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Janus Kinase (JAK) Inhibitor

Cellular Therapy + Ruxolitinib for Graft-versus-Host Disease

Phase < 1

Recruiting

Led By Partow Kebriaei

Research Sponsored by M.D. Anderson Cancer Center

Eligibility Criteria Checklist

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

Must have

Karnofsky/Lansky Performance score of at least 30 at the time of study entry

Patient (or legal representative where appropriate) must be capable of providing written informed consent, and assent if indicated

Must not have

Isolated acute GVHD of skin

De novo chronic GVHD

Timeline

Screening 3 weeks

Treatment Varies

Follow Up at 6 months

Awards & highlights

No Placebo-Only Group

Summary

This trial is testing whether adding cord blood-derived mesenchymal stromal cells to ruxolitinib can help treat patients with acute graft versus host disease that has not responded to steroid therapy.

Who is the study for?

This trial is for patients aged 12-80 with acute graft versus host disease that's not improving with steroids, specifically affecting the lower GI tract or liver. They must have a certain level of kidney function and be able to consent. Women who can get pregnant and men must use birth control. People with skin-only GVHD, uncontrolled infections, significant oxygen needs, allergies to certain animal products, or using other treatments are excluded.

What is being tested?

The study tests adding cord blood tissue-derived mesenchymal stromal cells (cb-MSCs) to ruxolitinib in treating steroid-refractory acute graft versus host disease after stem cell transplant. It explores whether cb-MSCs combined with ruxolitinib can better manage the condition compared to standard treatments.

What are the potential side effects?

Potential side effects include those related to ruxolitinib like anemia, low platelet count, dizziness and headache; plus risks from cellular therapy such as immune reactions or infection at injection site.

Eligibility Criteria

Inclusion Criteria

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

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I can care for myself but may need occasional help.

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I can give my consent, or my legal representative can, for this trial.

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I am between 12 and 80 years old.

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My kidneys are functioning well enough (creatinine clearance ≥ 30 mL/min).

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I have severe GVHD in my lower GI or liver that didn't improve with high-dose steroids.

Exclusion Criteria

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

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I have a skin condition caused by a recent transplant.

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I have been diagnosed with new onset chronic graft-versus-host disease.

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My primary treatment did not include AAT, steroids, or ruxolitinib.

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I need more than 6 liters of oxygen through a nasal tube.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ at 6 months

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~at 6 months

This trial's timeline: 3 weeks for screening, Varies for treatment, and at 6 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

Response

Secondary study objectives

Graft versus host disease status

Graft-vs-Host Disease

Incidence of chronic graft versus host disease

+6 more

Other study objectives

Cytokine biomarker analysis (optional)

Fecal samples analysis (optional)

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

3Treatment groups

Experimental Treatment

Active Control

Group I: Arm 3 (ruxolitinib, higher dose ds-MSCs)Experimental Treatment2 Interventions

Patients receive ruxolitinib PO BID as in Arm 1. Patients also receive higher dose of cb-MSCs IV for up to 60 minutes twice weekly (at least 3 days apart) over 4 consecutive weeks for 8 total doses.

Group II: Arm 2 (ruxolitinib, lower dose ds-MSCs)Experimental Treatment2 Interventions

Patients receive ruxolitinib PO BID as in Arm 1. Patients also receive lower dose of cb-MSCs IV for up to 60 minutes twice weekly (at least 3 days apart) over 4 consecutive weeks for 8 total doses.

Group III: Arm 1 (ruxolitinib)Active Control1 Intervention

Patients receive ruxolitinib PO BID for at least 3 days and may consider tapering after 6 months of therapy if response occurs and therapeutic corticosteroid doses have been discontinued.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Ruxolitinib

2018

Completed Phase 3

~1170

0 / 9Eligibility criteria met