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Anti-metabolites

ASTX727 for Peripheral Nerve Sheath Tumor

Phase 2

Recruiting

Led By Ping Chi, MD, PhD

Research Sponsored by Memorial Sloan Kettering Cancer Center

Eligibility Criteria Checklist

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

Must have

Patients must be at least 18 years of age

Patients must have advanced, unresectable or metastatic MPNSTs

Must not have

Left ventricular ejection fraction (LVEF) <50% as determined by a multigated acquisition (MUGA) scan or echocardiogram

Patients had a major surgery within 3 weeks prior to study entry or who have not recovered from side effects of such procedure

Timeline

Screening 3 weeks

Treatment Varies

Follow Up at the end of 16 weeks

Awards & highlights

No Placebo-Only Group

Summary

This trial is testing whether ASTX727 is an effective treatment for people who have MPNST with a PCR2 mutation.

Who is the study for?

This trial is for adults with malignant peripheral nerve sheath tumors (MPNST) that have a specific mutation called PCR2. Participants must have tried at least one standard treatment or be unsuitable for chemotherapy, and their cancer should be advanced and measurable. They need to be in fair health overall, able to take pills, not pregnant or breastfeeding, willing to use contraception, and without certain heart conditions or recent major surgeries.

What is being tested?

ASTX727 is being tested as a potential treatment for MPNST with PCR2 mutation. It's a combo of cedazuridine and decitabine designed to target cancer cells more effectively than decitabine alone by disrupting the tumor cells' survival mechanisms.

What are the potential side effects?

While the exact side effects are not listed here, ASTX727 includes decitabine which can cause low blood cell counts leading to increased infection risk, bleeding issues, fatigue; gastrointestinal symptoms like nausea; liver function changes; and infusion-related reactions.

Eligibility Criteria

Inclusion Criteria

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

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I am 18 years old or older.

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My cancer cannot be removed by surgery and has spread.

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

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My tumor is confirmed to lack PRC2.

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I can take pills by mouth.

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My cancer has worsened after treatment, or chemotherapy isn't right for me.

Exclusion Criteria

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

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My heart's pumping ability is below 50%.

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I had major surgery less than 3 weeks ago or am still recovering from it.

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I have a digestive system condition.

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I do not have active hepatitis C or B, nor an active HIV infection.

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I have active tuberculosis.

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I have a cancer that cannot be removed by surgery and is spreading.

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I have active brain metastasis or leptomeningeal disease.

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I haven't had any cancer treatment in the last 14 days.

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I have a serious heart condition.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ at the end of 16 weeks

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~at the end of 16 weeks

This trial's timeline: 3 weeks for screening, Varies for treatment, and at the end of 16 weeks for reporting.

Treatment Details

Study Objectives

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

Primary study objectives

the best clinical benefit rate (CBR)

Secondary study objectives

objective response rate (ORR)

Side effects data

From 2023 Phase 3 trial • 227 Patients • NCT03306264

69%

Thrombocytopenia

61%

Neutropenia

55%

Anaemia

52%

Fatigue

49%

Constipation

48%

Nausea

42%

Diarrhoea

38%

Headache

35%

Dyspnoea

33%

Decreased Appetite

32%

Dizziness

31%

Cough

28%

Leukopenia

28%

Oedema Peripheral

25%

Febrile neutropenia

25%

Arthralgia

22%

Asthenia

21%

Hypokalaemia

21%

Pyrexia

20%

Back Pain

20%

Oropharyngeal Pain

20%

Vomiting

18%

Contusion

18%

Stomatitis

16%

Myalgia

15%

Insomnia

15%

Abdominal Pain

15%

Alanine Aminotransferase Increased

14%

Pain In Extremity

13%

Rash Maculo-Papular

13%

Pneumonia

13%

Blood Creatinine Increased

12%

Rash

12%

Urinary Tract Infection

12%

Upper Respiratory Tract Infection

12%

Fall

12%

Nasal Congestion

12%

Epistaxis

12%

Hypotension

11%

Hyperglycaemia

11%

Hypocalcaemia

11%

Aspartate Aminotransferase Increased

10%

Chills

10%

Alopecia

10%

Muscle Spasms

Hyponatraemia

Petechiae

Haematuria

Sepsis

Blood Alkaline Phosphatase Increased

Cellulitis

Bone Pain

Anxiety

Nasopharyngitis

Blood Bilirubin Increased

Weight Decreased

Depression

Hypertension

Hypomagnesaemia

Haemorrhoids

Hypoalbuminaemia

Pruritus

Non-Cardiac Chest Pain

Toothache

Skin Lesion

Neck Pain

Pain

Lymphopenia

Musculoskeletal Pain

Dyspepsia

Rhinorrhoea

Febrile Neutropenia

Dehydration

Conjunctival Haemorrhage

Peripheral Swelling

Procedural Pain

Rhinitis Allergic

Pollakiuria

Dyspnoea Exertional

Night Sweats

Syncope

Influenza

Colitis

Myocardial infarction

Diverticulitis

Embolism

Gastrointestinal haemorrhage

Pneumothorax

Proctitis

Upper respiratory tract infection

Gingival bleeding

Enterobacter infection

Rib fracture

Sinusitis fungal

Spinal fracture

Haematoma

Rectal haemorrhage

Embolism arterial

Upper gastrointestinal haemorrhage

Respiratory failure

Device related infection

Peritonsillitis

Pneumonia cytomegaloviral

Pseudomonal bacteraemia

Soft tissue necrosis

Deep vein thrombosis

Colonic abscess

Gastroenteritis Escherichia coli

Proctalgia

Oedema peripheral

Pharyngitis

Urinary tract infection

Clostridium difficile colitis

Small intestinal obstruction

Aplasia pure red cell

Bronchopulmonary aspergillosis

Decreased appetite

Angioedema

Hypersensitivity vasculitis

Refractory cytopenia with unilineage dysplasia

Acute coronary syndrome

Enteritis

Cholelithiasis

Gastroenteritis viral

Oral candidiasis

Septic shock

Limb injury

Influenza A virus test positive

Cerebral haemorrhage

Pleural effusion

Pulmonary embolism

Bacteraemia

Post procedural haematoma

Chronic obstructive pulmonary disease

Adenocarcinoma gastric

Pancreatic carcinoma metastatic

Pyoderma gangrenosum

Thrombotic thrombocytopenic purpura

Parainfluenzae virus infection

Transient ischaemic attack

Suicide attempt

100%

80%

60%

40%

20%

Study treatment Arm

MDS or CMML: ASTX727

AML: ASTX727

MDS or CMML: IV Decitabine

MDS or CMML: Not Treated

AML: Not Treated

AML: IV Decitabine

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: ASTX727 (cedazuridine and decitabine)Experimental Treatment1 Intervention

Patients who meet the eligibility criteria will be treated with oral ASTX727 (INQOVI) on days 1-5 of each 21-day cycle with Pegfilgrastim support on day 7. A delay in the start of subsequent cycles due to holidays, weather, or other circumstances will be permitted up to 7 days and not considered a protocol deviation. Drug dosing will be interrupted for any Grade 4 adverse events or clinically significant laboratory abnormalities. For Grade 3 or 4 AE, if the AE returns to Grade 1 or baseline, the patient may be re-escalated.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

ASTX727

2018

Completed Phase 3

~270

0 / 15Eligibility criteria met