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Janus Kinase (JAK) Inhibitor

Ruxolitinib for Myelofibrosis

Phase 2

Waitlist Available

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

Off all MF-directed therapy at the time of enrollment, with the exception of ruxolitinib

ECOG performance status ≤2 (Karnofsky ≥60%, see Appendix A)

Must not have

Patients without normal organ function defined as follows: AST (SGOT), ALT (SGPT) and Alkaline Phosphatase ≥ 3 × institutional Upper Limit of Normal (ULN), Direct bilirubin >2.0 mg/dL, Adequate renal function as defined by calculated creatinine clearance≤60 mL/min (Cockcroft-Gault formula), Have a chronic or active infection that requires systemic antibiotics, antifungal or antiviral treatment, Have current or a history of congestive heart failure New York Heart Association (NYHA) class 3 or 4, or any history of documented diastolic or systolic dysfunction (LVEF < 40%, as measured by MUGA scan or echocardiogram), Pregnancy at the time of enrollment, Unable to give informed consent, Have an uncontrolled intercurrent illness including, but not limited to, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements, Subjects who require therapy with a strong CYP3A4 inhibitor prior to enrollment to this study, Not able to take oral medication

Had accelerated phase or leukemic transformation (≥10% blasts in PB or BM any time prior to HCT)

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 1 and 2 years

Awards & highlights

All Individual Drugs Already Approved

Approved for 10 Other Conditions

No Placebo-Only Group

Summary

This trial is testing Ruxolitinib as a possible treatment for Myelofibrosis, a disorder where the bone marrow doesn't make enough blood cells.

Who is the study for?

This trial is for adults aged 18-75 with primary or secondary myelofibrosis, who are either already taking Ruxolitinib or will start it. They must have symptoms like an enlarged spleen and meet certain blood count criteria. Participants should be fit enough for a stem cell transplant from a matched donor and not have severe organ damage, uncontrolled infections, heart failure, other recent cancers (except some skin cancers), or be pregnant.

What is being tested?

The study tests the drug Ruxolitinib in patients with myelofibrosis before, during, and after they receive a hematopoietic stem cell transplant (HSCT). The goal is to see how well Ruxolitinib works as part of the treatment process for this condition.

What are the potential side effects?

Ruxolitinib may cause side effects such as low blood counts leading to increased infection risk, bleeding problems, dizziness or headaches. It can also affect liver enzymes which might indicate liver issues. Some people might experience weight gain or swelling around their body.

Eligibility Criteria

Inclusion Criteria

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

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I am not on any treatment for myelofibrosis except ruxolitinib.

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

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I am scheduled for a stem cell transplant using donor cells.

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My condition is officially diagnosed as primary or secondary myelofibrosis.

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My donor for the transplant matches me closely in important genetic markers.

Exclusion Criteria

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

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My blood or bone marrow had a rapid increase in immature cells before my cell transplant.

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I have an infection that is not under control.

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I have had a bone marrow transplant from another person.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 1 and 2 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~1 and 2 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and 1 and 2 years for reporting.

Treatment Details

Study Objectives

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

Primary study objectives

GVHD free and relapse free survival at 1 year

Secondary study objectives

Cumulative incidence of aGVHD

Cumulative incidence of cGVHD

Hemopoietic stem cell transplant

+4 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

Acute pulmonary oedema

Peripheral artery thrombosis

Vertigo

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 Not Eligible pre-HSCTExperimental Treatment1 Intervention

* Ruxolitinib will be taken orally at a fixed dose twice every day after transplant * Dosing will be continuous, with a new cycle scheduled to start every 28 days. * There will be no break in dosing between cycles * Ruxolitinib can be administered with or without food.

Group II: Ruxolitinib Eligible pre-HSCTExperimental Treatment1 Intervention

* Ruxolitinib will be taken orally at a fixed dose twice every day * Dosing will be continuous, with a new cycle scheduled to start every 28 days. * There will be no break in dosing between cycles * Ruxolitinib can be administered with or without food.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Ruxolitinib

FDA approved

0 / 9Eligibility criteria met

Find a Location

Who is running the clinical trial?

Incyte CorporationIndustry Sponsor

391 Previous Clinical Trials

63,767 Total Patients Enrolled

Massachusetts General HospitalLead Sponsor

3,006 Previous Clinical Trials

13,307,238 Total Patients Enrolled

Gabriela Hobbs, MD4.04 ReviewsPrincipal Investigator - Massachusetts General Hospital

Massachusetts General Hospital

4 Previous Clinical Trials

116 Total Patients Enrolled

5Patient Review

I was always made to feel cared for, never rushed, and given as much time as I needed during appointments. Dr. Smith is also very knowledgeable and quick to return calls. I am very impressed with her as both a physician and a human being.