Momelotinib for Myelofibrosis
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 tolerability for people with myelofibrosis, a type of bone marrow cancer, during and after a bone marrow transplant. Participants will receive momelotinib alongside their usual transplant procedure to assess its effectiveness and how the body processes it. The trial seeks individuals with confirmed myelofibrosis who need a bone marrow transplant and may have specific risk factors, such as requiring frequent blood transfusions or possessing certain genetic markers. As a Phase 1 trial, the research focuses on understanding how the treatment works in people, offering participants the opportunity to be among the first to receive this new treatment.
Do I have to stop taking my current medications for the trial?
If you are currently taking JAK inhibitors other than momelotinib, you will need to switch to momelotinib before starting the trial. The protocol does not specify about other medications, so it's best to discuss with the trial team.
Is there any evidence suggesting that momelotinib is likely to be safe for humans?
Research has shown that momelotinib is generally well-tolerated by patients with myelofibrosis. Studies have found that patients taking momelotinib often experience a good response and improvements in quality of life, particularly for anemia-related symptoms. Long-term safety data indicate that many patients continued treatment for several years, suggesting that serious side effects might not be common.
In one study, only a small percentage of patients experienced severe issues over a long treatment period, suggesting the drug might be safe for long-term use. However, since this trial is in its early stages, discussing any concerns with the study team is advisable. They can provide detailed information about what to expect.12345Why do researchers think this study treatment might be promising?
Momelotinib is unique because it offers a fresh approach to treating myelofibrosis, a type of bone marrow cancer, by targeting specific pathways involved in disease progression. Unlike other treatments that primarily focus on symptom management and blood transfusions, Momelotinib works by inhibiting the JAK1, JAK2, and ACVR1 pathways, potentially reducing the need for transfusions and improving overall blood counts. Researchers are excited about Momelotinib because it could streamline the treatment process by being administered orally, enhancing patient convenience and adherence, especially when combined with standard hematopoietic cell transplantation.
What evidence suggests that momelotinib might be an effective treatment for myelofibrosis?
Research has shown that momelotinib may help treat myelofibrosis, a type of bone marrow disorder. In studies, patients taking momelotinib reported a better quality of life, particularly those with anemia, a condition characterized by a lack of healthy red blood cells. One study found that 71.5% of patients who took momelotinib as part of a combination treatment responded positively after three months. Another study showed that momelotinib helped 30% of patients stop needing blood transfusions after 24 weeks. In this trial, participants will receive momelotinib alongside standard of care hematopoietic cell transplantation (HCT). These findings suggest that momelotinib could be a useful option for managing symptoms of myelofibrosis.36789
Who Is on the Research Team?
Gabriela Hobbs, MD
Principal Investigator
Massachusetts General Hospital
Are You a Good Fit for This Trial?
This trial is for adults over 18 with myelofibrosis who haven't been treated with JAK inhibitors, are eligible for a specific type of bone marrow transplant (HCT), and meet certain disease severity criteria. They must not be pregnant, agree to use contraception, and have no recent major surgeries or other serious health conditions.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Conditioning and Pre-Transplant
Participants receive reduced intensity conditioning regimen before HCT, including Fludarabine and Mephalan, and start momelotinib 7 days before HCT
Hematopoietic Cell Transplantation (HCT)
Participants undergo standard of care allogeneic HCT and continue momelotinib treatment
Post-Transplant Treatment
Participants continue momelotinib treatment for up to 1 year post-HCT, with dose adjustments as needed
Follow-up
Participants are monitored for safety, effectiveness, and incidence of GVHD and other outcomes
What Are the Treatments Tested in This Trial?
Interventions
- Momelotinib
Trial Overview
The study tests the safety of momelotinib in patients with myelofibrosis during and after undergoing hematopoietic cell transplantation (HCT). It's an early-phase trial at one center where everyone knows what treatment they're getting.
How Is the Trial Designed?
1
Treatment groups
Experimental Treatment
Momelotinib will be administered orally once daily at a pre-determined dose starting 7 days before standard of care (SOC) hematopoietic cell transplantation (HCT) and for up to 1 year after HCT, for a total of 13 28-day cycles. Participants will receive SOC HCT and HCT treatment including: reduced intensity conditioning (RIC) regimen before HCT (Fludarabine, Mephalan), and tacrolimus and methotrexate after HCT, all administered according to SOC.
Find a Clinic Near You
Who Is Running the Clinical Trial?
Massachusetts General Hospital
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
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 ...
2.
ashpublications.org
ashpublications.org/blood/article/144/Supplement%201/6652/528227/Efficacy-and-Safety-of-Momelotinib-inEfficacy and Safety of Momelotinib in Myelofibrosis: A ...
Conclusions: MF patients treated with momelotinib demonstrated good response and improved quality of life, particularly in anemia-related ...
Momelotinib for the treatment of MF: Long-term data
At Week 24, transfusion independence response rates were 30% with momelotinib and 20% with danazol.6 At Week 48, 57% of patients who continued ...
Real world outcomes of momelotinib in myelofibrosis ...
MoReLife – real-life data support the potential of momelotinib as a safe and effective treat- ment option for cytopenic myelofibrosis patients.
Efficacy and Safety of Momelotinib in Myelofibrosis
Primary outcomes included spleen volume reduction (≥ 35%) and anemia response (transfusion independence). Secondary endpoints included symptom ...
Real world outcomes of momelotinib in myelofibrosis ...
Our results support that momelotinib is an effective and well-tolerated treatment for MF patients in routine clinical practice, demonstrating ...
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; ...
8.
mpn-hub.com
mpn-hub.com/medical-information/long-term-survival-and-safety-outcomes-with-momelotinibLong-term survival and safety outcomes with momelotinib
Long-term survival data · Death: 45.2% vs 44.2%. · Leukemic transformation: 6.7% vs 1.9%. · Median OS: 2.9 years vs 3.1 years (hazard ratio, [HR], ...
Efficacy and Safety of Momelotinib in Myelofibrosis: A ...
Conclusions: MF patients treated with momelotinib demonstrated good response and improved quality of life, particularly in anemia-related ...
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