A Study of Momelotinib in Participants With Low-risk Myelodysplastic Syndrome (MIDAS)

May 29, 2026 updated by: GlaxoSmithKline

A Phase 2, Randomized, Open-label, Study of Momelotinib in Participants With Anemia Due to Low-risk Myelodysplastic Syndrome

The goal of this clinical trial is to determine if momelotinib is safe and effective for people with low-risk myelodysplastic syndromes (LR-MDS). The trial will also examine how the body processes the drug. Participants will receive different doses of momelotinib to find the best dose by evaluating effectiveness in improving red blood cell transfusion requirements and safety.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Estimated)

80

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact Backup

Study Locations

  • Canada
    • Alberta
      • Calgary, Alberta, Canada, T2N 5G2
        • Recruiting
        • GSK Investigational Site
        • Contact:
        • Contact:
        • Principal Investigator:
          • Michelle Geddes
    • Ontario
      • Toronto, Ontario, Canada, M5G 2M9
        • Recruiting
        • GSK Investigational Site
        • Contact:
        • Contact:
        • Principal Investigator:
          • Karen Yee
  • France
      • Le Mans, France, 72015
        • Recruiting
        • GSK Investigational Site
        • Contact:
        • Contact:
        • Principal Investigator:
          • Kamel Laribi
      • Nice, France, 06202
        • Recruiting
        • GSK Investigational Site
        • Principal Investigator:
          • Thomas Cluzeau
        • Contact:
        • Contact:
      • Paris, France, 75010
        • Recruiting
        • GSK Investigational Site
        • Principal Investigator:
          • Pierre Fenaux
        • Contact:
        • Contact:
      • Poitiers, France, 86021
        • Recruiting
        • GSK Investigational Site
        • Contact:
        • Contact:
        • Principal Investigator:
          • Jose Torregrosa Diaz
      • Toulouse, France, 31059
        • Recruiting
        • GSK Investigational Site
        • Contact:
        • Contact:
        • Principal Investigator:
          • Thibault Comont
  • Germany
      • Dresden, Germany, 01307
        • Recruiting
        • GSK Investigational Site
        • Contact:
        • Contact:
        • Principal Investigator:
          • Ekaterina Balaian
      • Leipzig, Germany, 04103
        • Recruiting
        • GSK Investigational Site
        • Contact:
        • Contact:
        • Principal Investigator:
          • Madlen Jentzsch
    • North Rhine-Westphalia
      • Münster, North Rhine-Westphalia, Germany, 48149
        • Recruiting
        • GSK Investigational Site
        • Contact:
        • Contact:
        • Principal Investigator:
          • Jan-Henrik Mikesch
  • Italy
      • Catania, Italy, 95123
        • Recruiting
        • GSK Investigational Site
        • Contact:
        • Contact:
        • Principal Investigator:
          • Giuseppe Palumbo
      • Florence, Italy, 50134
        • Recruiting
        • GSK Investigational Site
        • Contact:
        • Contact:
        • Principal Investigator:
          • Valeria Santini
      • Milan, Italy, 20122
        • Recruiting
        • GSK Investigational Site
        • Contact:
        • Contact:
        • Principal Investigator:
          • Bruno Fattizzo
      • Orbassano to, Italy, 10043
        • Recruiting
        • GSK Investigational Site
        • Contact:
        • Contact:
        • Principal Investigator:
          • Marco Gobbi
      • Rozzano MI, Italy, 20089
        • Recruiting
        • GSK Investigational Site
        • Principal Investigator:
          • Matteo Della Porta
        • Contact:
        • Contact:
    • Friuli Venezia Giulia
      • Udine, Friuli Venezia Giulia, Italy, 33100
        • Recruiting
        • GSK Investigational Site
        • Contact:
        • Contact:
        • Principal Investigator:
          • Mario Tiribelli
  • Poland
      • Warsaw, Poland, 02-172
        • Recruiting
        • GSK Investigational Site
        • Contact:
        • Contact:
        • Principal Investigator:
          • Krzysztof Madry
    • Silesian Voivodeship
      • Chorzów, Silesian Voivodeship, Poland, 40-523
        • Recruiting
        • GSK Investigational Site
        • Contact:
