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

NCT04797000 | Active Not Recruiting Phase 2 DMC

• 1 other identifier: CETB115L11201
• Study Type: interventional
• Target: 36
• Locations: 1 country
• Sites: 20

Brief 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).

Conditions

Myelodysplastic Syndromes

Keywords

Phase II; eltrombopag; ETB115; lower-risk MDS patients with platelet transfusion dependence; TPO-RA; placebo; myelodysplastic syndromes (MDS); platelet transfusion dependence

Outcome Measures

Primary Outcomes (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.

  Week 24

Secondary Outcomes (19)

• Time to platelet transfusion independence

  Year 1, Year 2

• Duration of platelet transfusion independence

  Year 1, Year 2

• Percentage of participants with platelet transfusion independence

  Year 1, Year 2

• Percentage of participants with platelet transfusion frequency reduction at Week 24

  Week 24

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

  Week 24, Year 1, Year 2

Study Arms (2)

Eltrombopag Arm

EXPERIMENTAL

Participants randomized to a 1: 1 ratio will take eltrombopag.

Drug: Eltrombopag

Placebo Arm

PLACEBO COMPARATOR

Participants randomized to a 1: 1 ratio will take Placebo.

Drug: Placebo

Interventions

Eltrombopag DRUG

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

Also known as: ETB115

Eltrombopag Arm

Placebo DRUG

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

Placebo Arm

Eligibility Criteria

• Age: 20 Years - 100 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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.
• 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)

Sponsors & Collaborators

• Novartis Pharmaceuticals: lead

Study Sites (20)

Novartis Investigative Site

Narita, Chiba, 286-8523, Japan

Location

Novartis Investigative Site

Kurume, Fukuoka, 830-8543, Japan

Location

Novartis Investigative Site

Ohtake, Hiroshima, 739-0696, Japan

Location

Novartis Investigative Site

Nishinomiya, Hyōgo, 663 8501, Japan

Location

Novartis Investigative Site

Kanazawa, Ishikawa-ken, 920-8530, Japan

Location

Novartis Investigative Site

Isehara, Kanagawa, 259-1193, Japan

Location

Novartis Investigative Site

Yokohama, Kanagawa, 221-0855, Japan

Location

Novartis Investigative Site

Sendai, Miyagi, 9808574, Japan

Location

Novartis Investigative Site

Nagasaki, Nagasaki, 852-8501, Japan

Location

Novartis Investigative Site

Sakai, Osaka, 590-0197, Japan

Location

Novartis Investigative Site

Bunkyo-ku, Tokyo, 113-8603, Japan

Location

Novartis Investigative Site

Itabashi Ku, Tokyo, 173 8606, Japan

Location

Novartis Investigative Site

Shimonoseki, Yamaguchi, 750-0061, Japan

Location

Novartis Investigative Site

Aomori, 030 8553, Japan

Location

Novartis Investigative Site

Chiba, 260-0852, Japan

Location

Novartis Investigative Site

Fukushima, 9601295, Japan

Location

Novartis Investigative Site

Gifu, 5008513, Japan

Location

Novartis Investigative Site

Kumamoto, 860-0008, Japan

Location

Novartis Investigative Site

Osaka, 5340021, Japan

Location

Novartis Investigative Site

Yamagata, 990 9585, Japan

Location

MeSH Terms

Conditions

Myelodysplastic Syndromes

Interventions

eltrombopag

Condition Hierarchy (Ancestors)

Bone Marrow Diseases; Hematologic Diseases; Hemic and Lymphatic Diseases

Study Officials

• Novartis Pharmaceuticals

  Novartis Pharmaceuticals

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: NONE
• Masking Details: Participants, investigators, site staffs and site monitors will remain blinded to the identity of the treatment from the time of randomization until database lock for final analysis, and the clinical study team members and anyone involved in the Japan registration activities will be blinded until database lock for the primary analysis. Randomization data are kept strictly confidential until the time of unblinding and will not be accessible by anyone else involved in the study with the following exceptions: DMC members and who will perform data analysis for DMC. Once protocol amendment 5 becomes effective, all individuals, including participants, investigators, site staff, and site monitors, will be unblinded.
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 4, 2021
• First Posted: March 15, 2021
• Study Start: May 25, 2021
• Primary Completion: April 26, 2025
• Study Completion (Estimated): December 9, 2026
• Last Updated: March 19, 2026

Record last verified: 2026-03

Data Sharing

• IPD Sharing: Will share

Locations

JP (20)