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

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What is this study about?

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.

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 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 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 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 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 Study?

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 Study?

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

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

No sites found in this category

Other Sites

Site Name	City	Country
Universita’ Campus Bio-medico Di Roma	Rome	Italy
Universitaetsklinikum Mannheim GmbH	Mannheim	Germany
Hospital Universitario Fundacion Jimenez Diaz	Madrid	Spain
Netherlands Cancer Institute	Amsterdam	The Netherlands
Universitair Ziekenhuis Gent	Gent	Belgium
HELIOS Klinikum Berlin-Buch GmbH	Berlin	Germany
Hospital Universitario 12 De Octubre	Madrid	Spain
Fondazione I.R.C.C.S. Istituto Neurologico Besta	Milan	Italy
Narodowy Instytut Onkologii Im. Marii Sklodowskiej-Curie-Panstwowy Instytut Badawczy	Warsaw	Poland
Hsvfgkmb Vzwb dkgjasik	Barcelona	Spain
Ethwyyt Uwohcrhltylj Mookell Cwagzft Rguxfledy (mfwwkrm Mhk	Rotterdam	The Netherlands
Ioqhbjvb Cvladf Dsnfenoxmbjhjqygl	L'hospitalet De Llobregat	Spain

Trial status

Country	Status	Recruitment Start
Belgium	Not recruiting	15.03.2021
Germany	Not recruiting	15.03.2021
Italy	Not recruiting	15.03.2021
Poland	Not recruiting	15.03.2021
Spain	Not recruiting	15.03.2021
The Netherlands	Not recruiting	15.03.2021

Trial locations

Investigated Drugs:

Varegacestat

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.

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 ID:

2024-515909-26-00

Protocol code:

AL-DES-01

Trial Phase:

Therapeutic use (Phase IV)

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

What is this study about?

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