Trial Outcomes & Findings for A Trial of All-trans Retinoic Acid (ATRA) in Advanced Adenoid Cystic Carcinoma (NCT NCT03999684)

Results Overview

CR 0, PR 0, SD 11, PD 5, UE 2 Per Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.1) for target lesions and assessed by CT: Complete Response (CR), Disappearance of all target lesions; Partial Response (PR), \>=30% decrease in the sum of the longest diameter of target lesions; Overall Response (OR) = CR + PR. SD=stable disease, PD=progression of disease, UE=unevaluable

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

18 participants

Primary outcome timeframe

up to 8 months

Results posted on

2023-01-23

Participant Flow

Participant milestones

Participant milestones
Measure	Tretinoin -Participants will receive Tretinoin orally divided over two daily doses for days 1 through 14 of a 28-day cycle Tretinoin: ATRA control normal cell growth, cell differentiation (the normal process of making cell different from each other), and cell death during embryonic development and in certain tissues later in life. Retinoids effects on the cells are controlled by receptors on the nucleus of each cell (nuclear receptors)
Overall Study STARTED	18
Overall Study COMPLETED	18
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

A Trial of All-trans Retinoic Acid (ATRA) in Advanced Adenoid Cystic Carcinoma

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Tretinoin n=18 Participants -Participants will receive Tretinoin orally divided over two daily doses for days 1 through 14 of a 28-day cycle Tretinoin: ATRA control normal cell growth, cell differentiation (the normal process of making cell different from each other), and cell death during embryonic development and in certain tissues later in life. Retinoids effects on the cells are controlled by receptors on the nucleus of each cell (nuclear receptors)
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	14 Participants n=5 Participants
Age, Categorical >=65 years	4 Participants n=5 Participants
Age, Continuous	58 years n=5 Participants
Sex: Female, Male Female	11 Participants n=5 Participants
Sex: Female, Male Male	7 Participants n=5 Participants
Race/Ethnicity, Customized White/Caucasian	17 Participants n=5 Participants
Race/Ethnicity, Customized Other	1 Participants n=5 Participants
Race/Ethnicity, Customized Non-Hispanic	18 Participants n=5 Participants
Region of Enrollment United States	18 participants n=5 Participants

PRIMARY outcome

Timeframe: up to 8 months

CR 0, PR 0, SD 11, PD 5, UE 2 Per Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.1) for target lesions and assessed by CT: Complete Response (CR), Disappearance of all target lesions; Partial Response (PR), \>=30% decrease in the sum of the longest diameter of target lesions; Overall Response (OR) = CR + PR. SD=stable disease, PD=progression of disease, UE=unevaluable

Outcome measures

Outcome measures
Measure	Tretinoin n=18 Participants -Participants will receive Tretinoin orally divided over two daily doses for days 1 through 14 of a 28-day cycle Tretinoin: ATRA control normal cell growth, cell differentiation (the normal process of making cell different from each other), and cell death during embryonic development and in certain tissues later in life. Retinoids effects on the cells are controlled by receptors on the nucleus of each cell (nuclear receptors)
Best Overall Response Rate CR	0 Participants
Best Overall Response Rate PR	0 Participants
Best Overall Response Rate SD	11 Participants
Best Overall Response Rate PD	5 Participants
Best Overall Response Rate UE	2 Participants

SECONDARY outcome

Timeframe: up to 8 months

CR rate captured up to 8 months Per Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.1) for target lesions and assessed by CT: Complete Response (CR), Disappearance of all target lesions; Partial Response (PR), \>=30% decrease in the sum of the longest diameter of target lesions; Overall Response (OR) = CR + PR. SD=stable disease, PD=progression of disease, UE=unevaluable

Outcome measures

Outcome measures
Measure	Tretinoin n=18 Participants -Participants will receive Tretinoin orally divided over two daily doses for days 1 through 14 of a 28-day cycle Tretinoin: ATRA control normal cell growth, cell differentiation (the normal process of making cell different from each other), and cell death during embryonic development and in certain tissues later in life. Retinoids effects on the cells are controlled by receptors on the nucleus of each cell (nuclear receptors)
Complete Response	0 Participants

SECONDARY outcome

Timeframe: up to 8 months

PR rate captured up to 8 months Per Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.1) for target lesions and assessed by CT: Complete Response (CR), Disappearance of all target lesions; Partial Response (PR), \>=30% decrease in the sum of the longest diameter of target lesions; Overall Response (OR) = CR + PR. SD=stable disease, PD=progression of disease, UE=unevaluable