        • Contact:
        • Principal Investigator:
          • Sebastian Grosicki
  • South Korea
      • Seongnam-si Gyeonggi-do, South Korea, 13620
        • Recruiting
        • GSK Investigational Site
        • Contact:
        • Contact:
        • Principal Investigator:
          • Soo-Mee Bang
      • Seoul, South Korea, 03080
        • Recruiting
        • GSK Investigational Site
        • Principal Investigator:
          • JunShik Hong
        • Contact:
        • Contact:
      • Seoul, South Korea, 06591
        • Recruiting
        • GSK Investigational Site
        • Contact:
        • Contact:
        • Principal Investigator:
          • Yoo-Jin Kim
      • Seoul, South Korea, 05505
        • Recruiting
        • GSK Investigational Site
        • Contact:
        • Contact:
        • Principal Investigator:
          • JeHwan Lee
  • Spain
      • Barcelona, Spain, 8035
        • Recruiting
        • GSK Investigational Site
        • Contact:
        • Contact:
        • Principal Investigator:
          • David Valcarcel Ferreiras
      • Barcelona, Spain, 8907
        • Recruiting
        • GSK Investigational Site
        • Contact:
        • Contact:
        • Principal Investigator:
          • Montserrat Arnan
      • Madrid, Spain, 28027
        • Recruiting
        • GSK Investigational Site
        • Principal Investigator:
          • Ana Alfonso Pierola
        • Contact:
        • Contact:
      • Málaga, Spain, 29010
        • Recruiting
        • GSK Investigational Site
        • Contact:
        • Contact:
        • Principal Investigator:
          • Regina Garcia Delgado
      • Ourense, Spain, 32005
        • Recruiting
        • GSK Investigational Site
        • Contact:
        • Contact:
        • Principal Investigator:
          • José Luis Sastre Moral
      • PamplonaNavarra, Spain, 31008
        • Recruiting
        • GSK Investigational Site
        • Principal Investigator:
          • Ana Alfonso Pierola
        • Contact:
        • Contact:
      • Salamanca, Spain, 37007
        • Recruiting
        • GSK Investigational Site
        • Principal Investigator:
          • María Díez Campelo
        • Contact:
        • Contact:
      • Valencia, Spain, 46010
        • Recruiting
        • GSK Investigational Site
        • Principal Investigator:
          • María del Mar Tormo Díaz
        • Contact:
        • Contact:
  • United Kingdom
      • Boston, United Kingdom, PE21 9QS
        • Recruiting
        • GSK Investigational Site
        • Contact:
        • Contact:
        • Principal Investigator:
          • Ciro Rinaldi
      • London, United Kingdom, SE5 9RS
        • Recruiting
        • GSK Investigational Site
        • Principal Investigator:
          • Austin Kulasekararaj
        • Contact:
        • Contact:
      • Manchester, United Kingdom, M20 4BX
        • Recruiting
        • GSK Investigational Site
        • Contact:
        • Contact:
        • Principal Investigator:
          • Daniel Wiseman
  • United States
    • Arizona
      • Goodyear, Arizona, United States, 85338
        • Recruiting
        • GSK Investigational Site
        • Contact:
        • Contact:
        • Principal Investigator:
          • Zawit Misam
    • California
      • Duarte, California, United States, 91010-3012
        • Recruiting
        • GSK Investigational Site
        • Contact:
        • Contact:
        • Principal Investigator:
          • Brian Ball
      • Irvine, California, United States, 92618
        • Recruiting
        • GSK Investigational Site
        • Contact:
        • Contact:
        • Principal Investigator:
          • Brian Ball
    • Connecticut
      • New Haven, Connecticut, United States, 06519-1110
        • Recruiting
        • GSK Investigational Site
        • Principal Investigator:
          • Amer Zeidan
        • Contact:
        • Contact:
    • Ohio
      • Canton, Ohio, United States, 44718
        • Recruiting
        • GSK Investigational Site
        • Contact:
        • Contact:
        • Principal Investigator:
          • Nashat Gabrail
    • Texas
      • Houston, Texas, United States, 77030
        • Recruiting
        • GSK Investigational Site
        • Contact:
        • Contact:
        • Principal Investigator:
          • Guillermo Garcia-Manero

