What side effects are associated with this type of intervention?

Common treatments for Primary Myelofibrosis, particularly JAK2 inhibitors like Pacritinib, ruxolitinib, and fedratinib, have several known side effects. These include anemia, thrombocytopenia (low platelet count), and gastrointestinal issues such as diarrhea. Hydroxyurea, another treatment option, can cause cytopenias, mucocutaneous ulcers, diarrhea, peripheral neuropathy, and in rare cases, severe pulmonary toxicity. Patients should discuss these potential side effects with their healthcare provider to manage and mitigate risks effectively.

What prior FDA approvals exist?

The FDA has approved several JAK2 inhibitors for the treatment of Primary Myelofibrosis. Ruxolitinib was the first JAK2 inhibitor approved and is widely used for its efficacy in reducing spleen size and alleviating symptoms. Fedratinib is another JAK2 inhibitor that has been approved for patients with intermediate-2 or high-risk myelofibrosis, particularly those who are resistant or intolerant to Ruxolitinib. These treatments aim to manage symptoms and improve quality of life for patients with Primary Myelofibrosis.

What other types of research exist?

Promising alternatives to Pacritinib for Primary Myelofibrosis patients include Fedratinib and Imetelstat. Fedratinib has shown efficacy in patients previously treated with Ruxolitinib, and Imetelstat, a telomerase inhibitor, has demonstrated potential in clinical trials. Both options are worth discussing with a healthcare provider for suitability based on individual patient profiles.

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Tyrosine Kinase Inhibitor

NS-018 for Myelofibrosis

Phase 2

Waitlist Available

Research Sponsored by NS Pharma, Inc.

Eligibility Criteria Checklist

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

Must have

ECOG performance status ≤2

Total Symptom Score (TSS) ≥10 on the Myelofibrosis Symptom Assessment Form (MFSAF) version 4.0

Must not have

Active, uncontrolled systemic infection

History of splenectomy or planning to undergo splenectomy

Timeline

Screening 3 weeks

Treatment Varies

Follow Up from baseline to week 24

Awards & highlights

Summary

This trial tests NS-018, an oral medication taken regularly, in adults with severe low platelet counts caused by certain blood cancers. The drug aims to block signals that help cancer cells grow.

Who is the study for?

This trial is for adults with Myelofibrosis, including those who developed it after Polycythemia Vera or Essential Thrombocythemia. Participants must have severe thrombocytopenia, a life expectancy over 6 months, and significant symptoms. They shouldn't have had recent surgery, other cancers within 2 years, uncontrolled infections or heart issues, or multiple JAK inhibitor treatments.

What is being tested?

The study tests NS-018's effectiveness and safety against the Best Available Therapy (BAT) in treating Myelofibrosis with severe thrombocytopenia. It focuses on patients at intermediate-2 or high risk of disease progression according to DIPSS.

What are the potential side effects?

Potential side effects of NS-018 are not specified here but may include typical reactions to cancer therapies such as fatigue, digestive issues, blood count changes and increased infection risk. BAT side effects vary depending on the chosen therapy.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I can take care of myself but might not be able to do heavy physical work.

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My symptom score is 10 or higher on the MFSAF.

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My spleen is larger than normal, as confirmed by an MRI or CT scan.

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My condition is classified as intermediate-2 or high-risk myelofibrosis.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I currently have an untreated, serious infection.

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I have had my spleen removed or will have it removed.

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I have been treated with more than one JAK inhibitor.

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I am on medication that affects liver enzymes CYP1A2 or CYP3A4.

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I have no ongoing side effects from cancer treatment, except for blood-related ones.

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I haven't had surgery (except for minor procedures) in the last 4 weeks.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ from baseline to week 24

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~from baseline to week 24

This trial's timeline: 3 weeks for screening, Varies for treatment, and from baseline to week 24 for reporting.

Treatment Details

Study Objectives

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

Primary study objectives

Change in Total Symptom Score (TSS)

