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

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: NCT04797000

Novartis Reference Number: CETB115L11201

Last Update: Apr 21, 2022

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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).

Condition 
Myelodysplastic Syndromes
Phase 
Phase 2
Overall status 
Recruiting
Start date 
May 25, 2021
Completion date 
Oct 15, 2024
Gender 
All
Age(s)
20 Years and older (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 Locations

Japan
Novartis Investigative Site
Recruiting
Kitakyushu, 802-8533
Fukuoka
Japan
Novartis Investigative Site
Recruiting
Fukushima city, 960 1295
Fukushima
Japan
Novartis Investigative Site
Recruiting
Gifu shi, 500 8513
Gifu
Japan
Novartis Investigative Site
Recruiting
Ohtake, 739-0696
Hiroshima
Japan
Novartis Investigative Site
Recruiting
Nishinomiya, 663 8501
Hyogo
Japan
Novartis Investigative Site
Recruiting
Kanazawa, 920-0853
Ishikawa
Japan
Novartis Investigative Site
Recruiting
Isehara, 259-1193
Kanagawa
Japan
Novartis Investigative Site
Recruiting
Yokohama, 221-0855
Kanagawa
Japan
Novartis Investigative Site
Recruiting
Kumamoto-city, 860-0008
Kumamoto
Japan
Novartis Investigative Site
Recruiting
Sendai city, 980 8574
Miyagi
Japan
Novartis Investigative Site
Recruiting
Nagasaki-city, 852-8501
Nagasaki
Japan
Novartis Investigative Site
Recruiting
Osaka Sayama, 589 8511
Osaka
Japan
Novartis Investigative Site
Recruiting
Hamamatsu, 432-8580
Shizuoka
Japan
Novartis Investigative Site
Recruiting
Aomori, 030 8553
-
Japan
Novartis Investigative Site
Recruiting
Chiba, 260-0852
-
Japan
Novartis Investigative Site
Recruiting
Osaka, 534-0021
-
Japan
Novartis Investigative Site
Recruiting
Yamagata, 990 9585
-
Japan

Contacts

Name: 
Novartis Pharmaceuticals
Phone: 

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