Clinical Research Directory

Rivoceranib Plus Paclitaxel in Patients With Gastrointestinal Stromal Tumor

The purpose of this study is to evaluate the efficacy and safety of rivoceranib and paclitaxel combination therapy in patients with P-glycoprotein overexpressing GIST who failed standard treatment with imatinib, sunitinib, and regorafenib.

The Efficacy and Safety of Temozolomide in SDH-deficient GIST

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

GIST Oral Paclitaxel(Liporaxel)

The purpose of this study is to evaluate safety and efficacy of Liporaxel for patients with GIST who failed on prior standard treatments, including imatinib, sunitinib, and regorafenib, and with low P-glycoprotein expression.

Neoadjuvant Imatinib and Fampridine in KIT Mutant Gastrointestinal Stromal Tumor

The goal of this clinical trial is to learn what dose of the drug fampridine can be given safely together with imatinib (Gleevec) in patients with gastrointestinal stromal tumor (GIST) with a DNA mutation in exon 11 of the KIT gene. The main questions this study aims to answer are: * What is the maximum dose of fampridine that can be given safely together with imatinib (Gleevec)? * Is the combination of the two drugs efficacious against the tumor? Participants will: * Take the drugs before tumor surgery (neoadjuvant treatment) for at least 2 months with the option to continue for a longer period of time if treatment seems safe and effective. * Visit the clinic at the scheduled appointments for checkups and tests.

Assessment of Compliance With Monitoring Conducted by a Physician in Person or by a Nurse in Remote Monitoring

This is a multicenter, interventional, randomized study among adult patients recently diagnosed with a rare tumor (\<12 months). The study will aim to compare compliance with the personalized post-treatment surveillance plan, established for each patient according to national guidelines, when the surveillance is conducted in person by a hospital-based physician (control arm) or remotely by a trained nurse (experimental arm).

Imatinib TDM in GIST

Imatinib can lead to long recurrence free survival in patients diagnosed with gastrointestinal stromal tumors (GIST); however side effects can significantly hinder quality of life for our patients. This study will use therapeutic drug monitoring to improve quality of life and symptoms and assess how many patients maintain therapeutic levels. Free drug levels and pharmokinetics of imatinib will also be monitored.

A Study of Ripretinib vs Sunitinib in Advanced GIST Patients After Treatment With Imatinib

This is a 2-arm, randomized, open-label, international, multicenter study comparing the efficacy of ripretinib to sunitinib in GIST patients who progressed on or were intolerant to first-line anticancer treatment with imatinib. Approximately 426 patients will be randomized in a 1:1 ratio to ripretinib 150 mg once daily (continuous dosing for 6 week cycles) or sunitinib 50 mg once daily (6 week cycles, 4 weeks on, 2 weeks off).

Integrated Cancer Repository for Cancer Research

The iCaRe2 is a multi-institutional resource created and maintained by the Fred \& Pamela Buffett Cancer Center to collect and manage standardized, multi-dimensional, longitudinal data and biospecimens on consented adult cancer patients, high-risk individuals, and normal controls. The distinct characteristic of the iCaRe2 is its geographical coverage, with a significant percentage of small and rural hospitals and cancer centers. The iCaRe2 advances comprehensive studies of risk factors of cancer development and progression and enables the design of novel strategies for prevention, screening, early detection and personalized treatment of cancer. Centers with expertise in cancer epidemiology, genetics, biology, early detection, and patient care can collaborate by using the iCaRe2 as a platform for cohort and population studies.

