency according to WHO bleeding scale; CBC and differential; platelet count by phase-contrast microscopy; peripheral blood smear examination; plasma transaminases, bilirubin, and creatinine; urine analysis; ophthalmic assessment (only at some visits); measurement of serum thrombopoietin level; evaluation of HR-QoL (only at baseline and during Phase 2); evaluation of in vitro platelet aggregation in response to ADP, collagen and ristocetin whenever platelet count is over 100 x 10e9/L.
- Conditions
- Inherited Platelet Disorder
- Being tested
- Eltrombopag
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
EltrombopagDrug
| Study group | Role | What they receive |
|---|---|---|
| Eltrombopag | Experimental | Phase 1: Eltrombopag 50 mg/day for 3 weeks. At the end of these 3 weeks of treatment: platelet count higher than 100 x10e9/L and no spontaneous bleeding, end therapy. In the other cases, treatment with eltrombopag 75 mg/day for 3 additional weeks.
Phase 2: Eltrombopag will be administered for 16 weeks. Eltrombopag will be initially given at 25 mg/day for 4 weeks. Every 4 weeks, the dosage will be modified as follows. (1) Bleeding score (WHO bleeding scale) 0-1 and platelet count between 30 and 100 x10e9/L: continue at the current dose; (2) Bleeding score 0-1 and platelet count higher than 100 x10e9/L: switch to the next lower dose; (3) Bleeding score 2-4 or platelet count lower than 30 x 10e9/L: switch to the next higher dose. The following dosages of eltrombopag are considered: 12.5 mg/day; 25 mg/day; 50 mg/day; 75 mg/day. Receives: Drug: Eltrombopag |
What is being measured
The primary outcome is the main question the study is designed to answer.
platelet count
Phase 1: major response is defined by increase of platelet count over 100 x10e9/L and no bleeding tendency (grade 0 of the WHO bleeding scale) during the last week of treatment. Minor response is defined by a platelet at least two-fold increased with respect to baseline value without reaching the criteris for major response.
Measured: 3 or 6 weeks (Phase 1)
bleeding tendency according to the WHO bleeding scale
Phase 1: major response is defined by increase of platelet count over 100 x10e9/L and no bleeding tendency (grade 0 of the WHO bleeding scale) during the last week of treatment.
Phase 2: Major response is defined as a complete remission of hemorrhages. Minor response is defined as a reduction of bleeding according to the WHO bleeding scale without reaching the criteria for major response.
Measured: 3 or 6 weeks (Phase 1); 16 weeks (Phase 2)
Secondary outcomes (3)
Daily dose of eltrombopag (in milligrams) required for achievement of the primary endpoints
Measured: 3 or 6 weeks (Phase 1); 16 weeks (Phase 2)
number and severity of adverse events as a measure of safety and tolerability.
Measured: 7 or 10 weeks (Phase 1); 20 weeks (Phase 2)
patients' health-related quality of life as a result of reduction of spontaneous bleeding tendency.
Measured: 20 weeks (Phase 2)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Apr 2015 (estimated)
- Study startApr 2015
- Primary completionMar 2018
- Study completionMar 2018
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (6)Papers the sponsor has linked to this study.
- Other trials for Inherited Platelet DisorderStudies of the same condition, open and under way.
- More trials run by Fondazione IRCCS Policlinico San Matteo di PaviaThe 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
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 24 participants (actual)
Sponsor and investigators
- Lead sponsor
- Fondazione IRCCS Policlinico San Matteo di Pavia
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Alessandro Pecci, Fondazione IRCCS Policlinico San Matteo di Pavia
- Investigators
- Alessandro Pecci, M.D., Principal investigator, Unit of Internal Medicine 3, Department of Internal Medicine, IRCCS Policlinico San Matteo Foundation, Pavia, Italy
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02422394
- Sponsor's protocol ID
- Eltrombo-IT-2014
- Status last verified
- Aug 2018
- First submitted
- 14 Apr 2015
- Last updated
- 21 Aug 2018
- Results posted
- No
- Data monitoring committee
- No
Publications
Balduini CL, Pecci A, Noris P. Diagnosis and management of inherited thrombocytopenias. Semin Thromb Hemost. 2013 Mar;39(2):161-71. doi: 10.1055/s-0032-1333540. Epub 2013 Feb 8.
