Last Update: Sep 19, 2024
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
ClinicalTrials.gov Identifier:
Novartis Reference Number:CETB115L11201
All compounds are either investigational or being studied for (a) new use(s). Efficacy and safety have not been established. There is no guarantee that they will become commercially available for the use(s) under investigation.

Study Description

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). 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.

Myelodysplastic Syndromes
Phase2
Recruiting
36
May 25, 2021
Feb 28, 2027
All
20 Years - 99 Years (Adult, Older Adult)

Interventions

Drug

Eltrombopag

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

Placebo

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

Eligibility Criteria

Inclusion Criteria:

- 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)

- 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

Exclusion Criteria:

- 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) 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.

- 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.

Study Location

Novartis Investigative Site

Recruiting

Kanazawa,Ishikawa,920-0853,Japan

Novartis Investigative Site

Recruiting

Yamagata,990 9585,Japan

Novartis Investigative Site

Recruiting

Chiba,260-0852,Japan

Novartis Investigative Site

Recruiting

Nishinomiya,Hyogo,663 8501,Japan

Novartis Investigative Site

Recruiting

Kumamoto-city,Kumamoto,860-0008,Japan

Novartis Investigative Site

Recruiting

Fukushima city,Fukushima,960 1295,Japan

Novartis Investigative Site

Recruiting

Ohtake,Hiroshima,739-0696,Japan

Novartis Investigative Site

Recruiting

Kurume-city,Fukuoka,830-8543,Japan

Novartis Investigative Site

Recruiting

Gifu shi,Gifu,500 8513,Japan

Novartis Investigative Site

Recruiting

Osaka Sayama,Osaka,589 8511,Japan

Novartis Investigative Site

Recruiting

Matsumoto-city,Nagano,399-8701,Japan

Novartis Investigative Site

Recruiting

Hamamatsu,Shizuoka,432-8580,Japan

Novartis Investigative Site

Recruiting

Osaka,534-0021,Japan

Novartis Investigative Site

Recruiting

Mito,Ibaraki,310-0015,Japan

Novartis Investigative Site

Recruiting

Isehara,Kanagawa,259-1193,Japan

Novartis Investigative Site

Recruiting

Sendai city,Miyagi,980 8574,Japan

Novartis Investigative Site

Recruiting

Aomori,030 8553,Japan

Novartis Investigative Site

Recruiting

Nagasaki-city,Nagasaki,852-8501,Japan

Novartis Investigative Site

Recruiting

Itabashi ku,Tokyo,173 8606,Japan

Novartis Investigative Site

Recruiting

Shimonoseki,Yamaguchi,750-0061,Japan

Novartis Investigative Site

Recruiting

Bunkyo-ku,Tokyo,113-8603,Japan

Novartis Investigative Site

Recruiting

Narita,Chiba,286-8523,Japan

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