In this summary, we continue highlighting recent clinical trials that are still recruiting in the U.S. which investigate potential treatments for GIST patients. Our GIST Trials Database lists over 70 trials that are GIST-related and ongoing around the world. Currently there are 12 GIST-related trials recruiting in the United States. In our last article, we listed several for different types of GIST mutations. You can read the previous clinical trials update (December 2021) here:
https://liferaftgroup.org/2021/12/clinical-trials-update-december-2021/

For further information about GIST trials including international trials, visit: The Life Raft Group Clinical Trials Website: https://gisttrials.org/. If you want to speak with someone about clinical trials, please contact liferaft@liferaftgroup.org.

For patients with advanced KIT/PDGFRa mutant GIST:

THE-630 Phase 1/2 “A Study of THE-630 in Patients With Advanced Gastrointestinal Stromal Tumors (GIST)”  (https://gisttrials.org/iLRG/details.php?Trial=384) 

Site is at Dana-Farber Boston, MA Principal Investigator Suzanne George. Started January 3, 2022. The study will be conducted in two parts: a dose escalation phase, followed by an expansion phase. Plans to recruit 160 over a period of 42 months. 

In the Phase 1 dose escalation phase oral THE-630 will be administered once daily in a continuous regimen. Primary objectives during the 28-day dose escalation phase are: 1. Number of patients with dose-limiting toxicities. 2. Determination of the recommended Phase 2 dose. 3. Determination of the maximum tolerable dose. 4. Safety analysis – number of participants with treatment emergent adverse events. For the subsequent expansion Phase 2 the primary objective is confirmed Objective Response Rate over a period of up to 24 months after first dose. In the escalation Phase 1 participants with metastatic or unresectable GIST will be admitted. 

During the Expansion Phase 2 unresectable or metastatic GIST patients will be divided into 4 cohorts depending on prior TKI therapy. Cohort 4 will include patients who have progressed on or are intolerant to imatinib including patients in the adjuvant setting. The sponsor is Theseus Pharmaceuticals headquartered in Cambridge, MA. Theseus was founded in 2017 and emerged from stealth mode in 2021. On their web page Theseus describes THE-630 as “Our lead program, THE-630, is a pan-variant KIT inhibitor designed for patients with advanced gastrointestinal stromal tumors (GIST) whose cancer has developed resistance to earlier lines of kinase inhibitor therapy.” Patients interested in this clinical trial can contact: Theseus Pharmaceuticals, 857-400-9491, ClinicalTrials@theseusrx.com.  NCT05160168

About Phase 1 trials: It is important to recognize that Phase I studies are held to find the highest dose
of the new treatment that can be given safely without causing severe side effects.  

• The first few people in the study get a very low dose of the treatment and are watched very closely. If there are only minor side effects, the next feaw participants get a higher dose. This process continues until doctors find a dose that’s most likely to be and effective treatment while having an acceptable level of side effects.

• Safety is the main concern. The research team monitors participants and watches for severe side effects. Due to the small numbers of people in Phase I studies, rare side effects may not be seen until later phases of the trial when more people are receiving the treatment.

• While some people may benefit from being on one, disease response is not the main purpose of a Phase I trial.

Phase I trials carry the most potential risk, but these studies do help some patients. For those with life-threatening illnesses, weighing the potential risks and benefits carefully is key. Sometimes people choose to join Phase I trials when all other treatment options have already been tried.

Read Other Articles from February 2020 Newsletter