PubMed 23397552Pecci A. Pathogenesis and management of inherited thrombocytopenias: rationale for the use of thrombopoietin-receptor agonists. Int J Hematol. 2013 Jul;98(1):34-47. doi: 10.1007/s12185-013-1351-7. Epub 2013 May 1.
PubMed 23636669Pecci A, Gresele P, Klersy C, Savoia A, Noris P, Fierro T, Bozzi V, Mezzasoma AM, Melazzini F, Balduini CL. Eltrombopag for the treatment of the inherited thrombocytopenia deriving from MYH9 mutations. Blood. 2010 Dec 23;116(26):5832-7. doi: 10.1182/blood-2010-08-304725. Epub 2010 Sep 15.
PubMed 20844233Pecci A, Barozzi S, d'Amico S, Balduini CL. Short-term eltrombopag for surgical preparation of a patient with inherited thrombocytopenia deriving from MYH9 mutation. Thromb Haemost. 2012 Jun;107(6):1188-9. doi: 10.1160/TH12-01-0005. Epub 2012 Mar 8. No abstract available.
PubMed 22398565Favier R, Feriel J, Favier M, Denoyelle F, Martignetti JA. First successful use of eltrombopag before surgery in a child with MYH9-related thrombocytopenia. Pediatrics. 2013 Sep;132(3):e793-5. doi: 10.1542/peds.2012-3807. Epub 2013 Aug 12.
PubMed 23940247Zaninetti C, Gresele P, Bertomoro A, Klersy C, De Candia E, Veneri D, Barozzi S, Fierro T, Alberelli MA, Musella V, Noris P, Fabris F, Balduini CL, Pecci A. Eltrombopag for the treatment of inherited thrombocytopenias: a phase II clinical trial. Haematologica. 2020 Mar;105(3):820-828. doi: 10.3324/haematol.2019.223966. Epub 2019 Jul 4.
PubMed 31273088
Study at a glance
From the registry record, last updated by the sponsor on 21 Aug 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Eltrombopag for Inherited Thrombocytopenias
- ClinicalTrials.gov ID
- NCT02422394
- Acronym / sponsor ID
- Eltrombo-IT-2014
- Conditions
- Inherited Platelet Disorder
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 24 participants (actual)
- Lead sponsor
- Fondazione IRCCS Policlinico San Matteo di PaviaUniversity, hospital or other
- Study start
- Apr 2015 (actual)
- Primary completion
- Mar 2018 (actual)
- Study completion
- Mar 2018 (actual)
- First posted
- 21 Apr 2015 (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.
Inherited thrombocytopenias (ITs) are a heterogeneous group of disorders characterized by a reduced number of blood platelets and a consequent bleeding tendency that ranges from mild to life-threatening. Thrombocytopenia is caused by genetic mutations and therefore is present throughout life and can be transmitted to the progeny. Some patients with severely reduced platelet count present spontaneous bleeding, which represents a major clinical problem: in fact, bleeding diathesis exposes these subjects to the risk of severe hemorrhages, affects their quality of life and often requires hospitalization and/or transfusions. Conversely, other patients with ITs have absent or mild spontaneous bleeding tendency. However, even these patients are at risk of major bleeding on the occasion of surgery or other invasive procedures. Therefore, the potential for hemorrhages on the occasion of invasive procedures represent a clinical problem for all patients affected by ITs.
Eltrombopag is a drug, available in tablets, which stimulates the production of platelets by the bone marrow. A previous study demonstrated that a short course of eltrombopag was effective in increasing platelet count in most patients with the MYH9-related disease (MYH9-RD), the most frequent form of IT. Eltrombopag was given for 3 to 6 weeks to 12 patients with MYH9-RD and platelet counts lower than 50 x10e9/L. Eleven patients responded to the drug and 8 of them obtained platelet counts higher than 100 x10e9/L or three times the baseline value. Remission of spontaneous bleeding was achieved by 8 of 10 patients and treatment was well tolerated in all the cases. Based on these findings, short-term eltrombopag courses have been successfully used for preparing for major surgery two patients with MYH9-RD and less than 20 x10e9 platelets/L.
The present study has two main objectives. - To verify if eltrombopag is effective in transiently increasing platelet count over 100 x 10e9/L and abolishing bleeding tendency in patients with different forms of IT.
