AzTrials
AzTrials

DesmoidPhase 2/3RINGSIDENCT04871282

A Study of AL102 in Patients With Progressing Desmoid Tumors

A Phase 2/3 study testing AL102 in people with Desmoid and Desmoid Tumor, run by Immunome, Inc. at 53 sites in 11 countries.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Official record
Search all trials

Trial summary

The current study is designed to evaluate the efficacy and safety of AL102 in patients with progressive desmoid tumors. Read more about this study

Who is it for?
Ages 12 years and older
Conditions the study is for
Desmoid, Desmoid Tumor
What is being tested?
AL102
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
198 participants
Where
53 sitesin 11 countries
Study dates
1 Sep 2021to Dec 2026
Phase
Phase 2/3
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 12 years and older
  • All sexes
  • No healthy volunteers
  • Child, Adult, Older adult

Who may be able to join

  • At least 18 years of age (inclusive) at the time of signing the informed consent form (ICF).
  • Histologically confirmed desmoid tumor (aggressive fibromatosis) by local pathologist (prior to informed consent).
  • 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
  • At least 1 measurable lesion amenable to volume measurements by MRI at screening
Show all 21Show fewer
  • 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)
  • Agrees to provide formalin-fixed paraffin embedded archival or fresh tumor tissue for re-confirmation of disease.
  • Must be able to swallow whole capsules with no GI condition affecting absorption; nasogastric or G-tube administration is not allowed.
  • ≥12 years of age (inclusive) and ≥ 40 kg at the time of signing the ICF.
  • 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.
  • Evidence of measurable disease by CT/MRI scan. Measurable lesions are defined according to RECIST v1.1.
  • 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.
  • 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:
  • 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
  • Participating in Part B and were noted to have radiographic progressive disease by BICR, OR
  • Are on study treatment (placebo or varegacestat) after completion of Part B

Who cannot join

  • Diagnosed with a malignancy in the past 2 years with some exceptions.
  • 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.
  • 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.
  • 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.
  • 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.
  • Pregnant or breastfeeding or expecting to conceive children within the projected duration of the study.
Show all 28Show fewer
  • Eastern Cooperative Oncology Group (ECOG) performance status ≥2
  • Abnormal organ and marrow function at Screening defined as:
  • Neutrophils <1000/mm3,
  • Platelet count <100,000/mm3,
  • Hemoglobin <9 g/dL,
  • Total bilirubin >1.5x upper limit of normal (ULN) (except known Gilbert's syndrome),
  • Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) >2.5x ULN,
  • Serum creatinine > ULN and creatinine clearance (CrCl) <60 mL/min (calculation of CrCl will be based on acceptable institution standard)
  • Uncontrolled triglyceride ≥Grade 2 elevations per common terminology criteria for adverse events (CTCAE) v5.0 (>300 mg/dL or >3.42 mmol/L).
  • ECG Exclusions
  • Mean QT interval corrected for heart rate using Fridericia's formula (QTcF) ≥450 msec.
  • QRS duration > 110 ms
  • PR interval > 240 ms
  • Marked ST-T wave abnormalities which would make it difficult to measure the QT interval
  • 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:
  • Locoregional tumor directed therapies such as major surgery, radiation, radiofrequency ablation, or cryosurgery
  • Systemic therapy including chemotherapy, biologic (anti-neoplastic agent, antibodies), TKIs (e.g., sorafenib, pazopanib, imatinib), hormonal therapy, or investigational therapy
  • Chronic NSAIDs for the treatment of desmoid tumors within 4 weeks of first dose of IP
  • OLE Key Exclusion Criteria:
  • 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.
  • 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.
  • Breastfeeding or expecting to conceive children within the projected duration of the study.

Trial locations and contacts

53 sites in 11 countries.

53 sites in 11 countries

United States23 sites

  • University of Michigan

    Ann Arbor, Michigan, 48109

  • Massachusetts General Hospital

    Boston, Massachusetts, 02214

  • Levine Cancer Institute

    Charlotte, North Carolina, 28204

  • Cleveland Clinic

    Cleveland, Ohio, 44195

  • Ohio State University Wexner Medical Center

    Columbus, Ohio, 43210

  • UTSW Simmons Cancer Center

    Dallas, Texas, 75235

  • City of Hope

    Duarte, California, 91010

  • NorthShore University Health System

    Evanston, Illinois, 60201

  • MD Anderson Cancer Center

    Houston, Texas, 77005

  • Mayo Clinic

    Jacksonville, Florida, 32224

  • Jefferson City Medical Group

    Jefferson City, Missouri, 65109

  • Columbia University Irving Medical Center

    New York, New York, 10032

  • Memorial Sloan Kettering Cancer Center

    New York, New York, 10065

  • Mayo Clinic

    Pheonix, Arizona, 85054

  • Jefferson University Hospital

    Philadelphia, Pennsylvania, 19107

  • Fox Chase Cancer Center

    Philadelphia, Pennsylvania, 19111

  • University of Pittsburgh Medical Center, Hillman Cancer Center

    Pittsburgh, Pennsylvania, 15232

  • Oregon Health & Science University

    Portland, Oregon, 97239

  • Sarcoma Oncology Research Center

    Santa Monica, California, 90403

  • University of California at Los Angeles Hematology/Oncology

    Santa Monica, California, 90404

  • Fred Hutchinson Cancer Center

    Seattle, Washington, 98109

  • Washington University

    St Louis, Missouri, 63110

  • Stanford University Medical Center

    Stanford, California, 94305

Other trials in United States

Spain5 sites

  • Vall d´Hebrón University Hospital

    Barcelona, 08035

  • Catalan Institute of Oncology (ICO)

