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Alkylating agents

Temozolomide for Gastrointestinal Stromal Tumor

Phase 2
Waitlist Available
Led By Jason Sicklick, MD
Research Sponsored by Adam Burgoyne, MD, PhD
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Male patient with a partner of childbearing potential agrees to use an adequate method of contraception starting with the first dose of study therapy through 120 days after the last dose of study therapy
Patient has pathologically confirmed SDH-mutant/deficient GIST
Must not have
Patients who have had major surgery within 4 weeks of initiation of study medication
Evidence of severe or uncontrolled systemic diseases [e.g., unstable or uncompensated respiratory, cardiac (including life threatening arrhythmias)]
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 4 years
Awards & highlights
All Individual Drugs Already Approved
No Placebo-Only Group

Summary

This trial is investigating if the drug Temozolomide can be an effective treatment for SDH-Mutant/Deficient Gastrointestinal Stromal Tumor, which currently doesn't have any known effective treatments.

Who is the study for?
This trial is for male and female patients with a specific type of cancer called SDH-Mutant/Deficient Gastrointestinal Stromal Tumor (GIST) that cannot be removed by surgery or has spread. Participants must have recovered from previous treatments, not have severe diseases like uncontrolled heart conditions, and agree to use contraception. Pregnant or breastfeeding women and those unable to take oral medication are excluded.
What is being tested?
The study is testing the effectiveness of Temozolomide (TMZ), an FDA-approved drug for certain brain cancers but considered experimental for GIST. The goal is to see if TMZ can improve outcomes in patients with this particular subtype of GIST where other therapies are ineffective.
What are the potential side effects?
Temozolomide may cause side effects such as fatigue, nausea, vomiting, constipation, loss of appetite, headache, seizures or convulsions due to its impact on the brain and nervous system; it might also lower blood cell counts increasing infection risk.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I agree to use contraception during and up to 120 days after the study.
Select...
My GIST is confirmed to have an SDH mutation.
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I can take care of myself and am up and about more than half of the day.
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I agree to follow strict birth control measures or abstain from sex during and 120 days after the study.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
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I have not had major surgery in the last 4 weeks.
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I do not have severe or uncontrolled health issues like heart or lung problems.
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I cannot swallow pills.
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I am not on any cancer treatments like chemotherapy or immunotherapy when starting this study.
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I do not have brain metastases.
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I have severe heart issues or had a recent heart attack.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~4 years
This trial's timeline: 3 weeks for screening, Varies for treatment, and 4 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
Overall Response Rate
Secondary study objectives
Adverse events related to TMZ
Overall survival
Progression-free survival

