Momelotinib + Azacitidine for Myelodysplastic Syndrome
(M-HArbOr Trial)
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial tests a new treatment combination for individuals with certain blood disorders, such as myelodysplastic syndrome (MDS) or chronic neutrophilic leukemia (CNL). It combines a study drug, momelotinib, with the usual treatment, azacitidine, to determine if it can better manage the disease, improve symptoms, and increase the number of patients eligible for a transplant. Participants must have a diagnosis of MDS or CNL and may have tried other treatments, but not momelotinib. The study aims to assess the safety and effectiveness of this new treatment while monitoring for side effects, such as infections or bruising. As an Early Phase 1 trial, this research focuses on understanding how the treatment works in people, offering participants a chance to be among the first to receive this new treatment combination.
Is there any evidence suggesting that this trial's treatments are likely to be safe?
Research has shown that momelotinib, when used alone, appears safe for long-term use. Some individuals have taken it for over five years without major issues. However, combining momelotinib with azacitidine raises some concerns. Azacitidine has been linked to side effects such as infections and low blood cell counts, which can lead to bruising or bleeding. This trial is in its early stages, so limited information exists about the safety of using these drugs together. Participants might experience side effects, but researchers are diligently working to find the best way to use these drugs in combination.12345
Why do researchers think this study treatment might be promising?
Researchers are excited about Momelotinib combined with Azacitidine for treating Myelodysplastic Syndrome because it introduces a novel approach by combining a JAK1/2 inhibitor (Momelotinib) with a hypomethylating agent (Azacitidine). Unlike standard treatments, which typically rely solely on hypomethylating agents to alter DNA and slow disease progression, this combination targets both the disease's genetic and inflammatory pathways, potentially enhancing effectiveness. Additionally, the study explores dose escalation to optimize the balance between efficacy and safety, aiming to find the maximum tolerated dose that could offer better patient outcomes.
What evidence suggests that momelotinib plus azacitidine might be an effective treatment for myelodysplastic syndrome?
Research has shown that combining momelotinib with azacitidine may help treat myelodysplastic syndromes (MDS). In earlier studies, 95% of patients taking momelotinib remained healthy over 24 weeks. This combination effectively improved symptoms such as a swollen spleen and reduced disease indicators. Additionally, 54% of patients showed clear signs of improvement. This trial will evaluate the effectiveness of momelotinib with azacitidine, suggesting potential benefits for people with MDS.24567
Who Is on the Research Team?
Tania Jain, MD
Principal Investigator
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Are You a Good Fit for This Trial?
Inclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Dose Escalation
Participants receive momelotinib at 150 mg daily in combination with azacitidine. Dose may be escalated to 200 mg based on DLT assessment.
Expansion
Participants receive treatment at the maximum tolerated dose determined in the dose escalation phase.
Follow-up
Participants are monitored for safety and effectiveness after treatment completion.
What Are the Treatments Tested in This Trial?
Interventions
- Azacitidine
- Momelotinib
How Is the Trial Designed?
1
Treatment groups
Experimental Treatment
Combination of once daily oral momelotinib plus hypomethylating agents (HMA), e.g. azacitidine) starting cycle 1. Azacitidine will be administered as an injection at 75 mg/m2 for days 1-5 in a 28-day cycle. Dose escalation Phase: The first 3 patients will receive MMB 150 mg daily in combination with azacitidine. Depending on the number of dose limiting toxicities (DLTs) experienced during this period, participants in this phase of the study will receive either 150mg daily or have an escalated dose of 200mg daily. Depending on DLT assessment during this phase of the study, the maximum tolerated dose (MTD) of either 150mg or 200mg will be determined. Expansion Phase: Once the MTD is determined (either 150 mg or 200 mg), all remaining patients up to the total of 18 evaluable patients will be treated at the MTD.
Find a Clinic Near You
Who Is Running the Clinical Trial?
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Lead Sponsor
GlaxoSmithKline
Industry Sponsor
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
A phase II trial of ruxolitinib in combination with azacytidine in ...
As expected, the combination was particularly effective in improving proliferative features including ICP MDS/MPN reductions in palpable splenomegaly and ...
2.
ashpublications.org
ashpublications.org/blood/article/142/Supplement%201/1861/504885/Five-Year-Follow-up-Results-of-Phase-II-ClinicalFive-Year Follow up Results of Phase II Clinical Trial ...
Objective responses per the MDS/MPN international consensus response criteria were observed in 28 (54%) pts and included clinical improvement in ...
Momelotinib for Myelodysplastic Syndrome (MIDAS Trial)
Additionally, 95% of patients using momelotinib maintained their health over 24 weeks, indicating its potential effectiveness. This trial will explore different ...
A Pilot Study of Momelotinib in combination with ...
Study design: This is an open-label study of MMB-HMA in MDS/MPN and CNL. We will enroll a total of 18 evaluable patients using a modified ...
5.
ctv.veeva.com
ctv.veeva.com/study/a-study-of-momelotinib-in-participants-with-low-risk-myelodysplastic-syndromeA 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).
Safety and efficacy of azacitidine in myelodysplastic syndromes
Notably, 26% of patients with refractory disease had a documented complete response, and the median overall survival was 40 weeks. A prospective clinical trial ...
7.
hematologyadvisor.com
hematologyadvisor.com/news/azacitidine-myelodysplastic-syndrome-mds-adverse-events-safety-concerns/New Adverse Events Highlight Safety Concerns With ...
An analysis of 2 pharmacovigilance databases revealed a high prevalence of azacitidine-related adverse events in patients with MDS or AML.
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