Trial Outcomes & Findings for Immune Modulation by Abemaciclib in Head and Neck Squamous Cell Carcinoma (HNSCC). (AIM Trial) (NCT NCT04169074)
NCT ID: NCT04169074
Last Updated: 2026-05-22
Results Overview
To evaluate the clinical activity of abemaciclib in patients with operable, HPV-negative HNSCC as measured by quantitative change in tumor size (∆T) following 10-21 (+7) days of neoadjuvant exposure. ∆T will be measured as a continuous variable, based upon the percent change in RECIST v1.1 metrics for index lesions. Index lesions will be evaluated using the new international criteria proposed by the Response Evaluation Criteria in Solid Tumor (RECIST) Committee.
TERMINATED
PHASE2
7 participants
This outcome measure will be assessed at baseline and post 10-21 (+7) days of neoadjuvant exposure to abemaciclib.
2026-05-22
Participant Flow
The recruitment period was 06/16/2021-08/29/2025. The first patient was enrolled 07/07/2021. The last patient was enrolled 12/02/2024. Patients were recruited from the investigators' current clinical practice at University of Arizona Cancer Center and George Washington Cancer Center. The study was closed prematurely due to a change in standard of care for this patient population that was FDA approved June 2025.
Participant milestones
| Measure |
Treatment With Abemaciclib
Treatment will consist of a single neoadjuvant cycle of 10-21 days (+7 days if needed due to surgical delay). Abemaciclib will be administered from days 1-21. Abemaciclib may be continued for an additional 7 days, or up to 28 days, for delays in planned surgery. Abemaciclib 200 mg PO twice daily on Days 1-21 (+7 days).
Abemaciclib: Treatment will consist of a single neoadjuvant cycle of 10-21 days (+7 days). Abemaciclib will be administered from days 1-21. Abemaciclib may be continued for an additional 7 days, or up to 28 days, for delays in planned surgery.
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|---|---|
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Overall Study
STARTED
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7
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|
Overall Study
COMPLETED
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4
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|
Overall Study
NOT COMPLETED
|
3
|
Reasons for withdrawal
| Measure |
Treatment With Abemaciclib
Treatment will consist of a single neoadjuvant cycle of 10-21 days (+7 days if needed due to surgical delay). Abemaciclib will be administered from days 1-21. Abemaciclib may be continued for an additional 7 days, or up to 28 days, for delays in planned surgery. Abemaciclib 200 mg PO twice daily on Days 1-21 (+7 days).
Abemaciclib: Treatment will consist of a single neoadjuvant cycle of 10-21 days (+7 days). Abemaciclib will be administered from days 1-21. Abemaciclib may be continued for an additional 7 days, or up to 28 days, for delays in planned surgery.
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|---|---|
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Overall Study
Adverse Event
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1
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Overall Study
Withdrawal by Subject
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2
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Baseline Characteristics
Immune Modulation by Abemaciclib in Head and Neck Squamous Cell Carcinoma (HNSCC). (AIM Trial)
Baseline characteristics by cohort
| Measure |
Treatment With Abemaciclib
n=7 Participants
Treatment will consist of a single neoadjuvant cycle of 10-21 days (+7 days if needed due to surgical delay). Abemaciclib will be administered from days 1-21. Abemaciclib may be continued for an additional 7 days, or up to 28 days, for delays in planned surgery. Abemaciclib 200 mg PO twice daily on Days 1-21 (+7 days).
Abemaciclib: Treatment will consist of a single neoadjuvant cycle of 10-21 days (+7 days). Abemaciclib will be administered from days 1-21. Abemaciclib may be continued for an additional 7 days, or up to 28 days, for delays in planned surgery.