A Study of Pimitespib in Combination With Imatinib in Patients With GIST (CHAPTER-GIST-101)

This study consists of Dose escalation part and Expansion part. In Dose Escalation Part, the maximum tolerated dose of combination of pimitespib and imatinib in patients with gastrointestinal stromal tumors (GIST) who are judged to be refractory to imatinib, estimate the recommended dose, evaluate safety and pharmacokinetics, and observe the antitumor effect. Expansion part consists of 3 arms. In Arm A, the efficacy and safety will be evaluated, which of the combination of pimitespib and imatinib in patients with GIST who have failed imatinib at doses below the MTD determined in Dose Escalation Part. In Arm B, the efficacy and safety of pimitespib monotherapy will be evaluated and the therapeutic effect of imatinib administration after pimitespib will be evaluated in an exploratory manner. In Arm C, the efficacy and safety of sunitinib monotherapy will be evaluated as reference data.

Phase 2 Study to Evaluate the Efficacy of Regorafenib in Specific GIST Mutation Subsets (KIT Exon 17, 18, or 14 Mutation and SDHB Deficient GIST) in the Post-imatinib Second-line Setting.

To learn if regorafenib can help to control the disease.

SARC044: A Phase II Trial of Bezuclastinib in Combination With Sunitinib in Patients With GIST

This is an open label, single arm, phase 2 trial investigating bezuclastinib plus sunitinib in patients with GIST who have previously progressed on sunitinib.

A Study of the c-Kit Specific Antibody-Drug Conjugate NN3201 for Advanced and/or Metastatic Solid Tumors Known to Express c-Kit

This open-label clinical trial will evaluate the safety and tolerability of NN3201 in subjects with advanced and/or metastatic solid tumors known to express c-Kit.

ctDNA-Guided Sunitinib And Regorafenib Therapy for GIST

The purpose of this research is to test if mutations (changes in DNA) in exons (segment of DNA or RNA containing information that has the instructions for making proteins) in the KIT gene can be used to predict the body's response to standard of care treatment.

5 Years of Adjuvant Imatinib in Patients With Gastrointestinal Stromal Tumor With a High Risk

In this study, the investigators aim to investigate the efficacy and safety of 5 years of adjuvant imatinib treatment in patients with tumor rupture defined by Nishida classification or those with a tumor size 10cm or larger and a mitotic index of 10/50HPFs or higher.

Observational Registry Data on GIST Patients

This is a longitudinal, multi-center, registry study, collecting data via a web-based portal in patients with GIST (Gastrointestinal Stromal Tumor) from hospitals in Taiwan.

Observational Study, for Quality Assessment, of Sarcoma in European and Latin American Multidisciplinary NETWORK

Post-authorisation, multicentric, observational, retrospective and prospective study to assess quality of care of sarcoma patients in expert and non-expert centers by analysing correlation of quality items and outcomes such as relapse free survival, overall survival, percentage of amputation, etc. Expert pathology peer review will be performed to detect differences between expert and non-expert centers as well as differences in treatment and patient prognosis. Tumor samples of 4 types of sarcoma would also be included in translational research to detect biomarkers and produce preclinical models.

Avapritinib in the Treatment of Unresectable or Recurrent Metastatic GIST Non-exon18 Mutations of PDGFRA

This is a prospective, multicenter, observational real-world study to explore the Avapritinib therapy in GIST patients who definited Non-exon18 Mutations of PDGFRA.

A Multicenter Study of Avapritinib Efficacy and Safety of Metastatic or Unresectable Gastrointestinal Stromal Tumors

This is a prospective, multicenter, observational real-world study to explore the therapy patterns and clinical outcomes of Avapritinib in patients with metastatic or unresectable gastrointestinal stromal tumors.

The Correlation Between Ripretinib Exposure and the Efficacy and Safety in Patients with Advanced GISTs

This is a prospective, single-center, observational study to explore the correlation between ripretinib exposure and the efficacy and safety in patients with advanced gastrointestinal stromal tumors

Second-line Pharmacotherapy Patterns and Outcomes of Advanced Gastrointestinal Stromal Tumor: A Real-world Study

This is a prospective, multicenter, observational real-world study to explore the second-line Pharmacotherapy patterns and clinical outcomes in GIST patients who progressed on or were intolerant to first-line anticancer treatment.

