What is the mechanism of action of this treatment?

Desmoid tumors are often driven by mutations that activate the Notch signaling pathway, which promotes cell proliferation and survival. AL102, a Gamma-Secretase Inhibitor, works by inhibiting this pathway, thereby reducing tumor growth and progression. This mechanism is crucial for patients with Desmoid Tumors as it targets the underlying molecular drivers of the disease, potentially leading to more effective and targeted treatments with fewer side effects compared to traditional therapies.

What side effects are associated with this type of intervention?

Common treatments for Desmoid Tumors, including Gamma-Secretase Inhibitors like AL102, often lead to side effects such as gastrointestinal issues (nausea, diarrhea), fatigue, skin reactions (rash), and hematologic abnormalities (anemia, thrombocytopenia). Other treatments, such as NSAIDs, tyrosine kinase inhibitors, and chemotherapy, may cause additional side effects like cardiovascular issues, immunosuppression, and further gastrointestinal disturbances.

What prior FDA approvals exist?

As of now, there are no specific FDA-approved treatments for Desmoid Tumors that target the Notch signaling pathway through Gamma-Secretase Inhibition, like AL102. However, treatments for Desmoid Tumors generally include surgery, radiation therapy, and systemic therapies such as nonsteroidal anti-inflammatory drugs (NSAIDs), anti-hormonal therapies, and chemotherapy. Investigational treatments, including targeted therapies and novel agents like AL102, are currently being studied to provide more effective options for patients with progressive Desmoid Tumors.

What other types of research exist?

Promising novel alternatives to AL102 for Desmoid Tumors patients in 2024 may include other targeted therapies such as tyrosine kinase inhibitors (e.g., sorafenib, pazopanib), beta-catenin inhibitors, or investigational agents in clinical trials that target specific molecular pathways involved in desmoid tumor growth. Consultation with a healthcare provider for the latest clinical trial options and personalized treatment plans is recommended.

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AL102 for Desmoid Tumors (RINGSIDE Trial)

Phase 2 & 3

Waitlist Available

Led By Mrinal Gounder, MD

Research Sponsored by Immunome, Inc.

Eligibility Criteria Checklist

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

Must have

At least 18 years of age (inclusive) at the time of signing the ICF

Must be able to swallow whole capsules with no GI condition affecting absorption

Must not have

ECOG performance status ≥2

Diagnosed with a malignancy in the past 2 years

Timeline

Screening 3 weeks

Treatment Varies

Follow Up approximately 2 years

Summary

This trial is testing a new drug called AL102 to see if it can help people with desmoid tumors that are getting worse. The study will find the best dose and then see if AL102 can stop or slow down the growth of these tumors.

Who is the study for?

This trial is for adults with desmoid tumors that are growing and causing pain not controlled by non-opioid medication. Participants must have at least one tumor measurable by MRI, be able to swallow capsules, and can be treatment-naïve or have tried other treatments without success.

What is being tested?

The study tests the effectiveness and safety of a drug called AL102 compared to a placebo in patients with progressive desmoid tumors. The goal is to see if AL102 can help control the growth of these tumors better than no active treatment.

What are the potential side effects?

While specific side effects for AL102 aren't listed here, common ones may include digestive issues, fatigue, skin reactions, liver changes, muscle or joint pain. Placebo pills usually don't cause side effects but taking them means missing out on potential benefits from active drugs.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I am 18 years old or older.

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I can swallow pills and don't have stomach issues affecting drug absorption.

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My tumor has been confirmed as a desmoid tumor by a pathologist.

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My tumor has grown by 10% or more recently, or my tumor pain isn't controlled by regular painkillers.

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I have a tumor that can be measured with an MRI.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I need assistance with daily activities due to my health condition.

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I was diagnosed with cancer in the last 2 years.

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I have a history of serious heart conditions.

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I do not have any ongoing infections that aren't under control.

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I do not have any severe or uncontrolled health issues.

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My blood tests show abnormal organ or bone marrow function.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ approximately 2 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~approximately 2 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and approximately 2 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

Progression free survival

Secondary study objectives

Duration of response

Overall response rate

Patient reported outcome

Trial Design

6Treatment groups

Experimental Treatment

Placebo Group

Group I: Part B AL102Experimental Treatment1 Intervention

AL102, recommended dose regimen from Part A, 1.2 mg daily

Group II: Part A Main Study 4 mg IntermittentExperimental Treatment1 Intervention

AL102 4 mg

Group III: Part A Main Study 2 mg IntermittentExperimental Treatment1 Intervention

AL102 2 mg

Group IV: Part A Main Study 1.2 mg dailyExperimental Treatment1 Intervention

AL102 1.2 mg

Group V: Open Label ExtensionExperimental Treatment1 Intervention

AL102, recommended dose regimen from Part A, 1.2 mg daily

Group VI: Part B PlaceboPlacebo Group1 Intervention

Placebo to match recommended dose regimen from Part A

0 / 11Eligibility criteria met

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

Desmoid tumors are often driven by mutations that activate the Notch signaling pathway, which promotes cell proliferation and survival. AL102, a Gamma-Secretase Inhibitor, works by inhibiting this pathway, thereby reducing tumor growth and progression. This mechanism is crucial for patients with Desmoid Tumors as it targets the underlying molecular drivers of the disease, potentially leading to more effective and targeted treatments with fewer side effects compared to traditional therapies.

The proteins DLK1 and DLK2 modulate NOTCH1-dependent proliferation and oncogenic potential of human SK-MEL-2 melanoma cells.Dual inhibition of VEGFR and EGFR signaling reduces the incidence and size of intestinal adenomas in Apc(Min/+) mice.