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|---|---|
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Age, Continuous
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64 Years
STANDARD_DEVIATION 10 • n=2 Participants
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|
Sex: Female, Male
Female
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2 Participants
n=2 Participants
|
|
Sex: Female, Male
Male
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5 Participants
n=2 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
1 Participants
n=2 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
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6 Participants
n=2 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=2 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=2 Participants
|
|
Race (NIH/OMB)
Asian
|
1 Participants
n=2 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=2 Participants
|
|
Race (NIH/OMB)
Black or African American
|
1 Participants
n=2 Participants
|
|
Race (NIH/OMB)
White
|
5 Participants
n=2 Participants
|
|
Race (NIH/OMB)
More than one race
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0 Participants
n=2 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=2 Participants
|
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Primary site
Oral Cavity
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7 Participants
n=2 Participants
|
|
Primary site
Hypopharynx
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0 Participants
n=2 Participants
|
|
Primary site
larynx
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0 Participants
n=2 Participants
|
|
Primary site
p16 negative oropharynx
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0 Participants
n=2 Participants
|
|
p16 Status
p16 negative
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6 Participants
n=2 Participants
|
|
p16 Status
Unknown
|
1 Participants
n=2 Participants
|
|
Baseline Tumor Size
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3.5 cm
STANDARD_DEVIATION 1.6 • n=2 Participants
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Smoked >100 cigarettes in lifetime
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5 Participants
n=2 Participants
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|
Years smoked
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25 years
STANDARD_DEVIATION 13 • n=2 Participants
|
|
Clinical Stage
I
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1 Participants
n=2 Participants
|
|
Clinical Stage
III
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1 Participants
n=2 Participants
|
|
Clinical Stage
IVa
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5 Participants
n=2 Participants
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PRIMARY outcome
Timeframe: This outcome measure will be assessed at baseline and post 10-21 (+7) days of neoadjuvant exposure to abemaciclib.Population: 4 patients completed baseline and post treatment tumor measurements. Percent change in tumor size (∆T) from baseline to post-treatment were evaluated. Due to early termination, only 4 patients were evaluable; formal inference is limited and results should be interpreted descriptively.
To evaluate the clinical activity of abemaciclib in patients with operable, HPV-negative HNSCC as measured by quantitative change in tumor size (∆T) following 10-21 (+7) days of neoadjuvant exposure. ∆T will be measured as a continuous variable, based upon the percent change in RECIST v1.1 metrics for index lesions. Index lesions will be evaluated using the new international criteria proposed by the Response Evaluation Criteria in Solid Tumor (RECIST) Committee.
Outcome measures
| Measure |
Treatment With Abemaciclib
n=4 Participants
Treatment will consist of a single neoadjuvant cycle of 10-21 days (+7 days if needed due to surgical delay). Abemaciclib will be administered from days 1-21. Abemaciclib may be continued for an additional 7 days, or up to 28 days, for delays in planned surgery. Abemaciclib 200 mg PO twice daily on Days 1-21 (+7 days).
Abemaciclib: Treatment will consist of a single neoadjuvant cycle of 10-21 days (+7 days). Abemaciclib will be administered from days 1-21. Abemaciclib may be continued for an additional 7 days, or up to 28 days, for delays in planned surgery.
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|---|---|
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Measure Quantitative Change in Tumor Size to Assess the Clinical Activity of Abemaciclib.
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-0.01 % change
Standard Deviation 0.16
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SECONDARY outcome
Timeframe: Two monthsTo describe the safety and tolerability of neoadjuvant exposure to abemaciclib in accordance with NCI Common Terminology Criteria for Adverse Events (CTCAE) v.5.
Outcome measures
| Measure |
Treatment With Abemaciclib
n=7 Participants
Treatment will consist of a single neoadjuvant cycle of 10-21 days (+7 days if needed due to surgical delay). Abemaciclib will be administered from days 1-21. Abemaciclib may be continued for an additional 7 days, or up to 28 days, for delays in planned surgery. Abemaciclib 200 mg PO twice daily on Days 1-21 (+7 days).
Abemaciclib: Treatment will consist of a single neoadjuvant cycle of 10-21 days (+7 days). Abemaciclib will be administered from days 1-21. Abemaciclib may be continued for an additional 7 days, or up to 28 days, for delays in planned surgery.
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|---|---|
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Describe the Safety and Tolerability of Neoadjuvant Exposure to Abemaciclib in Accordance With NCI Common Terminology Criteria for Adverse Events (CTCAE) v.5.
Patients with any AE of grade ≥ 3
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2 Participants
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Describe the Safety and Tolerability of Neoadjuvant Exposure to Abemaciclib in Accordance With NCI Common Terminology Criteria for Adverse Events (CTCAE) v.5.
