Myelodysplastic SyndromesPhase 2MIDASNCT06847867
A Study of Momelotinib in Participants With Low-risk Myelodysplastic Syndrome
A Phase 2 study testing Momelotinib in people with Myelodysplastic Syndromes, run by GlaxoSmithKline at 39 sites in 9 countries.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Myelodysplastic Syndromes
- What is being tested?
- Momelotinib
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 80 participants
- Where
- 39 sitesin 9 countries
- Study dates
- 5 Jun 2025to 29 Dec 2027
- 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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
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- 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.
Who cannot join
- Prior treatment with the following with noted time periods:
- Janus kinase (JAK)1/2 inhibitors;
- ACVR1 inhibitors,
- ACTRII receptor ligand trap other than luspatercept
- Hypomethylating agents or other disease modifying agents (i.e., IMiDs) and immunosuppressive therapy for MDS
- ESA within 4 weeks, or 8 weeks for long-acting ESA.
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- Growth factors (i.e., G-CSF, GM-CSF) within 4 weeks.
- Luspatercept within 8 weeks.
- Investigational agents within 4 weeks or 5 half-lives, whichever is longer.
- 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.
- Other active anti-MDS therapy not otherwise listed within 28 days or 5 half-lives whichever is longer.
- Potent cytochrome P450 3A4 (CYP3A4) inducers, except for rifampin and rifampicin, within 14 days prior to the first dose of momelotinib.
- 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:
- 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.
- 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.
- 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:
- 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
- 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.
- 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:
- Unstable angina pectoris.
- Symptomatic congestive heart failure.
- 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:
- 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.
- 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.
Trial locations and contacts
39 sites in 9 countries. Contact the study team or a site near you to ask about taking part.
39 sites in 9 countries
Spain8 sites, 8 recruiting
- Recruiting
GSK Investigational Site
Barcelona, 8035
US GSK Clinical Trials Call Center, 877-379-3718, GSKClinicalSupportHD@gsk.com
EU GSK Clinical Trials Call Centre, +44 (0) 20 8990 4466, GSKClinicalSupportHD@gsk.com
- Recruiting
GSK Investigational Site
Barcelona, 8907
US GSK Clinical Trials Call Center, 877-379-3718, GSKClinicalSupportHD@gsk.com
EU GSK Clinical Trials Call Centre, +44 (0) 20 8990 4466, GSKClinicalSupportHD@gsk.com
- Recruiting
GSK Investigational Site
Madrid, 28027
US GSK Clinical Trials Call Center, 877-379-3718, GSKClinicalSupportHD@gsk.com
EU GSK Clinical Trials Call Centre, +44 (0) 20 8990 4466, GSKClinicalSupportHD@gsk.com
- Recruiting
GSK Investigational Site
Málaga, 29010
US GSK Clinical Trials Call Center, 877-379-3718, GSKClinicalSupportHD@gsk.com
EU GSK Clinical Trials Call Centre, +44 (0) 20 8990 4466, GSKClinicalSupportHD@gsk.com
- Recruiting
GSK Investigational Site
Ourense, 32005
