Trial Outcomes & Findings for A Study to Evaluate Abemaciclib in Advanced Biliary Tract Carcinoma (NCT NCT04003896)

Last Updated: 2024-10-08

Results Overview

overall response rate (ORR) is defined as the percentage of subjects with evidence of a confirmed complete response (CR) or partial response (PR) as per Response Evaluation Criteria In Solid Tumors (RECIST) Version 1.1.The scale of ORR ranges from 0% to 100%, where 0% indicates no subjects achieved CR or PR, and 100% indicates all evaluable subjects achieved CR or PR.

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

4 participants

Primary outcome timeframe

no longer than 7 months

Results posted on

2024-10-08

Participant Flow

This open-label pilot study aimed to evaluate the response and tolerability of abemaciclib (Verzenio) in patients with advanced biliary tract carcinoma. The primary purpose was to estimate the overall response rate in participants with locally advanced, unresectable, or metastatic biliary tract carcinoma who have failed first-line therapy.

Participant milestones

Participant milestones
Measure	Abemaciclib Abemaciclib will be given as a single oral agent A sample size of 10 subjects is determined to be minimally sufficient for these pilot study objectives. The starting dose will be 200 mg twice daily. Dosing will continue daily for 28 days, this being one cycle. There will be no protocol scheduled hiatus and daily dosing will be continuous unless there is unacceptable toxicity, disease progression, or death. Abemaciclib: Participant will take 200 mg capsules or tablets orally twice daily. The participant will be instructed to swallow abemaciclib as a whole tablet and not to chew, crush or split capsules or tablets before swallowing. Participants should not ingest abemaciclib tablets if broken, cracked, or otherwise not intact. Doses should be taken at a approximately the same time every day, twice daily. Capsules or tablets can be taken orally with or without food. If the participant vomits or misses a dose of abemaciclib, the participant will be instructed to take the next dose at its scheduled time. Participants will also be provided with a diary and instructed to keep a twice daily record of the times they have taken their medications and any other events such as vomiting, diarrhea, etc.
Overall Study STARTED	4
Overall Study COMPLETED	0
Overall Study NOT COMPLETED	4

Reasons for withdrawal

Reasons for withdrawal
Measure	Abemaciclib Abemaciclib will be given as a single oral agent A sample size of 10 subjects is determined to be minimally sufficient for these pilot study objectives. The starting dose will be 200 mg twice daily. Dosing will continue daily for 28 days, this being one cycle. There will be no protocol scheduled hiatus and daily dosing will be continuous unless there is unacceptable toxicity, disease progression, or death. Abemaciclib: Participant will take 200 mg capsules or tablets orally twice daily. The participant will be instructed to swallow abemaciclib as a whole tablet and not to chew, crush or split capsules or tablets before swallowing. Participants should not ingest abemaciclib tablets if broken, cracked, or otherwise not intact. Doses should be taken at a approximately the same time every day, twice daily. Capsules or tablets can be taken orally with or without food. If the participant vomits or misses a dose of abemaciclib, the participant will be instructed to take the next dose at its scheduled time. Participants will also be provided with a diary and instructed to keep a twice daily record of the times they have taken their medications and any other events such as vomiting, diarrhea, etc.
Overall Study Withdrawal by Subject	3
Overall Study Physician Decision	1

Baseline Characteristics

A Study to Evaluate Abemaciclib in Advanced Biliary Tract Carcinoma

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Abemaciclib n=4 tumor lesions Abemaciclib will be given as a single oral agent A sample size of 10 subjects is determined to be minimally sufficient for these pilot study objectives. The starting dose will be 200 mg twice daily. Dosing will continue daily for 28 days, this being one cycle. There will be no protocol scheduled hiatus and daily dosing will be continuous unless there is unacceptable toxicity, disease progression, or death. Abemaciclib: Participant will take 200 mg capsules or tablets orally twice daily. The participant will be instructed to swallow abemaciclib as a whole tablet and not to chew, crush or split capsules or tablets before swallowing. Participants should not ingest abemaciclib tablets if broken, cracked, or otherwise not intact. Doses should be taken at a approximately the same time every day, twice daily. Capsules or tablets can be taken orally with or without food. If the participant vomits or misses a dose of abemaciclib, the participant will be instructed to take the next dose at its scheduled time. Participants will also be provided with a diary and instructed to keep a twice daily record of the times they have taken their medications and any other events such as vomiting, diarrhea, etc.
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	1 Participants n=5 Participants
Age, Categorical >=65 years	3 Participants n=5 Participants
Age, Continuous	64 years STANDARD_DEVIATION 16.5 • n=5 Participants
Sex: Female, Male Female	3 Participants n=5 Participants
Sex: Female, Male Male	1 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	4 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	1 Participants n=5 Participants
Race (NIH/OMB) White	3 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Region of Enrollment United States	4 Participants n=5 Participants

