New hope for rare tumor patients: AL102 drug trial launches

NCT ID NCT04871282

Disease control Sponsor: Immunome, Inc. Source: ClinicalTrials.gov ↗

First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This study tests a drug called AL102 (varegacestat) in people with desmoid tumors that are growing. Desmoid tumors are non-cancerous but can be painful and invasive. The trial has two parts: Part A checks safety and tumor changes, and Part B compares AL102 to a placebo to see if it delays tumor growth. About 198 adults with progressing desmoid tumors are taking part.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance: AL102 (varegacestat), a gamma-secretase inhibitor
What this could lead to: If successful, AL102 could offer a new treatment option to slow or stop the growth of desmoid tumors, potentially reducing the need for surgery or other therapies.
What could go wrong: This is a mid-stage trial with only 198 participants, so results may not apply to all patients. The drug may cause side effects or fail to show meaningful benefit over placebo.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 2/3

Runs two stages together: whether the treatment works, then large-scale confirmation.

Participants

198 people

The number who actually took part.

Started

Sep 2021

Expected to finish

Dec 2026

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

12 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria Part A: 1. At least 18 years of age (inclusive) at the time of signing the informed consent form (ICF). 2. Histologically confirmed desmoid tumor (aggressive fibromatosis) by local pathologist (prior to informed consent). 3. Disease progression, assessed locally by the investigator, defined as having at least one of the following: * Unidimensional growth of desmoid tumor(s) by ≥10%, using the sum of the largest diameters of target lesion(s), within 18 months of the screening MRI * Having desmoid tumor-related pain that is not adequately controlled with nonopioid medication 4. At least 1 measurable lesion amenable to volume measurements by MRI at screening 5. One of the following: * Treatment naïve subjects for whom, in the opinion of the investigator, the IP is deemed appropriate, OR * Recurrent/refractory disease following at least one line of therapy (including surgery, radiation, or systemic therapy) 6. Agrees to provide formalin-fixed paraffin embedded archival or fresh tumor tissue for re-confirmation of disease. 7. Must be able to swallow whole capsules with no GI condition affecting absorption; nasogastric or G-tube administration is not allowed. Inclusion Criteria Part B 1. ≥12 years of age (inclusive) and ≥ 40 kg at the time of signing the ICF. 2. Histologically confirmed desmoid tumor (aggressive fibromatosis) by local pathologist (prior to informed consent) that has progressed by ≥ 20% as measured by RECIST v1.1 within 12 months of the screening visit scan. 3. Evidence of measurable disease by CT/MRI scan. Measurable lesions are defined according to RECIST v1.1. 4. Subject and/or legally authorized representative (i.e. parent/guardian) must be capable of giving signed informed consent, which includes compliance with the requirements and restrictions listed in the ICF. 5. Minor subjects must be capable of giving written assent as appropriate per the applicable age (per local regulatory requirements). OLE Key Inclusion Criteria: 1\. One of the following: 1. Participated in Part A (including MRI at Week 16) and were still on study at time that Part B/OLE dose selection was made, OR 2. Participating in Part B and were noted to have radiographic progressive disease by BICR, OR 3. Are on study treatment (placebo or varegacestat) after completion of Part B Exclusion Criteria Parts A and B: 1. Diagnosed with a malignancy in the past 2 years with some exceptions. 2. Current or recent (within 2 months of IP administration) GI disease or disorders that increase the risk of diarrhea, such as inflammatory bowel disease and Crohn's disease. 3. Evidence of uncontrolled, active infection, requiring systemic anti-bacterial, anti-viral or anti-fungal therapy ≤7 days prior to administration of IP such as known active infection with hepatitis B, hepatitis C, or human immunodeficiency virus (HIV) at Screening. 4. Myocardial infarction within 6 months prior to enrollment, greater than Class 1 angina pectoris, or has New York Heart Association (NYHA) Class III or IV heart failure, symptomatic ventricular arrhythmias, sustained ventricular tachycardia, Torsade's de Pointes (TdP), the long QT syndrome, pacemaker dependence, or electrocardiographic evidence of acute ischemia. 5. Unstable or severe uncontrolled medical condition (e.g., unstable cardiac or pulmonary function or uncontrolled diabetes) or any important medical illness or abnormal laboratory finding that would, in the investigator's judgment, increase the risk to the subject associated with his or her participation in the study. 6. Pregnant or breastfeeding or expecting to conceive children within the projected duration of the study. 7. Eastern Cooperative Oncology Group (ECOG) performance status ≥2 8. Abnormal organ and marrow function at Screening defined as: 1. Neutrophils \<1000/mm3, 2. Platelet count \<100,000/mm3, 3. Hemoglobin \<9 g/dL, 4. Total bilirubin \>1.5x upper limit of normal (ULN) (except known Gilbert's syndrome), 5. Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) \>2.5x ULN, 6. Serum creatinine \> ULN and creatinine clearance (CrCl) \<60 mL/min (calculation of CrCl will be based on acceptable institution standard) 7. Uncontrolled triglyceride ≥Grade 2 elevations per common terminology criteria for adverse events (CTCAE) v5.0 (\>300 mg/dL or \>3.42 mmol/L). 9. ECG Exclusions 1. Mean QT interval corrected for heart rate using Fridericia's formula (QTcF) ≥450 msec. 2. QRS duration \> 110 ms 3. PR interval \> 240 ms 4. Marked ST-T wave abnormalities which would make it difficult to measure the QT interval 10. Any treatments for desmoid tumors within 4 weeks prior to first dose of investigational therapy; subject must have recovered from therapy related toxicity to \< CTCAE Grade 2 or clinical baseline. Therapy includes: 1. Locoregional tumor directed therapies such as major surgery, radiation, radiofrequency ablation, or cryosurgery 2. Systemic therapy including chemotherapy, biologic (anti-neoplastic agent, antibodies), TKIs (e.g., sorafenib, pazopanib, imatinib), hormonal therapy, or investigational therapy 11. Chronic NSAIDs for the treatment of desmoid tumors within 4 weeks of first dose of IP OLE Key Exclusion Criteria: 1. Unstable or severe uncontrolled medical condition (e.g., unstable cardiac or pulmonary function or uncontrolled diabetes) or any important medical illness, abnormal ECG or abnormal laboratory finding that would, in the investigator's judgment, increase the risk to the participant associated with his or her participation in the study. 2. Any participant with an ongoing TEAE (including abnormal laboratory results) from Part A or Part B that requires discontinuation from the study, will not be eligible to enter the open-label extension. 3. Breastfeeding or expecting to conceive children within the projected duration of the study.