To this end, eltrombopag will be given for 3-6 weeks to patients with different forms of IT. Eltrombopag will be administered at the dose of 50 mg/day for 3 weeks. After 3 weeks of treatment, the patients who will obtain a platelet count higher than 100 x10e9/L and complete remission of bleeding tendency will stop therapy. In the other cases, patients will be treated with eltrombopag at a higher dose (75 mg/day) for 3 additional weeks. This treatment schedule is called "Phase 1" of the study.
If the study will achieve this goal, short-term eltrombopag could be potentially used in the future to prepare these patients for surgery or other invasive procedures
- To verify if eltrombopag can be used to stably reduce spontaneous bleeding tendency for long periods of time in patients with clinically significant spontaneous hemorrhages.
To this end, patients with clinically significant spontaneous bleedings at baseline and who had their bleeding tendency reduced during the Phase 1 of the study without severe side effects, will be admitted to the "Phase 2" of the study.
During the Phase 2, patients will be treated with eltrombopag for 16 weeks. In order to determine the lowest dose of eltrombopag that is able to reduce or abolish their bleeding tendency, patients will start treatment with eltrombopag 25 mg/day for 4 weeks. Then, every 4 weeks, patients will be re-evaluated and the dosage of eltrombopag will be adjusted according to bleeding tendency and platelet count. The dosages of eltrombopag that can be used in the Phase 2 range from 12.5 to 75 mg/day.
Other objectives of the study are:
- to evaluate safety and tolerability of Eltrombopag in patients affected with ITs.
- to identify the dosages of Eltrombopag required for achieving the primary endpoints of Phases 1 and 2.
- to study the effects of Phase 2 treatment on patients' health-related quality of life (HR-QoL);
- to study the effects of treatment on some laboratory parameters related to platelet production and function.
All patients will be undergo a follow-up visit 30 days after completion of treatment.
Patients will be treated as outpatients. The evaluation of patients at enrollment and at each subsequent on-treatment and post-treatment visits includes: medical history; physical examination; evaluation of bleeding tendency according to WHO bleeding scale; CBC and differential; platelet count by phase-contrast microscopy; peripheral blood smear examination; plasma transaminases, bilirubin, and creatinine; urine analysis; ophthalmic assessment (only at some visits); measurement of serum thrombopoietin level; evaluation of HR-QoL (only at baseline and during Phase 2); evaluation of in vitro platelet aggregation in response to ADP, collagen and ristocetin whenever platelet count is over 100 x 10e9/L.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Eltrombopag | Experimental | Drug: EltrombopagPhase 1: Eltrombopag 50 mg/day for 3 weeks. At the end of these 3 weeks of treatment: platelet count higher than 100 x10e9/L and no spontaneous bleeding, end therapy. In the other cases, treatment with eltrombopag 75 mg/day for 3 additional weeks. Phase 2: Eltrombopag will be administered for 16 weeks. Eltrombopag will be initially given at 25 mg/day for 4 weeks. Every 4 weeks, the dosage will be modified as follows. (1) Bleeding score (WHO bleeding scale) 0-1 and platelet count between 30 and 100 x10e9/L: continue at the current dose; (2) Bleeding score 0-1 and platelet count higher than 100 x10e9/L: switch to the next lower dose; (3) Bleeding score 2-4 or platelet count lower than 30 x 10e9/L: switch to the next higher dose. The following dosages of eltrombopag are considered: 12.5 mg/day; 25 mg/day; 50 mg/day; 75 mg/day. |
Who may qualify
- Ages
- 16 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (21 listed)
- Patients with the following forms of inherited thrombocytopenias will be considered for enrolment:
- MYH9-related disorders (OMIM 155100, 605249, 153640, 153650)
- Bernard-Soulier Syndrome (OMIM 231200) deriving from monoallelic mutations (at variance with biallelic classical BSS, monoallelic form does not present defective platelet function)
- Wiskott-Aldrich syndrome (OMIM 301000).
- X-linked thrombocytopenia (OMIM 313900).
Key exclusion criteria (12 listed)
- Phases 1 and 2
- Hypersensitivity to Eltrombopag or one of the excipients.
- History of thromboembolic events.
- Treatment with anti-platelet drugs or other drugs affecting platelet function and/or with anticoagulants.