US GSK Clinical Trials Call Center, 877-379-3718, GSKClinicalSupportHD@gsk.com
EU GSK Clinical Trials Call Centre, +44 (0) 20 8990 4466, GSKClinicalSupportHD@gsk.com
- Recruiting
GSK Investigational Site
PamplonaNavarra, 31008
US GSK Clinical Trials Call Center, 877-379-3718, GSKClinicalSupportHD@gsk.com
EU GSK Clinical Trials Call Centre, +44 (0) 20 8990 4466, GSKClinicalSupportHD@gsk.com
- Recruiting
GSK Investigational Site
Salamanca, 37007
US GSK Clinical Trials Call Center, 877-379-3718, GSKClinicalSupportHD@gsk.com
EU GSK Clinical Trials Call Centre, +44 (0) 20 8990 4466, GSKClinicalSupportHD@gsk.com
- Recruiting
GSK Investigational Site
Valencia, 46010
US GSK Clinical Trials Call Center, 877-379-3718, GSKClinicalSupportHD@gsk.com
EU GSK Clinical Trials Call Centre, +44 (0) 20 8990 4466, GSKClinicalSupportHD@gsk.com
United States6 sites, 6 recruiting
- Recruiting
GSK Investigational Site
Canton, Ohio, 44718
US GSK Clinical Trials Call Center, 877-379-3718, GSKClinicalSupportHD@gsk.com
EU GSK Clinical Trials Call Centre, +44 (0) 20 8990 4466, GSKClinicalSupportHD@gsk.com
- Recruiting
GSK Investigational Site
Duarte, California, 91010-3012
US GSK Clinical Trials Call Center, 877-379-3718, GSKClinicalSupportHD@gsk.com
EU GSK Clinical Trials Call Centre, +44 (0) 20 8990 4466, GSKClinicalSupportHD@gsk.com
- Recruiting
GSK Investigational Site
Goodyear, Arizona, 85338
US GSK Clinical Trials Call Center, 877-379-3718, GSKClinicalSupportHD@gsk.com
EU GSK Clinical Trials Call Centre, +44 (0) 20 8990 4466, GSKClinicalSupportHD@gsk.com
- Recruiting
GSK Investigational Site
Houston, Texas, 77030
US GSK Clinical Trials Call Center, 877-379-3718, GSKClinicalSupportHD@gsk.com
EU GSK Clinical Trials Call Centre, +44 (0) 20 8990 4466, GSKClinicalSupportHD@gsk.com
- Recruiting
GSK Investigational Site
Irvine, California, 92618
US GSK Clinical Trials Call Center, 877-379-3718, GSKClinicalSupportHD@gsk.com
EU GSK Clinical Trials Call Centre, +44 (0) 20 8990 4466, GSKClinicalSupportHD@gsk.com
- Recruiting
GSK Investigational Site
New Haven, Connecticut, 06519-1110
US GSK Clinical Trials Call Center, 877-379-3718, GSKClinicalSupportHD@gsk.com
EU GSK Clinical Trials Call Centre, +44 (0) 20 8990 4466, GSKClinicalSupportHD@gsk.com
Italy6 sites, 6 recruiting
- Recruiting
GSK Investigational Site
Catania, 95123
US GSK Clinical Trials Call Center, 877-379-3718, GSKClinicalSupportHD@gsk.com
EU GSK Clinical Trials Call Centre, +44 (0) 20 8990 4466, GSKClinicalSupportHD@gsk.com
- Recruiting
GSK Investigational Site
Florence, 50134
US GSK Clinical Trials Call Center, 877-379-3718, GSKClinicalSupportHD@gsk.com
EU GSK Clinical Trials Call Centre, +44 (0) 20 8990 4466, GSKClinicalSupportHD@gsk.com
- Recruiting
GSK Investigational Site
Milan, 20122
US GSK Clinical Trials Call Center, 877-379-3718, GSKClinicalSupportHD@gsk.com
EU GSK Clinical Trials Call Centre, +44 (0) 20 8990 4466, GSKClinicalSupportHD@gsk.com
- Recruiting
GSK Investigational Site
Orbassano to, 10043
US GSK Clinical Trials Call Center, 877-379-3718, GSKClinicalSupportHD@gsk.com
EU GSK Clinical Trials Call Centre, +44 (0) 20 8990 4466, GSKClinicalSupportHD@gsk.com
- Recruiting
GSK Investigational Site
Rozzano MI, 20089
US GSK Clinical Trials Call Center, 877-379-3718, GSKClinicalSupportHD@gsk.com
EU GSK Clinical Trials Call Centre, +44 (0) 20 8990 4466, GSKClinicalSupportHD@gsk.com