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Conditions

The condition(s) this trial relates to.

desmoid tumor

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Desmoid Desmoid tumor

Contacts and locations

Locations

ASAN Medical Center

Seoul, 43-gil, South Korea
Addenbrooke's Hospital

Cambridge, CB2 0QQ, United Kingdom
Adelaide Cancer Centre

Kurralta Park, South Australia, 5037, Australia
Campus Bio-Medico University Hospital

Rome, 00128, Italy
Catalan Institute of Oncology (ICO)

Barcelona, 08908, Spain
Chris O'Brien Lifehouse

Camperdown, New South Wales, 2050, Australia
City of Hope

Duarte, California, 91010, United States
Cleveland Clinic

Cleveland, Ohio, 44195, United States
Columbia University Irving Medical Center

New York, New York, 10032, United States
Erasmus Medisch Centrum

Rotterdam, 3015 AA, Netherlands
Fox Chase Cancer Center

Philadelphia, Pennsylvania, 19111, United States
Fred Hutchinson Cancer Center

Seattle, Washington, 98109, United States
Hadassah University Hospital - Ein Kerem

Jerusalem, 9112001, Israel
Helios Klinikum Berlin-Buch

Berlin, 13125, Germany
Hospital Universitario 12 de Octubre

Madrid, 28041, Spain
Hospital Universitario Fundacion Jimenez Diaz

Madrid, 28040, Spain
Hospital Universitario Miguel Servet

Zaragoza, 50009, Spain
IRCCS Fondazione Istituto Nazionale dei Tumori

Milan, 20133, Italy
IRCCS Istituto Ortopedico Rizzoli

Bologna, 40136, Italy
Jefferson City Medical Group

Jefferson City, Missouri, 65109, United States
Jefferson University Hospital

Philadelphia, Pennsylvania, 19107, United States
Leiden University Medical Center

Leiden, Netherlands
Levine Cancer Institute

Charlotte, North Carolina, 28204, United States
MD Anderson Cancer Center

Houston, Texas, 77005, United States
Mannheim university medical center

Mannheim, 68167, Germany
Maria Sklodowska-Curie National Research Institute of Oncology

Warsaw, 00-001, Poland
Massachusetts General Hospital

Boston, Massachusetts, 02214, United States
Mayo Clinic

Pheonix, Arizona, 85054, United States
Mayo Clinic

Jacksonville, Florida, 32224, United States
Memorial Sloan Kettering Cancer Center

New York, New York, 10065, United States
NorthShore University Health System

Evanston, Illinois, 60201, United States
Ohio State University Wexner Medical Center

Columbus, Ohio, 43210, United States
Oncology Institute Barzilai Medical Center

Ashkelon, Israel
Oregon Health & Science University

Portland, Oregon, 97239, United States
Peter MacCallum Cancer Centre

Melbourne, Victoria, 3000, Australia
Princess Alexandra Hospital

Woolloongabba, Queensland, 4102, Australia
Rambam MC

Haifa, 3109601, Israel
Sarcoma Oncology Research Center

Santa Monica, California, 90403, United States
Severance Hospital, Yonsei University Health System

Seoul, 03722, South Korea
Stanford University Medical Center

Stanford, California, 94305, United States
Tel Aviv Sourasky Medical Center

Tel Aviv, 64239, Israel
The Christie NHS Foundation Trust

Manchester, M20 4BX, United Kingdom
The Netherlands Cancer Institute

Amsterdam, 1066CX, Netherlands
The Royal Marsden Hospital

London, SW3 6JJ, United Kingdom
UTSW Simmons Cancer Center

Dallas, Texas, 75235, United States
Universitair Ziekenhuis

Ghent, 9000, Belgium
Universitaire Ziekenhuizen Leuven

Leuven, Belgium
University of California at Los Angeles Hematology/Oncology

Santa Monica, California, 90404, United States
University of Michigan

Ann Arbor, Michigan, 48109, United States
University of Pittsburgh Medical Center, Hillman Cancer Center

Pittsburgh, Pennsylvania, 15232, United States
Vall d´Hebrón University Hospital

Barcelona, 08035, Spain
Washington University

St Louis, Missouri, 63110, United States
Western General Hospital

Edinburgh, Scotland, EH4 2XU, United Kingdom

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