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Chemotherapy

CPX-351 for Myelodysplastic Syndrome

Phase 1

Waitlist Available

Led By Meagan Jacoby, M.D., Ph.D.

Research Sponsored by Washington University School of Medicine

Eligibility Criteria Checklist

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

Must have

Diagnosis of myelodysplastic syndrome (MDS) with an IPSS-R score of Intermediate, High or Very High (see Appendix A) AND ≥ 5% myeloblasts in the bone marrow

Left ventricular cardiac ejection fraction ≥ 50% by echocardiography or MUGA

Must not have

History of Wilson's disease or other copper-metabolism disorder

A history of allergic reactions attributed to compounds of similar chemical or biologic composition to CPX-351 or other agents used in the study

Timeline

Screening 3 weeks

Treatment Varies

Follow Up through 5 years

Awards & highlights

No Placebo-Only Group

Summary

This trial is testing a new cancer drug for safety and effectiveness in patients with myelodysplastic syndrome.

Who is the study for?

This trial is for adults aged 18-70 with higher risk Myelodysplastic Syndrome (MDS) who are eligible for a transplant. They must have good kidney, liver, and heart function, an ECOG performance status ≤2, and no history of Wilson's disease or certain viral infections. Women must not be pregnant and participants should agree to use contraception.

What is being tested?

The study tests two doses of CPX-351 (Vyxeos™) in MDS patients to see which is safer and more tolerable. It also looks at how many achieve complete remission and can proceed to a bone marrow transplant after treatment.

What are the potential side effects?

While the specific side effects aren't listed here, CPX-351 may include typical chemotherapy-related side effects such as fatigue, nausea, increased infection risk due to low blood cell counts, bleeding or bruising easily from low platelets.

Eligibility Criteria

Inclusion Criteria

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

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My MDS is rated Intermediate to Very High with more than 5% myeloblasts in my bone marrow.

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My heart pumps well, with an ejection fraction of 50% or higher.

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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 between 18 and 70 years old.

Exclusion Criteria

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

Select...

I have a history of Wilson's disease or a similar copper-metabolism disorder.

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I have allergies to drugs similar to CPX-351 or others used in this study.

Select...

I do not have any uncontrolled illnesses like infections or heart problems.

Select...

I have received specific treatments for my MDS before.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ through 5 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~through 5 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and through 5 years for reporting.

Treatment Details

Study Objectives

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

Secondary study objectives

Best overall response in MDS patients treated with CPX-351

Complete remission + marrow complete remission rates in patients treated with CPX-351