Outcome measures

Outcome measures
Measure	Tretinoin n=18 Participants -Participants will receive Tretinoin orally divided over two daily doses for days 1 through 14 of a 28-day cycle Tretinoin: ATRA control normal cell growth, cell differentiation (the normal process of making cell different from each other), and cell death during embryonic development and in certain tissues later in life. Retinoids effects on the cells are controlled by receptors on the nucleus of each cell (nuclear receptors)
Partial Response	0 Participants

SECONDARY outcome

Timeframe: up to 8 months

Median PFS or progression free survival Per Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.1) for target lesions and assessed by CT: Complete Response (CR), Disappearance of all target lesions; Partial Response (PR), \>=30% decrease in the sum of the longest diameter of target lesions; Overall Response (OR) = CR + PR. SD=stable disease, PD=progression of disease (Progression was defined as a 20% increase in the sum of the longest diameter of target lesions, or a measurable increase in a non-target lesion, or the appearance of new lesions), UE=unevaluable

Outcome measures

Outcome measures
Measure	Tretinoin n=18 Participants -Participants will receive Tretinoin orally divided over two daily doses for days 1 through 14 of a 28-day cycle Tretinoin: ATRA control normal cell growth, cell differentiation (the normal process of making cell different from each other), and cell death during embryonic development and in certain tissues later in life. Retinoids effects on the cells are controlled by receptors on the nucleus of each cell (nuclear receptors)
Progression Free Survival	3.2 months Interval 1.8 to 3.9

SECONDARY outcome

Timeframe: up to 8 months

Duration of CR+PR Per Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.1) for target lesions and assessed by CT: Complete Response (CR), Disappearance of all target lesions; Partial Response (PR), \>=30% decrease in the sum of the longest diameter of target lesions; Overall Response (OR) = CR + PR. SD=stable disease, PD=progression of disease, UE=unevaluable

Outcome measures

Outcome measures
Measure	Tretinoin n=18 Participants -Participants will receive Tretinoin orally divided over two daily doses for days 1 through 14 of a 28-day cycle Tretinoin: ATRA control normal cell growth, cell differentiation (the normal process of making cell different from each other), and cell death during embryonic development and in certain tissues later in life. Retinoids effects on the cells are controlled by receptors on the nucleus of each cell (nuclear receptors)
Duration Of Therapeutic Response	NA months No participants showed any complete or partial response

OTHER_PRE_SPECIFIED outcome

Timeframe: up to 8 months

Population: only 14 patients had samples evaluable for MYB IHC assessment (of a total of 18)

correlative measure of ATRA inhibitory effect measured by IHC (units: % MYB inhibition by IHC quantitative measurement; MYB IHC was performed on stained tissue slides from baseline tumors and scored (%) by an expert pathologist as no/low, medium, and high MYB expressing).

Outcome measures

Outcome measures
Measure	Tretinoin n=14 Participants -Participants will receive Tretinoin orally divided over two daily doses for days 1 through 14 of a 28-day cycle Tretinoin: ATRA control normal cell growth, cell differentiation (the normal process of making cell different from each other), and cell death during embryonic development and in certain tissues later in life. Retinoids effects on the cells are controlled by receptors on the nucleus of each cell (nuclear receptors)
Number of Participants With no/Low, Medium MYB Expression in ACC Tumors Low MYB expression	3 Participants
Number of Participants With no/Low, Medium MYB Expression in ACC Tumors Medium or high MYB expression	11 Participants

Adverse Events

Tretinoin

Serious events: 0 serious events

Other events: 14 other events

Deaths: 1 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	Tretinoin n=18 participants at risk -Participants will receive Tretinoin orally divided over two daily doses for days 1 through 14 of a 28-day cycle Tretinoin: ATRA control normal cell growth, cell differentiation (the normal process of making cell different from each other), and cell death during embryonic development and in certain tissues later in life. Retinoids effects on the cells are controlled by receptors on the nucleus of each cell (nuclear receptors)
Skin and subcutaneous tissue disorders Dry Skin	77.8% 14/18 • 8 months No difference
General disorders Headache	72.2% 13/18 • 8 months No difference

Additional Information

Dr. Glenn Hanna

Dana-Farber Cancer Institute

Phone: 6176323090

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place