What is the mechanism of action of this treatment?

JAK inhibitors, such as Momelotinib, Ruxolitinib, and Fedratinib, target the Janus kinase (JAK) signaling pathway, which is often dysregulated in Primary Myelofibrosis (PMF). By inhibiting JAK1 and JAK2, these drugs reduce the abnormal signaling that leads to excessive production of blood cells and fibrosis in the bone marrow. This mechanism helps to alleviate symptoms like splenomegaly (enlarged spleen) and constitutional symptoms (e.g., fatigue, night sweats), and can improve overall quality of life. For PMF patients, effective management of these symptoms is crucial as it directly impacts their daily functioning and long-term prognosis.

What side effects are associated with this type of intervention?

The most common treatments for Primary Myelofibrosis (PMF) include JAK inhibitors such as Ruxolitinib and Fedratinib. Known side effects of Ruxolitinib include anemia, thrombocytopenia, and increased risk of infections. Fedratinib can cause gastrointestinal symptoms, anemia, and thrombocytopenia, with a risk of Wernicke's encephalopathy. Momelotinib, another JAK inhibitor under study, has shown similar side effects, including anemia and thrombocytopenia, but aims to provide extended access and assess long-term safety in patients with PMF.

What prior FDA approvals exist?

The FDA has approved several JAK inhibitors for the treatment of Primary Myelofibrosis (PMF). Ruxolitinib (Jakafi) was the first JAK1/JAK2 inhibitor approved for PMF, providing significant relief from splenomegaly and symptoms. Fedratinib (Inrebic), another JAK2 inhibitor, was also approved for PMF, offering an alternative for patients who are intolerant or resistant to Ruxolitinib. These treatments, like Momelotinib, target the JAK-STAT pathway, which is crucial in the pathophysiology of myelofibrosis.

What other types of research exist?

Promising novel alternatives to Momelotinib for Primary Myelofibrosis patients include Ruxolitinib, which has shown efficacy in reducing spleen size and alleviating symptoms in clinical trials. Additionally, other JAK inhibitors like Fedratinib and Pacritinib are being explored for their potential benefits. Emerging therapies such as Luspatercept and novel agents targeting the bone marrow microenvironment are also under investigation and may offer new treatment options in 2024.

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JAK Inhibitor

MMB for Myelofibrosis

Phase 2

Waitlist Available

Research Sponsored by Sierra Oncology LLC - a GSK company

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Be older than 18 years old

Timeline

Screening 3 weeks

Treatment Varies

Follow Up participants will be assessed every 12 weeks until discontinuation. participation in this extended access study has been an average of approximately 8 months.

Awards & highlights

Summary

This trial aims to provide ongoing access to momelotinib for patients with certain types of myelofibrosis who are already taking the drug and have not seen their disease get worse. The study will also look at how long these patients live without developing leukemia. Momelotinib has been studied for myelofibrosis and other related conditions.

Who is the study for?

This trial is for adults with myelofibrosis who are already participating in specific earlier studies of momelotinib (MMB). They must understand and agree to the study's terms. People allergic to MMB or its ingredients cannot join.

What is being tested?

The trial provides continued access to MMB for patients from previous trials, assessing long-term safety. It also looks at how well participants live without their disease getting worse or developing into leukemia.

What are the potential side effects?

While not explicitly listed here, side effects may include those commonly associated with cancer treatments such as fatigue, nausea, and potential blood-related issues.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ participants will be assessed every 12 weeks until discontinuation. participation in this extended access study has been an average of approximately 8 months.

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~participants will be assessed every 12 weeks until discontinuation. participation in this extended access study has been an average of approximately 8 months.

This trial's timeline: 3 weeks for screening, Varies for treatment, and participants will be assessed every 12 weeks until discontinuation. participation in this extended access study has been an average of approximately 8 months. for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Number of Participants Who Had Access to, and Received the Intervention

Side effects data

From 2017 Phase 2 trial • 41 Patients • NCT02515630

29%

Cough

24%

Diarrhoea

22%

Nausea

20%

Fatigue

17%

Pruritus

17%

Vomiting

17%

Thrombocytopenia

15%

Abdominal pain

15%

Urinary tract infection

15%

Dizziness

15%

Headache

12%

Neutropenia

12%

Dyspnoea

12%

Contusion

10%

Decreased appetite

10%

Hyperuricaemia

10%

Fall

Night sweats

Blood creatinine increased

Chills

Constipation

Anemia

Acute kidney injury

Nasal congestion

Peripheral sensory neuropathy

Musculoskeletal pain

Pollakiuria

Hypotension

Presyncope

Lung infection

Cholecystitis

Metabolic encephalopathy

Asthenia

Death

Pyrexia

Pneumonia

Sepsis

Atrial fibrillation

Cardiac arrest

Colitis

Small intestinal obstruction

Acute respiratory failure

Respiratory failure

Transfusion reaction

Hepatic enzyme increased

Bone pain

100%

80%

60%

40%

20%

Study treatment Arm

Momelotinib (MMB)

Trial Design

4Treatment groups

Experimental Treatment

Group I: Cohort 4: Study SRA-MMB-301Experimental Treatment1 Intervention

Participants will continue to receive the same dosage regimen as in the previous MMB study SRA-MMB-301 until MMB receives regulatory approval and is commercially available, or development of the product ceases.

Group II: Cohort 3: Study GS-US-352-1154Experimental Treatment1 Intervention

Participants will continue to receive the same dosage regimen as in the previous MMB study GS-US-352-1154 until MMB receives regulatory approval and is commercially available, or development of the product ceases..

Group III: Cohort 2: Study GS-US-352-1214Experimental Treatment1 Intervention

Participants will continue to receive the same dosage regimen as in the previous MMB study GS-US-352-1214 until MMB receives regulatory approval and is commercially available, or development of the product ceases.

Group IV: Cohort 1: Study GS-US-352-0101Experimental Treatment1 Intervention

Participants will continue to receive the same dosage regimen as in the previous MMB study GS-US-352-0101 until MMB receives regulatory approval and is commercially available, or development of the product ceases.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

MMB

2016

Completed Phase 2

~50

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Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

JAK inhibitors, such as Momelotinib, Ruxolitinib, and Fedratinib, target the Janus kinase (JAK) signaling pathway, which is often dysregulated in Primary Myelofibrosis (PMF). By inhibiting JAK1 and JAK2, these drugs reduce the abnormal signaling that leads to excessive production of blood cells and fibrosis in the bone marrow. This mechanism helps to alleviate symptoms like splenomegaly (enlarged spleen) and constitutional symptoms (e.g., fatigue, night sweats), and can improve overall quality of life. For PMF patients, effective management of these symptoms is crucial as it directly impacts their daily functioning and long-term prognosis.