What is the mechanism of action of this treatment?

The most common treatments for Gastrointestinal Stromal Tumor (GIST) are tyrosine kinase inhibitors (TKIs) such as imatinib, sunitinib, and regorafenib. These drugs work by targeting specific mutations in the KIT and PDGFRA genes, which are responsible for the uncontrolled growth and survival of GIST cells. By inhibiting the activity of these mutated proteins, TKIs can effectively reduce tumor growth and spread. This targeted approach is crucial for GIST patients as it offers a more personalized treatment option, potentially leading to better outcomes and fewer side effects compared to traditional chemotherapy.

What side effects are associated with this type of intervention?

Common treatments for Gastrointestinal Stromal Tumor (GIST) include tyrosine kinase inhibitors (TKIs) such as imatinib, sunitinib, and regorafenib. These treatments are known to cause a range of side effects. Imatinib can lead to edema, nausea, muscle cramps, and fatigue. Sunitinib is associated with fatigue, diarrhea, hand-foot syndrome, and hypertension. Regorafenib may cause side effects like hypertension, hand-foot skin reactions, diarrhea, and fatigue. These side effects are important considerations for patients and healthcare providers when choosing a treatment plan.

What prior FDA approvals exist?

The FDA has approved several tyrosine kinase inhibitors (TKIs) for the treatment of Gastrointestinal Stromal Tumor (GIST). Imatinib (Gleevec) was the first TKI approved for GIST, targeting KIT and PDGFRA mutations. Sunitinib (Sutent) and regorafenib (Stivarga) were subsequently approved for patients with imatinib-resistant GIST. Avapritinib (Ayvakit) is specifically approved for GIST with the PDGFRA D842V mutation. Ripretinib (Qinlock) is another TKI approved for advanced GIST after prior TKI treatments. These drugs are similar to IDRX-42, which is being investigated for its safety, tolerability, and antitumor activity in advanced GIST.

What other types of research exist?

Promising alternatives to IDRX-42 for GIST patients include imatinib mesylate, which has been a first-line therapy for unresectable or recurrent GIST, and imatinib rechallenge, which has shown efficacy in patients who have progressed on other treatments like sunitinib and regorafenib. These alternatives have demonstrated significant antitumor activity and are supported by clinical data.

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IDRX-42 for GIST

Phase 1

Recruiting

Research Sponsored by IDRx, Inc.

Eligibility Criteria Checklist

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

Must have

Be older than 18 years old

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 5 years

Awards & highlights

No Placebo-Only Group

Summary

This trial tests IDRX-42, an oral drug designed to block proteins that help cancer grow, in adults with advanced GIST who haven't responded to other treatments. The goal is to see if the drug is safe and effective.

Who is the study for?

Adults with advanced GIST that can't be surgically removed or has spread, who have tried other treatments like imatinib and possibly others without success. They should be in good physical condition (ECOG 0-1) and have recovered from previous treatment side effects.

What is being tested?

The trial is testing IDRX-42's safety, how well it's tolerated by the body, its pharmacokinetics (how the drug moves through the body), and its initial effectiveness in treating GIST.

What are the potential side effects?

Since this is a first-in-human study for IDRX-42, specific side effects are not yet known but will likely include typical cancer drug reactions such as nausea, fatigue, risk of infection, and potential organ-related toxicities.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 5 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 5 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 5 years for reporting.

Treatment Details

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

5Treatment groups

Experimental Treatment

Group I: Dose Escalation (Phase I)Experimental Treatment1 Intervention

Participants should have advanced (metastatic and/or surgically unresectable) GIST, following failure of at least prior imatinib therapy due to progression of GIST.

Group II: (Phase 1b): Cohort 4Experimental Treatment1 Intervention

Participants with GIST progression who meet the same criteria as Cohort 2 (third line or greater TKI therapy) and have had prior treatment with investigational agents NB003 or THE-630 or a line of therapy of bezuclastinib plus sunitinib combination.

Group III: (Phase 1b): Cohort 3 - Participants with GIST who are treatment naïveExperimental Treatment1 Intervention

Participants with metastatic and/or surgically unresectable GIST who are treatment naïve (first line therapy) and refused or are ineligible for other standard of care (SOC) therapies.

Group IV: (Phase 1b): Cohort 2 - Participants with GIST progression after 2 or more lines of TKI therapyExperimental Treatment1 Intervention

Participants with metastatic and/or surgically unresectable GIST following progression EITHER after sequential imatinib then sunitinib (third-line therapy setting) OR after imatinib, sunitinib, and then an additional TKI agent (i.e., regorafenib or ripretinib) (fourth-line therapy setting) OR after imatinib, sunitinib, regorafenib, and ripretinib (5th line or greater therapy).

Group V: (Phase 1b) Cohort 1 - Participants with GIST progression after first-line imatinib therapyExperimental Treatment1 Intervention

Participants with advanced GIST who have had GIST progression after first-line imatinib only (second line therapy setting) and refused or are ineligible for other standard of care (SOC) therapies.

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Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

The most common treatments for Gastrointestinal Stromal Tumor (GIST) are tyrosine kinase inhibitors (TKIs) such as imatinib, sunitinib, and regorafenib. These drugs work by targeting specific mutations in the KIT and PDGFRA genes, which are responsible for the uncontrolled growth and survival of GIST cells. By inhibiting the activity of these mutated proteins, TKIs can effectively reduce tumor growth and spread. This targeted approach is crucial for GIST patients as it offers a more personalized treatment option, potentially leading to better outcomes and fewer side effects compared to traditional chemotherapy.

Pediatric and wild-type gastrointestinal stromal tumor: new therapeutic approaches.