PRIMARY outcome

Timeframe: no longer than 7 months

Population: No response data was collected as all participants were off treatment before the start of cycle 3, which is the earliest timepoint for ORR assessment.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: through study completion, an average of 2 years

Population: Only two subjects with follow-up data were analyzed.

the time interval from date of first dose of study drug to first documented disease progression or death from any cause, whichever occurs first. The PFS scale ranges from 0 days (for immediate progression or death) to the longest observed duration without progression or death.

Outcome measures

Outcome measures
Measure	Abemaciclib n=2 Participants Abemaciclib will be given as a single oral agent The starting dose will be 200 mg twice daily. Dosing will continue daily for 28 days, this being one cycle. There will be no protocol scheduled hiatus and daily dosing will be continuous unless there is unacceptable toxicity, disease progression, or death. Abemaciclib: Participant will take 200 mg capsules or tablets orally twice daily. The participant will be instructed to swallow abemaciclib as a whole tablet and not to chew, crush or split capsules or tablets before swallowing. Participants should not ingest abemaciclib tablets if broken, cracked, or otherwise not intact. Doses should be taken at a approximately the same time every day, twice daily. Capsules or tablets can be taken orally with or without food. If the participant vomits or misses a dose of abemaciclib, the participant will be instructed to take the next dose at its scheduled time. Participants will also be provided with a diary and instructed to keep a twice daily record of the times they have taken their medications and any other events such as vomiting, diarrhea, etc.
Progression-free Survival	110 days Standard Deviation 76

SECONDARY outcome

Timeframe: no longer than 7 months

Population: The number of subjects with CR, PR, or SD and the total number of subjects evaluable for response are detailed in the data tables.

Disease Control Rate (DCR) is defined as the proportion of subjects who achieve a Complete Response (CR), Partial Response (PR), or Stable Disease (SD) as determined by the Response Evaluation Criteria in Solid Tumors (RECIST) Version 1.1. The scale of DCR ranges from 0% to 100%, where 0% indicates no subjects achieved CR, PR, or SD, and 100% indicates all evaluable subjects achieved disease control (CR, PR, or SD). Subjects who do not have sufficient data to determine response status are excluded from the denominator.

Outcome measures

Outcome measures
Measure	Abemaciclib n=2 Participants Abemaciclib will be given as a single oral agent The starting dose will be 200 mg twice daily. Dosing will continue daily for 28 days, this being one cycle. There will be no protocol scheduled hiatus and daily dosing will be continuous unless there is unacceptable toxicity, disease progression, or death. Abemaciclib: Participant will take 200 mg capsules or tablets orally twice daily. The participant will be instructed to swallow abemaciclib as a whole tablet and not to chew, crush or split capsules or tablets before swallowing. Participants should not ingest abemaciclib tablets if broken, cracked, or otherwise not intact. Doses should be taken at a approximately the same time every day, twice daily. Capsules or tablets can be taken orally with or without food. If the participant vomits or misses a dose of abemaciclib, the participant will be instructed to take the next dose at its scheduled time. Participants will also be provided with a diary and instructed to keep a twice daily record of the times they have taken their medications and any other events such as vomiting, diarrhea, etc.
Disease Control Rate	1 Participants

SECONDARY outcome

Timeframe: up to 6 months

Population: Two out of two evaluable subjects were alive at 6 months. No data available for 12-month survival.

Overall survival rate (OSR) is is typically reported as a percentage (%), representing the proportion of subjects who are alive at a specific time point after receiving the first dose of the study drug. The scale of Overall Survival Rate (OSR) ranges from 0% to 100%, where 0% indicates that none of the study participants survived for the specified period after the first dose of the study drug, and 100% indicates that all participants are alive at that time point. A higher OSR percentage suggests a greater proportion of subjects who have survived, reflecting the potential effectiveness of the treatment under investigation.