    Barcelona, 08908

  • Hospital Universitario Fundacion Jimenez Diaz

    Madrid, 28040

  • Hospital Universitario 12 de Octubre

    Madrid, 28041

  • Hospital Universitario Miguel Servet

    Zaragoza, 50009

Other trials in Spain

Australia4 sites

  • Chris O'Brien Lifehouse

    Camperdown, New South Wales, 2050

  • Adelaide Cancer Centre

    Kurralta Park, South Australia, 5037

  • Peter MacCallum Cancer Centre

    Melbourne, Victoria, 3000

  • Princess Alexandra Hospital

    Woolloongabba, Queensland, 4102

Other trials in Australia

Israel4 sites

  • Oncology Institute Barzilai Medical Center

    Ashkelon

  • Rambam MC

    Haifa, 3109601

  • Hadassah University Hospital - Ein Kerem

    Jerusalem, 9112001

  • Tel Aviv Sourasky Medical Center

    Tel Aviv, 64239

Other trials in Israel

United Kingdom4 sites

  • Addenbrooke's Hospital

    Cambridge, CB2 0QQ

  • Western General Hospital

    Edinburgh, Scotland, EH4 2XU

  • The Royal Marsden Hospital

    London, SW3 6JJ

  • The Christie NHS Foundation Trust

    Manchester, M20 4BX

Other trials in United Kingdom

Italy3 sites

  • IRCCS Istituto Ortopedico Rizzoli

    Bologna, 40136

  • IRCCS Fondazione Istituto Nazionale dei Tumori

    Milan, 20133

  • Campus Bio-Medico University Hospital

    Rome, 00128

Other trials in Italy

Netherlands3 sites

  • The Netherlands Cancer Institute

    Amsterdam, 1066CX

  • Leiden University Medical Center

    Leiden

  • Erasmus Medisch Centrum

    Rotterdam, 3015 AA

Other trials in Netherlands

Belgium2 sites

  • Universitair Ziekenhuis

    Ghent, 9000

  • Universitaire Ziekenhuizen Leuven

    Leuven

Other trials in Belgium

Germany2 sites

  • Helios Klinikum Berlin-Buch

    Berlin, 13125

  • Mannheim university medical center

    Mannheim, 68167

Other trials in Germany

South Korea2 sites

  • Severance Hospital, Yonsei University Health System

    Seoul, 03722

  • ASAN Medical Center

    Seoul, 43-gil

Other trials in South Korea

Poland1 site

  • Maria Sklodowska-Curie National Research Institute of Oncology

    Warsaw, 00-001

Other trials in Poland

About this study

Official title: RINGSIDE: A Phase 2/3, Randomized, Multicenter Study to Evaluate AL102 in Patients With Progressing Desmoid Tumors

The current study is designed to evaluate the efficacy and safety of AL102 in patients with progressive desmoid tumors.

Read the detailed description

This is a Phase 2/3, randomized study in subjects with progressive desmoid tumors consisting of 2 parts. Phase2/Part A is an open-label, dose regimen finding study; Phase3/Part B is a double blind, placebo-controlled study and Open Label Extension utilizing the dose regimen selected in Phase2/Part A.

Being tested
AL102, Placebo
Keywords
RINGSIDE
Medical subject headings
Desmoid Tumors

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • AL102Drug

    Also called varegacestat

    AL102 is an inhibitor of gamma secretase-mediated Notch signaling.

  • PlaceboOther

    Placebo to match AL102

Study groups in NCT04871282
Study groupRoleWhat they receive
Part A Main Study 1.2 mg dailyExperimentalAL102 1.2 mg
Receives: Drug: AL102
Part A Main Study 2 mg IntermittentExperimentalAL102 2 mg
Receives: Drug: AL102
Part A Main Study 4 mg IntermittentExperimentalAL102 4 mg
Receives: Drug: AL102
Part B AL102ExperimentalAL102, recommended dose regimen from Part A, 1.2 mg daily
Receives: Drug: AL102
Part B PlaceboPlacebo comparatorPlacebo to match recommended dose regimen from Part A
Receives: Other: Placebo
Open Label ExtensionExperimentalAL102, recommended dose regimen from Part A, 1.2 mg daily
Receives: Drug: AL102

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Part A: Safety and Tolerability - Adverse Events

    Evaluation of the safety and tolerability of AL102 in subjects with progressing desmoid tumors as defined by as defined by the frequency and severity of treatment emergent adverse events (TEAEs) and serious adverse events (SAEs)

    Measured: Approximately 1.5 years

  2. Part B: Progression free survival (PFS)

    PFS as defined as the time from randomization until the date of assessment of progression as assessed by BICR based on Response Evaluation Criteria in Solid Tumors (RECIST) v1.1 or death by any cause

    Measured: Approximately 2 years

Secondary outcomes (16)

  1. Part A: Change in Tumor Volume

    Change from baseline to Week 16 in tumor volume as measured by centrally read magnetic resonance imaging (MRI) by Blinded Independent Central Review (BICR)

    Measured: Approximately 16 weeks

  2. Part B: Overall response rate (ORR)

    Defined as the proportion of subjects with confirmed ORR (complete response [CR] and partial response [PR]) by BICR based on RECIST v1.1.

