What is the mechanism of action of this treatment?

Common treatments for myeloid diseases, such as hypomethylating agents (HMAs) and tyrosine kinase inhibitors (TKIs), work by targeting the genetic and epigenetic abnormalities that drive these diseases. HMAs like azacitidine and decitabine inhibit DNA methylation, reactivating tumor suppressor genes and inducing cell differentiation and apoptosis. TKIs like ruxolitinib and dasatinib inhibit specific signaling pathways involved in cell proliferation and survival. These mechanisms are crucial for myeloid disease patients as they aim to control disease progression and improve outcomes by directly addressing the molecular drivers of the disease.

What side effects are associated with this type of intervention?

Common treatments for myeloid diseases include TPO receptor agonists like romiplostim and eltrombopag, lenalidomide, ruxolitinib, and hydroxyurea. TPO receptor agonists can reduce bleeding events and platelet transfusions but may have concerns about leukemic transformation. Lenalidomide is effective in promoting erythropoiesis but can cause adverse reactions such as cytopenias. Ruxolitinib can relieve symptoms of myelofibrosis but may lead to withdrawal syndrome and does not significantly prolong survival. Hydroxyurea can manage splenomegaly and other proliferative symptoms but has side effects including cytopenias, mucocutaneous ulcers, and potential teratogenicity.

What prior FDA approvals exist?

The FDA has approved several drugs for the treatment of myeloid diseases, including myelodysplastic syndromes (MDS) and chronic myelomonocytic leukemia (CMML). Notable approvals include azacitidine and decitabine, which are hypomethylating agents used to treat various forms of MDS. Lenalidomide has been approved for MDS with deletion 5q cytogenetic abnormality. Additionally, ruxolitinib, a JAK1/2 inhibitor, has been approved for myelofibrosis and polycythemia vera. These treatments aim to manage symptoms, improve blood counts, and modify disease progression.

What other types of research exist?

Promising novel alternatives for Myeloid Diseases patients include: 1) Venetoclax combined with low-dose cytarabine (LoDAC), which has shown superior outcomes compared to LoDAC monotherapy. 2) Pevonedistat, which targets malignant cells via NFκB pathway inhibition and has demonstrated efficacy in myeloproliferative neoplasms. 3) Ruxolitinib, particularly for patients with CSF3R mutations, as it has shown dramatic responses in some cases. These alternatives are based on recent clinical findings and ongoing trials.

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DFV890 for Myeloid Diseases

Phase 1

Recruiting

Research Sponsored by Novartis Pharmaceuticals

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 13 months

Awards & highlights

No Placebo-Only Group

Summary

This trial is testing a new drug called DFV890 to see if it can help patients with certain low-risk blood diseases. The goal is to find out if the drug is safe and effective, and to determine the best dose for treatment.

Who is the study for?

Adults over 18 with certain low-risk myeloid diseases (like MDS or CMML) who haven't responded well to other treatments can join. They must be able to have bone marrow tests and have a performance status indicating they can still do daily activities. People who've had recent cancer treatments, are sensitive to DFV890 or similar drugs, or take certain other medications cannot participate.

What is being tested?

The trial is testing DFV890's safety and effectiveness for myeloid diseases in two parts: one part tries different doses to find the best one, and the second part gives that dose to more people. It looks at how the drug works in the body and its impact on disease symptoms.

What are the potential side effects?

Possible side effects of DFV890 aren't listed here but generally could include reactions related to immune system activation, issues from bone marrow biopsies, and interactions with existing conditions or medications.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 13 months

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~13 months

This trial's timeline: 3 weeks for screening, Varies for treatment, and 13 months for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Incidence and severity of Adverse Events (AEs) and Serious Adverse Events (SAEs)

Incidence of Dose-limiting Toxicities (DLTs)

Incidence of dose interruptions, discontinuations and reductions

Secondary study objectives

Area under the plasma concentration-time curve from time zero to the last measurable concentration sampling time (AUClast) of DFV890

Best overall response (BOR) per 2006 IWG criteria for MDS and CMML

Change from baseline in Absolute Neutrophil Count/White Blood Cells (ANC/WBC)

+13 more

Side effects data

From 2020 Phase 2 trial • 143 Patients • NCT04382053

Anaemia

Respiratory failure

Diabetes mellitus

Hyperglycaemia

COVID-19 pneumonia

Sepsis

Shock haemorrhagic

Pulmonary embolism

Polyneuropathy

Renal failure

Dyspnoea

Multiple organ dysfunction syndrome

Condition aggravated

Septic shock

Pneumonia

Amylase increased

Peripheral artery thrombosis

100%

80%

60%

40%

20%

Study treatment Arm

DFV890 + SoC

Total

Standard of Care (SoC)

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

2Treatment groups

Experimental Treatment

Group I: DFV890 low doseExperimental Treatment1 Intervention

DFV890 given as single agent at a low dose

Group II: DFV890 high doseExperimental Treatment1 Intervention

DFV890 given as single agent at a high dose

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

DFV890

2021

Completed Phase 2

~180

Do I qualify?Apply below to find out

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

Common treatments for myeloid diseases, such as hypomethylating agents (HMAs) and tyrosine kinase inhibitors (TKIs), work by targeting the genetic and epigenetic abnormalities that drive these diseases. HMAs like azacitidine and decitabine inhibit DNA methylation, reactivating tumor suppressor genes and inducing cell differentiation and apoptosis. TKIs like ruxolitinib and dasatinib inhibit specific signaling pathways involved in cell proliferation and survival. These mechanisms are crucial for myeloid disease patients as they aim to control disease progression and improve outcomes by directly addressing the molecular drivers of the disease.

Treatment of myelodysplastic syndromes with cytokines and cytotoxic drugs.The levels of granulocyte colony-stimulating factor in the plasma of the bone marrow aspirate in various hematological disorders.Lenalidomide: Myelodysplastic syndromes with del(5q) and beyond.