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Eltrombopag for the treatment of thrombocytopenia due to low - and intermediate risk myelodysplastic syndromes.
EU CTIS ID: 2024-520133-72-00
What this study is testing
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
- Therapeutic exploratory (Phase II)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- 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.
You likely can't join if
- MDS with intermediate-2 or high IPSS risk.
- Treatment with an Investigational Product within 30 days or 5 half-lives (whichever is longer) preceding the first dose of study medication.
- 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.
- History of treatment with romiplostim or other TPO-R agonists.
See the full eligibility criteria
- 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%.
- MDS with intermediate-2 or high IPSS risk.
- Treatment with an Investigational Product within 30 days or 5 half-lives (whichever is longer) preceding the first dose of study medication.
- 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.
- History of treatment with romiplostim or other TPO-R agonists.
- 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)
- BM fibrosis that leads to an inability to aspirate marrow for assessment
- Peripheral monocytosis > 1000/uL prior to Day 1 of study medication.
- Leukocytosis >=25,000/uL prior to Day 1 of study medication.
- 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.
- Current alcohol or drug abuse.
The study team makes the final eligibility decision.
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18-64 years, 65+ years. The study team makes the final eligibility decision.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.