Secondary study objectives

Change in spleen volume

Comparison of treatment-emergent AEs

Side effects data

From 2020 Phase 1 & 2 trial • 77 Patients • NCT01423851

40%

Fatigue

40%

Diarrhoea

33%

Dyspnoea

33%

Upper respiratory tract infection

33%

Cough

33%

Oedema peripheral

33%

Back pain

33%

Dizziness

27%

Headache

20%

Nausea

20%

Abdominal discomfort

20%

Arthralgia

20%

Weight decreased

20%

Pneumonia

20%

Abdominal pain

20%

Blood alkaline phosphatase increased

20%

Epistaxis

20%

Musculoskeletal pain

20%

Pruritus

20%

Anaemia

20%

Hyperuricaemia

20%

Fall

13%

Pyrexia

13%

Oedema

13%

Muscle spasms

13%

Thrombocytopenia

13%

Decreased appetite

13%

Hyperhidrosis

13%

Hypertension

13%

Pain in extremity

13%

Platelet count decreased

13%

Vomiting

13%

Gastrooesophageal reflux disease

13%

Dyspnoea exertional

13%

Early satiety

13%

Memory impairment

13%

Dizziness postural

13%

Somnolence

13%

Hyperglycaemia

13%

Dehydration

13%

Oral candidiasis

13%

Petechiae

13%

Contusion

13%

Nocturia

Cellulitis

Angina pectoris

Myocardial infarction

Spinal stenosis

Glioblastoma multiforme

Hepatosplenomegaly

Abdominal distension

Constipation

Dyspepsia

Lipase increased

Blood cholesterol increased

Heart rate abnormal

Pleural effusion

Wheezing

Pulmonary oedema

Rales

Asthenia

Chest pain

Myalgia

Bursitis

Joint effusion

Musculoskeletal stiffness

Soft tissue mass

Paraesthesia

Balance disorder

Neuralgia

Hyperkalaemia

Infected seroma

Lip infection

Localised infection

Depression

Depressed mood

Emotional distress

Urinary tract pain

Dry eye

Meibomian gland dysfunction

Photophobia

Strabismus

Erectile dysfunction

Basal cell carcinoma

Muscle neoplasm

Skin cancer

Hypoacusis

Hypocalcaemia

Animal scratch

Lumbar spinal stenosis

Insomnia

Influenza

Chills

Hyponatraemia

Transient ischaemic attack

Tremor

Sinusitis

Night sweats

Animal bite

Agitation

Claustrophobia

Palpitations

Glaucoma

Eye swelling

Ear pain

Confusional state

Suicide attempt

Sepsis

Splenomegaly

Enterocolitis

Acute coronary syndrome

Coronary artery occlusion

Anal sphincter atony

Dysphagia

Flatulence

Weight increased

White blood cell count decreased

Blood potassium increased

Serum ferritin increased

Troponin I increased

Wound infection

Nasal congestion

Bronchospasm

Dysphonia

Ecchymosis

Pleuritic pain

Sleep apnoea syndrome

Chest discomfort

Non-cardiac chest pain

Fibromyalgia

Osteoarthritis

Synovial cyst

Neuropathy peripheral

Ataxia

Lethargy

Sensory disturbance

Increased tendency to bruise

Hyperphosphataemia

Nasopharyngitis

Bronchitis

Skin infection

Rash maculo-papular

Rash

Erythema

Neurodermatitis

Muscle strain

Skin wound

Dysuria

Haematuria

Sinus bradycardia

Hot flush

Blepharitis

Eyelid irritation

Lacrimation increased

100%

80%

60%

40%

20%

Study treatment Arm

Part 1: 300 mg QD

Part 1: 250 mg BID

Part 1: 400 mg BID

Part 2: 300 mg QD

Part 1: 75 mg QD

Part 1: 125 mg QD

Part 1: 200 mg QD

Part 1: 400 mg QD

Part 1: 100 mg BID

Part 1: 200 mg BID

Part 1: 300 mg BID

Trial Design

2Treatment groups

Experimental Treatment

Active Control

Group I: NS-018Experimental Treatment1 Intervention

Self-administered NS-018 300 mg orally, twice daily, preferably at the same time each day in consecutive 4-week (28-day) cycles

Group II: Best Available Therapy (BAT)Active Control1 Intervention

Single agent per Investigator discretion or no therapy

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

NS-018

2011

Completed Phase 2

~80

0 / 10Eligibility criteria met

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

The most common treatments for Primary Myelofibrosis (PMF) include JAK2 inhibitors such as ruxolitinib and fedratinib. These medications work by inhibiting the Janus kinase 2 (JAK2) enzyme, which plays a crucial role in the signaling pathways that regulate blood cell production. In PMF, mutations in the JAK2 gene lead to uncontrolled cell proliferation and fibrosis in the bone marrow. By inhibiting JAK2, these drugs help reduce abnormal blood cell production, decrease spleen size, and alleviate symptoms such as fatigue and pain. This is particularly important for PMF patients as it can significantly improve their quality of life and potentially slow disease progression. Other treatments, like hydroxyurea, work by reducing the overall blood cell count, but JAK2 inhibitors are more targeted in addressing the underlying pathophysiology of PMF.