Side effects data

From 2016 Phase 2 trial • 175 Patients • NCT01055314
36%
Febrile neutropenia
31%
Death NOS
30%
Diarrhea
22%
Pain
21%
Hyperglycemia
16%
Anorexia
16%
Infections and infestations - Other, specify
16%
Alanine aminotransferase increased
14%
Hypokalemia
13%
Nausea
11%
Hyponatremia
10%
Weight loss
9%
Aspartate aminotransferase increased
9%
Mucositis oral
9%
Anemia
9%
Vomiting
9%
Constipation
9%
Dehydration
9%
Hypophosphatemia
8%
Platelet count decreased
8%
Sepsis
7%
Neoplasms benign, malignant and unspecified (incl cysts and polyps) - Other, specify
7%
Catheter related infection
7%
Colitis
7%
Abdominal pain
6%
Hypotension
6%
White blood cell decreased
6%
GGT increased
6%
Hypocalcemia
6%
Urinary retention
6%
Hypoalbuminemia
6%
Fever
5%
Typhlitis
5%
Anxiety
5%
Neutrophil count decreased
5%
Urinary tract infection
4%
Peripheral motor neuropathy
4%
Enterocolitis
4%
Lipase increased
4%
Pleural effusion
4%
Serum amylase increased
4%
Skin infection
4%
Epistaxis
4%
Urinary tract obstruction
3%
Blood bilirubin increased
3%
Lymphocyte count decreased
3%
Syncope
3%
Wound infection
3%
Dermatitis radiation
3%
Hypertension
3%
Sinus tachycardia
3%
Edema limbs
3%
Bone pain
3%
Dyspnea
3%
Hematuria
3%
Hypercalcemia
2%
Vulval infection
2%
Upper gastrointestinal hemorrhage
2%
Depressed level of consciousness
2%
Thromboembolic event
2%
Stridor
2%
Allergic reaction
2%
Back pain
2%
Lung infection
2%
Urticaria
2%
Acute kidney injury
2%
Muscle weakness lower limb
2%
Musculoskeletal and connective tissue disorder - Other, specify
2%
Pain in extremity
2%
Peripheral sensory neuropathy
2%
Proctitis
2%
Skin ulceration
2%
Apnea
2%
Stoma site infection
2%
Tumor pain
2%
Left ventricular systolic dysfunction
2%
Pancreatitis
2%
Portal hypertension
2%
Rectal hemorrhage
2%
Creatinine increased
2%
Enterocolitis infectious
2%
Hyperkalemia
2%
Investigations - Other, specify
2%
Abdominal distension
1%
Heart failure
1%
Vascular disorders - Other, specify
1%
Anal hemorrhage
1%
Ascites
1%
Vasovagal reaction
1%
Penile pain
1%
Bone marrow hypocellular
1%
Gastrointestinal disorders - Other, specify
1%
Soft tissue infection
1%
Delirium
1%
Tracheitis
1%
Seizure
1%
Hepatobiliary disorders - Other, specify
1%
Esophageal pain
1%
Anal mucositis
1%
Menorrhagia
1%
Sore throat
1%
Anaphylaxis
1%
Fracture
1%
Hydrocephalus
1%
Device related infection
1%
Tooth infection
1%
Gastric ulcer
1%
Sinusitis
1%
Skin and subcutaneous tissue disorders - Other, specify
1%
Pharyngitis
1%
Pyramidal tract syndrome
1%
Anal ulcer
1%
Depression
1%
Ejection fraction decreased
1%
Rash maculo-papular
1%
Pruritus
1%
Myositis
1%
Nail infection
1%
Pain of skin
1%
Pleuritic pain
1%
Pneumonitis
1%
Pneumothorax
1%
Postoperative hemorrhage
1%
Renal and urinary disorders - Other, specify
1%
Respiratory, thoracic and mediastinal disorders - Other, specify
1%
Salivary duct inflammation
1%
Small intestine infection
1%
Alkaline phosphatase increased
1%
Appendicitis
1%
Spinal fracture
1%
Disseminated intravascular coagulation
1%
Ear and labyrinth disorders - Other, specify
1%
Endocrine disorders - Other, specify
1%
Esophageal stenosis
1%
Esophagitis
1%
Gastric hemorrhage
1%
Gum infection
1%
Tumor lysis syndrome
1%
Upper respiratory infection
1%
Hypertriglyceridemia
1%
Hypoxia
1%
Ileus
1%
INR increased
1%
Laryngeal edema
1%
Multi-organ failure
1%
Myelodysplastic syndrome
1%
Oral hemorrhage
1%
Oral pain
1%
Pulmonary edema
1%
Rectal fistula
1%
Rectal pain
1%
Respiratory failure
1%
Bladder spasm
1%
Chest wall pain
1%
Confusion
1%
Congenital, familial and genetic disorders - Other, specify
1%
CPK increased
1%
Dizziness
1%
Encephalopathy
1%
Eye disorders - Other, specify
1%
Generalized muscle weakness
1%
Hoarseness
1%
Hypernatremia
1%
Hypoglycemia
1%
Hypomagnesemia
1%
Insomnia
1%
Irregular menstruation
1%
Irritability
1%
Joint range of motion decreased cervical spine
1%
Kyphosis
1%
Lethargy
1%
Headache
1%
Laryngeal mucositis
1%
Pelvic pain
1%
Esophageal infection
1%
Abdominal infection
1%
Acidosis
1%
Anal fistula
1%
Fall
1%
Fatigue
1%
Gait disturbance
100%
80%
60%
40%
20%
0%
Study treatment Arm
Group 1 (Chemotherapy, Radiation Therapy, Cixutumumab)
Group 2 (Chemotherapy, Radiation Therapy, Temozolomide)

Awards & Highlights

All Individual Drugs Already Approved
Therapies where all constituent drugs have already been approved are likely to have better-understood side effect profiles.
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: TMZ 85 mg/m2 mg orallyExperimental Treatment1 Intervention
TMZ 85 mg/m2 mg orally once for 21 days followed by 7 days without treatment in 28 day cycles. Treatment will continue for 6 months (with option to continue if benefiting treatment) or until disease progression or unacceptable toxicity (whichever occurs first). All patients will have regular evaluations for assessment of safety parameters. Temozolomide dose may be held and/or modified for the management of adverse treatment effects according to pre-specified criteria. Patients will have radiographic imaging (CT or MRI) every 8 weeks to assess tumor resection. An end of treatment visit for clinical evaluations and safety assessments will be performed approximately 28 days after the last dose of study drug. Patients discontinuing study treatment will be followed every 3-6 months for disease recurrence and survival.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Temozolomide
FDA approved

Find a Location

Who is running the clinical trial?

Adam Burgoyne, MD, PhDLead Sponsor
Jason Sicklick, MDPrincipal InvestigatorUniversity of California, San Diego

Media Library

Temozolomide (Alkylating agents) Clinical Trial Eligibility Overview. Trial Name: NCT03556384 — Phase 2
Cancer Research Study Groups: TMZ 85 mg/m2 mg orally
Cancer Clinical Trial 2023: Temozolomide Highlights & Side Effects. Trial Name: NCT03556384 — Phase 2
Temozolomide (Alkylating agents) 2023 Treatment Timeline for Medical Study. Trial Name: NCT03556384 — Phase 2
~2 spots leftby Jun 2025
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