What side effects are associated with this type of intervention?

Ruxolitinib, a Janus kinase (JAK) inhibitor, is being studied for its efficacy in treating Large Granular Lymphocyte Leukemia (LGL). Common side effects of Ruxolitinib include cytopenias (such as thrombocytopenia and neutropenia), gastrointestinal issues, and an increased risk of infections. Other enzyme inhibitors used in similar contexts may also cause fatigue, diarrhea, and liver function abnormalities.

What prior FDA approvals exist?

There are no specific FDA-approved treatments mentioned for Large Granular Lymphocyte Leukemia (LGLL) in the provided context. However, treatments targeting the JAK/STAT pathway, such as JAK-3 inhibitors, are noted as promising, particularly in relapse settings. Ruxolitinib, which is being studied in a phase II trial for its efficacy in shrinking tumors in LGLL by blocking enzymes needed for cell growth, represents a similar approach.

What other types of research exist?

Promising novel alternatives to Ruxolitinib for LGL leukemia patients in 2024 may include: 1) Venetoclax, a BCL-2 inhibitor, which has shown efficacy in various lymphoid malignancies. 2) BTK inhibitors like Acalabrutinib or Zanubrutinib, which are effective in CLL and may offer benefits in LGL leukemia. 3) CAR-T cell therapies, which are being explored for various hematologic cancers and could provide a novel approach for LGL leukemia.

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Janus Kinase (JAK) Inhibitor

Ruxolitinib for Large Granular Lymphocytic Leukemia

Phase 2

Recruiting

Led By Jonathan Brammer, MD

Research Sponsored by Jonathan Brammer

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 up to 12 months

Awards & highlights

No Placebo-Only Group

Summary

This trial tests if ruxolitinib can shrink tumors in patients with a specific type of leukemia called T-cell large granular lymphocyte leukemia. Ruxolitinib works by blocking enzymes that cancer cells need to grow. The goal is to see if this medication can help these patients when other treatments might not work. Ruxolitinib is an oral medication used to treat myelofibrosis and polycythemia vera, but its effects in these conditions are uncertain.

Who is the study for?

Adults with T-cell large granular lymphocyte leukemia (T-LGLL) who have tried at least one treatment without success and are not currently on therapy. They must have symptoms like low hemoglobin, need regular blood transfusions, or suffer from frequent infections due to low neutrophil counts. Their liver and kidney functions should be near normal, they can't be pregnant, and must agree to use birth control.

What is being tested?

The trial is testing Ruxolitinib's effectiveness in shrinking tumors for patients with T-LGLL. It's a phase II study where the drug aims to inhibit enzymes that cancer cells need to grow.

What are the potential side effects?

Ruxolitinib may cause side effects such as infection risk due to lowered immune response, anemia, changes in liver enzyme levels indicating potential liver issues, and possibly other reactions related to altering cell growth processes.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 12 months

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 12 months

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 12 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

Overall response rate (ORR)

Secondary study objectives

Incidence of treatment-emergent adverse events

Leukemia-free survival (LFS)

Patient quality-of-life (QOL) EORTC

+3 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

No Placebo-Only Group

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

Trial Design

1Treatment groups

Experimental Treatment

Group I: Treatment (ruxolitinib)Experimental Treatment1 Intervention

Patients receive ruxolitinib PO BID on days 1-28. Cycles repeat every 28 days for 12 months in the absence of disease progression or unacceptable toxicity. Patients who achieve a response (CR or PR) may receive an additional 12 months of ruxolitinib, for a maximum of 24 months.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Ruxolitinib

2018

Completed Phase 3

~1170

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

Ruxolitinib, a JAK1/2 inhibitor, works by blocking enzymes essential for the growth and survival of cancer cells, thereby reducing tumor size in patients with T-cell Large Granular Lymphocyte Leukemia (LGL). Other common treatments for LGL include immunosuppressive therapies like methotrexate and cyclosporine, which reduce the activity of the immune system to control the overproduction of large granular lymphocytes. Understanding these mechanisms is crucial for LGL patients as it helps in selecting the most effective treatment strategy, managing side effects, and improving overall outcomes by targeting the specific pathways involved in the disease.

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