- Recruiting
GSK Investigational Site
Udine, Friuli Venezia Giulia, 33100
US GSK Clinical Trials Call Center, 877-379-3718, GSKClinicalSupportHD@gsk.com
EU GSK Clinical Trials Call Centre, +44 (0) 20 8990 4466, GSKClinicalSupportHD@gsk.com
France5 sites, 5 recruiting
- Recruiting
GSK Investigational Site
Le Mans, 72015
US GSK Clinical Trials Call Center, 877-379-3718, GSKClinicalSupportHD@gsk.com
EU GSK Clinical Trials Call Centre, +44 (0) 20 8990 4466, GSKClinicalSupportHD@gsk.com
- Recruiting
GSK Investigational Site
Nice, 06202
US GSK Clinical Trials Call Center, 877-379-3718, GSKClinicalSupportHD@gsk.com
EU GSK Clinical Trials Call Centre, +44 (0) 20 8990 4466, GSKClinicalSupportHD@gsk.com
- Recruiting
GSK Investigational Site
Paris, 75010
US GSK Clinical Trials Call Center, 877-379-3718, GSKClinicalSupportHD@gsk.com
EU GSK Clinical Trials Call Centre, +44 (0) 20 8990 4466, GSKClinicalSupportHD@gsk.com
- Recruiting
GSK Investigational Site
Poitiers, 86021
US GSK Clinical Trials Call Center, 877-379-3718, GSKClinicalSupportHD@gsk.com
EU GSK Clinical Trials Call Centre, +44 (0) 20 8990 4466, GSKClinicalSupportHD@gsk.com
- Recruiting
GSK Investigational Site
Toulouse, 31059
US GSK Clinical Trials Call Center, 877-379-3718, GSKClinicalSupportHD@gsk.com
EU GSK Clinical Trials Call Centre, +44 (0) 20 8990 4466, GSKClinicalSupportHD@gsk.com
South Korea4 sites, 4 recruiting
- Recruiting
GSK Investigational Site
Seongnam-si Gyeonggi-do, 13620
US GSK Clinical Trials Call Center, 877-379-3718, GSKClinicalSupportHD@gsk.com
EU GSK Clinical Trials Call Centre, +44 (0) 20 8990 4466, GSKClinicalSupportHD@gsk.com
- Recruiting
GSK Investigational Site
Seoul, 03080
US GSK Clinical Trials Call Center, 877-379-3718, GSKClinicalSupportHD@gsk.com
EU GSK Clinical Trials Call Centre, +44 (0) 20 8990 4466, GSKClinicalSupportHD@gsk.com
- Recruiting
GSK Investigational Site
Seoul, 05505
US GSK Clinical Trials Call Center, 877-379-3718, GSKClinicalSupportHD@gsk.com
EU GSK Clinical Trials Call Centre, +44 (0) 20 8990 4466, GSKClinicalSupportHD@gsk.com
- Recruiting
GSK Investigational Site
Seoul, 06591
US GSK Clinical Trials Call Center, 877-379-3718, GSKClinicalSupportHD@gsk.com
EU GSK Clinical Trials Call Centre, +44 (0) 20 8990 4466, GSKClinicalSupportHD@gsk.com
Germany3 sites, 3 recruiting
- Recruiting
GSK Investigational Site
Dresden, 01307
US GSK Clinical Trials Call Center, 877-379-3718, GSKClinicalSupportHD@gsk.com
EU GSK Clinical Trials Call Centre, +44 (0) 20 8990 4466, GSKClinicalSupportHD@gsk.com
- Recruiting
GSK Investigational Site
Leipzig, 04103
US GSK Clinical Trials Call Center, 877-379-3718, GSKClinicalSupportHD@gsk.com
EU GSK Clinical Trials Call Centre, +44 (0) 20 8990 4466, GSKClinicalSupportHD@gsk.com
- Recruiting
GSK Investigational Site
Münster, North Rhine-Westphalia, 48149
US GSK Clinical Trials Call Center, 877-379-3718, GSKClinicalSupportHD@gsk.com
EU GSK Clinical Trials Call Centre, +44 (0) 20 8990 4466, GSKClinicalSupportHD@gsk.com
United Kingdom3 sites, 3 recruiting
- Recruiting
GSK Investigational Site
Boston, PE21 9QS
US GSK Clinical Trials Call Center, 877-379-3718, GSKClinicalSupportHD@gsk.com
EU GSK Clinical Trials Call Centre, +44 (0) 20 8990 4466, GSKClinicalSupportHD@gsk.com
- Recruiting
GSK Investigational Site
London, SE5 9RS
