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Momelotinib Dihydrochloride Monohydrate

This article discusses clinical trials investigating the use of momelotinib dihydrochloride monohydrate, a drug being studied for the treatment of myelofibrosis. Myelofibrosis is a rare bone marrow cancer that disrupts the body’s normal production of blood cells. The trials aim to assess the safety, effectiveness, and long-term outcomes of momelotinib in patients with different types of myelofibrosis.

Table of Contents

What is Momelotinib?

Momelotinib is an investigational drug being developed for the treatment of myelofibrosis, a rare blood cancer[1]. Its full chemical name is momelotinib dihydrochloride monohydrate. Momelotinib is taken orally in the form of tablets[2].

Medical Conditions Treated

Momelotinib is being studied for the treatment of:

  • Primary myelofibrosis (PMF)
  • Post-polycythemia vera myelofibrosis (Post-PV MF)
  • Post-essential thrombocythemia myelofibrosis (Post-ET MF)

These conditions are all forms of myelofibrosis, a serious bone marrow disorder that disrupts the body’s normal production of blood cells[1].

How Momelotinib Works

Momelotinib is a type of drug called a JAK inhibitor. It works by blocking certain proteins (called Janus kinases or JAKs) that are involved in signaling within cells. In myelofibrosis, these JAK proteins become overactive, leading to the symptoms of the disease. By inhibiting these proteins, momelotinib aims to reduce symptoms and potentially slow the progression of myelofibrosis[1].

Clinical Trials

Momelotinib is currently being studied in several clinical trials:

  1. A Phase 1/2 study combining momelotinib with another drug called TP-3654 in patients with intermediate or high-risk myelofibrosis[1].
  2. An extended access study for patients who have been receiving momelotinib in previous clinical trials and have not experienced disease progression[2].

These trials aim to further evaluate the safety and effectiveness of momelotinib in treating myelofibrosis.

Potential Benefits

Based on the clinical trials, momelotinib may offer several potential benefits for patients with myelofibrosis:

  • Reduction in spleen size: One of the main goals of treatment is to achieve a 35% or greater reduction in spleen volume (SVR35)[1].
  • Improvement in symptoms: Researchers are measuring changes in total symptom score (TSS) using a specific assessment tool (MFSAF v4.0)[1].
  • Potential to help with anemia: Some patients receiving momelotinib in combination with other drugs are required to have low hemoglobin levels (anemia) to participate in the study, suggesting that the treatment may help address this common problem in myelofibrosis[1].

Safety and Side Effects

As with any medication, momelotinib may cause side effects. The clinical trials are closely monitoring patients for:

  • Adverse events (AEs): These are any undesirable experiences associated with the use of the drug[2].
  • Changes in heart function: Researchers are monitoring for any effects on heart rhythm (QT interval changes)[1].
  • Dose-limiting toxicities (DLTs): These are side effects severe enough to prevent increasing the dose of the medication[1].

It’s important to note that the full safety profile of momelotinib is still being established through these clinical trials.

Ongoing Research

Momelotinib is still considered an investigational drug, which means it has not yet been approved by regulatory agencies for general use. The ongoing clinical trials are designed to:

  • Determine the best dose of momelotinib (alone and in combination with other drugs)
  • Assess its long-term safety and effectiveness
  • Evaluate its impact on patient survival and quality of life

Researchers are also studying how momelotinib interacts with other medications and how it’s processed by the body (pharmacokinetics)[1][2].

If you have myelofibrosis and are interested in learning more about momelotinib or participating in clinical trials, it’s important to discuss this with your healthcare provider. They can provide more information about whether this investigational treatment might be appropriate for your specific situation.

