Eltrombopag for the Treatment of Thrombocytopenia Due to Low- and Intermediate Risk Myelodysplastic Syndromes
NCT ID: NCT02912208
Last Updated: 2022-01-28
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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ACTIVE_NOT_RECRUITING
PHASE2
174 participants
INTERVENTIONAL
2011-06-11
2026-10-31
Brief Summary
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Eltrombopag is an orally bioavailable agonist of the thrombopoietin receptor. In adult patients with chronic immune thrombocytopenia (ITP), Eltrombopag rapidly increases platelet counts and significantly reduces bleeding episodes during treatment. Eltrombopag is well tolerated. In 2007, Eltrombopag has received the Orphan Drug Designation for the treatment of ITP (EMEA/OD/031/07), and in 2008 the Food and Drug Association approved Eltrombopag for the treatment of ITP refractory or resistant. It has been shown that in patients affected by MDS and by acute myeloid leukemia, Eltrombopag neither increases the proliferation, nor the clonogenic growth capacity of bone marrow blasts. Furthermore, Eltrombopag induces an increase in the megakaryocytic differentiation and in the formation of normal megakaryocytic colonies. These results provide the rationale for pursuing further research on Eltrombopag for the treatment of thrombocytopenia in case of MDS.
The study is open to adult patients with myelodysplastic syndrome (MDS) with thrombocytopenia and low- or intermediate-1 IPSS risk (Index Prognostic Score System).
Severe thrombocytopenia associated with MDS may lead to death from hemorrhage, even in low prognostic risk patients. The benefit of platelet transfusion is short-termed. Patients become refractory in the long term. The availability of a treatment that induces the increase of platelet count is extremely important, either in terms of quality of life, and in overall survival.
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Detailed Description
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Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
SINGLE
Study Groups
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Arm 1 (Eltrombopag)
Arm 1 is the active treatment arm
Eltrombopag/Revolade
Eltrombopag 50 mg once daily has been selected as the starting dose for this study. Thereafter, dependent on platelet response the dose of study medication can be increased by 50 mg every 2 weeks, up to a maximum dose of 300 mg once daily (150 mg in subjects of East Asian ethnicity).
Arm 2 (Placebo)
Arm 2 is the control arm
Placebo
The administration is the same of eltrombopag
Interventions
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Eltrombopag/Revolade
Eltrombopag 50 mg once daily has been selected as the starting dose for this study. Thereafter, dependent on platelet response the dose of study medication can be increased by 50 mg every 2 weeks, up to a maximum dose of 300 mg once daily (150 mg in subjects of East Asian ethnicity).
Placebo
The administration is the same of eltrombopag
Eligibility Criteria
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Inclusion Criteria
2. Subjects must have a platelet count taken within the 4 weeks prior to randomization that is \<30 Gi/L.
3. Subjects must be ineligible or relapsed or refractory to receive other treatment options (such as azacitidine or lenalidomide) and must be ineligible to receive intensive chemotherapy or autologous/allogeneic stem cell transplantation.
4. Subjects must have platelet count and platelet transfusion data available over a period of 8 weeks prior to randomization.
5. During the 2 months prior to randomization, subjects must have a baseline Bone Marrow examination which includes cytomorphology and cytogenetics. Histopathology should be performed.
6. Erythropoiesis-stimulating agents (ESAs) in anemic subjects or granulocyte colonystimulating factor (G-CSF) in subjects with severe neutropenia and recurrent infections are allowed during the study as per accepted standards. Subjects who enter the study on ESAs or G-CSF should continue at the same dose schedule until the optimal dose of study medication has been established.
7. ECOG (Eastern Cooperative Oncology Group) Performance Status 0-3
8. Subject is able to understand and comply with protocol requirements and instructions.
9. Subject has signed and dated informed consent.
10. Adequate baseline organ function defined by the criteria below:
total bilirubin (except for Gilbert's Syndrome) ≤ 1.5 x Upper Limit Normal Alanine aminotransferase and Aspartate aminotransferase ≤ 3 x Upper Limit Normal creatinine ≤ 2 x Upper Limit Normal albumin must not be below the lower limit of normal by more than 20%.
11. Subject is practicing an acceptable method of contraception. Female subjects (or female partners of male subjects) must either be of non-childbearing potential (hysterectomy, bilateral oophorectomy, bilateral tubal ligation or post-menopausal \>1 year), or of childbearing potential and use of an highly effective method of contraception from 2 weeks prior to administration of study medication, throughout the study, and 28 days after completion or premature discontinuation from the study.
Exclusion Criteria
2. History of treatment for cancer other than MDS with systemic chemotherapy and/or radiotherapy within the last 2 years.
3. History of treatment with romiplostim or other Thrombopoietin receptor agonists.
4. Pre-existing cardiovascular disease (including congestive heart failure, New York Heart Association Grade III/IV), or arrhythmia known to increase the risk of thromboembolic events (e.g. persistent atrial fibrillation), or subjects with a QTc \>450 msec (QTc \>480 msec for subjects with Bundle Branch Block).
5. BM fibrosis that leads to an inability to aspirate marrow for assessment.
6. Peripheral monocytosis \> 1000/uL prior to Day 1 of study medication.
7. Leukocytosis \>=25,000/uL prior to Day 1 of study medication.
8. Female subjects who are nursing or pregnant (positive serum or urine Beta-human chorionic gonadotropin \[B-hCG\] pregnancy test) at screening or pre-dose on Day 1.
9. Current alcohol or drug abuse.
10. Treatment with an Investigational Product within 30 days or 5 half-lives (whichever is longer) preceding the first dose of study medication.
