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

NCT06847867 | Recruiting Phase 2 FDA Drug

• 2 other identifiers: 2235842024-519928-24
• Study Type: interventional
• Target: 80
• Locations: 9 countries
• Sites: 39

Brief 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. 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

Keywords

Momelotinib; Low-risk Myelodysplastic Syndrome; MIDAS

Outcome Measures

Primary Outcomes (4)

• 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.

  Up to 24 weeks

• Number of participants with Grade 3 Adverse events (AEs), AE leading to treatment discontinuation and AEs leading to dose modifications

  Up to approximately 109 weeks

• Maximum plasma concentration (Cmax) of momelotinib and major metabolite of momelotinib (M21)

  Up to 24 weeks

• Area under the plasma concentration versus time curve (AUC) of momelotinib and M21

  Up to 24 weeks

Secondary Outcomes (10)

• Percentage of participants with ≥16 weeks of RBC-TI by the end of Week 24

  At week 24

• Percentage of participants with ≥1.5 grams per deciliter (g/dL) increase in hemoglobin

  Up to 24 weeks

• Percentage of participants with ≥24 weeks of RBC-TI 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

  Up to 24 weeks

• Time to RBC-TI by Weeks 24 and 48

  Up to Weeks 24 and 48

Study Arms (2)

Momelotinib dose level 1

EXPERIMENTAL

Drug: Momelotinib

Momelotinib dose level 2

EXPERIMENTAL

Drug: Momelotinib

Interventions

Momelotinib DRUG

Momelotinib will be administered.

Also known as: GSK3070785

Momelotinib dose level 1; Momelotinib dose level 2

Eligibility Criteria

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

You may qualify if:

• 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%.
• 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.

You may not qualify if:

• 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.
• 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.

Sponsors & Collaborators

• GlaxoSmithKline: lead

Study Sites (39)

GSK Investigational Site

Goodyear, Arizona, 85338, United States

RECRUITING

GSK Investigational Site

Duarte, California, 91010-3012, United States

RECRUITING

GSK Investigational Site

Irvine, California, 92618, United States

RECRUITING

GSK Investigational Site

New Haven, Connecticut, 06519-1110, United States

RECRUITING

GSK Investigational Site

Canton, Ohio, 44718, United States

RECRUITING

GSK Investigational Site

Houston, Texas, 77030, United States

RECRUITING

GSK Investigational Site

Calgary, Alberta, T2N 5G2, Canada

RECRUITING

GSK Investigational Site

Toronto, Ontario, M5G 2M9, Canada

RECRUITING

GSK Investigational Site

Le Mans, 72015, France

RECRUITING

GSK Investigational Site

Nice, 06202, France

RECRUITING

GSK Investigational Site

Paris, 75010, France

RECRUITING

GSK Investigational Site

Poitiers, 86021, France

RECRUITING

GSK Investigational Site

Toulouse, 31059, France

RECRUITING

GSK Investigational Site

Münster, North Rhine-Westphalia, 48149, Germany

RECRUITING

GSK Investigational Site

Dresden, 01307, Germany

RECRUITING

GSK Investigational Site

Leipzig, 04103, Germany

RECRUITING

GSK Investigational Site

Udine, Friuli Venezia Giulia, 33100, Italy

RECRUITING

GSK Investigational Site

Catania, 95123, Italy

RECRUITING

GSK Investigational Site

Florence, 50134, Italy

RECRUITING

GSK Investigational Site

Milan, 20122, Italy

RECRUITING

GSK Investigational Site

Orbassano to, 10043, Italy

RECRUITING

GSK Investigational Site

Rozzano MI, 20089, Italy

RECRUITING

GSK Investigational Site

Chorzów, Silesian Voivodeship, 40-523, Poland

RECRUITING

GSK Investigational Site

Warsaw, 02-172, Poland

RECRUITING

GSK Investigational Site

Seongnam-si Gyeonggi-do, 13620, South Korea

RECRUITING

GSK Investigational Site

Seoul, 03080, South Korea

RECRUITING

GSK Investigational Site

Seoul, 05505, South Korea

RECRUITING

GSK Investigational Site

Seoul, 06591, South Korea

RECRUITING

GSK Investigational Site

Barcelona, 8035, Spain

RECRUITING

GSK Investigational Site

Barcelona, 8907, Spain

RECRUITING

GSK Investigational Site

Madrid, 28027, Spain

RECRUITING

GSK Investigational Site

Málaga, 29010, Spain

RECRUITING

GSK Investigational Site

Ourense, 32005, Spain

RECRUITING

GSK Investigational Site

PamplonaNavarra, 31008, Spain

RECRUITING

GSK Investigational Site

Salamanca, 37007, Spain

RECRUITING

GSK Investigational Site

Valencia, 46010, Spain

RECRUITING

GSK Investigational Site

Boston, PE21 9QS, United Kingdom

RECRUITING

GSK Investigational Site

London, SE5 9RS, United Kingdom

RECRUITING

GSK Investigational Site

Manchester, M20 4BX, United Kingdom

RECRUITING

MeSH Terms

Conditions

Myelodysplastic Syndromes

Interventions

N-(cyanomethyl)-4-(2-((4-(4-morpholinyl)phenyl)amino)-4-pyrimidinyl)benzamide

Condition Hierarchy (Ancestors)

Bone Marrow Diseases; Hematologic Diseases; Hemic and Lymphatic Diseases

Central Study Contacts

US GSK Clinical Trials Call Center

CONTACT

877-379-3718; GSKClinicalSupportHD@gsk.com

EU GSK Clinical Trials Call Center

CONTACT

+44 (0) 20 89904466; GSKClinicalSupportHD@gsk.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 21, 2025
• First Posted: February 26, 2025
• Study Start: June 5, 2025
• Primary Completion (Estimated): July 14, 2027
• Study Completion (Estimated): December 29, 2027
• Last Updated: June 2, 2026

Record last verified: 2026-05

Data Sharing

• IPD Sharing: Will not share

Locations

US (6); DE (3); ES (8); IT (6); PL (2); FR (5); KR (4); CA (2); GB (3)