US GSK Clinical Trials Call Center, 877-379-3718, GSKClinicalSupportHD@gsk.com
EU GSK Clinical Trials Call Centre, +44 (0) 20 8990 4466, GSKClinicalSupportHD@gsk.com
- Recruiting
GSK Investigational Site
Manchester, M20 4BX
US GSK Clinical Trials Call Center, 877-379-3718, GSKClinicalSupportHD@gsk.com
EU GSK Clinical Trials Call Centre, +44 (0) 20 8990 4466, GSKClinicalSupportHD@gsk.com
Canada2 sites, 2 recruiting
- Recruiting
GSK Investigational Site
Calgary, Alberta, T2N 5G2
US GSK Clinical Trials Call Center, 877-379-3718, GSKClinicalSupportHD@gsk.com
EU GSK Clinical Trials Call Centre, +44 (0) 20 8990 4466, GSKClinicalSupportHD@gsk.com
- Recruiting
GSK Investigational Site
Toronto, Ontario, M5G 2M9
US GSK Clinical Trials Call Center, 877-379-3718, GSKClinicalSupportHD@gsk.com
EU GSK Clinical Trials Call Centre, +44 (0) 20 8990 4466, GSKClinicalSupportHD@gsk.com
Poland2 sites, 2 recruiting
- Recruiting
GSK Investigational Site
Chorzów, Silesian Voivodeship, 40-523
US GSK Clinical Trials Call Center, 877-379-3718, GSKClinicalSupportHD@gsk.com
EU GSK Clinical Trials Call Centre, +44 (0) 20 8990 4466, GSKClinicalSupportHD@gsk.com
- Recruiting
GSK Investigational Site
Warsaw, 02-172
US GSK Clinical Trials Call Center, 877-379-3718, GSKClinicalSupportHD@gsk.com
EU GSK Clinical Trials Call Centre, +44 (0) 20 8990 4466, GSKClinicalSupportHD@gsk.com
About this study
Official title: 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.
- Conditions
- Myelodysplastic Syndromes
- Being tested
- Momelotinib
- Keywords
- Momelotinib, Low-risk Myelodysplastic Syndrome, MIDAS
- Medical subject headings
- Myelodysplastic Syndromes
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MomelotinibDrug
Also called GSK3070785
Momelotinib will be administered.
| Study group | Role | What they receive |
|---|---|---|
| Momelotinib dose level 1 | Experimental | Not described Receives: Drug: Momelotinib |
| Momelotinib dose level 2 | Experimental | Not described Receives: Drug: Momelotinib |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percentage of participants with Red Blood Cells - transfusion independence (RBC-TI) for at least 12 weeks, rolling over 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.
Measured: Up to 24 weeks
Number of participants with Grade 3 Adverse events (AEs), AE leading to treatment discontinuation and AEs leading to dose modifications
Measured: Up to approximately 109 weeks
Maximum plasma concentration (Cmax) of momelotinib and major metabolite of momelotinib (M21)
Measured: Up to 24 weeks
Area under the plasma concentration versus time curve (AUC) of momelotinib and M21
Measured: Up to 24 weeks
Secondary outcomes (10)
Percentage of participants with ≥16 weeks of RBC-TI by the end of 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.
Measured: At week 24
Percentage of participants with ≥1.5 grams per deciliter (g/dL) increase in hemoglobin
Measured: Up to 24 weeks
Percentage of participants with ≥24 weeks of RBC-TI by the end of 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.
Measured: At week 48
Number of participants with hematologic improvement (HI-E) response per International Working Group (IWG) 2018 criteria
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
Measured: Up to 24 weeks
Time to RBC-TI by 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).
