Adenoid Cystic CarcinomaPhase 1NCT04973683
AL101 Before Surgery for the Treatment of Notch Activated Adenoid Cystic Cancer
A Phase 1 study testing AL101 and Therapeutic Conventional Surgery in people with Adenoid Cystic Carcinoma and Metastatic Adenoid Cystic Carcinoma, run by M.D. Anderson Cancer Center at 1 site in United States.
Trial summary
This phase Ib trial studies the side effects and possible benefits of AL101 before surgery in treating patients with notch activated adenoid cystic cancer. AL101 may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Adenoid Cystic Carcinoma, Metastatic Adenoid Cystic Carcinoma
- What is being tested?
- AL101 and Therapeutic Conventional Surgery
- Type of treatment
- Injection or infusion, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 13 participants
- Where
- 1 siteUnited States
- Study dates
- 30 Sep 2021to 16 Jun 2026
- Phase
- Phase 1
- Are there results?
- None posted in the registry
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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Capable of giving signed informed consent which includes compliance with the requirements and restrictions listed in the informed consent and in this protocol
- Age >= 18 years old
- Histologically/cytological confirmed adenoid cystic carcinoma (ACC) of any primary site
- Evidence of NOTCH1 pathway activation as determined by NICD1 IHC nuclear staining in >= 70% of tumor cells
- Patients must have surgically resectable disease, either with a curative intent or for local control in the setting of metastatic disease, in the opinion of the treating physician
- Patients must be willing to undergo baseline biopsy to obtain tumor material
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- Disease must be measurable by RECIST 1.1
- Performance status Eastern Cooperative Oncology Group (ECOG) 0 or 1
- Neutrophils < 1500/mm^3
- Platelet count < 100,000/mm^3
- Hemoglobin < 9 g/dL
- Total bilirubin > 1.5 upper limit of normal (ULN) (except known Gilbert's syndrome)
- Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) > 2.5 of upper limit of normality (ULN) OR > 5 ULN for patients with liver metastases
- Creatinine clearance < 40 mL/min (Calculation of creatinine clearance [CrCl] will be based on acceptable institution standard)
- Female patients with reproductive potential must practice two effective contraceptive measures for the duration of study drug therapy and for at least 90 days after completion of AL101 therapy. The two birth control methods can be either two barrier methods or a barrier method plus a hormonal method to prevent pregnancy. The following are considered adequate barrier methods of contraception: diaphragm, condom, copper intrauterine device, sponge, or spermicide. Appropriate hormonal contraceptives will include any registered and marketed contraceptive agent that contains an estrogen and/or a progestational agent (including oral, subcutaneous, intrauterine, or intramuscular agents)
- Male patients who are sexually active with women with reproductive potential must agree to use contraception for the duration of treatment and for at least 90 days after completion of AL101 therapy
Who cannot join
- Prior radiotherapy, chemotherapy, or biologic therapy is allowed in patients with loco-regional recurrent disease, if administered at least 4 weeks prior to study enrollment
- Prior treatment with gamma-secretase inhibitor
- History of previous malignancy other than malignancy treated with curative intent and with no evidence of active disease >= 2 years before the first dose of study drug and of low potential risk for recurrence. Patients with the following diagnoses represents an exception and may enroll:
- Non-melanoma skin cancers with no current evidence of disease
- Melanoma in situ with no current evidence of disease
- Localized cancer of the prostate with prostate-specific antigen of < 0.1 ng/mL
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- Treated or localized well-differentiated thyroid cancer
- Treated cervical carcinoma in situ
- Treated ductal/lobular carcinoma in situ of the breast
- Current or recent (within 2 months of investigational product administration) gastrointestinal disease such as disorders that increase the risk of diarrhea (e.g.: inflammatory bowel disease). Non-chronic conditions (e.g., infectious diarrhea) that are completely resolved for at least 2 weeks prior to starting investigational product are not exclusionary
- Evidence of clinically significant bleeding diathesis or coagulopathy (in the absence of therapeutic anticoagulation)
- Evidence of uncontrolled, active infection, requiring systemic anti-bacterial, anti-viral or anti-fungal therapy =< 7 days prior to administration of investigational product such as known active infection with hepatitis B and hepatitis C (HCV) at Screening
- Symptomatic central nervous system (CNS) metastases. Patients with asymptomatic CNS metastases as well as those with previously treated CNS metastases are eligible for enrollment in the study if at least four weeks has elapsed since last whole brain radiation treatment or at least two weeks has elapsed since last focal radiation treatment and the patient is deemed clinically stable by the investigator
- 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 patient associated with his or her participation in the study
- Mean QT interval corrected for heart rate using Fridericia's formula (QTcF) >= 480 msec
- Female subjects who are pregnant or breast-feeding
- Hypersensitivity and/or history of allergy to the investigational product excipients
Trial locations and contacts
1 site in 1 country.