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

Inclusion criteria

  • Age ≥18 years or of legal age of consent in the jurisdiction in which the study is taking place, at the time of signing the informed consent form (ICF).
  • Documented diagnosis of MDS according to the World Health Organization classifications with an Revised International Prognostic Scoring System (IPSS-R) classification of very low, low, or intermediate risk disease, with an overall risk score ≤3.5 and bone marrow blasts < 5%.

  • Received only one prior line of treatment with either Erythropoiesis-stimulating agent (ESA) or luspatercept for LR-MDS-related anemia that is relapsed/refractory to therapy. Participants intolerant OR ineligible to prior ESA or luspatercept will fulfill this inclusion criterion provided the definition below is met.

    • Refractory to prior treatment: documentation of loss of erythroid (E) response or never achieved HI-E response as defined by the IWG 2018 criteria.
    • Intolerant to prior treatment: documentation of reasons for discontinuation of prior ESA containing regimen, either as single agent or combination (e.g., G-CSF) or luspatercept due to intolerance or adverse event.
    • ESA ineligible: low chance of response to ESA based on endogenous serum erythropoietin level > 200 U/L for participants not previously treated with ESAs.
  • Red blood cell transfusion dependence, defined as requiring ≥3 units of Packed red blood cells (pRBC) transfused over 16-week period in at least 2 transfusions episodes during the 16 weeks preceding randomization. Documentation of a participant's transfusion policy during this 16-week period is required.
  • A female participant is eligible to participate if she is not pregnant or breastfeeding and one of the following conditions applies:
  • Is a woman of non-childbearing potential (WONCBP). OR
  • Is a woman of childbearing potential (WOCBP) and using a contraceptive method.
  • Is capable of giving signed informed consent.
  • Eastern Cooperative Oncology Group performance status ≤2.
  • Adequate organ function.

Exclusion criteria

  • Prior treatment with the following with noted time periods:

    1. Janus kinase (JAK)1/2 inhibitors;
    2. ACVR1 inhibitors,
    3. ACTRII receptor ligand trap other than luspatercept
    4. Hypomethylating agents or other disease modifying agents (i.e., IMiDs) and immunosuppressive therapy for MDS
    5. ESA within 4 weeks, or 8 weeks for long-acting ESA.
    6. Growth factors (i.e., G-CSF, GM-CSF) within 4 weeks.
    7. Luspatercept within 8 weeks.
    8. Investigational agents within 4 weeks or 5 half-lives, whichever is longer.
    9. Corticosteroids for treatment of the underlying disease within 28 days. Supportive care use of steroids for non-MDS indications may be used provided participant is on a stable dose equivalent to ≤10 mg prednisone per day.
    10. Other active anti-MDS therapy not otherwise listed within 28 days or 5 half-lives whichever is longer.
    11. Potent cytochrome P450 3A4 (CYP3A4) inducers, except for rifampin and rifampicin, within 14 days prior to the first dose of momelotinib.
    12. Has received a live vaccine within 30 days.
  • Prior allogeneic or autologous stem cell transplant.
  • Has had any major surgery within 28 days prior to randomization.
  • Ongoing adverse reaction(s) from prior therapy that have not recovered to ≤Grade 1 or to the baseline status preceding prior therapy, except if the investigator, with the agreement of the sponsor, considers to be not clinically relevant for the tolerability of study intervention in the current clinical study.
  • MDS associated with del 5q cytogenetic abnormality.
  • MDS/ Myeloproliferative neoplasm (MPN) overlap disorders (e.g., Chronic Myelomonocytic Leukemia [CMML]).
  • Secondary MDS (i.e., MDS that is known to have arisen as the result of chemical injury, treatment with chemotherapy, and/or radiation for other diseases).
  • Known history of diagnosis of acute myeloid leukemia.
  • Known clinically significant anemia due to iron, vitamin B12, or folate deficiencies, or autoimmune or hereditary hemolytic anemia, gastrointestinal bleeding, or thalassemia.