Temozolomide (TMZ) in Advanced Succinate Dehydrogenase (SDH)-Mutant/Deficient Gastrointestinal Stromal Tumor (GIST)

Funding Source - FDA OOPD FDA-approved products for patients with unresectable or metastatic GIST include therapies such as imatinib and sunitinib. Although there are FDA-approved products for the treatment of advanced/metastatic GIST, these therapies are known to be ineffective in the SDH-mutant/deficient subtype and no known effective therapies exist. The purpose of this study is to investigate SDH-Mutant/Deficient Gastrointestinal Stromal cancer's response to the drug Temozolomide (TMZ) and aim to improve patient outcomes. Temozolomide is approved by the FDA for the treatment of newly diagnosed glioblastoma multiforme (GBM) and refractory anaplastic astrocytoma cancers. Temozolomide is considered experimental because it is not approved by the FDA for the treatment of SDH-Mutant/Deficient Gastrointestinal Stromal Tumor.

Targeted Agent Evaluation in Digestive Cancers in China Based on Molecular Characteristics

This a prospective real-world navigation study using tumor DNA sequencing technology to sequence genes of previously treated and refractory gastrointestinal tumors, which are generally considered to be highly heterogeneous and complex, to screen potential molecular targeted drugs for individualized treatment. This study may provide feasibility and response information, which will be the basis for designing better randomized trials, which may change the pattern of cancer treatment. If the hypothesis is finally proved, it will help doctors and molecular biologists to choose the best drug (or combination of drugs) based on the individual oncogenomics of each patient.

Genetic Bases of Neuroendocrine Neoplasms in Mexican Patients

Neuroendocrine neoplasms (NENs) are a heterogeneous group of lesions derived from cells with the ability to produce hormones that may arise from multiple different organs. Their clinical behavior is quite variable, encompassing both benign lesions and aggressive tumors that invade surrounding and/or distant structures. NENs may also cause serious morbidity due to hormone oversecretion. NENs are among the most frequently inherited human tumors, presenting either isolated or as part of syndromes in which a single patient or family develops multiple tumors. There are also non-inherited changes in the genetic information of the tumor cells that are potential targets for treatment. Both inherited and non-inherited DNA defects can be identified using modern routine genetic tests which, unfortunately, are not widely available in Mexico. This project seeks to uncover the genetic defects causing NENs in a large cohort of Mexican patients, using three different methods for genetic testing. Adult individuals with various types of NENs from two reference hospitals in Mexico City will be invited to participate. After completing informed consent, blood and, if possible, tissue samples will be obtained from all participants. Clinical details, laboratory results, imaging studies, and histopathological data at disease presentation will be retrieved. An initial screening will be performed by analyzing changes in the sequence of multiple genes that have been associated with the occurrence of NENs. In cases with negative screening, a specific method to assess changes in the number of copies of the same genes will also be employed. Finally, sequences of all DNA regions encoding information required to make proteins will be obtained in selected cases. Analyses will be carried out in blood and, if available, also in tumor tissue samples from study participants. Screening of additional family members will be offered. This project will accurately describe the repertoire of specific defects causing NENs in the study population, and will likely uncover and characterize novel genetic associations. The results will contribute for a better understanding of the alterations within and outside known driver genes that shape syndromic presentations, tumor behaviors, and inheritance patterns in individuals with NENs. These data will contribute to improve the information on the molecular bases of NENs, including alterations that can be used as therapeutic targets.

Evaluate the Efficacy and Safety of Radiotherapy Combined With Ripretinib in the Treatment of Unresectable Advanced GIST

The goal of this prospective study is to learn whether there is a synergistic effect when radiotherapy is combined with ripretinib in the treatment of patients with unresectable advanced GIST. It will also learn about the safety of this regimen.The main questions it aim to answer is: Dose radiotherapy combined with ripretinib prolong the time to disease progression for patients with advanced GIST?