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Janus Kinase (JAK) Inhibitor

Ruxolitinib for Thrombocythemia and Polycythemia

Phase 2

Recruiting

Led By Gabriela Hobbs, MD

Research Sponsored by Massachusetts General Hospital

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 all patients who initiate treatment with study drug up to 60 months

Awards & highlights

All Individual Drugs Already Approved

Approved for 10 Other Conditions

No Placebo-Only Group

Summary

This trial will assess the efficacy of ruxolitinib in reducing symptoms in patients with low-risk essential thrombocythemia (ET) or polycythemia vera (PV).

Who is the study for?

Adults diagnosed with low-risk essential thrombocythemia or polycythemia vera, without a history of high-risk factors like thrombosis or certain mutations. Participants must have specific blood count levels, organ function within normal ranges, and no severe cardiac issues. They should not be on conflicting medications, pregnant, breastfeeding, or at risk of pregnancy without contraception.

What is being tested?

The trial is testing Ruxolitinib's effectiveness in reducing symptoms of essential thrombocythemia and polycythemia vera. It involves patients who meet specific criteria for these conditions and requires them to have a particular symptom score before starting the treatment.

What are the potential side effects?

Ruxolitinib may cause side effects such as changes in blood counts leading to an increased risk of infections, liver problems indicated by abnormal lab values, potential harm to unborn babies (hence the need for contraception), and possibly other reactions similar to drugs with comparable chemical structures.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ all patients who initiate treatment with study drug up to 60 months

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~all patients who initiate treatment with study drug up to 60 months

This trial's timeline: 3 weeks for screening, Varies for treatment, and all patients who initiate treatment with study drug up to 60 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

Percentage of patients who achieve >50% reduction from baseline to Myeloproliferative Neoplasm Symptom Assessment Total Symptom Score

Secondary study objectives

Best MPN-SAF TSS score

Number of Participants with Treatment Related Adverse Events as Assessed by CTCAE v 5.0

Percentage of change in Spleen Volume

+2 more

Side effects data

From 2020 Phase 3 trial • 149 Patients • NCT02038036

33%

Anaemia

19%

Hypertension

17%

Nasopharyngitis

16%

Weight increased

14%

Herpes zoster

14%

Constipation

14%

Abdominal pain

14%

Headache

12%

Pruritus

12%

Back pain

12%

Epistaxis

12%

Pyrexia

12%

Dizziness

10%

Asthenia

10%

Fatigue

10%

Cough

10%

Oedema peripheral

10%

Arthralgia

Thrombocytosis

Upper respiratory tract infection

Hypercholesterolaemia

Haematoma

Dyslipidaemia

Pain in extremity

Abdominal discomfort

Diarrhoea

Dyspepsia

Vomiting

Blood lactate dehydrogenase increased

Memory impairment

Dyspnoea

Tinnitus

Osteoarthritis

Leukocytosis

Thrombocytopenia

Flatulence

Nausea

Sinusitis

Basal cell carcinoma

Neuropathy peripheral

Hyperuricaemia

Paraesthesia

Bronchitis

Cystitis

Blood creatine phosphokinase increased

Skin ulcer

Abdominal pain upper

Pulmonary embolism

Pneumonia

Influenza

Myalgia

Urinary tract infection

Depression

Peripheral artery thrombosis

Vertigo

Acute pulmonary oedema

Night sweats

Intervertebral disc protrusion

Urethral stenosis

Ureterolithiasis

Localised infection

Pericardial effusion

Acute myocardial infarction

Syncope

Gastrooesophageal reflux disease

General physical health deterioration

Atrial fibrillation

Cardiac disorder

Mitral valve incompetence

Vertigo positional

Retinal artery occlusion

Visual acuity reduced

Gastrointestinal haemorrhage

Oesophageal varices haemorrhage

Lower respiratory tract infection

Pyelonephritis

Respiratory tract infection

Sepsis

Tendon rupture

Ulna fracture

Weight decreased

Decreased appetite

Hyponatraemia

Blast cell crisis

Bone marrow tumour cell infiltration

Lung adenocarcinoma

Metastases to spine

Myelofibrosis

Prostatic adenoma

Squamous cell carcinoma of skin

Nephrolithiasis

Gamma-glutamyltransferase increased

Haematocrit increased

Musculoskeletal pain

Ischaemic stroke

Diabetes mellitus

100%

80%

60%

40%

20%

Study treatment Arm

All Crossover Patients

Best Available Therapy

Ruxolitinib

Awards & Highlights

All Individual Drugs Already Approved

Therapies where all constituent drugs have already been approved are likely to have better-understood side effect profiles.

Approved for 10 Other Conditions

This treatment demonstrated efficacy for 10 other conditions.

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: Ruxolitinib Stage 2Experimental Treatment1 Intervention

Stage 2 will commence based on 3 or more participants in Stage 1 showing a predetermined positive response to Ruxolitinib. In stage 2, participants will be divided into two cohorts: * Very low, Low, and Intermediate-risk ET patients with significant symptom burden and Low-risk PV patients with significant symptom burden * Study cycles are 28 days long, participants in both cohorts will receive: * Ruxolitinib 2x daily for 6 study cycles.

Group II: Ruxolitinib Stage 1Experimental Treatment1 Intervention

In stage 1, participants will be divided into two cohorts: * Very low, Low, and Intermediate-risk ETpatients with significant symptom burden and Low-risk PV patients with significant symptom burden * Study cycles are 28 days long, participants in both cohorts will receive: * Ruxolitinib 2x daily for 6 study cycles.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Ruxolitinib

FDA approved

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