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Myelodysplastic SyndromesPhase 2NCT04797000

Study of Efficacy and Safety of Eltrombopag in Lower-risk MDS Patients With Platelet Transfusion Dependence

A Phase 2 study testing Eltrombopag in people with Myelodysplastic Syndromes, run by Novartis Pharmaceuticals at 20 sites in Japan.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Official record
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Trial summary

This study is designed to evaluate the efficacy and safety of eltrombopag monotherapy in Japanese adult patients with platelet transfusion-dependent lower-risk Myelodysplastic syndromes (LR-MDS). Read more about this study

Who is it for?
Ages 20 to 100 years
Conditions the study is for
Myelodysplastic Syndromes
What is being tested?
Eltrombopag
Type of treatment
Pill or oral medicine
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
36 participants
Where
20 sitesJapan
Study dates
25 May 2021to 2 Dec 2026
Phase
Phase 2
Are there results?
Not yet: the study is under way

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 20 to 100 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Patients diagnosed with MDS according to the WHO classification revised 4th edition by investigator assessment with one of the following prognostic risk categories, based on the International
  • Prognostic Scoring System (IPSS-R):
  • very low (0-1.5)
  • low (2-3)
  • intermediate risks (3.5-4.5) All following criteria for prognostic variables per IPSS-R should be met.
  • Bone marrow blast < 5% (per both investigator's assessment and central review)
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  • Cytogenetic very good, good or intermediate risk corresponding to IPSS-R
  • Platelet transfusion dependence
  • Refractory, intolerant to, or ineligible for MDS treatments
  • Eastern Cooperative Oncology Group (ECOG) Performance status (PS) 0 or 1

Who cannot join

  • Patients with a history of prior administration of eltrombopag, romiplostim, or other TPO-RA
  • Therapy-related MDS per WHO classification revised 4th edition
  • MDS/myeloproliferative neoplasms including chronic myelomonocytic leukaemia per the WHO classification revised 4th edition
  • MDS with excess blasts (EB) per WHO classification revised 4th edition
  • Known history of IPSS-R high or very high risk MDS
  • Currently receiving treatments for MDS (e.g., HMA, cyclosporine A (CsA) or lenalidomide). Supportive treatment with erythropoiesis-stimulating agents (ESAs) or erythroid mutation agent in anemic patients or granulocyte-colony stimulating factor (G-CSF) in patients with severe neutropenia and recurrent infections is allowed if at stable dosage for 3 months prior to screening and continued at the same dosing/schedule until the optimal dose of eltrombopag has been established.
Show all 10Show fewer
  • Patients scheduled for hematopoietic stem cell transplantation
  • Bone marrow fibrosis that leads to an inability to aspirate adequate bone marrow sample
  • Known thrombophilic risk factors (except in cases where potential benefits of participating in the study outweighed potential risks of thromboembolic events (TEE), as determined by the investigator)
  • Other protocol-defined inclusion/exclusion criteria may apply.

Trial locations and contacts

20 sites in 1 country.

20 sites in 1 country

Japan20 sites

  • Novartis Investigative Site

    Aomori, 0308553

  • Novartis Investigative Site

    Bunkyo-ku, Tokyo, 1138603

  • Novartis Investigative Site

    Chiba, 2600852

  • Novartis Investigative Site

    Fukushima, 9601295

  • Novartis Investigative Site

    Gifu, 5008513

  • Novartis Investigative Site

    Isehara, Kanagawa, 2591193

  • Novartis Investigative Site

    Kanazawa, Ishikawa-ken, 9208530

  • Novartis Investigative Site

    Kumamoto, 8600008

  • Novartis Investigative Site

    Kurume, Fukuoka, 8308543

  • Novartis Investigative Site

    Nagasaki, Nagasaki, 8528501

  • Novartis Investigative Site

    Narita, Chiba, 2868523

  • Novartis Investigative Site

    Nishinomiya, Hyōgo, 6638501

  • Novartis Investigative Site

    Ohtake, Hiroshima, 7390696

  • Novartis Investigative Site

    Osaka, 5340021

  • Novartis Investigative Site

    Sakai, Osaka, 5900197

  • Novartis Investigative Site

    Sendai, Miyagi, 9808574

  • Novartis Investigative Site

    Shimonoseki, Yamaguchi, 7500061

  • Novartis Investigative Site

    tabashi City, Tokyo, 1738606

  • Novartis Investigative Site

    Yamagata, 9909585

  • Novartis Investigative Site

    Yokohama, Kanagawa, 2210855

Other trials in Japan

About this study

Official title: A Randomized, Double-blind, Placebo-controlled, Japan Local Phase II Clinical Study Comparing Eltrombopag Monotherapy Versus Placebo in Adult Lower-risk Myelodysplastic Syndromes (MDS) Patients With Platelet Transfusion Dependence

This study is designed to evaluate the efficacy and safety of eltrombopag monotherapy in Japanese adult patients with platelet transfusion-dependent lower-risk Myelodysplastic syndromes (LR-MDS).