    Measured: Approximately 2 years

  3. Part B: Change in Tumor Volume

    Change from baseline at Week 24 in estimated tumor volume measured by T2 weighted (T2W) MRI or CT by BICR

    Measured: Approximately 24 weeks

  4. Part B: Duration of response (DOR)

    Defined by the time from confirmed CR or PR (by BICR based on RECIST v1.1) until the earlier of the first documentation of disease progression or death from any cause

    Measured: Approximately 2 years

  5. Part B: PFS with inclusion of Clinical Progression as an event

    Defined as the time from randomization until the date of radiographic progression as assessed by BICR or clinical progression as assessed by the investigator or death by any cause

    Measured: Approximately 2 years

  6. Part B: Patient Reported Outcome (PRO)

    Change from baseline to Week 12 in the worst pain intensity (WPI) using GOunder/Desmoid Tumor Research Foundation (DTRF) DEsmoid Symptom Scale and Impact Scale (GODDESS) Desmoid Tumor Symptom Scale (DTSS)

    Measured: Approximately 12 weeks

  7. Part B: Safety and Tolerability - Adverse Events

    Evaluation of the safety and tolerability of AL102 in subjects with progressing desmoid tumors as defined by the frequency and severity of TEAEs and SAEs

    Measured: Approximately 2 years

  8. Part B: Safety and Tolerability - Time to Treatment Discontinuation

    Evaluation of the safety and tolerability of AL102 in subjects with progressing desmoid tumors as defined by the time to treatment discontinuation due to TEAE

    Measured: Approximately 2 years

  9. Open Label Extension (OLE): Safety and Tolerability

    Evaluation of the safety and tolerability of AL102 in subjects with progressing desmoid tumors as defined by the frequency and severity of TEAEs and SAEs

    Measured: Approximately 12 months

  10. OLE: PFS

    PFS as defined as the time to radiographic progression as assessed by BICR based on RECIST v1.1 or death by any cause

    Measured: Approximately 12 months

  11. OLE: Confirmed ORR

    Proportion of participants with confirmed ORR (CR and PR) by BICR based on RECIST v1.1

    Measured: Approximately 12 months

  12. OLE: DOR

    DOR as defined by the time from confirmed CR or PR by BICR based on RECIST v1.1 until the earlier of the first documentation of disease progression or death from any cause

    Measured: Approximately 12 months

  13. OLE: PFS

    PFS as defined as the time to radiographic progression as assessed by BICR based on RECIST v1.1 or clinical progression as assessed by the investigator or death by any cause

    Measured: Approximately 12 months

  14. OLE: PRO - GODDESS DTSS Total Symptom Score

    Change from baseline in quality of life (QoL) as determined by GODDESS DTSS Total Symptom Score

    Measured: Approximately 12 months

  15. OLE: PRO - GODDESS DTSS Physical Functioning Domain Score

    Change from baseline in QoL as determined by GODDESS DTSS Physical Functioning Domain Score

    Measured: Approximately 12 months

  16. OLE: PRO - WPI using GODDESS DTSS Item 1

    Change from baseline in QoL as determined by WPI using GODDESS DTSS Item 1

    Measured: Approximately 12 months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted4 May 2021
  2. Study start1 Sep 2021
  3. Primary completion3 Dec 2025
  4. Study completionDec 2026 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2/3
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
Quadruple blind
Who is masked
Participant, Care provider, Investigator, Outcomes assessor
Enrolment
198 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04871282
Sponsor's protocol ID
AL-DES-01
Other IDs
2024-515909-26-00
Acronym
RINGSIDE
Status last verified
Jun 2026
First submitted
23 Apr 2021
Last updated
8 Jun 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Phase 2/3 study testing AL102 in people with Desmoid and Desmoid Tumor, run by Immunome, Inc. at 53 sites in 11 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 21 inclusion and 28 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: under way, not looking for participants, as of the sponsor's last update on 8 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 53 sites in United States, Spain, Australia, Israel, United Kingdom and 6 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this combined phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Part A: Safety and Tolerability - Adverse Events (time frame: Approximately 1.5 years). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT04871282.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 8 Jun 2026.

AzTrials uses cookies for analytics and advertising. You can accept or reject them; the site works either way. Privacy Policy.

Free account

Log in to keep searching

You’ve used your 3 free searches. AzTrials is free: log in or create a free account to keep searching clinical trials and save the ones you want to follow.

Create a free accountLog in