← Back to Search

Methyltransferase Inhibitor

ASTX727 for Brain Cancer

Phase 1

Recruiting

Led By Isabel Arrillaga-Romany

Research Sponsored by Massachusetts General Hospital

Eligibility Criteria Checklist

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

Must have

Participants must have archived primary tumor biopsies or surgical specimens for additional exploratory translational studies

Female participants with reproductive potential must have a negative serum pregnancy test within 14 days prior to the first study drug administration

Must not have

Participants with known additional malignancy that is progressing or requires active treatment within 2 years of start of study drug

Participants with an active severe infection that requires anti-infective therapy or with an unexplained fever >38.5°C during screening visits or on their first day of study drug administration

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 1 year

Awards & highlights

No Placebo-Only Group

Summary

This trial is testing if a higher dose of ASTX727 is safe for patients with a certain type of brain tumor.

Who is the study for?

Adults with recurrent/progressive non-enhancing IDH mutant gliomas who've completed radiation at least 12 weeks prior, understand the consent process, have good liver and kidney function, stable brain scans within 28 days of starting treatment, and a life expectancy over 6 months. Not for pregnant/breastfeeding individuals or those with certain medical conditions that could interfere with the trial.

What is being tested?

The study is testing ASTX727 to find the highest safe dose for patients with specific types of brain tumors (non-enhancing IDH mutant gliomas). It involves people who've had previous treatments as well as those untreated. Some participants may need accessible tumors for surgical evaluation.

What are the potential side effects?

While not explicitly listed in the provided information, potential side effects of ASTX727 based on its class may include gastrointestinal issues like nausea or vomiting, fatigue, liver problems, and blood count abnormalities. Specific side effects will be monitored throughout the trial.

Eligibility Criteria

Inclusion Criteria

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

Select...

I have saved samples from my initial cancer biopsy or surgery.

Select...

I am a woman who can have children and have a recent negative pregnancy test.

Select...

My bone marrow is functioning well.

Select...

My low-grade brain tumor has grown or come back, shown by scans.

Select...

I have not received any treatment for my brain tumor.

Select...

I am 18 years old or older.

Select...

My glioma has a confirmed IDH1 or IDH2 mutation.

Select...

I am mostly able to take care of myself.

Select...

My liver is working well.

Exclusion Criteria

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

Select...

I have another cancer that is getting worse or needs treatment.

Select...

I do not have a severe infection or unexplained fever over 38.5°C.

Select...

My heart's electrical activity is normal and does not pose a risk for irregular heartbeats.

Select...

I have never been treated with Avastin.

Select...

I have a history of severe heart rhythm problems.

Select...

I haven't had cancer treatment in the last 4 weeks.

Select...

I have a condition that affects my ability to swallow or absorb pills.

Select...

I have HIV or active hepatitis B or C.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ from the time of randomization until disease progression or death, up to five years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~from the time of randomization until disease progression or death, up to five years

This trial's timeline: 3 weeks for screening, Varies for treatment, and from the time of randomization until disease progression or death, up to five 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

Maximum tolerated dose

Secondary study objectives

Median Progression Free Survival

Overall Survival

Response Rate

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

Cellulitis

Sepsis

Blood Alkaline Phosphatase Increased

Bone Pain

Anxiety

Nasopharyngitis

Blood Bilirubin Increased

Weight Decreased

Depression

Hypertension

Hypomagnesaemia

Haemorrhoids

Hypoalbuminaemia

Non-Cardiac Chest Pain

Pruritus

Toothache

Skin Lesion

Neck Pain

Pain

Lymphopenia

Musculoskeletal Pain

Dyspepsia

Rhinorrhoea

Dyspnoea Exertional

Febrile Neutropenia

Dehydration

Conjunctival Haemorrhage

Peripheral Swelling

Procedural Pain

Rhinitis Allergic

Pollakiuria

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

Upper gastrointestinal haemorrhage

Rectal haemorrhage

Embolism arterial

Respiratory failure

Device related infection

Peritonsillitis

Pneumonia cytomegaloviral

Pseudomonal bacteraemia

Soft tissue necrosis

Refractory cytopenia with unilineage dysplasia

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

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

2Treatment groups

Experimental Treatment

Group I: Expansion CohortExperimental Treatment1 Intervention

* Oral ASTX727 will be administered daily for 4, 5 or 6 consecutive days * Surgical resection will take place 12 days (+/- 1 day) after initiation of treatment

Group II: ASTX727 (Cedazuridine + Cytidine Antimetabolite Decitabine)Experimental Treatment1 Intervention

-ASTX727 administered orally for 5 or 6 consecutive days every 28d cycle and will de-escalate to 4 consecutive days every 28 d cycle.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

ASTX727

2018

Completed Phase 3

~270

0 / 17Eligibility criteria met