Patients with any serious AE
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2 Participants
|
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Describe the Safety and Tolerability of Neoadjuvant Exposure to Abemaciclib in Accordance With NCI Common Terminology Criteria for Adverse Events (CTCAE) v.5.
Patients with any AE possibly attributed to abemaciclib
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2 Participants
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OTHER_PRE_SPECIFIED outcome
Timeframe: Two monthsEvaluate baseline and pharmacodynamic biomarkers within the TME associated with ∆T, including IFN-ɣ gene expression signature in baseline and post-treatment tumor biopsies. The primary biomarker hypothesis is that abemaciclib will increase the proportion of tumors that are T-cell inflamed.
Outcome measures
Outcome data not reported
OTHER_PRE_SPECIFIED outcome
Timeframe: Two monthsEvaluate tumor-intrinsic genetic biomarkers associated with ∆T, including somatic genetic or epigenetic alterations in CCND1, CDKN2A, and TP53 utilizing tumor specimen obtained during planned oncologic head and neck cancer surgery.
Outcome measures
Outcome data not reported
OTHER_PRE_SPECIFIED outcome
Timeframe: Two monthsEvaluate baseline and pharmacodynamic biomarkers within the TME associated with ∆T, including The distribution of tumor-infiltrating immune cell subtypes and their activation status, including lymphocytes and myeloid-derived stem cells, as measured by nanostring.
Outcome measures
Outcome data not reported
OTHER_PRE_SPECIFIED outcome
Timeframe: Two monthsEvaluate baseline and pharmacodynamic biomarkers within the TME associated with ∆T, including The distribution of tumor-infiltrating immune cell subtypes and their activation status, including lymphocytes and myeloid-derived stem cells, as measured by immunohistochemistry (IHC).
Outcome measures
Outcome data not reported
OTHER_PRE_SPECIFIED outcome
Timeframe: Two monthsEvaluate baseline and pharmacodynamic biomarkers within the TME associated with ∆T, including The distribution of tumor-infiltrating immune cell subtypes and their activation status, including lymphocytes and myeloid-derived stem cells, as measured by flow cytometry.
Outcome measures
Outcome data not reported
OTHER_PRE_SPECIFIED outcome
Timeframe: Two monthsTo evaluate the distribution of peripheral immune cell subtypes and their activation status, and how this is altered by abemaciclib.
Outcome measures
Outcome data not reported
OTHER_PRE_SPECIFIED outcome
Timeframe: Two monthsTo evaluate serum Th1 and Th2 cytokine profiles, and how they are altered by abemaciclib.
Outcome measures
Outcome data not reported
OTHER_PRE_SPECIFIED outcome
Timeframe: Two monthsEvaluate tumor-intrinsic molecular mediators of response and resistance to abemaciclib in baseline and post-treatment tumor biopsies, including expression of CDKN2A (p16), CCND1 (cyclin D1), and retinoblastoma tumor suppressor protein (pRB) retinoblastoma.
Outcome measures
Outcome data not reported
OTHER_PRE_SPECIFIED outcome
Timeframe: Two monthsEvaluate the anti-proliferative activity of abemaciclib as measured by change in the proliferative index (∆Ki67) in pre- and post-treatment tumor specimens.
Outcome measures
Outcome data not reported
OTHER_PRE_SPECIFIED outcome
Timeframe: Two monthsTo describe features of the oral and intestinal microbiomes that are associated with the clinical activity of abemaciclib.
Outcome measures
Outcome data not reported
Adverse Events
Treatment With Abemaciclib
Serious adverse events
| Measure |
Treatment With Abemaciclib
n=7 participants at risk
Treatment will consist of a single neoadjuvant cycle of 10-21 days (+7 days if needed due to surgical delay). Abemaciclib will be administered from days 1-21. Abemaciclib may be continued for an additional 7 days, or up to 28 days, for delays in planned surgery. Abemaciclib 200 mg PO twice daily on Days 1-21 (+7 days).
Abemaciclib: Treatment will consist of a single neoadjuvant cycle of 10-21 days (+7 days). Abemaciclib will be administered from days 1-21. Abemaciclib may be continued for an additional 7 days, or up to 28 days, for delays in planned surgery.