About this study
Official title: AL101 Prior to Standard-of-Care Surgery in Patients With Notch Activated Adenoid Cystic Carcinoma (ACC)
This phase Ib trial studies the side effects and possible benefits of AL101 before surgery in treating patients with notch activated adenoid cystic cancer. AL101 may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. Giving AL101 before surgery may help to control adenoid cystic cancer that has a NOTCH pathway activation.
Read the detailed description
PRIMARY OBJECTIVES:
- To evaluate the safety and feasibility of AL101 administered weekly for 6 to 8 weeks in the preoperative setting using National Cancer Institute (NCI)-Common Terminology Criteria for Adverse Events (CTCAE) version 5.0.
II. To determine the differences between NICD1 levels by immunohistochemistry (IHC) in the post-treatment surgical specimens as compared to baseline in patients treated with AL101.
SECONDARY OBJECTIVES:
- To evaluate the objective response rate (ORR) to AL101 by Response Evaluation Criteria in Solid Tumors (RECIST) 1.1 at 6 to 8 weeks.
II. To assess percentage of patients undergoing the initially proposed surgery. III. To assess percentage of viable tumor cells in the surgical specimen (pathologic response) following AL101 treatment.
EXPLORATORY OBJECTIVE:
- To evaluate pre- and post- treatment tumor and blood biomarkers and correlate with clinical and pathologic response and toxicity.
OUTLINE:
Patients receive AL101 intravenously (IV) over 60 minutes once weekly (QW) for 6-8 weeks in the absence of disease progression or unacceptable toxicity. Within 24-72 hours after the last infusion of AL101, patients undergo surgery per standard of care. Patients may continue AL101 after surgery at the discretion of the study doctor.
After completion of study, patients are followed up within 6 weeks after surgery or within 30 days after last dose of AL101, and then every 6 months thereafter.
- Being tested
- AL101, Therapeutic Conventional Surgery
- Medical subject headings
- Carcinoma, Adenoid Cystic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AL101Drug
Also called AL 101, AL-101, BM-0018, BM0018
Given IV
Therapeutic Conventional SurgeryProcedure or surgery
Undergo standard of care surgery
| Study group | Role | What they receive |
|---|---|---|
| Treatment (AL101) | Experimental | Patients receive AL101 IV over 60 minutes QW for 6-8 weeks in the absence of disease progression or unacceptable toxicity. Within 24-72 hours after the last infusion of AL101, patients undergo surgery per standard of care. Patients may continue AL101 after surgery at the discretion of the study doctor. Receives: Drug: AL101; Procedure: Therapeutic Conventional Surgery |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of adverse events
Adverse events will be graded by National Cancer Institute Common Terminology Criteria for Adverse Events version 5.0. Frequency tables will summarize toxicity data for all patients. Logistic regression may be utilized to assess the effect of patient prognostic factors on the response and toxicity.
Measured: Up to 2 years
Changes in NICD1 levels
Evaluated by immunohistochemistry in the post-treatment surgical specimen (at 6 to 8 weeks) as compared to baseline (pre-treatment) in patients treated with AL101
Measured: Baseline and after surgery
Secondary outcomes (1)
Overall response rate
Evaluated per Response Evaluation Criteria in Solid Tumors. Logistic regression may be utilized to assess the effect of patient prognostic factors on the response and toxicity.
Measured: At 6-8 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Jul 2021
- Study start30 Sep 2021
- Primary completion16 Jun 2026
- Study completion16 Jun 2026
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Adenoid Cystic CarcinomaStudies of the same condition, open and under way.
- More trials run by M.D. Anderson Cancer CenterThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
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 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 13 participants (actual)
Sponsor and investigators
- Lead sponsor
- M.D. Anderson Cancer Center
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Renata Ferrarotto, Principal investigator, M.D. Anderson Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04973683
- Sponsor's protocol ID
- 2021-0296
- Other IDs
- NCI-2021-05878, 2021-0296
- Status last verified
- Jun 2026
- First submitted
- 18 Jun 2021
- Last updated
- 24 Jun 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 24 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- AL101 Prior to Standard-of-Care Surgery in Patients With Notch Activated Adenoid Cystic Carcinoma (ACC)
- ClinicalTrials.gov ID
- NCT04973683
- Acronym / sponsor ID
- 2021-0296
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 13 participants (actual)
- Lead sponsor
- M.D. Anderson Cancer CenterUniversity, hospital or other
- Study start
- 30 Sep 2021 (actual)
- Primary completion
- 16 Jun 2026 (actual)
- Study completion
- 16 Jun 2026 (actual)
- First posted
- 22 Jul 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This phase Ib trial studies the side effects and possible benefits of AL101 before surgery in treating patients with notch activated adenoid cystic cancer. AL101 may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. Giving AL101 before surgery may help to control adenoid cystic cancer that has a NOTCH pathway activation.