Outcome measures

Outcome measures
Measure	Abemaciclib n=2 Participants Abemaciclib will be given as a single oral agent The starting dose will be 200 mg twice daily. Dosing will continue daily for 28 days, this being one cycle. There will be no protocol scheduled hiatus and daily dosing will be continuous unless there is unacceptable toxicity, disease progression, or death. Abemaciclib: Participant will take 200 mg capsules or tablets orally twice daily. The participant will be instructed to swallow abemaciclib as a whole tablet and not to chew, crush or split capsules or tablets before swallowing. Participants should not ingest abemaciclib tablets if broken, cracked, or otherwise not intact. Doses should be taken at a approximately the same time every day, twice daily. Capsules or tablets can be taken orally with or without food. If the participant vomits or misses a dose of abemaciclib, the participant will be instructed to take the next dose at its scheduled time. Participants will also be provided with a diary and instructed to keep a twice daily record of the times they have taken their medications and any other events such as vomiting, diarrhea, etc.
Overall Survival Rate	2 Participants

SECONDARY outcome

Timeframe: up to 12 months

Population: No data available for 12-month survival.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Prior to treatment with Abemaciclib (baseline)

Population: Four subjects completed EORTC QLQ-C30.

Quality of Life (QoL) will be assessed from the date of baseline and then every 4 weeks using the EORTC QLQ-C30. The QLQ-C30 comprises both multi-item scales and single-item measures, including a global health status/QoL scale, five functional scales, three multi-item symptom scales, and six single-item scales. For the multi-item scales, the raw score (RS) is calculated by taking the mean of the individual item scores. A linear transformation is then applied to standardize the raw score, resulting in a standardized score ranging from 0 to 100, where a score of 0 indicates the worst possible QoL and a score of 100 indicates the best possible QoL.

Outcome measures

Outcome measures
Measure	Abemaciclib n=4 Participants Abemaciclib will be given as a single oral agent The starting dose will be 200 mg twice daily. Dosing will continue daily for 28 days, this being one cycle. There will be no protocol scheduled hiatus and daily dosing will be continuous unless there is unacceptable toxicity, disease progression, or death. Abemaciclib: Participant will take 200 mg capsules or tablets orally twice daily. The participant will be instructed to swallow abemaciclib as a whole tablet and not to chew, crush or split capsules or tablets before swallowing. Participants should not ingest abemaciclib tablets if broken, cracked, or otherwise not intact. Doses should be taken at a approximately the same time every day, twice daily. Capsules or tablets can be taken orally with or without food. If the participant vomits or misses a dose of abemaciclib, the participant will be instructed to take the next dose at its scheduled time. Participants will also be provided with a diary and instructed to keep a twice daily record of the times they have taken their medications and any other events such as vomiting, diarrhea, etc.
Quality of Life Score	58.3 score on a scale Standard Deviation 37.9

SECONDARY outcome

Timeframe: Cycle 1 (28 days) treatment with Abemaciclib

Population: Three subjects completed EORTC QLQ-C30.

Outcome measures

Outcome measures
Measure	Abemaciclib n=3 Participants Abemaciclib will be given as a single oral agent The starting dose will be 200 mg twice daily. Dosing will continue daily for 28 days, this being one cycle. There will be no protocol scheduled hiatus and daily dosing will be continuous unless there is unacceptable toxicity, disease progression, or death. Abemaciclib: Participant will take 200 mg capsules or tablets orally twice daily. The participant will be instructed to swallow abemaciclib as a whole tablet and not to chew, crush or split capsules or tablets before swallowing. Participants should not ingest abemaciclib tablets if broken, cracked, or otherwise not intact. Doses should be taken at a approximately the same time every day, twice daily. Capsules or tablets can be taken orally with or without food. If the participant vomits or misses a dose of abemaciclib, the participant will be instructed to take the next dose at its scheduled time. Participants will also be provided with a diary and instructed to keep a twice daily record of the times they have taken their medications and any other events such as vomiting, diarrhea, etc.
Quality of Life Score	66.7 score on a scale Standard Deviation 30

SECONDARY outcome

Timeframe: Cycle 2 (56 days) treatment with Abemaciclib

Population: Three subjects completed EORTC QLQ-C30.

