Tests eltrombopag for low platelet counts caused by myelodysplastic syndromes
Tests eltrombopag against placebo in adults with low platelets from myelodysplastic syndromes.
Plain-language title generated from the registry record
Registry title: Eltrombopag for the treatment of thrombocytopenia due to low - and intermediate risk myelodysplastic syndromes.
Key facts
- Where
- Bari, Firenze, Genova, Milano, Reggio Calabria, Roma, and Rome, ItalySee the 9 sites
- Phase
- Phase 2
Where in Italy
- A.O.U Maggiore della CaritàItalyTaking people
- Istituto Tumori Bari Giovanni Paolo IIBari, ItalyTaking people
- Azienda Ospedaliera Universitaria CareggiFirenze, ItalyTaking people
- Ircss Ospedale Policlinico San MartinoGenova, ItalyTaking people
- ASST Grande Ospedale Metropolitano NiguardaMilano, ItalyTaking people
- Grande Ospedale Metropolitano Bianchi Melacrino MorelliReggio Calabria, ItalyTaking people
- Ospedale S. Eugenio, ASL Roma 2Roma, ItalyTaking people
- AOU Policlinico Umberto I -Università degli studi di Roma "La Sapienza"Rome, ItalyTaking people
- IFO-Regina Elena Institute for Cancer ResearchRome, ItalyTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Associazione Qol-One
- Trial code
- NCT02912208EU 2024-520133-72-00
- Record updated
- 24 June 2025
- Original description
Phase 1 Primary Objectives To evaluate the effect of treatment relative to placebo on: 1. response rate: the proportion of patients achieving a complete response (CR) or response (R) during the treatment period 2. safety and tolerability in terms of frequency of adverse events (AE) and serious adverse events (SAE).Read full original descriptionShow less
Eltrombopag for the treatment of thrombocytopenia due to low - and intermediate risk myelodysplastic syndromes.
Phase 1 Primary Objectives To evaluate the effect of treatment relative to placebo on: 1. response rate: the proportion of patients achieving a complete response (CR) or response (R) during the treatment period 2. safety and tolerability in terms of frequency of adverse events (AE) and serious adverse events (SAE).
Phase 2 Primary objectives To evaluate the effect of treatment relative to placebo on: 1. duration of platelet response; 2. long-term safety and tolerability
Inclusion: Adult subjects (18 years of age or older) with low or intermediate-1 IPSS risk MDS and stable disease; 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; Subjects must have a platelet count taken within the 4 weeks prior to randomization that is <30 Gi/L; 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; Subjects must have platelet count and platelet transfusion data available over a period of 8 weeks prior to randomization; During the 2 months prior to randomization, subjects must have a baseline BM examination which includes cytomorphology and cytogenetics. Histopathology should be performed; Erythropoiesis-stimulating agents (ESAs) in anemic subjects or granulocyte colony stimulating 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; ECOG Performance Status 0-3; Subject is able to understand and comply with protocol requirements and instructions; Adequate baseline organ function defined by the criteria below: total bilirubin (except for Gilbert’s Syndrome) ≤ 1.5xULN ALT and AST ≤ 3xULN creatinine ≤ 2xULN albumin must not be below the lower limit of normal by more than 20%.
Exclusion: Treatment with an Investigational Product within 30 days or 5 half-lives (whichever is longer) preceding the first dose of study medication; History of treatment with romiplostim or other TPO-R agonists; Peripheral monocytosis > 1000/uL prior to Day 1 of study medication; Leukocytosis >=25,000/uL prior to Day 1 of study medication; Current alcohol or drug abuse; Active and uncontrolled infections; Subjects infected with Hepatitis B, C or Human Immunodeficiency Virus (HIV); History of treatment for cancer other than MDS with systemic chemotherapy and/or radiotherapy within the last 2 years; Pre-existing cardiovascular disease (including congestive heart failure, New York Heart Association [NYHA] 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); 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; MDS with intermediate-2 or high IPSS risk; BM fibrosis that leads to an inability to aspirate marrow for assessment.