11. Active and uncontrolled infections.
12. Subjects infected with Hepatitis B, C or Human Immunodeficiency Virus (HIV).
18 Years
ALL
No
Sponsors
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Associazione Qol-one
OTHER
Responsible Party
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Principal Investigators
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Esther Natalie Oliva
Role: STUDY_CHAIR
QOL-ONE Associazione Culturale e di Ricerca
Locations
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CHU Amiens
Amiens, , France
Centre d'Avignon
Avignon, , France
Hôpital de la Côte Basque
Bayonne, , France
Centre d'Avicenne, Hôpital d'Avicenne
Bobigny, , France
CHU de Haut-Lévèque
Bordeaux, , France
Centre Hospitalier de Boulogne Sur Mer
Boulogne-sur-Mer, , France
CHU Clémenceau
Caen, , France
Centre Henri Mondor
Créteil, , France
CHU de Grenoble
Grenoble, , France
Centre Le Mans
Le Mans, , France
Hôpital Saint Vincent de Paul
Lille, , France
CHRU de Limoges
Limoges, , France
Centre de Marseille
Marseille, , France
CHU Brabois
Nancy, , France
Centre de Nantes
Nantes, , France
Hopital Archet 1
Nice, , France
Centre Hospitalier Universitaire de Nimes
Nîmes, , France
Centre de St Louis, Hôpital St Louis
Paris, , France
Centre Hospitalier de la Région d'Annecy
Pringy, , France
Centre de Rouen, Centre Henri Becquerel
Rouen, , France
CHU Purpan
Toulouse, , France
CHU de Bretonneau
Tours, , France
Heinrich-Heine-Universität Düsseldorf
Düsseldorf, , Germany
Universitätsmedizin Mannheim
Mannheim, , Germany
A.O. SS. Antonio e Biagio e Cesare Arrigo
Alessandria, AL, Italy
Ospedale Riuniti
Ancona, AN, Italy
Ospedale Cardinal Massaia
Asti, AT, Italy
A.O. S. Giovanni Moscati
Avellino, AV, Italy
Policlinico Università di Bari
Bari, BA, Italy
Ospedale A. Perrino
Brindisi, BR, Italy
Ospedale "Roberto Binaghi"
Cagliari, CA, Italy
Ospedale L'Annunziata
Cosenza, CS, Italy
Ospedale Ferrarotto
Catania, CT, Italy
Ospedale Garibaldi
Catania, CT, Italy
Ospedale Casa Sollievo della Sofferenza
San Giovanni Rotondo, FG, Italy
Azienda Ospedaliera Universitaria Careggi
Florence, FI, Italy
Università degli Studi di Genova
Genova, GE, Italy
Ospedale Vito Fazzi
Lecce, LE, Italy
IRCCS Ospedale Maggiore Policlinico
Milan, MI, Italy
Ospedale Niguarda
Milan, MI, Italy
Ospedale Civile Spirito Santo
Pescara, PE, Italy
Azienda Ospedaliera Bianchi-Melacrino-Morelli
Reggio Calabria, RC, Italy
Arcispedale di Santa Maria Nuova
Reggio Emilia, RE, Italy
A.O. San Camillo Forlanini
Roma, RM, Italy
Ospedale Sant'Eugenio
Roma, RM, Italy
Policlinico Agostino Gemelli
Roma, RM, Italy
Università Campus Bio Medico di Roma
Roma, RM, Italy
Azienda Ospedaliera Sant'Andrea
Rome, RM, Italy
IRCCS Istituto Regina Elena
Rome, RM, Italy
Ospedale Nuova Regina Margherita
Rome, RM, Italy
Policlinico Umberto I
Rome, RM, Italy
Policlinico Universitario Tor Vergata
Rome, RM, Italy
A.O.U. San Giovanni di Dio e Ruggì D'Aragona
Salerno, SA, Italy
Policlinico Santa Maria alle Scotte
Siena, SI, Italy
A.O. Santa Maria
Terni, TE, Italy
A.O. Citta' della Salute e della Scienza di Torino
Torino, TO, Italy
U.O. Citta' della Salute e della Scienza di Torino
Torino, TO, Italy
General Hospital Celje
Celje, , Slovenia
Univerzitetni klinini center Ljubljana
Ljubljana, , Slovenia
University Medical Centre Maribor
Maribor, , Slovenia
General Hospital Murska Sobota
Murska Sobota, , Slovenia
General Hospital Nova Gorica
Nova Gorica, , Slovenia
General Hospital Novo mesto
Novo Mesto, , Slovenia
General Hospital Slovenj Gradec
Slovenj Gradec, , Slovenia
Countries
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References
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Oliva EN, Riva M, Niscola P, Santini V, Breccia M, Giai V, Poloni A, Patriarca A, Crisa E, Capodanno I, Salutari P, Reda G, Cascavilla N, Ferrero D, Guarini A, Tripepi G, Ianni G, Russo E, Castelli A, Fattizzo B, Beltrami G, Bocchia M, Molteni A, Fenaux P, Germing U, Ricco A, Palumbo GA, Impera S, Di Renzo N, Rivellini F, Buccisano F, Stamatoullas-Bastard A, Liberati AM, Candoni A, Delfino IM, Arcadi MT, Cufari P, Rizzo L, Bova I, D'Errigo MG, Zini G, Latagliata R. Eltrombopag for Low-Risk Myelodysplastic Syndromes With Thrombocytopenia: Interim Results of a Phase II, Randomized, Placebo-Controlled Clinical Trial (EQOL-MDS). J Clin Oncol. 2023 Oct 1;41(28):4486-4496. doi: 10.1200/JCO.22.02699. Epub 2023 Jun 9.
Other Identifiers
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EQoL-MDS
Identifier Type: -
Identifier Source: org_study_id
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