Skip to content
Clinical Trials – home

Rare diseases

Investigational molecules

Locations

Our offerNewsroom

Not recruitingRare disease

Study of varegacestat (AL102) in patients with progressing desmoid tumors

Verified siteInvestigational
Clinical Trials Concierge

Prefer not to search? Our Concierge searches the trials for you.

What is this trial about?

A plain-language summary of the goals, design and what participants do

This study focuses on patients with desmoid tumors, which are rare growths that can develop in soft tissues. The research evaluates a new medication called AL102 (also known as varegacestat) in people whose tumors are getting worse. The medication comes in the form of capsules that are taken by mouth.

The research is divided into different parts. In Part A, researchers study how safe the medication is and how well patients tolerate it. In Part B, they examine how effective the medication is at stopping tumor growth. Some patients will receive AL102, while others will receive a placebo. The study also includes an extended treatment period where all participants can receive the active medication.

Throughout the study, doctors will monitor the size of tumors using medical imaging. They will also track any changes in symptoms and how patients feel while taking the medication. The medication is being tested at different dose levels to find the most effective and safe amount for treating desmoid tumors.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Initial assessment

    Your eligibility for the study will be confirmed through medical examination and review of your desmoid tumor diagnosis

    The tumor must show measurable growth within the past 12 months, confirmed by CT or MRI scan

    You must be able to swallow capsules whole, as other methods of taking the medication are not permitted

  2. Step 2

    Study assignment

    You will be assigned to either receive AL102 (varegacestat) capsules or placebo capsules

    The medication or placebo will be taken by mouth

    A tissue sample from your tumor will be required for study purposes

  3. Step 3

    Treatment period

    Regular assessments will monitor your tumor using imaging scans

    Your pain levels will be evaluated, particularly during the first 12 weeks

    The study team will track any side effects you may experience

    Your quality of life will be assessed using specific questionnaires

  4. Step 4

    Extended treatment option

    If you meet specific criteria, you may be eligible to continue treatment in the Open Label Extension (OLE) phase

    During the OLE phase, all participants receive the active medication (not placebo)

    Continued monitoring of tumor progression and side effects will occur

  5. Step 5

    Study completion

    The study is expected to continue until September 2026

    Your participation may end earlier if your tumor shows progression or if you experience significant side effects

    Final assessments will evaluate the medication's effect on your tumor and overall health status

Who can join the trial?

14 criteria

  • Must be at least 18 years old for Part A of the study, or at least 12 years old and weigh at least 40 kg for Part B
  • Must have a confirmed diagnosis of desmoid tumor verified by a pathologist
  • For Part B: Must show evidence that the tumor has grown by at least 20% or developed new tumors within the last 12 months
  • Must have tumors that can be measured by CT scan or MRI
  • Must meet one of these conditions:
    • Have previously received treatment (surgery, radiation, or other therapy) that didn't work
    • Be newly diagnosed and not suitable for surgery or radiation treatment
    • Must have a tumor that is not immediately life-threatening
    • Must agree to provide a tumor tissue sample
    • Must be able to swallow whole capsules
    • Must not have digestive system conditions that affect medicine absorption (previous bowel surgery is acceptable)
    • Must be able to provide informed consent to participate in the study
    • For minors: must be able to provide written permission to participate, as required by local regulations
    • For Part A: Must have either:
      • Tumor growth of at least 10% within 18 months before screening
      • Tumor-related pain not controlled by non-opioid pain medication

Who cannot join the trial?

15 criteria

  • Previous treatment with gamma secretase inhibitors (medications that block certain proteins)
  • Prior treatment with systemic therapy (medications that travel through the bloodstream) within 4 weeks before starting the study
  • Presence of metastatic disease (cancer that has spread to other parts of the body)
  • Pregnant or breastfeeding women
  • Known allergies to the study medication or its components
  • Serious heart conditions or uncontrolled high blood pressure
  • Major surgery within 4 weeks before starting the study
  • Active infections requiring treatment
  • Significant liver or kidney problems
  • History of other cancers within the past 3 years (except for successfully treated skin cancer or cervical cancer)
  • Mental health conditions that could interfere with study participation
  • Use of medications that could interact with the study drug
  • Participation in another clinical trial within 30 days before this study
  • Immunocompromised condition (weakened immune system)
  • Unable to swallow oral medications
Clinical Trials Concierge

Prefer not to search? Our Concierge searches the trials for you.

Tell us about your condition – we search every trial in Europe and connect you with the right site.

We usually reply within a few days

Verified sites

All sites with verified contact details – recruitment status may not be available; ask directly

Trial locations

Where you can join this trial

Countries are shaded by recruitment status. Click a recruiting country to ask about joining there.

Not recruiting
Not finding your country?

Not sure what to do next?

Joining a clinical trial can seem overwhelming. We guide you step by step, so you know exactly what to expect and how we support you through the process.

See the full process and FAQ

Investigated drugs

Based on the provided source data, there is one medication involved in this clinical trial: AL102 is an investigational drug being studied for the treatment of desmoid tumors that are getting worse (progressing). This medication is being tested to see how safe it is and how well it works in patients whose tumors continue to grow. The drug is currently known by its code name as it is still in the research phase and hasn't been assigned a standard generic name yet.

What is already known about the treatment

AL102 - An investigational medication being studied in clinical trials for the treatment of progressing desmoid tumors, which are rare soft tissue tumors. The drug is currently undergoing Phase 2/3 clinical trials (RINGSIDE study) to evaluate its safety, tolerability, and effectiveness in patients with progressive disease. While the exact mechanism of action has not been fully disclosed in public sources, AL102 is being investigated as a targeted therapy specifically designed to address the underlying causes of desmoid tumor growth. The medication is being evaluated to determine its potential to stop or slow tumor progression in affected patients, with ongoing research to establish its optimal dosing and administration protocols.

Investigated diseases

Desmoid Tumor - A rare type of soft tissue growth that develops in the connective tissue beneath the skin or in deeper structures of the body. These tumors, also known as aggressive fibromatosis, are not cancerous but can grow and invade nearby tissues and organs. Desmoid tumors typically develop in the abdomen, shoulders, arms, legs, or chest wall. They can be slow-growing or aggressive, and their behavior can be unpredictable, with some tumors growing rapidly while others remain stable or even shrink on their own. These tumors most commonly affect young adults, particularly women of childbearing age.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IVTrial ID2024-515909-26-00Protocol codeAL-DES-01Estimated enrolment198 patientsSponsorImmunome Inc.

sourced from the EU Clinical Trials Register and site verification

Want to learn more about this trial or check if you can participate?

Clinical Trials Concierge

Prefer not to search? Our Concierge searches the trials for you.

Tell us about your condition – we search every trial in Europe and connect you with the right site.