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Trial record 118 of 350 for:    Gastrointestinal Stromal Tumors
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The Efficacy and Safety of Temozolomide in SDH-deficient GIST (GIST)

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ClinicalTrials.gov Identifier: NCT05661643
Recruitment Status : Recruiting
First Posted : December 22, 2022
Last Update Posted : March 15, 2024
Sponsor:
Information provided by (Responsible Party):
Min-Hee Ryu, Asan Medical Center

Study Description
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Brief Summary:
The goal of this clinical trial is to investigate the efficacy and safety of temozolomide in SDH deficiency GIST patients.

Condition or disease Intervention/treatment Phase
Gastrointestinal Stromal Tumors Drug: Temozolomide capsule Phase 2

Detailed Description:

Wild type GISTs are less responsive to imatinib with a response rate of 23.1-44.6% and a median progressiion-free survival of 12.3-12.8 months. The efficacy of imatinib is limited in particular in SDH deficienctGIST with a reported response of 2%. Therefore, the development of a new therapeutic agents is urgently needed.

Recently, a study of TKI-resistant SDH-deficient preclinical model showed that temozolomide, an alkylating agent, promotes DNA damage in tumor cells, leading to tumor cell killing. In a retrospective analysis, 2 out of 5 SDH deficient GIST patients treated with temozolomide showed partial response, suggesting its efficacy in this patient population.

Based on these findings,The goal of this clinical trial is to investigate the efficacy and safety of temozolomide in SDH deficiency GIST patients. In addition, for exploratory purposes, aim to investigate the efficacy and safety of temozolomide in KIT and PDGFRA wild-type GIST without SDH deficiency.

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 29 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase 2 Study to Evaluate the Efficacy and Safety of Temozolomide in Advanced Gastrointestinal Stromal Tumor Patients With SDH Deficiency
Actual Study Start Date : June 28, 2023
Estimated Primary Completion Date : December 1, 2026
Estimated Study Completion Date : December 31, 2027

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: temozolomide treatment Drug: Temozolomide capsule
Temozolomide 200 mg/m2 is administered orally for 1-5 days of each cycle, and then canceled for 23 days (a total of 28 days is 1 cycle)


Outcome Measures
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Primary Outcome Measures :
  1. Objective respone rate in SDH deficiency wild type GIST [ Time Frame: up to 4 years ]
    complet response+partial response defined by RECIST v1.1


Eligibility Criteria
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Ages Eligible for Study:   20 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Age 20 years or older, at the time of acquisition of informed consent
  2. Histologically confirmed GIST with CD117(+), DOG-1(+)
  3. Wild type GIST without KIT or PDGFRα gene mutations determined by Sanger sequencing and panel sequencing
  4. Eastern Cooperative Oncology Group (ECOG) performance status 0 ~ 2
  5. Resolution of all adverse events with prior treatments to grade 0 or 1 by NCI-CTCAE version 5.0
  6. At least one measurable lesion by RECIST version 1.1.

  7. Adequate bone marrow, hepatic, renal, and other organ functions, before adjuvant imatinib treatment

    • Neutrophil >1,500/mm3
    • Platelet > 100,000/mm3
    • Hemoglobin >8.0 g/dL
    • Total bilirubin < 1.5 x upper limit of normal (ULN)
    • AST/ALT < 2.5 x ULN
    • Creatinine <1.5 x ULN
  8. Life expectancy ≥12 weeks
  9. Disease progression or discontinuation of treatment due to intolerable toxicity at least with palliative 1st line imatinib .
  10. Washout period of previous TKIs or chemotherapy for more than 4 times the half life ((Imitinib and regorafenib need 1 week and sunitinib need 2 weeks.)
  11. Provision of a signed written informed consent

Exclusion Criteria:

  1. Confirmed GIST with KIT or PDGFRα gene mutations determined by Sanger sequencing and panel sequencing
  2. Women of child-bearing potential who are pregnant or breast feeding
  3. Women or men who are not willing to use effective contraception entering the study period or until at least 6 months after the last study drug administration
  4. If any of the following applies within ≤ 6 months prior to starting study enrollment : Myocardial Infarction, severe instable angina, coronary/peripheral bypass, NYHA class III or IV congestive heart failure, stroke or transient ischemic attack, treatment required severe arrhythmia
  5. Uncontrolled infection
  6. Acute and chronic liver disease and all chronic liver impairment.(But Patients with stable chronic hepatitis B are eligible
  7. Acute, or chronic medical or psychiatric condition or laboratory abnormality such as active uncontrolled infection that difficult to study participation in the judgment of the investigator
  8. Known diagnosis of HIV infection (HIV testing is not mandatory).
  9. History of another primary malignancy that is currently clinically significant or currently requires active intervention.
  10. Alcohol or substance abuse disorder
  11. The patients with NTRK fusion

5)

Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05661643


Contacts
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Contact: Min-Hee Ryu, MD, PhD 82-2-3010-5936 miniryu@amc.seoul.kr
Contact: Hyung-Don Kim, MD, PhD 82-2-3010-0236 kimhdmd@amc.seoul.kr

Locations
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Korea, Republic of
Asan Medical Center, University of Ulsan College of Medicine Recruiting
Seoul, Korea, Republic of, 138-736
Contact: Min-Hee Ryu, MD, PhD    +82-2-3010-5935    miniryu@amc.seoul.kr   
Sponsors and Collaborators
Asan Medical Center
More Information
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Responsible Party: Min-Hee Ryu, Professor, Asan Medical Center
ClinicalTrials.gov Identifier: NCT05661643    
Other Study ID Numbers: AMC2203
First Posted: December 22, 2022    Key Record Dates
Last Update Posted: March 15, 2024
Last Verified: March 2024

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Gastrointestinal Stromal Tumors
Neoplasms, Connective Tissue
Neoplasms, Connective and Soft Tissue
Neoplasms by Histologic Type
Neoplasms
Gastrointestinal Neoplasms
Digestive System Neoplasms
 Digestive System Diseases
Gastrointestinal Diseases
Temozolomide
Antineoplastic Agents, Alkylating
Alkylating Agents
Molecular Mechanisms of Pharmacological Action
Antineoplastic Agents