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|---|---|
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Cardiac disorders
Myocardial Infarction
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14.3%
1/7 • Number of events 1 • Adverse event data was collected for each patient from the start of study drug to 4 weeks (+/- 1 week) after the last dose of study drug. For patients who completed the study this ranges from 10 days + 4 weeks (+/- 1 week) to 28 days + 4 weeks (+/1 week).
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Surgical and medical procedures
Surgical and medical procedures - Other, specify - flap graft failure
|
14.3%
1/7 • Number of events 1 • Adverse event data was collected for each patient from the start of study drug to 4 weeks (+/- 1 week) after the last dose of study drug. For patients who completed the study this ranges from 10 days + 4 weeks (+/- 1 week) to 28 days + 4 weeks (+/1 week).
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Vascular disorders
Thromboembolic event
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14.3%
1/7 • Number of events 1 • Adverse event data was collected for each patient from the start of study drug to 4 weeks (+/- 1 week) after the last dose of study drug. For patients who completed the study this ranges from 10 days + 4 weeks (+/- 1 week) to 28 days + 4 weeks (+/1 week).
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|
Infections and infestations
Skin infection
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14.3%
1/7 • Number of events 1 • Adverse event data was collected for each patient from the start of study drug to 4 weeks (+/- 1 week) after the last dose of study drug. For patients who completed the study this ranges from 10 days + 4 weeks (+/- 1 week) to 28 days + 4 weeks (+/1 week).
|
Other adverse events
| Measure |
Treatment With Abemaciclib
n=7 participants at risk
Treatment will consist of a single neoadjuvant cycle of 10-21 days (+7 days if needed due to surgical delay). Abemaciclib will be administered from days 1-21. Abemaciclib may be continued for an additional 7 days, or up to 28 days, for delays in planned surgery. Abemaciclib 200 mg PO twice daily on Days 1-21 (+7 days).
Abemaciclib: Treatment will consist of a single neoadjuvant cycle of 10-21 days (+7 days). Abemaciclib will be administered from days 1-21. Abemaciclib may be continued for an additional 7 days, or up to 28 days, for delays in planned surgery.
|
|---|---|
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Gastrointestinal disorders
Diarrhea
|
57.1%
4/7 • Number of events 4 • Adverse event data was collected for each patient from the start of study drug to 4 weeks (+/- 1 week) after the last dose of study drug. For patients who completed the study this ranges from 10 days + 4 weeks (+/- 1 week) to 28 days + 4 weeks (+/1 week).
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Gastrointestinal disorders
Oral Mucositis
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14.3%
1/7 • Number of events 1 • Adverse event data was collected for each patient from the start of study drug to 4 weeks (+/- 1 week) after the last dose of study drug. For patients who completed the study this ranges from 10 days + 4 weeks (+/- 1 week) to 28 days + 4 weeks (+/1 week).
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General disorders
Fatigue
|
14.3%
1/7 • Number of events 1 • Adverse event data was collected for each patient from the start of study drug to 4 weeks (+/- 1 week) after the last dose of study drug. For patients who completed the study this ranges from 10 days + 4 weeks (+/- 1 week) to 28 days + 4 weeks (+/1 week).
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Blood and lymphatic system disorders
Anemia
|
14.3%
1/7 • Number of events 1 • Adverse event data was collected for each patient from the start of study drug to 4 weeks (+/- 1 week) after the last dose of study drug. For patients who completed the study this ranges from 10 days + 4 weeks (+/- 1 week) to 28 days + 4 weeks (+/1 week).
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Metabolism and nutrition disorders
Anorexia
|
14.3%
1/7 • Number of events 1 • Adverse event data was collected for each patient from the start of study drug to 4 weeks (+/- 1 week) after the last dose of study drug. For patients who completed the study this ranges from 10 days + 4 weeks (+/- 1 week) to 28 days + 4 weeks (+/1 week).
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Gastrointestinal disorders
Dysphagia
|
14.3%
1/7 • Number of events 1 • Adverse event data was collected for each patient from the start of study drug to 4 weeks (+/- 1 week) after the last dose of study drug. For patients who completed the study this ranges from 10 days + 4 weeks (+/- 1 week) to 28 days + 4 weeks (+/1 week).
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Additional Information
Investigator Initiated Trials Manager
University of Arizona Cancer Center
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place