Measured: 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
Measured: Up to approximately 133 weeks
Number of participants with AEs, SAEs, AEs leading to treatment discontinuation, and AEs leading to dose modifications by Severity
Measured: Up to approximately 133 weeks
Number of participants with clinically significant changes in laboratory parameters
Measured: Up to approximately 133 weeks
Number of participants with clinically significant changes in vital signs
Measured: Up to approximately 133 weeks
Plasma concentration of momelotinib and M21
Measured: Up to 24 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Feb 2025
- Study start5 Jun 2025
- Primary completion14 Jul 2027 (estimated)
- Study completion29 Dec 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Myelodysplastic SyndromesStudies of the same condition, open and under way.
- More trials run by GlaxoSmithKlineThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
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
- 80 participants (estimated)
Sponsor and investigators
- Lead sponsor
- GlaxoSmithKline
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06847867
- Sponsor's protocol ID
- 223584
- Other IDs
- 2024-519928-24
- Acronym
- MIDAS
- Status last verified
- May 2026
- First submitted
- 21 Feb 2025
- Last updated
- 2 Jun 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No: 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
Study at a glance
From the registry record, last updated by the sponsor on 2 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2, Randomized, Open-label, Study of Momelotinib in Participants With Anemia Due to Low-risk Myelodysplastic Syndrome
- ClinicalTrials.gov ID
- NCT06847867
- Acronym / sponsor ID
- MIDAS / 223584
- Conditions
- Myelodysplastic Syndromes
- Keywords
- Momelotinib, Low-risk Myelodysplastic Syndrome, MIDAS
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 80 participants (estimated)
- Lead sponsor
- GlaxoSmithKlineIndustry (pharma, biotech, devices)
- Study start
- 5 Jun 2025 (actual)
- Primary completion
- 14 Jul 2027 (estimated)
- Study completion
- 29 Dec 2027 (estimated)
- First posted
- 26 Feb 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Momelotinib dose level 1 | Experimental | Drug: Momelotinib |
| Momelotinib dose level 2 | Experimental | Drug: Momelotinib |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (13 listed)
- 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.
- 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:
Key exclusion criteria (42 listed)
- Prior treatment with the following with noted time periods:
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| MomelotinibAlso: GSK3070785Momelotinib will be administered. | Drug | Momelotinib dose level 1, Momelotinib dose level 2 |
Primary outcome
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.
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
Maximum plasma concentration (Cmax) of momelotinib and major metabolite of momelotinib (M21)
Time frame: Up to 24 weeks
Area under the plasma concentration versus time curve (AUC) of momelotinib and M21
Time frame: Up to 24 weeks
10 secondary outcomes
Percentage of participants with ≥16 weeks of RBC-TI by the end of Week 24
Time frame: At week 24
Percentage of participants with ≥1.5 grams per deciliter (g/dL) increase in hemoglobin
Time frame: Up to 24 weeks
Percentage of participants with ≥24 weeks of RBC-TI by the end of Week 48
Time frame: 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
Time to RBC-TI by Weeks 24 and 48
Time frame: 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
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
Number of participants with clinically significant changes in laboratory parameters
Time frame: Up to approximately 133 weeks
Number of participants with clinically significant changes in vital signs
Time frame: Up to approximately 133 weeks
Plasma concentration of momelotinib and M21
Time frame: Up to 24 weeks
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 Jun 2026.
- Individual participant dataSharing planned: No. 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
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
39 sites in 9 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Momelotinib in people with Myelodysplastic Syndromes, run by GlaxoSmithKline at 39 sites in 9 countries.
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 13 inclusion and 42 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: looking for participants now, as of the sponsor's last update on 2 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 39 sites in Spain, United States, Italy, France, South Korea and 4 more countries. 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. 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?
Contact US GSK Clinical Trials Call Center (877-379-3718, GSKClinicalSupportHD@gsk.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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). The study lists 4 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 2 Jun 2026.