Read the detailed description

This is a randomized, double-blind, placebo-controlled, Japanese local phase II study to evaluate the efficacy and safety of eltrombopag monotherapy in Japanese adult patients with platelet transfusion-dependent lower-risk MDS (IPSS-R very low, low, intermediate risk with bone marrow blast count < 5% and cytogenetic very good, good or intermediate risk). Platelet transfusion dependence at baseline is defined as receiving platelet transfusion regularly with a frequency of 2 or more times within 4 weeks prior to randomization. Platelet transfusion should be performed for a patient with platelet counts < 20 X 10^9/L, or with hemorrhagic symptoms and platelet counts < 30 X 10^9/L.

The primary objective is to demonstrate superiority of eltrombopag versus placebo in terms of the proportion of participants who achieve platelet transfusion independence at Week 24.

Being tested
Eltrombopag, Placebo
Keywords
Phase II, eltrombopag, ETB115, lower-risk MDS patients with platelet transfusion dependence, TPO-RA, placebo, myelodysplastic syndromes (MDS), platelet transfusion dependence
Medical subject headings
Myelodysplastic Syndromes

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • EltrombopagDrug

    Also called ETB115

    Eltrombopag comes in 12.5 mg \& 25 mg tablets and is taken orally once per day (QD)

  • PlaceboDrug

    Placebo comes in 12.5 mg \& 25 mg tablets and is taken orally once per day (QD)

Study groups in NCT04797000
Study groupRoleWhat they receive
Eltrombopag ArmExperimentalParticipants randomized to a 1: 1 ratio will take eltrombopag.
Receives: Drug: Eltrombopag
Placebo ArmPlacebo comparatorParticipants randomized to a 1: 1 ratio will take Placebo.
Receives: Drug: Placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Percentage of participants who achieve platelet transfusion independence at Week 24

    Platelet transfusion independence is defined as the absence of platelet transfusion for at least 8 weeks. Platelet count should be higher than the baseline count.

    Measured: Week 24

Secondary outcomes (19)

  1. Time to platelet transfusion independence

    This is defined as time from the date of randomization to the first day of a platelet transfusion-free period lasting at least 8 weeks.

    Measured: Year 1, Year 2

  2. Duration of platelet transfusion independence

    This is defined for participants who have achieved platelet transfusion independence only and will be calculated from the first date of platelet transfusion-free resulting in achievement of transfusion independence to the date before becoming platelet transfusion dependent.

    Measured: Year 1, Year 2

  3. Percentage of participants with platelet transfusion independence

    Platelet transfusion independence is defined as the absence of platelet transfusion for at least 8 weeks. Platelet count should be higher than the baseline count.

    Measured: Year 1, Year 2

  4. Percentage of participants with platelet transfusion frequency reduction at Week 24

    This is defined as a reduction by at least 50 percent during the 4 prior weeks as compared to the 4 weeks prior to randomization.

    Measured: Week 24

  5. Percentage of participants with platelet response (Hematologic improvement (HI) - platelet))

    Platelet response is defined as HI-platelet per International Working Group criteria.

    Measured: Week 24, Year 1, Year 2

  6. Time to platelet response

    This is defined as time from the date of randomization to the first date of achieving the platelet response criteria per modified IWG criteria.

    Measured: Week 24, Year 1, Year 2

  7. Duration of platelet response

    This is defined for participants who have achieved platelet response and will be calculated from the first date of achieving the platelet response criteria to the date before loss of platelet response per modified IWG criteria.

    Measured: Week 24, Year 1, Year 2

  8. Percentage of hematologic improvement-erythroid and -neutrophil

    per modified IWG criteria at 24 weeks, Year 1 and 2

    Measured: Week 24, Year 1, Year 2

  9. Percentage of participants with disease progression excluding relapse after HI

    This is with regards to blast counts for both investigator assessment and central review per modified IWG criteria.