  • Diagnosis of invasive malignancy or history of invasive malignancy other than the disease under study within the last 5 years, except as noted below:

    1. History of an invasive malignancy for which the participant was definitively treated, and in which the participant has been disease free for at least 2 years, and which, in the opinion of the principal investigator and medical monitor, is not expected to affect the evaluation of the effects of the study intervention on the currently targeted disease under study.
    2. Curatively treated basal cell carcinoma of the skin, superficial bladder cancer, squamous cell carcinoma of the skin, in situ cervical cancer, and/or in situ breast cancer may be enrolled.
    3. Incidental histologic finding of prostate cancer (T1a or T1b using the Tumor, nodes, metastasis [TNM] clinical staging system).

  • Uncontrolled intercurrent illness including, but not limited to:

    1. Active uncontrolled infection (participants receiving outpatient antibacterial and/or antiviral treatments for infection that is under control or as infection prophylaxis may be included in the trial); or
    2. Significant active or chronic bleeding event ≥Grade 2 per Common Terminology Criteria for Adverse Events version 5.0 (CTCAE v5.0) within 4 weeks prior randomization.
    3. Uncontrolled acute and chronic liver disease (e.g., Child-Pugh score ≥10) OR has current unstable liver or biliary disease per investigator assessment defined by the presence of ascites, encephalopathy, coagulopathy, hypoalbuminemia, esophageal or gastric varices, persistent jaundice, or cirrhosis.

  • Any of the following conditions within 6 months prior to randomization:

    1. Unstable angina pectoris.
    2. Symptomatic congestive heart failure.
    3. Uncontrolled cardiac arrhythmia.
  • QTc interval >480 milliseconds (msec) (corrected using Fridericia formula).
  • Psychiatric illness, social situation, or any other condition that would limit compliance with trial requirements or may interfere with the interpretation of study results, as judged by investigator or sponsor.
  • Presence of peripheral neuropathy ≥Grade 2 per CTCAE v5.0.
  • Known positive status for human immunodeficiency virus (HIV).

  • Hepatitis B or C status as defined below:

    1. Active Hepatitis B infection indicated by the presence of hepatitis B surface antigen (HBsAg) at screening or within 3 months prior to the first dose of study intervention.
    2. Positive hepatitis C antibody test result at screening or within 3 months before the first dose of study intervention. Has any clinically significant gastrointestinal conditions or abnormalities that may alter absorption, e.g., uncontrolled nausea, vomiting, malabsorption syndrome or major resection of the stomach and/or bowels.
  • Is unable to swallow and/or retain oral medications.
  • Known contraindication or hypersensitivity to momelotinib and its metabolites, or any of their excipients.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Momelotinib dose level 1
Drug: Momelotinib
Momelotinib will be administered.
Other Names:
  • GSK3070785
Experimental: Momelotinib dose level 2
Drug: Momelotinib
Momelotinib will be administered.
Other Names:
  • GSK3070785