Aspect Details
Drug Name Momelotinib dihydrochloride monohydrate
Condition Studied Primary Myelofibrosis (PMF), Post-polycythemia Vera Myelofibrosis (post-PV MF), Post-essential Thrombocythemia Myelofibrosis (post-ET MF)
Trial Phases Phase 1/2 and Extended Access
Main Objectives Assess safety, determine optimal dosing, evaluate effectiveness, study long-term outcomes
Key Endpoints Spleen volume reduction, symptom improvement, adverse events, overall survival, leukemia-free survival
Patient Population Adults with intermediate or high-risk myelofibrosis, including those previously treated with other drugs
Administration Oral tablets

FAQ

  • What is momelotinib and how does it work? Momelotinib is an investigational drug for treating myelofibrosis. It works by inhibiting certain proteins involved in the disease process, potentially helping to reduce symptoms and improve blood cell production.
  • What types of myelofibrosis are being studied in these trials? The trials are studying primary myelofibrosis (PMF) as well as post-polycythemia vera myelofibrosis and post-essential thrombocythemia myelofibrosis (post-PV/ET MF).
  • What are the main goals of these clinical trials? The main goals are to evaluate the safety and effectiveness of momelotinib, determine the appropriate dosing, assess its effects on spleen size and symptoms, and study long-term outcomes like overall survival.
  • Who is eligible to participate in these trials? Eligibility varies, but generally includes adult patients with intermediate or high-risk myelofibrosis. Some trials focus on patients who have previously been treated with other drugs, while others are studying newly diagnosed patients.
  • What are some of the key measurements being taken in these studies? Key measurements include changes in spleen volume, improvement in disease symptoms, safety assessments, drug levels in the body, and long-term outcomes like overall survival and leukemia-free survival.

Ongoing Clinical Trials on Momelotinib Dihydrochloride Monohydrate

  • Study of momelotinib tablets in patients with anemia caused by low-risk myelodysplastic syndrome

    Recruiting

    2 1 1
    Investigated diseases:
    Investigated drugs:
    France Germany Italy Poland Spain

  • Study of momelotinib in patients with VEXAS syndrome and myelodysplastic syndrome who are dependent on or do not respond to steroid treatment

    Recruiting

    2 1 1
    Investigated diseases:
    Investigated drugs:
    France

  • Study on the Safety of Roginolisib and Ruxolitinib for Patients with Myelofibrosis Unresponsive to JAK Inhibitors

    Recruiting

    1 1 1 1
    Investigated diseases:
    Investigated drugs:
    Italy Spain

  • Study of TP-3654 and Momelotinib for Patients with Intermediate or High-risk Myelofibrosis

    Recruiting

    1 1 1
    Investigated drugs:
    Austria Belgium Bulgaria Czechia Denmark France +7

  • Study of Momelotinib and Luspatercept for Patients with Transfusion-Dependent Myelofibrosis

    Not yet recruiting

    2 1 1 1
    Investigated diseases:
    Investigated drugs:
    France Germany Italy Spain

  • Study on Extended Access to Momelotinib for Patients with Myelofibrosis

    Not recruiting

    2 1 1
    Investigated diseases:
    Investigated drugs:
    Belgium Bulgaria Denmark France Germany Hungary +4

Glossary

  • Myelofibrosis: A rare type of blood cancer that affects the bone marrow, leading to scarring, abnormal blood cell production, and various symptoms.
  • JAK inhibitor: A type of drug that blocks the action of Janus kinase enzymes, which are involved in cell signaling and can be overactive in myelofibrosis.
  • Splenomegaly: Enlargement of the spleen, which is a common symptom in myelofibrosis.
  • DIPSS: Dynamic International Prognostic Scoring System, a tool used to assess the risk level and prognosis of myelofibrosis patients.
  • Pharmacokinetics (PK): The study of how a drug moves through the body, including its absorption, distribution, metabolism, and excretion.
  • Dose-limiting toxicity (DLT): Side effects of a drug that are severe enough to prevent increasing the dose or require a dose reduction.
  • Recommended Phase 2 dose (RP2D): The dose of a drug determined to be appropriate for further testing in Phase 2 clinical trials based on safety and effectiveness data from Phase 1.
  • MFSAF: Myelofibrosis Symptom Assessment Form, a tool used to measure the severity of symptoms experienced by myelofibrosis patients.
  • Leukemia-free survival: The length of time after treatment during which no signs of leukemia (a type of blood cancer) are detected in a person who has been treated for myelofibrosis.
  • Anemia: A condition in which there are not enough healthy red blood cells to carry adequate oxygen to the body's tissues.

References

  1. http://clinicaltrials.eu/trial/study-of-tp-3654-and-momelotinib-for-patients-with-intermediate-or-high-risk-myelofibrosis/
  2. http://clinicaltrials.eu/trial/study-on-extended-access-to-momelotinib-for-patients-with-myelofibrosis/