    Measured: Week 24, Year 1, Year 2

  10. Percentage of participants with relapse after HI and transfusion independence

    The percentage of participants with relapse after HI and transfusion independence at Week 24, Year 1 and 2.

    Measured: Week 24, Year 1, Year 2

  11. Percentage of participants with progression to Acute myeloid leukemia (AML)

    This is defined as ≥ 20% blasts at Week 24, Year 1 and 2 for both investigator assessment and central review per WHO classification revised 4th edition.

    Measured: Week 24, Year 1, Year 2

  12. Leukemia free survival (LFS)

    This is defined as time from the date of randomization to progression to AML per WHO classification revised 4th edition of ≥ 20 percent blasts or death due to any cause for both investigator assessment and central review.

    Measured: Week 24, Year 1, Year 2

  13. Clinically significant bleeding events

    This is defined as ≥ grade 2 events as per WHO bleeding scale.

    Measured: Week 24, Year 1, Year 2

  14. Overall survival (OS)

    OS is defined as time from randomization to death due to any cause.

    Measured: Week 24, Year 1, Year 2

  15. Quality of Life measured using QLQ-C30

    The changes from baseline to each visit where measured using QLQ-C30. EORTC-QLQ-C30 is a 30-item questionnaire developed to assess the quality of life of cancer patients. Subject's responses to 28 questions about their physical functioning, disease symptoms, global health status and utilities are scored on a 4-point scale (1=Not at all to 4=Very much), a low score indicates a high / healthy level of functioning. And the responses to 2 questions about health-related QoL are scored on a 7-point scale (1=Very poor to 7=Excellent), a high score indicates a high / healthy level of functioning.

    Using linear transformation, raw scores are standardized, so that scores range from 0 to 100.

    Measured: Baseline, every 4 weeks until week 52, every 8 weeks after week 52 up to end of treatment or end of post-treatment follow-up, approximately 3.5 years.

  16. Pharmacokinetics (PK): Cmax

    Full PK profile of eltrombopag over 24 hours at steady state at the initial dose, and at 125 mg and 150 mg when applicable, in subset of participants.

    Measured: Intensive: pre-dose, 1, 2, 4, 6, 8, and 24 hours post-dose. Trough: 15th days after starting the initial dose or each dose modification until reaching the maximum dose.

  17. PK: Tmax

    Full PK profile of eltrombopag over 24 hours at steady state at the initial dose, and at 125 mg and 150 mg when applicable, in subset of participants.

    Measured: Intensive: pre-dose, 1, 2, 4, 6, 8, and 24 hours post-dose. Trough: 15th days after starting the initial dose or each dose modification until reaching the maximum dose.

  18. PK: AUC

    Full PK profile of eltrombopag over 24 hours at steady state at the initial dose, and at 125 mg and 150 mg when applicable, in subset of participants.

    Measured: Intensive: pre-dose, 1, 2, 4, 6, 8, and 24 hours post-dose. Trough: 15th days after starting the initial dose or each dose modification until reaching the maximum dose.

  19. Trough concentrations of eltrombopag at steady state

    at each dose level in all the participants

    Measured: Intensive: pre-dose, 1, 2, 4, 6, 8, and 24 hours post-dose. Trough: 15th days after starting the initial dose or each dose modification until reaching the maximum dose.

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted15 Mar 2021
  2. Study start25 May 2021
  3. Primary completion26 Apr 2025
  4. Study completion2 Dec 2026 (estimated)

Resources

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
Randomised
Intervention model
Parallel groups
Masking
None (open label)
Enrolment
36 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04797000
Sponsor's protocol ID
CETB115L11201
Status last verified
Oct 2026
First submitted
4 Mar 2021
Last updated
20 Oct 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
Yes: Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations. This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Eltrombopag in people with Myelodysplastic Syndromes, run by Novartis Pharmaceuticals at 20 sites in Japan.

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 10 inclusion and 10 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: under way, not looking for participants, as of the sponsor's last update on 20 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 20 sites in Japan. 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. Some participants in this study may receive a placebo. 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?

This study is still under way. Results are not available in the source record yet.

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?

Percentage of participants who achieve platelet transfusion independence at Week 24 (time frame: Week 24). 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 NCT04797000.

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 20 Oct 2026.

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