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Percentage of participants with Red Blood Cells - transfusion independence (RBC-TI) for at least 12 weeks, rolling over 24 weeks
Time Frame: Up to 24 weeks
RBC-TI defined as not requiring RBC transfusions (except in the case of clinically overt bleeding). Percentage of participants with RBC-TI will be measured for any consecutive 12-week interval over 24-week duration.
Up to 24 weeks
Number of participants with Grade 3 Adverse events (AEs), AE leading to treatment discontinuation and AEs leading to dose modifications
Time Frame: Up to approximately 109 weeks
Up to approximately 109 weeks
Maximum plasma concentration (Cmax) of momelotinib and major metabolite of momelotinib (M21)
Time Frame: Up to 24 weeks
Up to 24 weeks
Area under the plasma concentration versus time curve (AUC) of momelotinib and M21
Time Frame: Up to 24 weeks
Up to 24 weeks

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Percentage of participants with ≥16 weeks of RBC-TI by the end of Week 24
Time Frame: At week 24
RBC-TI defined as not requiring RBC transfusions (except in the case of clinically overt bleeding). Percentage of participants with RBC-TI will be measured for any consecutive ≥16-week interval by the end of Week 24.
At week 24
Percentage of participants with ≥1.5 grams per deciliter (g/dL) increase in hemoglobin
Time Frame: Up to 24 weeks
Up to 24 weeks
Percentage of participants with ≥24 weeks of RBC-TI by the end of Week 48
Time Frame: At week 48
RBC-TI defined as not requiring RBC transfusions (except in the case of clinically overt bleeding). Percentage of participants with RBC-TI will be measured for any consecutive ≥24-week interval by the end of Week 48.
At week 48
Number of participants with hematologic improvement (HI-E) response per International Working Group (IWG) 2018 criteria
Time Frame: Up to 24 weeks
HI-E response is measured based on the combined incidence of: Low transfusion burden (LTB) patients: absence of any transfusion for ≥ 8 consecutive weeks. High transfusion burden (HTB) patients: minor response defined as reduction by ≥ 50% of red blood cell (RBC) units for ≥ 8 consecutive weeks. Major response defined as absence of RBC transfusions for ≥ 8 consecutive weeks or longer up to 24 weeks
Up to 24 weeks
Time to RBC-TI by Weeks 24 and 48
Time Frame: Up to Weeks 24 and 48
Time to RBC-TI defined as the time from the first dose of study intervention until the first date of TI (absence of RBC transfusion requirement).
Up to Weeks 24 and 48
Number of participants with AEs, Serious adverse events (SAEs), AEs leading to treatment discontinuation, and AEs leading to dose modifications
Time Frame: Up to approximately 133 weeks
Up to approximately 133 weeks
Number of participants with AEs, SAEs, AEs leading to treatment discontinuation, and AEs leading to dose modifications by Severity
Time Frame: Up to approximately 133 weeks
Up to approximately 133 weeks
Number of participants with clinically significant changes in laboratory parameters
Time Frame: Up to approximately 133 weeks
Up to approximately 133 weeks
Number of participants with clinically significant changes in vital signs
Time Frame: Up to approximately 133 weeks
Up to approximately 133 weeks
Plasma concentration of momelotinib and M21
Time Frame: Up to 24 weeks
Up to 24 weeks

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

GlaxoSmithKline

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

June 5, 2025

Primary Completion (Estimated)

July 14, 2027

Study Completion (Estimated)

December 29, 2027

Study Registration Dates

First Submitted

February 21, 2025

First Submitted That Met QC Criteria

February 21, 2025

First Posted (Actual)

February 26, 2025

Study Record Updates

Last Update Posted (Actual)

June 2, 2026

Last Update Submitted That Met QC Criteria

May 29, 2026

Last Verified

May 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 223584
  • 2024-519928-24 (Other Identifier: EU CT Number)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

IPD Plan Description

GSK will assess requests from qualified researchers for anonymized individual patient-level data and related study documents. Data sharing is subject to certain criteria, conditions, and exceptions. For further information, refer to https://www.gsk-studyregister.com/gsk-patient-level-data-sharing-july2025.pdf

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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