- Concurrent diseases or conditions that significantly increase the risk of thromboembolic events.
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 |
|---|---|---|
| Eltrombopag | Drug | Eltrombopag |
Primary outcome
platelet count
Time frame: 3 or 6 weeks (Phase 1)
Phase 1: major response is defined by increase of platelet count over 100 x10e9/L and no bleeding tendency (grade 0 of the WHO bleeding scale) during the last week of treatment. Minor response is defined by a platelet at least two-fold increased with respect to baseline value without reaching the criteris for major response.
bleeding tendency according to the WHO bleeding scale
Time frame: 3 or 6 weeks (Phase 1); 16 weeks (Phase 2)
Phase 1: major response is defined by increase of platelet count over 100 x10e9/L and no bleeding tendency (grade 0 of the WHO bleeding scale) during the last week of treatment. Phase 2: Major response is defined as a complete remission of hemorrhages. Minor response is defined as a reduction of bleeding according to the WHO bleeding scale without reaching the criteria for major response.
3 secondary outcomes
Daily dose of eltrombopag (in milligrams) required for achievement of the primary endpoints
Time frame: 3 or 6 weeks (Phase 1); 16 weeks (Phase 2)
number and severity of adverse events as a measure of safety and tolerability.
Time frame: 7 or 10 weeks (Phase 1); 20 weeks (Phase 2)
patients' health-related quality of life as a result of reduction of spontaneous bleeding tendency.
Time frame: 20 weeks (Phase 2)
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 21 Aug 2018.
- Background: PubMed 23397552Balduini CL, Pecci A, Noris P. Diagnosis and management of inherited thrombocytopenias. Semin Thromb Hemost. 2013 Mar;39(2):161-71. doi: 10.1055/s-0032-1333540. Epub 2013 Feb 8.
- Background: PubMed 23636669Pecci A. Pathogenesis and management of inherited thrombocytopenias: rationale for the use of thrombopoietin-receptor agonists. Int J Hematol. 2013 Jul;98(1):34-47. doi: 10.1007/s12185-013-1351-7. Epub 2013 May 1.
- Background: PubMed 20844233Pecci A, Gresele P, Klersy C, Savoia A, Noris P, Fierro T, Bozzi V, Mezzasoma AM, Melazzini F, Balduini CL. Eltrombopag for the treatment of the inherited thrombocytopenia deriving from MYH9 mutations. Blood. 2010 Dec 23;116(26):5832-7. doi: 10.1182/blood-2010-08-304725. Epub 2010 Sep 15.
- Background: PubMed 22398565Pecci A, Barozzi S, d'Amico S, Balduini CL. Short-term eltrombopag for surgical preparation of a patient with inherited thrombocytopenia deriving from MYH9 mutation. Thromb Haemost. 2012 Jun;107(6):1188-9. doi: 10.1160/TH12-01-0005. Epub 2012 Mar 8. No abstract available.
- Background: PubMed 23940247Favier R, Feriel J, Favier M, Denoyelle F, Martignetti JA. First successful use of eltrombopag before surgery in a child with MYH9-related thrombocytopenia. Pediatrics. 2013 Sep;132(3):e793-5. doi: 10.1542/peds.2012-3807. Epub 2013 Aug 12.
- Derived from this study: PubMed 31273088Zaninetti C, Gresele P, Bertomoro A, Klersy C, De Candia E, Veneri D, Barozzi S, Fierro T, Alberelli MA, Musella V, Noris P, Fabris F, Balduini CL, Pecci A. Eltrombopag for the treatment of inherited thrombocytopenias: a phase II clinical trial. Haematologica. 2020 Mar;105(3):820-828. doi: 10.3324/haematol.2019.223966. Epub 2019 Jul 4.
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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.
Alessandro Pecci, M.D. principal investigator
Unit of Internal Medicine 3, Department of Internal Medicine, IRCCS Policlinico San Matteo Foundation, Pavia, Italy
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Eltrombopag in people with Inherited Platelet Disorder, run by Fondazione IRCCS Policlinico San Matteo di Pavia at 3 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 21 inclusion and 12 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 21 Aug 2018. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 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.
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 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?
platelet count (time frame: 3 or 6 weeks (Phase 1)). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02422394, which also lists 6 linked publications.
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 21 Aug 2018.