Outcome measures

Outcome measures
Measure	Abemaciclib n=3 Participants Abemaciclib will be given as a single oral agent The starting dose will be 200 mg twice daily. Dosing will continue daily for 28 days, this being one cycle. There will be no protocol scheduled hiatus and daily dosing will be continuous unless there is unacceptable toxicity, disease progression, or death. Abemaciclib: Participant will take 200 mg capsules or tablets orally twice daily. The participant will be instructed to swallow abemaciclib as a whole tablet and not to chew, crush or split capsules or tablets before swallowing. Participants should not ingest abemaciclib tablets if broken, cracked, or otherwise not intact. Doses should be taken at a approximately the same time every day, twice daily. Capsules or tablets can be taken orally with or without food. If the participant vomits or misses a dose of abemaciclib, the participant will be instructed to take the next dose at its scheduled time. Participants will also be provided with a diary and instructed to keep a twice daily record of the times they have taken their medications and any other events such as vomiting, diarrhea, etc.
Quality of Life Score	44.4 score on a scale Standard Deviation 25.5

SECONDARY outcome

Timeframe: Assessment at the end of treatment with Abemaciclib, approximately 60 days after starting treatment.

Population: Two subjects completed EORTC QLQ-C30.

Outcome measures

Outcome measures
Measure	Abemaciclib n=2 Participants Abemaciclib will be given as a single oral agent The starting dose will be 200 mg twice daily. Dosing will continue daily for 28 days, this being one cycle. There will be no protocol scheduled hiatus and daily dosing will be continuous unless there is unacceptable toxicity, disease progression, or death. Abemaciclib: Participant will take 200 mg capsules or tablets orally twice daily. The participant will be instructed to swallow abemaciclib as a whole tablet and not to chew, crush or split capsules or tablets before swallowing. Participants should not ingest abemaciclib tablets if broken, cracked, or otherwise not intact. Doses should be taken at a approximately the same time every day, twice daily. Capsules or tablets can be taken orally with or without food. If the participant vomits or misses a dose of abemaciclib, the participant will be instructed to take the next dose at its scheduled time. Participants will also be provided with a diary and instructed to keep a twice daily record of the times they have taken their medications and any other events such as vomiting, diarrhea, etc.
Quality of Life Score	29.2 score on a scale Standard Deviation 41.2