Detailed description
PRIMARY OBJECTIVES:
- To evaluate the safety and feasibility of AL101 administered weekly for 6 to 8 weeks in the preoperative setting using National Cancer Institute (NCI)-Common Terminology Criteria for Adverse Events (CTCAE) version 5.0.
II. To determine the differences between NICD1 levels by immunohistochemistry (IHC) in the post-treatment surgical specimens as compared to baseline in patients treated with AL101.
SECONDARY OBJECTIVES:
- To evaluate the objective response rate (ORR) to AL101 by Response Evaluation Criteria in Solid Tumors (RECIST) 1.1 at 6 to 8 weeks.
II. To assess percentage of patients undergoing the initially proposed surgery. III. To assess percentage of viable tumor cells in the surgical specimen (pathologic response) following AL101 treatment.
EXPLORATORY OBJECTIVE:
- To evaluate pre- and post- treatment tumor and blood biomarkers and correlate with clinical and pathologic response and toxicity.
OUTLINE:
Patients receive AL101 intravenously (IV) over 60 minutes once weekly (QW) for 6-8 weeks in the absence of disease progression or unacceptable toxicity. Within 24-72 hours after the last infusion of AL101, patients undergo surgery per standard of care. Patients may continue AL101 after surgery at the discretion of the study doctor.
After completion of study, patients are followed up within 6 weeks after surgery or within 30 days after last dose of AL101, and then every 6 months thereafter.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Treatment (AL101) | Experimental | Drug: AL101, Procedure: Therapeutic Conventional SurgeryPatients receive AL101 IV over 60 minutes QW for 6-8 weeks in the absence of disease progression or unacceptable toxicity. Within 24-72 hours after the last infusion of AL101, patients undergo surgery per standard of care. Patients may continue AL101 after surgery at the discretion of the study doctor. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (16 listed)
- Capable of giving signed informed consent which includes compliance with the requirements and restrictions listed in the informed consent and in this protocol
- Age >= 18 years old
- Histologically/cytological confirmed adenoid cystic carcinoma (ACC) of any primary site
- Evidence of NOTCH1 pathway activation as determined by NICD1 IHC nuclear staining in >= 70% of tumor cells
- Patients must have surgically resectable disease, either with a curative intent or for local control in the setting of metastatic disease, in the opinion of the treating physician
Key exclusion criteria (17 listed)
- Prior radiotherapy, chemotherapy, or biologic therapy is allowed in patients with loco-regional recurrent disease, if administered at least 4 weeks prior to study enrollment
- Prior treatment with gamma-secretase inhibitor
- History of previous malignancy other than malignancy treated with curative intent and with no evidence of active disease >= 2 years before the first dose of study drug and of low potential risk for recurrence. Patients with the following diagnoses represents an exception and may enroll:
- Current or recent (within 2 months of investigational product administration) gastrointestinal disease such as disorders that increase the risk of diarrhea (e.g.: inflammatory bowel disease). Non-chronic conditions (e.g., infectious diarrhea) that are completely resolved for at least 2 weeks prior to starting investigational product are not exclusionary
- Evidence of clinically significant bleeding diathesis or coagulopathy (in the absence of therapeutic anticoagulation)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| AL101Also: AL 101, AL-101, BM-0018, BM0018Given IV | Drug | Treatment (AL101) |
| Therapeutic Conventional SurgeryUndergo standard of care surgery | Procedure or surgery | Treatment (AL101) |
Primary outcome
Incidence of adverse events
Time frame: Up to 2 years
Adverse events will be graded by National Cancer Institute Common Terminology Criteria for Adverse Events version 5.0. Frequency tables will summarize toxicity data for all patients. Logistic regression may be utilized to assess the effect of patient prognostic factors on the response and toxicity.
Changes in NICD1 levels
Time frame: Baseline and after surgery
Evaluated by immunohistochemistry in the post-treatment surgical specimen (at 6 to 8 weeks) as compared to baseline (pre-treatment) in patients treated with AL101
1 secondary outcome
Overall response rate
Time frame: At 6-8 weeks
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Jun 2026.
- MD Anderson Cancer Center WebsiteLink provided by the sponsor.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Renata Ferrarotto principal investigator
M.D. Anderson Cancer Center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing AL101 and Therapeutic Conventional Surgery in people with Adenoid Cystic Carcinoma and Metastatic Adenoid Cystic Carcinoma, run by M.D. Anderson Cancer Center at 1 site in United States.
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 16 inclusion and 17 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: finished, as of the sponsor's last update on 24 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 1 site in United States. 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. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Incidence of adverse events (time frame: Up to 2 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?
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 24 Jun 2026.