Eltrombopag for the treatment of thrombocytopenia due to low- and intermediate risk myelodysplastic syndromes. - EQol-MDS

Status

Active, not recruiting

Phases

Phase 2

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2010-022890-33-IT

Enrollment

Registered

2010-11-30

Start date

2010-11-23

Completion date

Unknown

Last updated

2025-02-17

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Adult patients with low or intermediate-1 IPSS risk MDS and thrombocytopenia MedDRA version: 9.1 Level: HLGT Classification code 10035534

Interventions

Trade Name: REVOLADE Pharmaceutical Form: Film-coated tablet INN or Proposed INN: ELTROMBOPAG Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 50- Pharmaceutical fo

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: 1. Adult subjects (18 years of age or older) with low or intermediate-1 IPSS risk MDS and stable disease. 2. Subjects must have a platelet count taken within the 4 weeks prior to randomization that is 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. Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range

Exclusion criteria

Exclusion criteria: 1. MDS with intermediate-2 or high IPSS risk 2. History of treatment for cancer other than MDS or sAML/MDS with systemic chemotherapy and/or radiotherapy within the last 2 years. 3. History of treatment with romiplostim or other TPO-R agonists. 4. 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). 5. BM fibrosis that leads to an inability to aspirate marrow for assessment. 6. Spleen size >14 cm (length as per ultrasound examination). 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 drug 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).

Design outcomes

Primary

Measure	Time frame
Main Objective: To evaluate 1. Response rate: The proportion of patients achieving a complete response (CR) or response (R) during the six month treatment period, for subjects receiving eltrombopag relative to placebo (see Platelet Response Section 5.4.4) 2. Safety and tolerability in terms of frequency of adverse events (AE)s and serious adverse events (SAE), for subjects receiving eltrombopag relative to placebo.;Secondary Objective: To evaluate the effect of treatment on: 1. Quality of life (QoL) scores for subjects receiving eltrombopag relative to placebo 2. The number of monthly platelet transfusions in subjects receiving eltrombopag compared to the placebo group 3. The duration of transfusion independence as measured in weeks and months for subjects receiving eltrombopag relative to placebo. 4. Time to response (time from starting treatment to time of achievement of CR or PR) between treatment groups as measured by the MDS response criteria 5. The incidence and severity of bleeding using the WHO Bleeding Scale (see Section 8: WHO Bleeding Scale).for subjects receiving eltrombopag relative to placebo 6. Overall survival (OS) at 2 years. Event for OS in both arms is death and patients are censored at the date of last contact if alive. 7. Leukemia-free survival (LFS) at 2 years [22]. Events for LFS in both arms are death and progression to AML. 8. To evaluate eltrombopag population pharmacokinet;Primary end point(s): 1. Proportion of patients obtaining CR or R during the six month treatment period, for subjects receiving eltrombopag relative to placebo 2. Safety and tolerability parameters including non-hematological laboratory Grade 3/Grade 4 toxicities, change in bone marrow blast counts from baseline and adverse events reporting for subjects receiving eltrombopag relative to placebo	—

Measure

Time frame

Main Objective: To evaluate 1. Response rate: The proportion of patients achieving a complete response (CR) or response (R) during the six month treatment period, for subjects receiving eltrombopag relative to placebo (see Platelet Response Section 5.4.4) 2. Safety and tolerability in terms of frequency of adverse events (AE)s and serious adverse events (SAE), for subjects receiving eltrombopag relative to placebo.;Secondary Objective: To evaluate the effect of treatment on: 1. Quality of life (QoL) scores for subjects receiving eltrombopag relative to placebo 2. The number of monthly platelet transfusions in subjects receiving eltrombopag compared to the placebo group 3. The duration of transfusion independence as measured in weeks and months for subjects receiving eltrombopag relative to placebo. 4. Time to response (time from starting treatment to time of achievement of CR or PR) between treatment groups as measured by the MDS response criteria 5. The incidence and severity of bleeding using the WHO Bleeding Scale (see Section 8: WHO Bleeding Scale).for subjects receiving eltrombopag relative to placebo 6. Overall survival (OS) at 2 years. Event for OS in both arms is death and patients are censored at the date of last contact if alive. 7. Leukemia-free survival (LFS) at 2 years [22]. Events for LFS in both arms are death and progression to AML. 8. To evaluate eltrombopag population pharmacokinet;Primary end point(s): 1. Proportion of patients obtaining CR or R during the six month treatment period, for subjects receiving eltrombopag relative to placebo 2. Safety and tolerability parameters including non-hematological laboratory Grade 3/Grade 4 toxicities, change in bone marrow blast counts from baseline and adverse events reporting for subjects receiving eltrombopag relative to placebo

—

Countries

France, Italy, Slovenia

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026