Adverse Events

Abemaciclib

Serious events: 0 serious events

Other events: 4 other events

Deaths: 3 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	Abemaciclib n=4 participants at risk Abemaciclib will be given as a single oral agent A sample size of 10 subjects is determined to be minimally sufficient for these pilot study objectives. The starting dose will be 200 mg twice daily. Dosing will continue daily for 28 days, this being one cycle. There will be no protocol scheduled hiatus and daily dosing will be continuous unless there is unacceptable toxicity, disease progression, or death. Abemaciclib: Participant will take 200 mg capsules or tablets orally twice daily. The participant will be instructed to swallow abemaciclib as a whole tablet and not to chew, crush or split capsules or tablets before swallowing. Participants should not ingest abemaciclib tablets if broken, cracked, or otherwise not intact. Doses should be taken at a approximately the same time every day, twice daily. Capsules or tablets can be taken orally with or without food. If the participant vomits or misses a dose of abemaciclib, the participant will be instructed to take the next dose at its scheduled time. Participants will also be provided with a diary and instructed to keep a twice daily record of the times they have taken their medications and any other events such as vomiting, diarrhea, etc.
Gastrointestinal disorders Diarrhea	75.0% 3/4 • Number of events 6 • 1st subject: 1 month 2nd subject: 1 month 3rd subject: 2 months 4th subject: 2 months
Gastrointestinal disorders Nausea	50.0% 2/4 • Number of events 5 • 1st subject: 1 month 2nd subject: 1 month 3rd subject: 2 months 4th subject: 2 months
Gastrointestinal disorders Abdominal Distension	25.0% 1/4 • Number of events 2 • 1st subject: 1 month 2nd subject: 1 month 3rd subject: 2 months 4th subject: 2 months
Gastrointestinal disorders Dyspepsia	25.0% 1/4 • Number of events 1 • 1st subject: 1 month 2nd subject: 1 month 3rd subject: 2 months 4th subject: 2 months
Gastrointestinal disorders Vomiting	25.0% 1/4 • Number of events 1 • 1st subject: 1 month 2nd subject: 1 month 3rd subject: 2 months 4th subject: 2 months
General disorders Fatigue	100.0% 4/4 • Number of events 5 • 1st subject: 1 month 2nd subject: 1 month 3rd subject: 2 months 4th subject: 2 months
General disorders Chills	50.0% 2/4 • Number of events 2 • 1st subject: 1 month 2nd subject: 1 month 3rd subject: 2 months 4th subject: 2 months
General disorders Flu like symptoms	25.0% 1/4 • Number of events 1 • 1st subject: 1 month 2nd subject: 1 month 3rd subject: 2 months 4th subject: 2 months
General disorders Night sweat	25.0% 1/4 • Number of events 1 • 1st subject: 1 month 2nd subject: 1 month 3rd subject: 2 months 4th subject: 2 months
Metabolism and nutrition disorders Anorexia	75.0% 3/4 • Number of events 4 • 1st subject: 1 month 2nd subject: 1 month 3rd subject: 2 months 4th subject: 2 months
General disorders Dehydration	25.0% 1/4 • Number of events 4 • 1st subject: 1 month 2nd subject: 1 month 3rd subject: 2 months 4th subject: 2 months
Psychiatric disorders Insomnia	50.0% 2/4 • Number of events 2 • 1st subject: 1 month 2nd subject: 1 month 3rd subject: 2 months 4th subject: 2 months
Psychiatric disorders Depression	25.0% 1/4 • Number of events 1 • 1st subject: 1 month 2nd subject: 1 month 3rd subject: 2 months 4th subject: 2 months
Infections and infestations Sepsis	25.0% 1/4 • Number of events 1 • 1st subject: 1 month 2nd subject: 1 month 3rd subject: 2 months 4th subject: 2 months
Infections and infestations Urinary tract infection	25.0% 1/4 • Number of events 1 • 1st subject: 1 month 2nd subject: 1 month 3rd subject: 2 months 4th subject: 2 months
Investigations Alanine aminotransferase increased	25.0% 1/4 • Number of events 1 • 1st subject: 1 month 2nd subject: 1 month 3rd subject: 2 months 4th subject: 2 months
Investigations Aspartate aminotransferase increased	25.0% 1/4 • Number of events 1 • 1st subject: 1 month 2nd subject: 1 month 3rd subject: 2 months 4th subject: 2 months
Investigations Blood bilirubin increased	25.0% 1/4 • Number of events 2 • 1st subject: 1 month 2nd subject: 1 month 3rd subject: 2 months 4th subject: 2 months
Investigations Creatinine increased	25.0% 1/4 • Number of events 2 • 1st subject: 1 month 2nd subject: 1 month 3rd subject: 2 months 4th subject: 2 months
Investigations BUN increased	25.0% 1/4 • Number of events 1 • 1st subject: 1 month 2nd subject: 1 month 3rd subject: 2 months 4th subject: 2 months
Investigations Platelet count decreased	25.0% 1/4 • Number of events 1 • 1st subject: 1 month 2nd subject: 1 month 3rd subject: 2 months 4th subject: 2 months
Musculoskeletal and connective tissue disorders Generalized muscle weakness	25.0% 1/4 • Number of events 1 • 1st subject: 1 month 2nd subject: 1 month 3rd subject: 2 months 4th subject: 2 months
Musculoskeletal and connective tissue disorders Pain in extremity	25.0% 1/4 • Number of events 1 • 1st subject: 1 month 2nd subject: 1 month 3rd subject: 2 months 4th subject: 2 months
Nervous system disorders Headache	50.0% 2/4 • Number of events 4 • 1st subject: 1 month 2nd subject: 1 month 3rd subject: 2 months 4th subject: 2 months
Nervous system disorders Dizziness	25.0% 1/4 • Number of events 1 • 1st subject: 1 month 2nd subject: 1 month 3rd subject: 2 months 4th subject: 2 months
Musculoskeletal and connective tissue disorders Movements involuntary	25.0% 1/4 • Number of events 1 • 1st subject: 1 month 2nd subject: 1 month 3rd subject: 2 months 4th subject: 2 months
Blood and lymphatic system disorders Anemia	25.0% 1/4 • Number of events 2 • 1st subject: 1 month 2nd subject: 1 month 3rd subject: 2 months 4th subject: 2 months
Cardiac disorders Sinus tachycardia	25.0% 1/4 • Number of events 1 • 1st subject: 1 month 2nd subject: 1 month 3rd subject: 2 months 4th subject: 2 months
Endocrine disorders Hypothyroidism	25.0% 1/4 • Number of events 1 • 1st subject: 1 month 2nd subject: 1 month 3rd subject: 2 months 4th subject: 2 months
Respiratory, thoracic and mediastinal disorders Epistaxis	25.0% 1/4 • Number of events 1 • 1st subject: 1 month 2nd subject: 1 month 3rd subject: 2 months 4th subject: 2 months

Additional Information

Dr. Nelson Yee

Penn State Health

Phone: 7175310003

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place