192 Participants Needed

AL102 for Desmoid Tumors

(RINGSIDE Trial)

Recruiting at 58 trial locations
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Overseen ByYelena Lalazar, RN, MPH
Prior Safety DataThis treatment has passed at least one previous human trial

Trial Summary

What is the purpose of this trial?

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.

Will I have to stop taking my current medications?

The trial requires that you stop any treatments for desmoid tumors, including chronic NSAIDs, at least 4 weeks before starting the study medication. However, the protocol does not specify if you need to stop other medications, so it's best to discuss this with the study team.

What data supports the effectiveness of the drug AL102 for desmoid tumors?

A case report showed that a similar drug, AL101, which also targets the Notch pathway, led to significant tumor shrinkage in patients with desmoid tumors. Additionally, in a phase 1 study, AL102, which is structurally similar to AL101, also showed tumor shrinkage in a patient with a desmoid tumor.12345

How does the drug AL102 differ from other treatments for desmoid tumors?

AL102 is unique because it is an orally administered gamma secretase inhibitor that targets the Notch pathway, which is involved in the development of desmoid tumors. This approach is novel as there are currently no approved systemic therapies for desmoid tumors, and AL102 offers a potential new treatment option for tumors that are not suitable for surgery or have recurred after other treatments.12356

Research Team

MG

Mrinal Gounder, MD

Principal Investigator

MSKCC

Eligibility Criteria

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.

Inclusion Criteria

I am 18 years old or older.
I agree to give a sample of my tumor for testing.
I can swallow pills and don't have stomach issues affecting drug absorption.
See 4 more

Exclusion Criteria

Pregnant, breastfeeding, or expecting to conceive
I need assistance with daily activities due to my health condition.
Uncontrolled triglyceride elevations
See 9 more

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Phase 2/Part A - Dose Regimen Finding

Open-label study to determine the optimal dose regimen of AL102 in patients with desmoid tumors

Approximately 6 months

Phase 3/Part B - Double Blind

Double blind, placebo-controlled study to evaluate the efficacy and safety of AL102

Approximately 18 months

Open Label Extension

Participants may opt into continuation of treatment with AL102 long-term

Long-term

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

  • AL102
  • Placebo
Trial OverviewThe 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.
Participant Groups
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

Find a Clinic Near You

Who Is Running the Clinical Trial?

Immunome, Inc.

Lead Sponsor

Trials
7
Recruited
420+

Ayala Pharmaceuticals, Inc,

Lead Sponsor

Trials
4
Recruited
340+

References

Activity of the Gamma Secretase Inhibitor AL101 in Desmoid Tumors: A Case Report of 2 Adult Cases. [2021]
The impact of radiotherapy in the treatment of desmoid tumours. An international survey of 110 patients. A study of the Rare Cancer Network. [2022]
A Novel Method to Treat Progressive Desmoid Tumors Involving Neurovascular Bundles: A Retrospective Cohort Study. [2022]
Recent Advances in Desmoid Tumor Therapy. [2020]
Desmoid tumor: prognostic factors and outcome after surgery, radiation therapy, or combined surgery and radiation therapy. [2023]
The management of desmoid tumours: A joint global consensus-based guideline approach for adult and paediatric patients. [2021]