Muscular Dystrophy

+7 more

Side effects data

From 2015 Phase 3 trial • 309 Patients • NCT01696084

68%

Febrile Neutropenia

49%

Nausea

46%

Diarrhoea

42%

Constipation

41%

Oedema Peripheral

35%

Headache

35%

Epistaxis

35%

Fatigue

33%

Cough

33%

Decreased Appetite

29%

Rash

27%

Chills

25%

Vomiting

24%

Dyspnoea

24%

Insomnia

22%

Abdominal Pain

22%

Pyrexia

21%

Dizziness

20%

Hypotension

20%

Hypoxia

19%

Hypertension

18%

Pneumonia

18%

Mucosal Inflammation

18%

Oropharyngeal Pain

17%

Pleural Effusion

16%

Arthralgia

15%

Pruritus

15%

Anxiety

14%

Tachycardia

14%

Petechiae

14%

Back Pain

13%

Confusional State

13%

Pain In Extremity

12%

Abdominal Distension

12%

Haemorrhoids

10%

Mouth Haemorrhage

Rash Maculo-Papular

Erythema

Stomatitis

Dyspepsia

Asthenia

Night Sweats

Blood Blister

Dysgeusia

Fluid Overload

Bacteraemia

Transfusion Reaction

Haemoptysis

Sepsis

Gingival Bleeding

Oedema

Procedural Pain

Fall

Neck Pain

Pulmonary Oedema

Rales

Respiratory Failure

Hyperhidrosis

Wheezing

Vision Blurred

Dry Mouth

Chest Pain

Catheter Site Pain

Musculoskeletal Pain

Depression

Renal Failure Acute

Haematuria

Rash Pruritic

Urinary Incontinence

Ecchymosis

Abdominal Pain Upper

Nasal Congestion

Mouth Ulceration

Ejection Fraction Decreased

Dysphagia

Catheter Site Erythema

Cellulitis

Contusion

Pollakiuria

Deep Vein Thrombosis

Myalgia

Dry Skin

Hiccups

Tachypnoea

Dysuria

Atrial Fibrillation

Conjunctival Haemorrhage

Chest Discomfort

Agitation

Acute Respiratory Failure

Disease Progression

Delirium

Rash Erythematous

Syncope

Muscular Weakness

Gastrooesophageal Reflux Disease

Skin Lesion

Oral Pain

Hallucination

Alopecia

Weight Decreased

Central Nervous System Haemorrhage

Myocardial Infarction

Somnolence

Streptococcus Test Positive

Cerebral Haemorrhage

Mental Status Changes

Haemorrhage Intracranial

Fungal Test Positive

Stenotrophomonas Test Positive

Pancytopenia

Euthyroid Sick Syndrome

Hypothyroidism

Urinary Tract Infection

Enterococcus Test Positive

Hepatic Enzyme Increased

Neutropenia

Pneumonia Bacterial

Bacteroides Bacteraemia

Streptococcal Sepsis

Pseudomonas Test Positive

Staphylococcus Test Positive

Staphylococcal Bacteraemia

Bronchopulmonary Aspergillosis

Sinusitis Fungal

Skin Infection

Pneumonia Aspiration

Pneumothorax

Transfusion-Related Acute Lung Injury

Alloimmunisation

Anaemia

Thrombocytopenia

Cardiac Failure

Cardiac Arrest

Cardiac Failure Congestive

Cardiomyopathy

Mitral Valve Incompetence

Pericarditis

Small Intestinal Disorders

Chron's Disease

Gastric Haemorrhage

Lower Gastrointestinal Haemorrhage

Multi-Organ Failure

Death

Non-Cardiac Chest Pain

Cholecystitis Acute

Bile Duct Stone

Septic Shock

Enterococcal Bacteraemia

Diverticulitis

Enterobacter Bacteraemia

Mycotic Aneurysm

Neutropenic Infection

Pseudomonal Bacteraemia

Sinusitis

Dehydration

Lactic Acidosis

Acute Myeloid Leukaemia

Acute Myeloid Leukaemia Recurrent

Myelodysplastic Syndrome

Renal Cell Carcinoma

Carotid Artery Stenosis

Cerebral Infarction

Convulsion

Presyncope

Radiculopathy

Acute Respiratory Distress Syndrome

100%

80%

60%

40%

20%

Study treatment Arm

Arm A (CPX-351)

Arm B (7+3)

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: CPX-351Experimental Treatment4 Interventions

* CPX-351 will be given according to the assigned dose level over a minimum of a 90-minutes via IV infusion on Days 1, 3, and 5 of the first induction * If the treating physician elects to perform a day 14 bone marrow biopsy then, a second induction may be considered for patients in the absence of a chemoablated, hypocellular marrow on the Day 14 bone marrow assessment, if the patient has failed to achieve a marrow CR, and it is deemed safe to administer by the treating physician. The second induction uses a modified schedule in which CPX-351 will be given according to the assigned dose level on Days 1 and 3 * In the absence of disease progression or unacceptable toxicity, the patient may continue to consolidation at the discretion of the treating physician or the patient may proceed to alloHCT after induction at the discretion of the treating physician

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Research blood draw

2022

Completed Phase 2

~240

CPX-351

2022

Completed Phase 3

~1170