Momelotinib for Myelodysplastic Syndrome

(MIDAS Trial)

Overseen By EU GSK Clinical Trials Call Center

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: GlaxoSmithKline

Must not be taking: JAK inhibitors, Hypomethylating agents

Disqualifiers: Stem cell transplant, Acute myeloid leukemia, Uncontrolled infection, Unstable liver disease, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

80 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a drug called momelotinib to determine its safety and effectiveness for individuals with low-risk myelodysplastic syndromes (LR-MDS), a condition where the bone marrow fails to produce enough healthy blood cells. Researchers aim to find the optimal dose by evaluating the drug's impact on red blood cell transfusion needs and its safety. Eligible participants should have LR-MDS that has not responded well to previous treatments and require regular blood transfusions. As a Phase 2 trial, the research focuses on assessing the treatment's effectiveness in an initial, smaller group of participants.

Will I have to stop taking my current medications?

The trial requires stopping certain medications before participating. You must stop taking erythropoiesis-stimulating agents (ESAs) at least 4 weeks before, and luspatercept 8 weeks before. Other medications like corticosteroids, growth factors, and certain investigational agents also have specific timeframes for discontinuation.

Is there any evidence suggesting that momelotinib is likely to be safe for humans?

Research has shown that momelotinib is generally well-tolerated. Patients with myelofibrosis, a type of bone marrow disorder, have used momelotinib, and it has maintained a consistent safety record. Specifically, one study found that some patients took momelotinib for over five years, suggesting it might be safe for long-term use. Another study suggested that momelotinib could be safer than a similar drug, fedratinib.

These findings are promising, but it's important to remember that momelotinib is still under study in this trial for its safety in people with low-risk myelodysplastic syndromes (LR-MDS). For those considering joining the trial, this past data can offer some reassurance about its safety. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for myelodysplastic syndrome?

Momelotinib is unique because it offers a new approach to treating myelodysplastic syndrome (MDS), a condition traditionally managed with treatments like azacitidine and lenalidomide. Unlike these standard therapies, which primarily target cancer cells, momelotinib works by inhibiting the JAK1 and JAK2 enzymes. This mechanism not only helps control abnormal cell growth but also reduces inflammation and improves anemia-related symptoms. Researchers are excited about momelotinib because it has the potential to offer a more comprehensive solution by addressing multiple aspects of MDS, potentially improving patient outcomes more effectively than current options.

What evidence suggests that momelotinib might be an effective treatment for low-risk myelodysplastic syndromes?

Research has shown that momelotinib may help treat blood-related conditions. In studies on myelofibrosis, a disease affecting bone marrow, momelotinib reduced anemia, and many patients no longer needed blood transfusions. Specifically, 45.8% of these patients experienced a smaller spleen, and 30.8% reported improved symptoms. Additionally, 95% of patients using momelotinib maintained their health over 24 weeks, indicating its potential effectiveness. This trial will explore different dosages of momelotinib for low-risk myelodysplastic syndromes (LR-MDS). While data for LR-MDS may be limited, these results suggest momelotinib could help manage symptoms like anemia in similar conditions. ^{1 4 6 7 8}

Are You a Good Fit for This Trial?

This trial is for individuals with low-risk myelodysplastic syndromes (LR-MDS) who have had prior treatments like ESAs or luspatercept but didn't respond well or couldn't tolerate them. They must need regular blood transfusions and be in relatively good health overall. Women participating should either not be able to bear children or use contraception.

Inclusion Criteria

I can sign and understand the consent form.

My organs are working well.

I stopped my previous ESA or luspatercept treatment due to side effects.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment Part 1

Participants receive different doses of momelotinib to find the best dose by evaluating effectiveness in improving red blood cell transfusion requirements and safety

24 weeks

Treatment Part 2

Participants receive the dose selected from Part 1 to assess its impact on improving red blood cell transfusion requirements and safety in LR-MDS

24 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Momelotinib

Trial Overview The study tests the safety and effectiveness of Momelotinib, a drug for LR-MDS patients. It has two parts: first, finding the best dose by looking at how it helps reduce the need for blood transfusions and its safety; second, using this dose to further assess its impact on reducing transfusion needs.

How Is the Trial Designed?

Group I: Part 2- Dose Exploration: Momelotinib Experimental Treatment 1 Intervention

Group II: Part 1- Dose Optimisation: Momelotinib Experimental Treatment 1 Intervention

Who Is Running the Clinical Trial?

GlaxoSmithKline

Lead Sponsor

Trials

4,834

Recruited

8,389,000+

Headquarters

London, UK

Known For

Vaccines & Medicines

Top Products

**Advair (salmeterol, fluticasone propionate)**, **Shingrix (shingles vaccine)**, **Augmentin (amoxicillin/clavulanate potassium)**, **Ventolin (salbutamol sulfate)

Dame Emma Walmsley

GlaxoSmithKline

Chief Executive Officer since 2017

MA in Classics and Modern Languages from Oxford University

Dr. Hal Barron

GlaxoSmithKline

Chief Medical Officer since 2018

MD from Harvard Medical School

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT06847867

A Study of Momelotinib in Participants With Low-risk ... The goal of this clinical trial is to determine if momelotinib is safe and effective for people with low-risk myelodysplastic syndromes (LR-MDS).

2. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC12006532/

Real world outcomes of momelotinib in myelofibrosis patients ... At the 3-month follow-up, the overall response rate (major + minor) was 71.5% in the combination treatment group and 50% in the monotherapy ...

3. link.springer.com link.springer.com/article/10.1007/s12672-024-01252-1

Momelotinib in myelofibrosis and beyond The outcomes included anemia response in 17% and 21% by IWG-MRT criteria, spleen response in 45.8%, symptom improvement in 30.8%, and a 28.2% ...

4. bloodcancerstoday.com bloodcancerstoday.com/post/efficacy-of-momelotinib-in-reducing-anemia-in-mf-confirmed-with-real-world-data

Efficacy of Momelotinib in Reducing Anemia in MF ... A real-world, retrospective analysis showed that momelotinib reduces anemia in patients with myelofibrosis (MF), with many achieving transfusion independence.

5. ajmc.com ajmc.com/view/ash-2023-coverage-in-mds-myeloproliferative-disease

ASH 2023: Coverage in MDS, Myeloproliferative Disease Among these patients, 95% (142 of 150) taking momelotinib maintained their status over 24 weeks compared with 57% (93 of 163) of patients taking ...

6. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC10368854/

Momelotinib long-term safety and survival in myelofibrosis As of the data cutoff (3 December 2021), 12.1% of patients were treated for ≥5 years, including 75% of patients (88/118) who continued momelotinib in the XAP; ...

7. ashpublications.org ashpublications.org/blood/article/140/Supplement%201/9670/491483/Momelotinib-MMB-Long-Term-Safety-Pooled-Data-from

Momelotinib (MMB) Long-Term Safety: Pooled Data from ... Conclusion: We report the largest trial safety database to date for a JAK inhibitor in MF. MMB demonstrated a consistent safety profile without ...

8. rarediseaseadvisor.com rarediseaseadvisor.com/news/momelotinib-appears-favorable-safety-profile-mf-care/

Momelotinib Appears to Have a Favorable Safety Profile in ... Momelotinib seems to have a more favorable safety profile than fedratinib in patients with myelofibrosis (MF), a study found.